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Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP

Primary Purpose

Erythropoietic Protoporphyria

Status
Not yet recruiting
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
DISC-1459
DISC-1459
Sponsored by
Disc Medicine, Inc
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Erythropoietic Protoporphyria focused on measuring EPP, DISC-1459, porphyria, RO4917838

Eligibility Criteria

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

Inclusion Criteria: Participants with diagnosis of EPP who are participating (or who have participated) in a prior Disc Medicine bitopertin study and who have completed the randomized treatment phase and End-of-Study visit Aged ≥12 years upon study consent Body weight ≥32 kg for participants <18 years of age and BMI ≥18.5 kg/m2 for adult participants Exclusion Criteria: Participants who have an ongoing SAE from a clinical study that is assessed by the investigator as related to bitopertin Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgement of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude participation in the study Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, or participant diary data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months Planned treatment with afamelanotide or dersimelagon during the study Planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of cytochrome p450 (CYP)3A4 enzymes throughout the study If female, pregnant, or breastfeeding Participation in any other clinical protocol or investigational trial, other than Disc Medicine bitopertin trials, that involves administration of experimental therapy and/or therapeutic devices within 30 days of Day 1 Score of PHQ-8 ≥10 at screening or any response of "yes" on the C-SSRS Grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the stud

Sites / Locations

  • University of Alabama Hospital
  • University of California San Francisco
  • University of Miami Miller School of Medicine
  • Massachusetts General Hospital
  • Mount Sinai Hospital
  • Atrium Health Wake Forest Baptist
  • Einstein Medical Center
  • University of Pittsburgh Cancer Pavilion
  • University of Texas
  • Fred Hutchinson Cancer Center
  • Royal Prince Alfred Hospital
  • The Royal Melbourne Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Experimental

Arm Label

DISC-1459 Oral Dose Level 1

DISC-1459 Oral Dose Level 2

Arm Description

Oral dose, once a day

Oral dose, once a day

Outcomes

Primary Outcome Measures

Incidence of treatment-emergent adverse events
Incidence of clinically abnormal vital signs
Incidence of clinically abnormal physical exam
Incidence of abnormal laboratory test results
Assessment of Patient Health Questionnaire (PHQ-8)
The Patient Health Questionnaire (PHQ-8), an 8-item participant-report measure for screening for depression and for establishing depression severity. The total score ranges from 0-24, with a higher score indicating greater depression symptom severity.
Assessment of C-SSRS
The C-SSRS is a clinician-rated scale that assesses suicidality from ideation to behaviors and monitors the potential emergence of suicidality in clinical studies.

Secondary Outcome Measures

Change from baseline in daily daylight tolerance, as assessed by total hours spent in the sunlight without pain and average time to first prodromal syndrome in sunlight
Change from baseline in whole blood metal-free PPIX levels
Plasma Bitopertin Concentrations

Full Information

First Posted
May 22, 2023
Last Updated
September 5, 2023
Sponsor
Disc Medicine, Inc
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1. Study Identification

Unique Protocol Identification Number
NCT05883748
Brief Title
Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP
Official Title
An Open-Label, Long-Term Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With Erythropoietic Protoporphyria (EPP)
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Not yet recruiting
Study Start Date
September 2023 (Anticipated)
Primary Completion Date
June 2028 (Anticipated)
Study Completion Date
June 2028 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Disc Medicine, Inc

4. Oversight

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

5. Study Description

Brief Summary
This is an open-label, long-term extension study to investigate the safety, tolerability and efficacy of DISC-1459 in participants with EPP.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Erythropoietic Protoporphyria
Keywords
EPP, DISC-1459, porphyria, RO4917838

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
200 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
DISC-1459 Oral Dose Level 1
Arm Type
Experimental
Arm Description
Oral dose, once a day
Arm Title
DISC-1459 Oral Dose Level 2
Arm Type
Experimental
Arm Description
Oral dose, once a day
Intervention Type
Drug
Intervention Name(s)
DISC-1459
Other Intervention Name(s)
Bitopertin, RO4917838
Intervention Description
DISC-1459 dose level 1
Intervention Type
Drug
Intervention Name(s)
DISC-1459
Other Intervention Name(s)
Bitopertin, RO4917838
Intervention Description
DISC-1459 dose level 2
Primary Outcome Measure Information:
Title
Incidence of treatment-emergent adverse events
Time Frame
up to 5 Years
Title
Incidence of clinically abnormal vital signs
Time Frame
up to 5 Years
Title
Incidence of clinically abnormal physical exam
Time Frame
up to 5 Years
Title
Incidence of abnormal laboratory test results
Time Frame
up to 5 Years
Title
Assessment of Patient Health Questionnaire (PHQ-8)
Description
The Patient Health Questionnaire (PHQ-8), an 8-item participant-report measure for screening for depression and for establishing depression severity. The total score ranges from 0-24, with a higher score indicating greater depression symptom severity.
Time Frame
up to 5 Years
Title
Assessment of C-SSRS
Description
The C-SSRS is a clinician-rated scale that assesses suicidality from ideation to behaviors and monitors the potential emergence of suicidality in clinical studies.
Time Frame
up to 5 Years
Secondary Outcome Measure Information:
Title
Change from baseline in daily daylight tolerance, as assessed by total hours spent in the sunlight without pain and average time to first prodromal syndrome in sunlight
Time Frame
up to 5 Years
Title
Change from baseline in whole blood metal-free PPIX levels
Time Frame
up to 5 Years
Title
Plasma Bitopertin Concentrations
Time Frame
up to 5 Years

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Participants with diagnosis of EPP who are participating (or who have participated) in a prior Disc Medicine bitopertin study and who have completed the randomized treatment phase and End-of-Study visit Aged ≥12 years upon study consent Body weight ≥32 kg for participants <18 years of age and BMI ≥18.5 kg/m2 for adult participants Exclusion Criteria: Participants who have an ongoing SAE from a clinical study that is assessed by the investigator as related to bitopertin Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgement of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude participation in the study Condition or concomitant medication that would confound the ability to interpret clinical, clinical laboratory, or participant diary data, including a major psychiatric condition that has had an exacerbation or required hospitalization in the last 6 months Planned treatment with afamelanotide or dersimelagon during the study Planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of cytochrome p450 (CYP)3A4 enzymes throughout the study If female, pregnant, or breastfeeding Participation in any other clinical protocol or investigational trial, other than Disc Medicine bitopertin trials, that involves administration of experimental therapy and/or therapeutic devices within 30 days of Day 1 Score of PHQ-8 ≥10 at screening or any response of "yes" on the C-SSRS Grapefruit/Seville orange and products containing these for 14 days prior to first dose of study drug and throughout the stud
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Disc Medicine Clinical Trials
Phone
(617) 674 9274
Email
clinicaltrials@discmedicine.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Will Savage, MD PhD
Organizational Affiliation
Disc Medicine
Official's Role
Study Director
Facility Information:
Facility Name
University of Alabama Hospital
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Brendan McGuire, MD
Phone
205-934-3411
First Name & Middle Initial & Last Name & Degree
Brendan McGuire, MD
Facility Name
University of California San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94117
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Bruce Wang, MD
First Name & Middle Initial & Last Name & Degree
Bruce Wang, MD
Facility Name
University of Miami Miller School of Medicine
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Cynthia Levy, MD
Phone
305-243-4615
First Name & Middle Initial & Last Name & Degree
Cynthia Levy, MD
Facility Name
Massachusetts General Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Amy Dickey, MD
Phone
206-681-0218
First Name & Middle Initial & Last Name & Degree
Amy Dickey, MD
Facility Name
Mount Sinai Hospital
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Manisha Balwani, MD
Phone
212-241-0915
First Name & Middle Initial & Last Name & Degree
Manisa Balwani, MD
Facility Name
Atrium Health Wake Forest Baptist
City
Winston-Salem
State/Province
North Carolina
ZIP/Postal Code
27157
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Herbert Bonkovsky, MD
Phone
336-713-1442
First Name & Middle Initial & Last Name & Degree
Herbert Bonkovksy, MD
Facility Name
Einstein Medical Center
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19141
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Manish Thapar, MD
First Name & Middle Initial & Last Name & Degree
Manish Thapar
Facility Name
University of Pittsburgh Cancer Pavilion
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15260
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Roy Smith, MD
First Name & Middle Initial & Last Name & Degree
Roy Smith
Facility Name
University of Texas
City
Galveston
State/Province
Texas
ZIP/Postal Code
77550
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Karl Anderson, MD
Phone
409-772-4661
First Name & Middle Initial & Last Name & Degree
Karl Anderson, MD
Facility Name
Fred Hutchinson Cancer Center
City
Seattle
State/Province
Washington
ZIP/Postal Code
98109
Country
United States
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Ryan Donnelly
Phone
206-606-1286
Email
rbd18@seattlecca.org
First Name & Middle Initial & Last Name & Degree
Sioban Keel, MD
Facility Name
Royal Prince Alfred Hospital
City
Camperdown
State/Province
New South Wales
ZIP/Postal Code
2050
Country
Australia
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Peter Stewart
First Name & Middle Initial & Last Name & Degree
Peter Stewart
Facility Name
The Royal Melbourne Hospital
City
Parkville
State/Province
Victoria
ZIP/Postal Code
3050
Country
Australia
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Gayle Ross
Phone
+61 3 93424542
First Name & Middle Initial & Last Name & Degree
Gayle Ross

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP

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