An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)

Part of paid clinical trials in Pittsburgh, Pennsylvania.

Sponsor
Jerry Vockley, MD, PhD
Study ID
NCT07125066
Phase
PHASE1
Status
Enrolling By Invitation

Conditions

  • Combined D,L-2-hydroxyglutaric Aciduria

Eligibility Criteria

Sex
MALE
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Sodium phenylbutyrate — DRUG
    open-label design with doses of sodium phenylbutyrate

Study Details

This is an individual patient research protocol to treat a patient diagnosed with Combined D,L-2-hydroxyglutaric aciduria (C-2HGA) with ACER-001.

Key Dates

Start date
Jul 30, 2025
Status verified
Jun 2026
Primary completion
Jul 31, 2027
Completion
Dec 31, 2027

Study Design

Enrollment
1 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: sodium phenylbutyrate
    a single patient will take sodium phenylbutyrate (ACER-001) current at a dose of 12 g/day; dose will be modified if the patient gains more than 5 kg of weight

Primary Outcome Measure

Number of treatment related adverse events [ Time Frame: 2 years ]

Locations (1)

FacilityCityStateZIPSite coordinators
Children's Hospital of Pittsburgh of UPMCPittsburghPennsylvania15224-

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By research site
Children's Hospital of Pittsburgh of UPMC· Pittsburgh, PA