Enrolling by invitationPhase 1NCT07125066

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

Studying 2-hydroxyglutaric aciduria

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Key facts

Sponsor
Jerry Vockley, MD, PhD
Principal Investigator
Gerard Vockley, MD, PhD, MD, PhD
UPMC Children's Hospital of Pittsburgh
Intervention
Sodium phenylbutyrate(drug)
Enrollment
1 enrolled
Eligibility
MALE
Timeline
20252027

Study locations (1)

Collaborators

Zevra Therapeutics

Primary source

Recruitment status, site addresses, contacts, and full eligibility criteria can change between syncs. Always verify with the trial team before planning travel or treatment.

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