Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

75 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-10-31

Study Completion Date

2024-08-23

Brief Summary

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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.

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Detailed Description

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Conditions

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Erythropoietic Protoporphyria

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

TRIPLE

Participants Investigators Outcome Assessors

Study Groups

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Placebo

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

Oral dose, once a day for 120 days

DISC-1459 oral dose level 1

Group Type EXPERIMENTAL

DISC-1459

Intervention Type DRUG

Oral dose level 1, once a day for 120 days

DISC-1459 oral dose level 2

Group Type EXPERIMENTAL

DISC-1459

Intervention Type DRUG

Oral dose level 2, once a day for 120 days

Interventions

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DISC-1459

Oral dose level 1, once a day for 120 days

Intervention Type DRUG

DISC-1459

Oral dose level 2, once a day for 120 days

Intervention Type DRUG

Placebo

Oral dose, once a day for 120 days

Intervention Type DRUG

Other Intervention Names

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Bitopertin RO4917838 Bitopertin RO4917838

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No