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

NCT07125066 · clinicaltrials.gov ↗

PHASE1

Phase

ENROLLING_BY_INVITATION

Status

1

Enrollment

OTHER

Sponsor class

Conditions

Combined D,L-2-hydroxyglutaric Aciduria

Interventions

DRUG: Sodium phenylbutyrate

Sponsor

Jerry Vockley, MD, PhD

Collaborators

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