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Trial record 1 of 1 for:    NCT05308472
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Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP

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ClinicalTrials.gov Identifier: NCT05308472
Recruitment Status : Recruiting
First Posted : April 4, 2022
Last Update Posted : March 24, 2023
Sponsor:
Information provided by (Responsible Party):
Disc Medicine, Inc

Study Description
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Brief Summary:
This is a Phase 2, multi-center, double-blind, placebo-controlled, parallel group study of bitopertin to evaluate the safety, tolerability, efficacy, and PPIX concentration change in participants with EPP. Participants may roll over to an open label extension portion after completing the double-blind treatment period.

Condition or disease Intervention/treatment Phase
Erythropoietic Protoporphyria Drug: DISC-1459 Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 75 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: (AURORA) A Randomized, Double-blind, Placebo-Controlled Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and Protoporphyrin IX (PPIX) Concentrations in Participants With Erythropoietic Protoporphyria (EPP)
Actual Study Start Date : October 31, 2022
Estimated Primary Completion Date : November 2023
Estimated Study Completion Date : July 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Placebo Comparator: Placebo Drug: Placebo
Oral dose, once a day for 120 days
Experimental: DISC-1459 oral dose level 1 Drug: DISC-1459
Oral dose level 1, once a day for 120 days
Other Names:
  • Bitopertin
  • RO4917838
Experimental: DISC-1459 oral dose level 2 Drug: DISC-1459
Oral dose level 2, once a day for 120 days
Other Names:
  • Bitopertin
  • RO4917838
Experimental: Open-Label Extension (optional) Drug: DISC-1459
Oral dose level 1, once a day for up to 8 months
Other Names:
  • Bitopertin
  • RO4917838


Outcome Measures
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Primary Outcome Measures :
  1. Percent change from baseline in whole blood metal-free PPIX levels [ Time Frame: 121 days ]

Secondary Outcome Measures :
  1. Total hours of sunlight exposure to skin on days with no pain from 1000 to 1800 hours (10:00am to 6:00pm) [ Time Frame: 121 days ]
  2. Daily sunlight exposure time (minutes) to first prodromal symptom (burning, tingling, itching, or stinging) associated with sunlight exposure between 1 hour post-sunrise and 1 hour pre-sunset [ Time Frame: 121 days ]
  3. Pain intensity of phototoxic reactions according to a Likert scale (0-10) [ Time Frame: 121 days ]
  4. Incidence of treatment-emergent adverse events [ Time Frame: 121 days ]
  5. Erythrocyte total PPIX concentrations [ Time Frame: 121 days ]
  6. Plasma total PPIX concentrations [ Time Frame: 121 days ]
  7. Whole blood total PPIX concentrations [ Time Frame: 121 days ]
  8. Plasma bitopertin concentrations [ Time Frame: 121 days ]

Other Outcome Measures:
  1. Plasma maximum measured drug concentration (Cmax) [ Time Frame: 121 days ]
  2. Time of maximum concentration (Tmax) [ Time Frame: 121 days ]
  3. Area under the concentration-time curve (AUC) [ Time Frame: 121 days ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Aged 18 years or older at the time of signing the informed consent form (ICF).
  2. Diagnosis of EPP, based on medical history by ferrochelatase ( FECH) genotyping or by biochemical porphyrin analysis.
  3. Body weight ≥50 kg.
  4. Washout of at least 2 months prior to Screening of afamelanotide and dersimelagon, if applicable.
  5. Aspartate aminotransferase (AST) and alanine transaminase (ALT) <2× upper limit of normal (ULN) and total bilirubin <ULN (unless documented Gilbert syndrome) at Screening. Albumin >lower limit of normal (LLN).

Exclusion Criteria:

Medical History:

  1. Major surgery within 8 weeks before Screening or incomplete recovery from any previous surgery.
  2. Other than EPP, an inherited or acquired red cell disease associated with anemia.
  3. A history or known allergic reaction to any investigational product excipients or history of anaphylaxis to any food or drug.
  4. History of liver transplantation.
  5. History of alcohol dependence or excessive alcohol consumption, as assessed by the Investigator.
  6. Human immunodeficiency virus (HIV), active Hepatitis B, or C.
  7. Other medical or psychiatric condition or laboratory finding not specifically noted above that, in the judgment of the Investigator or Sponsor, would put the participant at unacceptable risk or otherwise preclude the participant from participating in the study

  8. 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.

    Treatment History:

  9. Concurrent or planned treatment with afamelanotide or dersimelagon during the study period.
  10. Treatment with opioids for any period >7 days in the 2 months prior to screening or anticipated to require opioid use for >7 days at any point during the study.
  11. New treatment for anemia, including initiation of iron supplementation, in the 2 months prior to Screening.

  12. Current or planned use of any drugs or herbal remedies known to be strong inhibitors or inducers of CYP3A4 enzymes for 28 days prior to the first dose and throughout the study.

    Laboratory Exclusions:

  13. Hemoglobin <10 g/dL at Screening.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05308472


Contacts
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Contact: Disc Medicine Clinical Trials (617) 674 9274 clinicaltrials@discmedicine.com

Locations
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United States, Alabama
University of Alabama Hospital Recruiting
Birmingham, Alabama, United States, 35233
Contact: Brendan McGuire, MD    205-934-3411      
Principal Investigator: Brendan McGuire, MD         
United States, California
University of California San Francisco Not yet recruiting
San Francisco, California, United States, 94117
Contact: Bruce Wang, MD         
Principal Investigator: Bruce Wang, MD         
United States, Florida
University of Miami Miller School of Medicine Recruiting
Miami, Florida, United States, 33136
Contact: Cynthia Levy, MD    305-243-4615      
Principal Investigator: Cynthia Levy, MD         
United States, Massachusetts
Massachusetts General Hospital Recruiting
Boston, Massachusetts, United States, 02114
Contact: Amy Dickey, MD    206-681-0218      
Principal Investigator: Amy Dickey, MD         
United States, New York
Mount Sinai Hospital Recruiting
New York, New York, United States, 10029
Contact: Manisha Balwani, MD    212-241-0915      
Principal Investigator: Manisa Balwani, MD         
United States, North Carolina
Atrium Health Wake Forest Baptist Recruiting
Winston-Salem, North Carolina, United States, 27157
Contact: Herbert Bonkovsky, MD    336-713-1442      
Principal Investigator: Herbert Bonkovksy, MD         
United States, Pennsylvania
Einstein Medical Center Recruiting
Philadelphia, Pennsylvania, United States, 19141
Contact: Manish Thapar, MD         
Principal Investigator: Manish Thapar         
University of Pittsburgh Cancer Pavilion Not yet recruiting
Pittsburgh, Pennsylvania, United States, 15260
Contact: Roy Smith, MD         
Principal Investigator: Roy Smith         
United States, Texas
University of Texas Recruiting
Galveston, Texas, United States, 77550
Contact: Karl Anderson, MD    409-772-4661      
Principal Investigator: Karl Anderson, MD         
United States, Washington
Fred Hutchinson Cancer Center Recruiting
Seattle, Washington, United States, 98109
Contact: Ryan Donnelly    206-606-1286    rbd18@seattlecca.org   
Principal Investigator: Sioban Keel, MD         
Sponsors and Collaborators
Disc Medicine, Inc
Investigators
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Study Director: Will Savage, MD PhD Disc Medicine
More Information
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Responsible Party: Disc Medicine, Inc
ClinicalTrials.gov Identifier: NCT05308472    
Other Study ID Numbers: DISC-1459-201
First Posted: April 4, 2022    Key Record Dates
Last Update Posted: March 24, 2023
Last Verified: March 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Disc Medicine, Inc:
EPP
DISC-1459
RO4917838
porphyria
Additional relevant MeSH terms:
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Protoporphyria, Erythropoietic
Porphyrias, Hepatic
Liver Diseases
Digestive System Diseases
Skin Diseases, Genetic
 Genetic Diseases, Inborn
Skin Diseases
Porphyrias
Metabolic Diseases