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Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF) (DeFi)

Primary Purpose

Desmoid Tumor, Aggressive Fibromatosis

Status
Active
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Nirogacestat oral tablet
Placebo Oral Tablet
Sponsored by
SpringWorks Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Desmoid Tumor focused on measuring PF-03084014, GSI, gamma secretase inhibitor, notch pathway

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Double-Blind Key Inclusion Criteria:

  • Participant has histologically confirmed DT/AF (by local pathologist prior to informed consent) that has progressed by ≥ 20% as measured by RECIST v1.1 within 12 months of the screening visit scan.
  • Participant has:

    1. Treatment naïve, measurably progressing DT/AF that is deemed not amenable to surgery without the risk of significant morbidity; OR
    2. Recurrent, measurably progressing DT/AF following at least one line of therapy; OR
    3. Refractory, measurably progressing DT/AF following at least one line of therapy.
  • Participant has a DT/AF tumor where continued progressive disease will not result in immediate significant risk to the participant.
  • Participant agrees to provide archival or new tumor tissue for re-confirmation of disease.
  • If participant is currently being treated with any therapy for the treatment of DT/AF, this must be completed at least 28 days (or 5 half-lives, whichever is longer) prior to first dose of study treatment. All toxicities from prior therapy must be resolved to ≤ Grade 1 or clinical baseline.
  • Participants who are receiving chronic nonsteroidal anti-inflammatory drugs (NSAIDs) as treatment for conditions other than DT/AF must be receiving them prior to the documented DT/AF progressive disease (inclusion criteria 2) and on a stable dose for at least 28 days prior to first dose of study treatment.
  • Participant has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 at screening.
  • Participant has adequate organ and bone marrow function.

Double-Blind Key Exclusion Criteria:

  • Participant has known malabsorption syndrome or preexisting gastrointestinal conditions that may impair absorption of nirogacestat.
  • Participant has experienced any of the following within 6 months of signing informed consent: clinically significant cardiac disease (New York Heart Association Class III or IV), myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident, transient ischemic attack, or symptomatic pulmonary embolism.
  • Participant has an abnormal QT interval at screening.
  • Participant is using concomitant medications that are known to prolong the QT/QTcF interval including Class Ia and Class III antiarrhythmics at the time of informed consent. Non-antiarrhythmic medications which may prolong the QT/QTcF interval are allowed provided the participant does not have additional risk factors for Torsades de Pointes (TdP)
  • Participant has congenital long QT syndrome.
  • Participant has a history of additional risk factors for Torsades de Pointes (TdP) (e.g., heart failure, hypokalemia, family history of Long QT Syndrome).
  • Participant has had lymphoma, leukemia, or any malignancy within the past 5 years at the time of informed consent, except for any locally recurring cancer that has been treated curatively (e.g., resected basal or squamous cell skin cancer, superficial bladder cancer, carcinoma in situ of the cervix or breast), with no evidence of metastatic disease for 3 years at the time of informed consent.
  • Participant has current or chronic history of liver disease or known hepatic or biliary abnormalities (except for Gilbert's syndrome or asymptomatic gallstones).
  • Participant previously received or is currently receiving therapy with GS inhibitors or anti-Notch antibody therapy.
  • Participant is currently using any treatment for DT/AF including tyrosine kinase inhibitors (TKIs), NSAIDs (chronic daily use) or any investigational treatment 28 days (or 5 half-lives, whichever is longer) prior to the first dose of study treatment.

OR

Participant has started any treatment for DT/AF after the documented DT/AF progressive disease.

  • Participant is currently using or anticipates using food or drugs that are known strong/moderate cytochrome P450 3A4 (CYP3A4) inhibitors, or strong CYP3A inducers within 14 days prior to the first dose of study treatment.
  • Participant has a positive human immunodeficiency virus antibody test.
  • Participant has presence of Hepatitis B surface antigen at screening.
  • Participant has a positive Hepatitis C antibody or Hepatitis C ribonucleic acid (RNA) test result at screening or within 3 months prior to starting study treatment.
  • Participant is unable to tolerate MRI or for whom MRI is contraindicated.
  • Participant with active or chronic infection at the time of informed consent and during the screening period.
  • Participant has experienced other severe acute or chronic medical or psychiatric conditions within 1 year of signing informed consent.
  • Participant is unable to comply with study related procedures (including, but not limited to, the completion of electronic patient report outcomes (ePROs), or the ePRO questionnaires are not available in the participant's preferred language).

Open-Label Key Inclusion

  • Participant is enrolled in the double-blind phase when the estimated number of PFS events have been observed and the primary PFS analysis has been completed; OR
  • Participant is randomized to receive placebo in the double-blind phase and Central Imaging Review determines that the participant has radiographic progressive disease; OR
  • Participant is randomized to receive nirogacestat in the double-blind phase and Central Imaging Review determines that the participant has radiographic progressive disease but the participant is deriving clinical benefit without significant toxicity (as determined by the investigator).
  • Participant has adequate organ and bone marrow function

Open-Label Key Exclusion

  • Participant requires surgery to prevent organ dysfunction.
  • Participant has prematurely discontinued from the double-blind phase for any reason other than radiographic progressive disease (as determined by Central Imaging Review).
  • Participant developed a concurrent illness/condition that, in the opinion of the investigator, would represent a risk to overall health if they enroll in this study.
  • Participant has initiated a new treatment for DT/AF after the Central Imaging Review determines that a participant has radiographic progressive disease.

Sites / Locations

  • Arkansas Children's Hospital
  • USC/Norris Comprehensive Cancer Center
  • Ronald Regan UCLA Medical Center
  • Stanford Cancer Center
  • UCSF Mission Bay
  • Sarcoma Oncology Research Center
  • University of Colorado Hospital-Anschutz Cancer Pavillion (ACP)
  • Smilow Cancer Hospital at Yale-New Haven
  • Washington Cancer Institute at MedStar Washington Hospital Center
  • Mayo Clinic Florida
  • Sylvester Comprehensive Cancer Center
  • Northwestern Memorial Hospital
  • Johns Hopkins Hospital
  • Massachusetts General Hosptial (MGH)
  • Dana-Farber Cancer Institute (DFCI)
  • University of Michigan
  • Mayo Clinic Rochester
  • Washington Univerisity School of Medicine
  • Northwell Health
  • Columbia University Medical Center
  • Memorial Sloan Kettering Cancer Center
  • DUMC/Duke Cancer Center
  • Cincinnati Childrens's Hospital Medical Center
  • James Cancer Hospital and Solove Research Institute at The Ohio State University Comprehensive Cancer Center
  • Oregon Health & Science Univeristy-Center for Health & Healing
  • Abramson Cancer Center at Pennsylvania Hospital
  • Fox Chase Cancer Center
  • UPMC Hillman Cancer Ceter
  • Henry-Joyce Cancer Clinic
  • UT Southwestern Medical Center
  • The University of Texas MD Anderson Cancer Center
  • Seattle Cancer Care Alliance
  • University of Wisconsin Clinical Science Center
  • Froedtert Hospital & the Medical College of Wisconsin
  • Institut Jules Bordet-Medical Onocology
  • Cliniques Universitaires Saint-Luc, Institut Roi Albert II
  • UZ Gent
  • UZ Leuven
  • Princess Margaret Cancer Centre
  • McGill University Health Centre
  • Helios Klinikum Berlin-Buch
  • Universitaetsklinikum Hamburg-Eppendorf
  • Universitätsmedizin Mannheim
  • IRCCS Istituto Ortopedico Rizzoli
  • Istituto di Candiolo IRCCS Oncologia Medica
  • Fondazione IRCCS Instituto Nazionale dei Tumori di Milano
  • Policlinico Unvrsitario Campus Bio-Medico
  • Radboud University Medical Centre
  • The Netherlands Cancer Institute
  • Leiden University Medical Center (LUMC)
  • Department of Oncology, University College of London Hospital
  • The Royal Marsden NHS Foundation Trust

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Placebo Comparator

Experimental

Arm Label

Double-Blind Phase - Nirogacestat

Double-Blind Phase - Placebo

Open-Label Phase - Nirogacestat

Arm Description

Nirogacestat 150 mg by mouth, twice daily

Placebo 150 mg by mouth, twice daily

Nirogacestat 150 mg by mouth, twice daily

Outcomes

Primary Outcome Measures

Number of progression free survival (PFS) events as defined as the time from randomization until date of assessment of progression or death by any cause.
Progression will be determined radiographically using RECIST v1.1 criteria by an independent, blinded, central radiologic review, or clinically as assessed by the Investigator.

Secondary Outcome Measures

The incidence of adverse events (AEs) according to toxicities graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Overall response rate using RECIST Version 1.1 criteria.
Overall response rate is defined as the proportion of participants with complete response (CR) + partial response (PR) assessed by central reader using RECIST v1.1 criteria.
Duration of response for participants whose best response is CR or PR.
Tumor volume changes from baseline as measured by MRI volumetric.
Change from baseline in patient reported outcome (PRO) scores using the GOunder/Desmoid Tumor Research Tumor Foundation (DTRF) DEsmoid Symptom Scale (GODDESS);.
This PRO will measure desmoid tumor symptoms by evaluating change from baseline. The items are evaluated on an 11-point numeric rating scale (NRS) form 0-10 measure severity from "none" to "as bad as you can imagine," with a 24-hour recall period.
Change from baseline in PRO scores using the Brief Pain Inventory (BPI) short form.
This PRO will measure clinical pain by evaluating change from baseline. It consists of 9 questions and will utilize an 11-point NRS from 0-10 measure severity from "no pain" to "pain as bad as you can imagine," with a 24-hour recall period.
Change in baseline in PRO scores using the Patient-Reported Outcomes Measurement Information System Physical Function (PROMIS PF) short form 10a plus 3 additional items from PROMIS item banks.
This PRO will measure self-reported capability of physical activities by evaluating change from baseline. This includes the functioning of one's upper extremities (dexterity), lower extremities (walking or mobility), and central regions (neck, back), as well as instrumental activities of daily living, such as running errands. The PROMIS PF short form 10a plus (consisting) of 10 questions plus 3 additional questions from the PROMIS item bank will be used with a 7-day recall period.
Change from baseline in PRO scores using the GOunder/Desmoid Tumor Research Tumor Foundation (DTRF) DEsmoid Impact Scale (GODDESS);
This PRO will measure desmoid tumor impacts by evaluating change from baseline. The items are evaluated either on an 11-point NRS to measure severity, or a 5-point Likert Scale ranging from "none of the time" to "all of the time" to measure frequency, with a 7-day recall period.
Change from baseline in PRO scores using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC) QLQ-C30.
This PRO will measure the health-related quality of life of cancer patients by evaluated change from baseline. It consists of 30 questions overall with a 4-point scale and incorporates 5 functional scales (physical, role, cognitive, emotional and social), 3 symptom scales (fatigue, pain, and nausea and vomiting), a global health status/quality of life scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnea, loss of appetite, insomnia, constipation and diarrhea) and perceived financial impact of disease.

Full Information

First Posted
December 17, 2018
Last Updated
June 26, 2023
Sponsor
SpringWorks Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03785964
Brief Title
Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF)
Acronym
DeFi
Official Title
A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Trial of Nirogacestat Versus Placebo in Adult Patients With Progressing Desmoid Tumors/Aggressive Fibromatosis (DT/AF)
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
April 17, 2019 (Actual)
Primary Completion Date
April 7, 2022 (Actual)
Study Completion Date
December 31, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
SpringWorks Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
This study evaluates nirogacestat (PF-03084014) in the treatment of desmoid tumor/aggressive fibromatosis (DT/AF). In the double-blind phase, half of the participants will receive nirogacestat while the other half will receive placebo. Once participants are eligible to roll into the open-label phase, they will receive nirogacestat.
Detailed Description
Desmoid tumors, also referred to as aggressive fibromatosis, are rare, locally invasive, slow growing soft tissue tumors. Although considered benign because of their inability to metastasize, desmoid tumors can cause significant morbidity and occasionally mortality in patients. Nirogacestat (PF-03084014) is a potent, small molecule, selective, reversible, noncompetitive inhibitor of γ-secretase (GS) with a potential antitumor activity. Nirogacestat is being investigated for the treatment of desmoid tumors due to its ability to bind to GS, blocking proteolytic activation of Notch receptors. Previous clinical study data have shown that Notch signaling plays an important role in cancer development. Hence, inhibition of Notch signaling is an important strategy for therapeutic treatment.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Desmoid Tumor, Aggressive Fibromatosis
Keywords
PF-03084014, GSI, gamma secretase inhibitor, notch pathway

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Masking Description
For the double-blind phase, the participant, investigator, and all other clinical site personnel will be blinded to the assigned treatment allocation. All sponsor personnel will also be blinded except for the sponsor's quality assurance designee(s), safety designee(s), and clinical supply material designee(s). Once participants are eligible, they will roll into the open-label phase where they will receive nirogacestat. In the open-label phase, the participant, investigator, all other clinical site personnel, and the sponsor are not blinded.
Allocation
Randomized
Enrollment
142 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Double-Blind Phase - Nirogacestat
Arm Type
Experimental
Arm Description
Nirogacestat 150 mg by mouth, twice daily
Arm Title
Double-Blind Phase - Placebo
Arm Type
Placebo Comparator
Arm Description
Placebo 150 mg by mouth, twice daily
Arm Title
Open-Label Phase - Nirogacestat
Arm Type
Experimental
Arm Description
Nirogacestat 150 mg by mouth, twice daily
Intervention Type
Drug
Intervention Name(s)
Nirogacestat oral tablet
Other Intervention Name(s)
PF-03084014
Intervention Description
Nirogacestat tablet
Intervention Type
Drug
Intervention Name(s)
Placebo Oral Tablet
Intervention Description
Sugar pill manufactured to mimic nirogacestat 50 mg tablet
Primary Outcome Measure Information:
Title
Number of progression free survival (PFS) events as defined as the time from randomization until date of assessment of progression or death by any cause.
Description
Progression will be determined radiographically using RECIST v1.1 criteria by an independent, blinded, central radiologic review, or clinically as assessed by the Investigator.
Time Frame
On the first day of every 3 cycles (each cycle is 28 days) until disease progression is observed or death, whichever comes first, assessed up to approximately 2 years
Secondary Outcome Measure Information:
Title
The incidence of adverse events (AEs) according to toxicities graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Time Frame
Weekly for cycle 1, last day of cycle 2, first day of cycle 4 and then the first day of every 3 cycles (each cycle is 28 days) through study completion, an average of 2 years
Title
Overall response rate using RECIST Version 1.1 criteria.
Description
Overall response rate is defined as the proportion of participants with complete response (CR) + partial response (PR) assessed by central reader using RECIST v1.1 criteria.
Time Frame
On the first day of every 3 cycles (each cycle is 28 days) through study completion, an average of 2 years
Title
Duration of response for participants whose best response is CR or PR.
Time Frame
On the first day of every 3 cycles (each cycle is 28 days) through study completion, an average of 2 years
Title
Tumor volume changes from baseline as measured by MRI volumetric.
Time Frame
On the first day of every 6 cycles (each cycle is 28 days) through study completion, an average of 2 years
Title
Change from baseline in patient reported outcome (PRO) scores using the GOunder/Desmoid Tumor Research Tumor Foundation (DTRF) DEsmoid Symptom Scale (GODDESS);.
Description
This PRO will measure desmoid tumor symptoms by evaluating change from baseline. The items are evaluated on an 11-point numeric rating scale (NRS) form 0-10 measure severity from "none" to "as bad as you can imagine," with a 24-hour recall period.
Time Frame
Daily for the last 7 days of every cycle (each cycle is 28 days) through study completion, an average of 2 years
Title
Change from baseline in PRO scores using the Brief Pain Inventory (BPI) short form.
Description
This PRO will measure clinical pain by evaluating change from baseline. It consists of 9 questions and will utilize an 11-point NRS from 0-10 measure severity from "no pain" to "pain as bad as you can imagine," with a 24-hour recall period.
Time Frame
Daily for the last 7 days of every cycle (each cycle is 28 days) through study completion, an average of 2 years
Title
Change in baseline in PRO scores using the Patient-Reported Outcomes Measurement Information System Physical Function (PROMIS PF) short form 10a plus 3 additional items from PROMIS item banks.
Description
This PRO will measure self-reported capability of physical activities by evaluating change from baseline. This includes the functioning of one's upper extremities (dexterity), lower extremities (walking or mobility), and central regions (neck, back), as well as instrumental activities of daily living, such as running errands. The PROMIS PF short form 10a plus (consisting) of 10 questions plus 3 additional questions from the PROMIS item bank will be used with a 7-day recall period.
Time Frame
On the last day of every cycle (each cycle is 28 days) through study completion, an average of 2 years
Title
Change from baseline in PRO scores using the GOunder/Desmoid Tumor Research Tumor Foundation (DTRF) DEsmoid Impact Scale (GODDESS);
Description
This PRO will measure desmoid tumor impacts by evaluating change from baseline. The items are evaluated either on an 11-point NRS to measure severity, or a 5-point Likert Scale ranging from "none of the time" to "all of the time" to measure frequency, with a 7-day recall period.
Time Frame
On the last day of every cycle (each cycle is 28 days) through study completion, an average of 2 years
Title
Change from baseline in PRO scores using the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire-Core 30 (EORTC) QLQ-C30.
Description
This PRO will measure the health-related quality of life of cancer patients by evaluated change from baseline. It consists of 30 questions overall with a 4-point scale and incorporates 5 functional scales (physical, role, cognitive, emotional and social), 3 symptom scales (fatigue, pain, and nausea and vomiting), a global health status/quality of life scale, and a number of single items assessing additional symptoms commonly reported by cancer patients (dyspnea, loss of appetite, insomnia, constipation and diarrhea) and perceived financial impact of disease.
Time Frame
Last day of every cycle (each cycle is 28 days) through study completion, an average of 2 years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Double-Blind Key Inclusion Criteria: Participant has histologically confirmed DT/AF (by local pathologist prior to informed consent) that has progressed by ≥ 20% as measured by RECIST v1.1 within 12 months of the screening visit scan. Participant has: Treatment naïve, measurably progressing DT/AF that is deemed not amenable to surgery without the risk of significant morbidity; OR Recurrent, measurably progressing DT/AF following at least one line of therapy; OR Refractory, measurably progressing DT/AF following at least one line of therapy. Participant has a DT/AF tumor where continued progressive disease will not result in immediate significant risk to the participant. Participant agrees to provide archival or new tumor tissue for re-confirmation of disease. If participant is currently being treated with any therapy for the treatment of DT/AF, this must be completed at least 28 days (or 5 half-lives, whichever is longer) prior to first dose of study treatment. All toxicities from prior therapy must be resolved to ≤ Grade 1 or clinical baseline. Participants who are receiving chronic nonsteroidal anti-inflammatory drugs (NSAIDs) as treatment for conditions other than DT/AF must be receiving them prior to the documented DT/AF progressive disease (inclusion criteria 2) and on a stable dose for at least 28 days prior to first dose of study treatment. Participant has an Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 at screening. Participant has adequate organ and bone marrow function. Double-Blind Key Exclusion Criteria: Participant has known malabsorption syndrome or preexisting gastrointestinal conditions that may impair absorption of nirogacestat. Participant has experienced any of the following within 6 months of signing informed consent: clinically significant cardiac disease (New York Heart Association Class III or IV), myocardial infarction, severe/unstable angina, coronary/peripheral artery bypass graft, symptomatic congestive heart failure, cerebrovascular accident, transient ischemic attack, or symptomatic pulmonary embolism. Participant has an abnormal QT interval at screening. Participant is using concomitant medications that are known to prolong the QT/QTcF interval including Class Ia and Class III antiarrhythmics at the time of informed consent. Non-antiarrhythmic medications which may prolong the QT/QTcF interval are allowed provided the participant does not have additional risk factors for Torsades de Pointes (TdP) Participant has congenital long QT syndrome. Participant has a history of additional risk factors for Torsades de Pointes (TdP) (e.g., heart failure, hypokalemia, family history of Long QT Syndrome). Participant has had lymphoma, leukemia, or any malignancy within the past 5 years at the time of informed consent, except for any locally recurring cancer that has been treated curatively (e.g., resected basal or squamous cell skin cancer, superficial bladder cancer, carcinoma in situ of the cervix or breast), with no evidence of metastatic disease for 3 years at the time of informed consent. Participant has current or chronic history of liver disease or known hepatic or biliary abnormalities (except for Gilbert's syndrome or asymptomatic gallstones). Participant previously received or is currently receiving therapy with GS inhibitors or anti-Notch antibody therapy. Participant is currently using any treatment for DT/AF including tyrosine kinase inhibitors (TKIs), NSAIDs (chronic daily use) or any investigational treatment 28 days (or 5 half-lives, whichever is longer) prior to the first dose of study treatment. OR Participant has started any treatment for DT/AF after the documented DT/AF progressive disease. Participant is currently using or anticipates using food or drugs that are known strong/moderate cytochrome P450 3A4 (CYP3A4) inhibitors, or strong CYP3A inducers within 14 days prior to the first dose of study treatment. Participant has a positive human immunodeficiency virus antibody test. Participant has presence of Hepatitis B surface antigen at screening. Participant has a positive Hepatitis C antibody or Hepatitis C ribonucleic acid (RNA) test result at screening or within 3 months prior to starting study treatment. Participant is unable to tolerate MRI or for whom MRI is contraindicated. Participant with active or chronic infection at the time of informed consent and during the screening period. Participant has experienced other severe acute or chronic medical or psychiatric conditions within 1 year of signing informed consent. Participant is unable to comply with study related procedures (including, but not limited to, the completion of electronic patient report outcomes (ePROs), or the ePRO questionnaires are not available in the participant's preferred language). Open-Label Key Inclusion Participant is enrolled in the double-blind phase when the estimated number of PFS events have been observed and the primary PFS analysis has been completed; OR Participant is randomized to receive placebo in the double-blind phase and Central Imaging Review determines that the participant has radiographic progressive disease; OR Participant is randomized to receive nirogacestat in the double-blind phase and Central Imaging Review determines that the participant has radiographic progressive disease but the participant is deriving clinical benefit without significant toxicity (as determined by the investigator). Participant has adequate organ and bone marrow function Open-Label Key Exclusion Participant requires surgery to prevent organ dysfunction. Participant has prematurely discontinued from the double-blind phase for any reason other than radiographic progressive disease (as determined by Central Imaging Review). Participant developed a concurrent illness/condition that, in the opinion of the investigator, would represent a risk to overall health if they enroll in this study. Participant has initiated a new treatment for DT/AF after the Central Imaging Review determines that a participant has radiographic progressive disease.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bernd Kasper, MD
Organizational Affiliation
Mannheim University Medical Center
Official's Role
Principal Investigator
Facility Information:
Facility Name
Arkansas Children's Hospital
City
Little Rock
State/Province
Arkansas
ZIP/Postal Code
72202
Country
United States
Facility Name
USC/Norris Comprehensive Cancer Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90033
Country
United States
Facility Name
Ronald Regan UCLA Medical Center
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Stanford Cancer Center
City
Palo Alto
State/Province
California
ZIP/Postal Code
94304
Country
United States
Facility Name
UCSF Mission Bay
City
San Francisco
State/Province
California
ZIP/Postal Code
94158
Country
United States
Facility Name
Sarcoma Oncology Research Center
City
Santa Monica
State/Province
California
ZIP/Postal Code
90403
Country
United States
Facility Name
University of Colorado Hospital-Anschutz Cancer Pavillion (ACP)
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
Smilow Cancer Hospital at Yale-New Haven
City
New Haven
State/Province
Connecticut
ZIP/Postal Code
06510
Country
United States
Facility Name
Washington Cancer Institute at MedStar Washington Hospital Center
City
Washington
State/Province
District of Columbia
ZIP/Postal Code
20010
Country
United States
Facility Name
Mayo Clinic Florida
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Facility Name
Sylvester Comprehensive Cancer Center
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Northwestern Memorial Hospital
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60611
Country
United States
Facility Name
Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Massachusetts General Hosptial (MGH)
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Dana-Farber Cancer Institute (DFCI)
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215
Country
United States
Facility Name
University of Michigan
City
Ann Arbor
State/Province
Michigan
ZIP/Postal Code
48109
Country
United States
Facility Name
Mayo Clinic Rochester
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Washington Univerisity School of Medicine
City
Saint Louis
State/Province
Missouri
ZIP/Postal Code
63110
Country
United States
Facility Name
Northwell Health
City
Lake Success
State/Province
New York
ZIP/Postal Code
11042
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Memorial Sloan Kettering Cancer Center
City
New York
State/Province
New York
ZIP/Postal Code
10065
Country
United States
Facility Name
DUMC/Duke Cancer Center
City
Durham
State/Province
North Carolina
ZIP/Postal Code
27710
Country
United States
Facility Name
Cincinnati Childrens's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
James Cancer Hospital and Solove Research Institute at The Ohio State University Comprehensive Cancer Center
City
Columbus
State/Province
Ohio
ZIP/Postal Code
43210
Country
United States
Facility Name
Oregon Health & Science Univeristy-Center for Health & Healing
City
Portland
State/Province
Oregon
ZIP/Postal Code
97201
Country
United States
Facility Name
Abramson Cancer Center at Pennsylvania Hospital
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19106
Country
United States
Facility Name
Fox Chase Cancer Center
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19111
Country
United States
Facility Name
UPMC Hillman Cancer Ceter
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15232
Country
United States
Facility Name
Henry-Joyce Cancer Clinic
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37232
Country
United States
Facility Name
UT Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
The University of Texas MD Anderson Cancer Center
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Seattle Cancer Care Alliance
City
Seattle
State/Province
Washington
ZIP/Postal Code
98109
Country
United States
Facility Name
University of Wisconsin Clinical Science Center
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53792
Country
United States
Facility Name
Froedtert Hospital & the Medical College of Wisconsin
City
Milwaukee
State/Province
Wisconsin
ZIP/Postal Code
53226
Country
United States
Facility Name
Institut Jules Bordet-Medical Onocology
City
Brussels
ZIP/Postal Code
1000
Country
Belgium
Facility Name
Cliniques Universitaires Saint-Luc, Institut Roi Albert II
City
Brussels
ZIP/Postal Code
1200
Country
Belgium
Facility Name
UZ Gent
City
Gent
ZIP/Postal Code
9000
Country
Belgium
Facility Name
UZ Leuven
City
Leuven
ZIP/Postal Code
3000
Country
Belgium
Facility Name
Princess Margaret Cancer Centre
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G2M9
Country
Canada
Facility Name
McGill University Health Centre
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H4A3JI
Country
Canada
Facility Name
Helios Klinikum Berlin-Buch
City
Berlin
ZIP/Postal Code
13125
Country
Germany
Facility Name
Universitaetsklinikum Hamburg-Eppendorf
City
Hamburg
ZIP/Postal Code
20246
Country
Germany
Facility Name
Universitätsmedizin Mannheim
City
Mannheim
ZIP/Postal Code
D-68167
Country
Germany
Facility Name
IRCCS Istituto Ortopedico Rizzoli
City
Bologna
ZIP/Postal Code
40136
Country
Italy
Facility Name
Istituto di Candiolo IRCCS Oncologia Medica
City
Candiolo
ZIP/Postal Code
10060
Country
Italy
Facility Name
Fondazione IRCCS Instituto Nazionale dei Tumori di Milano
City
Milano
ZIP/Postal Code
20133
Country
Italy
Facility Name
Policlinico Unvrsitario Campus Bio-Medico
City
Roma
ZIP/Postal Code
00128
Country
Italy
Facility Name
Radboud University Medical Centre
City
Nijmegen
State/Province
Gelderland
ZIP/Postal Code
6525GA
Country
Netherlands
Facility Name
The Netherlands Cancer Institute
City
Amsterdam
ZIP/Postal Code
1066
Country
Netherlands
Facility Name
Leiden University Medical Center (LUMC)
City
Leiden
ZIP/Postal Code
23333
Country
Netherlands
Facility Name
Department of Oncology, University College of London Hospital
City
London
ZIP/Postal Code
NW12PG
Country
United Kingdom
Facility Name
The Royal Marsden NHS Foundation Trust
City
London
ZIP/Postal Code
SW36JJ
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
SpringWorks Therapeutics is committed to data transparency and sharing data to further research while maintaining the privacy and confidentiality of research participants. Pertinent patient-level data from completed registrational clinical trials will be made available by SpringWorks to qualified researchers upon approval of reasonable requests following de-identification/anonymization pursuant to applicable law.

Learn more about this trial

Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF)

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