A Phase 1/Phase 2 Open-label Study to Evaluate the Safety, Tolerability, and Efficacy of a Single Intravenous Administration of SAR444836 in Adult Participants With Phenylketonuria (NCT05972629)
A Phase 1/Phase 2, Open-label, Dose-escalation, and Dose Expansion Study to Evaluate the Safety, Tolerability, and Efficacy of SAR444836, an Adeno-associated Viral Vector-mediated Gene Transfer of Human Phenylalanine Hydroxylase, in Adult Participants With Phenylketonuria
🩺 Plain-English Summary (8th-Grade Level)
This is a single group Phase 1/Phase 2, 1-arm, open-label study with SAR444836, an adeno-associated virus (AAV) vector-mediated gene transfer of human phenylalanine hydroxylase (PAH), for the treatment of adult participants with phenylketonuria (PKU) on a chronic, stable diet. The purpose of the study is to evaluate the safety and efficacy of SAR444836 in reducing phenylalanine (Phe) levels and in the elimination of a Phe restricted diet. Participants will receive a one-time intravenous (IV) administration of SAR444836. The study is constituted of 2 separate parts: a dose escalation part, and a dose expansion part where subsequent participants will be administered a safe and effective dose level identified during the dose escalation part. In both study parts, clinical and laboratory assessments will be collected to: a) assess the incidence of adverse events, and b) evaluate the effect of SAR444836 on reductions in blood Phe levels and maintenance of these Phe levels after elimination of a Phe restricted diet. The study duration will be for a minimum duration of 5 years following SAR444836 administration, for each participant and includes a 6-week screening phase, a 96-week treatment follow-up period after SAR444836 administration, followed by an additional 3 years of long-term safety (and efficacy) monitoring. There will be a total of 47 study visits. Many study visits may occur as remote visits and be performed by a qualified in-home service provider. Actual study duration for an individual participant may be longer than 5 years due to the administration of SAR444836 to participants in Stage 1A in a serial fashion, the duration of the screening period, and/or other factors such as delays in scheduling study visits.
Who can join: Review the eligibility criteria below.
Location: University of California San Francisco- Site Number : 8400007 — San Francisco, California
Age range: 18 Years to 65 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (11 Sites)
- University of California San Francisco- Site Number : 8400007 — San Francisco, California
- Children's Hospital IMD Clinic- Site Number : 8400015 — Aurora, Colorado
- University of Florida-Genetics- Site Number : 8400010 — Gainesville, Florida
- UHCMC- Site Number : 8400014 — Cleveland, Ohio
- UPMC Children's Hospital of Pittsburgh-4401 Penn Ave- Site Number : 8400018 — Pittsburgh, Pennsylvania
- Medical University of SC- Site Number : 8400004 — Charleston, South Carolina
- University of Texas- Site Number : 8400002 — Houston, Texas
- Investigational Site Number : 0320002 — Ciudad Autonoma Buenos Aires, Buenos Aires
- Hospital de Clinicas de Porto Alegre - HCPA- Site Number : 0760001 — Porto Alegre, Rio Grande do Sul
- Investigational Site Number : 3760001 — Tel Litwinsky,
- Investigational Site Number : 7920001 — Ankara,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Phenylketonuria
- A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria — Aurora, Colorado
- Rapid Drug Desensitization Study in Adults Experiencing Hypersensitivity Reactions to Palynziq — Chevy Chase, Maryland
- Immune Modulation During Palynziq® Treatment in Adults (IMPALA) — Los Angeles, California
- A Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AG-181 in Subjects With Phenylketonuria — Indianapolis, Indiana
- A Study of JNT-517 in Participants With Phenylketonuria (PKU) — Los Angeles, California
Explore by condition: All Phenylketonuria clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.