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Bitopertin for EPP

Q: Are participants still being accepted for the current trial?

<p>Evidently, the trial posted on June 1st 2023 is no longer recruiting patients. This information was last updated on May 22nd 2023 and there are currently 6 other trials that require enrolment.</p>

Q: How many health centers in the state are holding this research investigation?

<p>At the moment, 12 sites are recruiting participants for this medical trial. These locations include San Francisco, Miami and Boston with 9 other clinical centres dispersed around the country. Given that travelling for trials could be considerable, you should consider joining a local clinic to reduce any travel time or costs involved.</p>

Phase 2 & 3

Waitlist Available

Research Sponsored by Disc Medicine, Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Body weight ≥32 kg for participants <18 years of age and BMI ≥18.5 kg/m2 for adult participants

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

Study Summary

This trial will look at how safe & effective a drug is in treating EPP, a rare genetic disorder.

Who is the study for?

This trial is for individuals aged ≥12 years with a confirmed diagnosis of Erythropoietic Protoporphyria (EPP) who have previously participated in a bitopertin study. Adults must have a BMI ≥18.5 kg/m2, and minors must weigh at least 32 kg. Exclusions include ongoing severe side effects from previous studies related to bitopertin, certain medical or psychiatric conditions, pregnancy, breastfeeding, recent participation in other clinical trials, and use of specific drugs.Check my eligibility

What is being tested?

The trial is testing the long-term safety and effectiveness of DISC-1459 (Bitopertin) for treating EPP. It's an open-label extension study which means everyone knows they are receiving Bitopertin without any placebo control.See study design

What are the potential side effects?

While not explicitly listed here, potential side effects may include those commonly associated with medications affecting blood properties since Bitopertin is being tested for EPP—a condition that affects blood enzymes.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I weigh at least 32 kg if under 18, or have a BMI of at least 18.5 if adult.

Select...

I have EPP and have completed a prior Disc Medicine bitopertin study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Assessment of C-SSRS

Assessment of Patient Health Questionnaire (PHQ-8)

Incidence of abnormal laboratory test results

+3 more

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

Trial Design

2Treatment groups

Experimental Treatment

Group I: DISC-1459 Oral Dose Level 2Experimental Treatment1 Intervention

Oral dose, once a day

Group II: DISC-1459 Oral Dose Level 1Experimental Treatment1 Intervention

Oral dose, once a day

0 / 2Eligibility criteria met

Find a Location

Who is running the clinical trial?

Disc Medicine, IncLead Sponsor

5 Previous Clinical Trials

269 Total Patients Enrolled

Will Savage, MD PhDStudy DirectorDisc Medicine

4 Previous Clinical Trials

227 Total Patients Enrolled

Media Library

DISC-1459 (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05883748 — Phase 2 & 3

Erythropoietic Protoporphyria Research Study Groups: DISC-1459 Oral Dose Level 1, DISC-1459 Oral Dose Level 2

Erythropoietic Protoporphyria Clinical Trial 2023: DISC-1459 Highlights & Side Effects. Trial Name: NCT05883748 — Phase 2 & 3

DISC-1459 (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05883748 — Phase 2 & 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are participants still being accepted for the current trial?

"Evidently, the trial posted on June 1st 2023 is no longer recruiting patients. This information was last updated on May 22nd 2023 and there are currently 6 other trials that require enrolment."

Answered by AI

How many health centers in the state are holding this research investigation?

"At the moment, 12 sites are recruiting participants for this medical trial. These locations include San Francisco, Miami and Boston with 9 other clinical centres dispersed around the country. Given that travelling for trials could be considerable, you should consider joining a local clinic to reduce any travel time or costs involved."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2023. "Open-Label, Long-Term Extension Study to Investigate the Safety, Tolerability, and Efficacy of DISC-1459 (Bitopertin) in Participants With EPP". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05883748.