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NCT06773026 · ClinicalTrials.gov registry record · Phase 2
Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)
A Phase 2 study of Medium-chain Acyl-CoA Dehydrogenase Deficiency, sponsored by Jerry Vockley, MD, PhD.
- Recruiting
- Registry status
- Phase 2
- Development phase
- 24
- Enrollment target
- 1
- Study location
NCT06773026 is a Phase 2 study of Medium-chain Acyl-CoA Dehydrogenase Deficiency that is actively recruiting participants, run by Jerry Vockley, MD, PhD. The registered enrollment target is 24 participants. The trial reports 1 study location across 1 state.
The verdict
NCT06773026, a Phase 2 study of Medium-chain Acyl-CoA Dehydrogenase Deficiency, is actively recruiting participants, sponsored by Jerry Vockley, MD, PhD.
- RECRUITING
- Registry status
- Phase 2
- Development phase
- 24 participants
- Enrollment target
- 1
- Study location
Study Summary
This is a medical research study to test a medication in patients 4 years of age and older with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD) caused by the common ACADM c.985 A\>G (K304E) mutation. The medication is sodium phenylbutyrate (ACER-001), which is currently FDA approved for the treatment of Urea Cyle Disorders. Previous research suggests that sodium phenylbutyrate may also be effective in the treatment MCADD. This study will investigate the safety and efficacy (how well it works) of sodium phenylbutyrate in patients with MCADD.
Conditions Studied
Interventions
- DRUG Sodium phenylbutyrate
Study Locations (1)
Pennsylvania
- UPMC Children's Hospital of Pittsburgh - Pittsburgh
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 24 participants |
| Start Date | 2025-06-30 |
| Est. Completion | 2027-07 |
| Phase | Phase 2 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT06773026
The ClinicalTrials.gov registry entry for NCT06773026 describes a study currently listed as recruiting, categorized as Phase 2. The registered enrollment target is 24 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Jerry Vockley, MD, PhD, which has 2 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Medium-chain Acyl-CoA Dehydrogenase Deficiency appearing as the primary indexed condition, and to 1 intervention - of which Sodium phenylbutyrate is the first listed.
NCT06773026 reports 1 study location spanning 1 distinct geographic area - top geographies include Pennsylvania.
Frequently Asked Questions
What is clinical trial NCT06773026 about?
NCT06773026 is a clinical study titled "Study of Sodium Phenylbutyrate (ACER-001) for the Treatment of Pediatric and Adults Patients With Medium Chain Acyl-CoA Dehydrogenase Deficiency (MCADD)". This is a medical research study to test a medication in patients 4 years of age and older with a disease called medium-chain acyl-CoA dehydrogenase deficiency (MCADD) caused by the common ACADM c.985 A\>G (K304E) mutation. The medication is sodium phenylbutyrate (ACER-001), which is currently FDA a...
What is the current status of trial NCT06773026?
This trial is currently recruiting. It is a Phase 2 study. The enrollment target is 24 participants. The study started on 2025-06-30. Estimated completion is 2027-07.
What conditions does trial NCT06773026 study?
This clinical trial studies the following conditions: Medium-chain Acyl-CoA Dehydrogenase Deficiency.
What interventions are being tested in trial NCT06773026?
The interventions under investigation include: Sodium phenylbutyrate (DRUG).
Who is sponsoring clinical trial NCT06773026?
This trial is sponsored by Jerry Vockley, MD, PhD, which has 2 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT06773026 being conducted?
This trial has 1 study location across Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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