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

> **NCT07125066** · PHASE1 · ENROLLING_BY_INVITATION · sponsor: **Jerry Vockley, MD, PhD** · enrollment: 1 (estimated)

## Conditions studied

- Combined D,L-2-hydroxyglutaric Aciduria

## Interventions

- **DRUG:** Sodium phenylbutyrate

## Key facts

- **NCT ID:** NCT07125066
- **Lead sponsor:** Jerry Vockley, MD, PhD
- **Sponsor class:** OTHER
- **Phase:** PHASE1
- **Study type:** INTERVENTIONAL
- **Status:** ENROLLING_BY_INVITATION
- **Start date:** 2025-07-30
- **Primary completion:** 2027-07
- **Final completion:** 2027-12
- **Target enrollment:** 1 (ESTIMATED)
- **Last updated:** 2025-08-15

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT07125066

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT07125066, "An Individual Patient, Open Label Study to Use ACER-001 to Treat Combined D,L-2 Hydroxyglutaric Aciduria (C-2HGA)". Retrieved via AI Analytics 2026-06-26 from https://api.ai-analytics.org/clinical/NCT07125066. Licensed CC0.

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*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*