Back to resultsA Phase 2 Study of Intravenous AMB-05X in Tenosynovial Giant Cell Tumor Patients
Primary Purpose
Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis, TGCT
Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
AMB-05X
Sponsored by
About this trial
This is an interventional treatment trial for Tenosynovial Giant Cell Tumor focused on measuring Tenosynovial Giant Cell Tumor, TGCT, PVNS
Eligibility Criteria
Inclusion Criteria:
- Subject ≥ 18 years
- A confirmed diagnosis of TGCT
- Measurable disease based on RECIST v1.1
- Symptomatic disease
- Stable prescription of analgesic regimen
- Agrees to follow contraception guidelines
- Adequate hematologic, hepatic, and renal function, at Screening
- Willing and able to complete self-assessment instruments throughout the study
Exclusion Criteria:
- Prior investigational drug use within 4 weeks or 5 half-lives of Baseline
- Current or prior radiotherapy within 3 months before Baseline
- Current or prior active cancer within 3 years before Baseline that requires/required therapy (eg, surgery, chemotherapy, or radiation therapy)
- Known metastatic TGCT or malignant transformation of diffuse-type TGCT
- Hepatitis C virus (HCV) or hepatitis B virus (HBV) or known active or chronic infection with human immuno deficiency virus (HIV)
- Known active tuberculosis (TB)
- Significant concomitant arthropathy in the affected joint, serious illness, uncontrolled infection, or a medical or psychiatric history
- Women who are pregnant or breastfeeding
- Screening Fridericia-corrected QT interval(QTcF) ≥450ms (men) or ≥470ms (women)
- MRI contraindications (eg, pacemaker, loose metallic implants)
- History of hypersensitivity to any ingredient in the study drug
- History of drug or alcohol abuse within 3 months before Baseline
- Has any other severe acute or chronic medical or psychiatric condition or clinically significant laboratory abnormality that may increase the risk associated with study participation/treatment, interfere with interpretation of study results, or, in the Investigator's opinion, make the subject inappropriate for this study
- A person who is held in detention as the result of a judicial or official decision or who is in a subordinate relationship to the Sponsor or Investigator
- A subject who, in the opinion of the Investigator, should not participate in the study for any reason, including if there is a question about the subject's ability to comply with study requirements
Sites / Locations
- AmMax Bio Clinical Site
- AmMax Bio Clinical Site
- AmMax Bio Clinical Site
- AmMax Bio Clinical Site
- AmMax Bio Clinical Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Cohort A
Arm Description
Low-Dose AMB-05X Each subject will receive a low dose of AMB-05X every 2 weeks, for a total of 6 doses over the 12-week treatment period.
Outcomes
Primary Outcome Measures
Overall tumor response per RECIST
Proportion of subjects achieving overall tumor response per the Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1
Secondary Outcome Measures
Treatment-emergent adverse events
Frequency and severity of reported treatment-emergent adverse events
Tumor response based on tumor volume
Proportion of subjects with overall response based on tumor volume score
Mean change from baseline in range of motion (ROM)
ROM of the joint will be assessed by qualified assessors. Measurements will be recorded in degrees. At baseline, the plane of movement with the smallest (worst) relative value will be identified; only this plane will be used for evaluating the change in ROM subsequently.
Mean change from Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function score
The PROMIS Physical Function Scale will be used to assess physical function. The scale ranges from 1 ('unable to do' or 'cannot do') to 5 ('without any difficulty' or 'not at all'), where higher scores represent better outcomes.
Mean change from Baseline in Worst Stiffness Numeric Rating Scale (NRS) score
The Worst Stiffness NRS is a 1-item, self-administered questionnaire assessing the "worst" stiffness within the last 24 hours. The NRS for this item ranges from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).
Mean change from Baseline in Worst Pain NRS score
The Worst Pain NRS is a component of the Brief Pain Inventory assessing the "worst" pain in the last 24 hours. The NRS for this item ranges from 0 (no pain) to 10 (pain as bad as you can imagine).
Mean change from Baseline in the Brief Pain Inventory (BPI) score
Brief Pain Inventory (BPI) Short Form is a self-administered questionnaire used to evaluate the severity of a subject's pain and the impact of this pain on the subject's daily functioning. The subject is asked to rate their worst, least, average, and current pain intensity, list current treatments and their perceived effectiveness, and rate the degree that pain interferes with general activity, mood, walking ability, normal work, relations with other persons, sleep, and enjoyment of life on a scale from 0 to 10.
EQ-5D-5L (EuroQol-5 Dimension-5 Level) Health Assessment
EQ-5D-5L (EuroQol-5 Dimension-5 Level) is a widely used quality of life instrument that includes questions in each of 5 domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The choices include 5 levels of severity for each domain followed by a general health visual analogue scale (VAS).
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04938180
Brief Title
A Phase 2 Study of Intravenous AMB-05X in Tenosynovial Giant Cell Tumor Patients
Official Title
A Phase 2, Adaptive, Open-Label, Multiple-Dose, Dose-Escalation Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of Intravenous AMB-05X in Subjects With Tenosynovial Giant Cell Tumor
Study Type
Interventional
2. Study Status
Record Verification Date
July 2022
Overall Recruitment Status
Active, not recruiting
Study Start Date
September 16, 2021 (Actual)
Primary Completion Date
April 20, 2022 (Actual)
Study Completion Date
October 31, 2022 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
AmMax Bio, 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
The purpose of this Phase 2, open-label, multiple-dose, dose-escalation study is to evaluate intravenous AMB-05X in the treatment of patients with TGCT.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Tenosynovial Giant Cell Tumor, Pigmented Villonodular Synovitis, TGCT, PVNS - Pigmented Villonodular Synovitis
Keywords
Tenosynovial Giant Cell Tumor, TGCT, PVNS
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Sequential Assignment
Model Description
A data monitoring committee will review study data on an ongoing basis and advise on the appropriate dose for each cohort.
Masking
None (Open Label)
Allocation
N/A
Enrollment
4 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Cohort A
Arm Type
Experimental
Arm Description
Low-Dose AMB-05X Each subject will receive a low dose of AMB-05X every 2 weeks, for a total of 6 doses over the 12-week treatment period.
Intervention Type
Biological
Intervention Name(s)
AMB-05X
Intervention Description
A fully human monoclonal immunoglobulin (IgG2) directed against c-fms
Primary Outcome Measure Information:
Title
Overall tumor response per RECIST
Description
Proportion of subjects achieving overall tumor response per the Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1
Time Frame
Week 12
Secondary Outcome Measure Information:
Title
Treatment-emergent adverse events
Description
Frequency and severity of reported treatment-emergent adverse events
Time Frame
Up to week 24
Title
Tumor response based on tumor volume
Description
Proportion of subjects with overall response based on tumor volume score
Time Frame
Week 12
Title
Mean change from baseline in range of motion (ROM)
Description
ROM of the joint will be assessed by qualified assessors. Measurements will be recorded in degrees. At baseline, the plane of movement with the smallest (worst) relative value will be identified; only this plane will be used for evaluating the change in ROM subsequently.
Time Frame
Week 12
Title
Mean change from Baseline in the Patient-Reported Outcomes Measurement Information System (PROMIS) Physical Function score
Description
The PROMIS Physical Function Scale will be used to assess physical function. The scale ranges from 1 ('unable to do' or 'cannot do') to 5 ('without any difficulty' or 'not at all'), where higher scores represent better outcomes.
Time Frame
Week 12
Title
Mean change from Baseline in Worst Stiffness Numeric Rating Scale (NRS) score
Description
The Worst Stiffness NRS is a 1-item, self-administered questionnaire assessing the "worst" stiffness within the last 24 hours. The NRS for this item ranges from 0 (no stiffness) to 10 (stiffness as bad as you can imagine).
Time Frame
Week 12
Title
Mean change from Baseline in Worst Pain NRS score
Description
The Worst Pain NRS is a component of the Brief Pain Inventory assessing the "worst" pain in the last 24 hours. The NRS for this item ranges from 0 (no pain) to 10 (pain as bad as you can imagine).
Time Frame
Week 12
Title
Mean change from Baseline in the Brief Pain Inventory (BPI) score
Description
Brief Pain Inventory (BPI) Short Form is a self-administered questionnaire used to evaluate the severity of a subject's pain and the impact of this pain on the subject's daily functioning. The subject is asked to rate their worst, least, average, and current pain intensity, list current treatments and their perceived effectiveness, and rate the degree that pain interferes with general activity, mood, walking ability, normal work, relations with other persons, sleep, and enjoyment of life on a scale from 0 to 10.
Time Frame
Week 12
Title
EQ-5D-5L (EuroQol-5 Dimension-5 Level) Health Assessment
Description
EQ-5D-5L (EuroQol-5 Dimension-5 Level) is a widely used quality of life instrument that includes questions in each of 5 domains: mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. The choices include 5 levels of severity for each domain followed by a general health visual analogue scale (VAS).
Time Frame
Week 12
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Subject ≥ 18 years
A confirmed diagnosis of TGCT
Measurable disease based on RECIST v1.1
Symptomatic disease
Stable prescription of analgesic regimen
Agrees to follow contraception guidelines
Adequate hematologic, hepatic, and renal function, at Screening
Willing and able to complete self-assessment instruments throughout the study
Exclusion Criteria:
Prior investigational drug use within 4 weeks or 5 half-lives of Baseline
Current or prior radiotherapy within 3 months before Baseline
Current or prior active cancer within 3 years before Baseline that requires/required therapy (eg, surgery, chemotherapy, or radiation therapy)
Known metastatic TGCT or malignant transformation of diffuse-type TGCT
Hepatitis C virus (HCV) or hepatitis B virus (HBV) or known active or chronic infection with human immuno deficiency virus (HIV)
Known active tuberculosis (TB)
Significant concomitant arthropathy in the affected joint, serious illness, uncontrolled infection, or a medical or psychiatric history
Women who are pregnant or breastfeeding
Screening Fridericia-corrected QT interval(QTcF) ≥450ms (men) or ≥470ms (women)
MRI contraindications (eg, pacemaker, loose metallic implants)
History of hypersensitivity to any ingredient in the study drug
History of drug or alcohol abuse within 3 months before Baseline
Has any other severe acute or chronic medical or psychiatric condition or clinically significant laboratory abnormality that may increase the risk associated with study participation/treatment, interfere with interpretation of study results, or, in the Investigator's opinion, make the subject inappropriate for this study
A person who is held in detention as the result of a judicial or official decision or who is in a subordinate relationship to the Sponsor or Investigator
A subject who, in the opinion of the Investigator, should not participate in the study for any reason, including if there is a question about the subject's ability to comply with study requirements
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dorothy Nguyen, MD
Organizational Affiliation
AmMax Bio, Inc.
Official's Role
Study Chair
Facility Information:
Facility Name
AmMax Bio Clinical Site
City
Berlin
Country
Germany
Facility Name
AmMax Bio Clinical Site
City
Warsaw
Country
Poland
Facility Name
AmMax Bio Clinical Site
City
Dnipro
Country
Ukraine
Facility Name
AmMax Bio Clinical Site
City
Kharkiv
Country
Ukraine
Facility Name
AmMax Bio Clinical Site
City
Kyiv
Country
Ukraine
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
A Phase 2 Study of Intravenous AMB-05X in Tenosynovial Giant Cell Tumor Patients
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