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DISC-1459 for Erythropoietic Protoporphyria

Phase 2

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

Be older than 18 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 121 days

Awards & highlights

Study Summary

This trialstudies a new drug to treat EPP. It's double-blind, meaning neither patient nor doctor knows if they get drug or placebo. Results used to evaluate safety, effectiveness, and changes in bodily substance.

Who is the study for?

Adults over 18 with Erythropoietic Protoporphyria (EPP), weighing at least 50 kg, and normal albumin levels can join this trial. They must not have used certain medications recently and should have stable liver function tests. People with severe allergies, recent major surgery, psychiatric conditions requiring hospitalization within six months, or those using strong CYP3A4 enzyme affecting drugs cannot participate.Check my eligibility

What is being tested?

The study is testing Bitopertin's safety and effectiveness in treating EPP compared to a placebo. Participants will be randomly assigned to receive either the drug or placebo in a double-blind setup where neither they nor the researchers know who gets what until after the results are collected.See study design

What are the potential side effects?

While specific side effects for Bitopertin are not listed here, common ones may include reactions at the injection site, gastrointestinal issues like nausea or diarrhea, potential liver enzyme changes, fatigue, headache or allergic reactions.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 121 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~121 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and 121 days for reporting.

Treatment Details

Study Objectives

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

Primary outcome measures

Beryllium

Secondary outcome measures

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

Erythrocyte total PPIX concentrations

Incidence of treatment-emergent adverse events

+5 more

Other outcome measures

Area under the concentration-time curve (AUC)

Plasma maximum measured drug concentration (Cmax)

Time of maximum concentration (Tmax)

Trial Design

4Treatment groups

Experimental Treatment

Placebo Group

Group I: Open-Label Extension (optional)Experimental Treatment1 Intervention

Group II: DISC-1459 oral dose level 2Experimental Treatment1 Intervention

Group III: DISC-1459 oral dose level 1Experimental Treatment1 Intervention

Group IV: PlaceboPlacebo Group1 Intervention

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Disc Medicine, IncLead Sponsor

5 Previous Clinical Trials

394 Total Patients Enrolled

Will Savage, MD PhDStudy DirectorDisc Medicine

4 Previous Clinical Trials

352 Total Patients Enrolled

Media Library

Bitopertin (Other) Clinical Trial Eligibility Overview. Trial Name: NCT05308472 — Phase 2

Erythropoietic Protoporphyria Research Study Groups: DISC-1459 oral dose level 2, Open-Label Extension (optional), Placebo, DISC-1459 oral dose level 1

Erythropoietic Protoporphyria Clinical Trial 2023: Bitopertin Highlights & Side Effects. Trial Name: NCT05308472 — Phase 2

Bitopertin (Other) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05308472 — Phase 2

Frequently Asked Questions

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

How many participants has this trial been limited to?

"Affirmative. According to clinicaltrials.gov, recruitment for this experiment is still ongoing as of November 18th 2022; the research was first posted on October 31st 2020 and requires 75 participants across 7 centres."

Answered by AI

Does this research program still accept volunteers?

"Affirmative, according to the clinicaltrials.gov website, this medical study is actively recruiting participants that meet inclusion criteria. The trial was issued on October 31st of 2022 and most recently updated November 18th of 2022. Ultimately, 75 people need to be enrolled across seven different sites for completion of the trial."

Answered by AI

Does the FDA sanction Open-Label Extension as an optional procedure?

"Our team at Power have assigned a score of 2 to Open-Label Extension (optional) due to the available evidence that suggests it is safe, yet there is no proof demonstrating its efficacy."

Answered by AI

How many sites are presently executing this clinical investigation in the state?

"Currently, 7 trial sites are enlisting participants with locations in Miami, Boston, and Winston-Salem plus other nearby towns. To reduce your need to travel when participating it is best to choose the site located closest to you."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2022. "Study of Bitopertin to Evaluate the Safety, Tolerability, Efficacy, and PPIX Concentrations in Participants With EPP". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT05308472.