A Study of Sepiapterin in Participants With Phenylketonuria (PKU) (NCT06302348)
A Phase 3b Open-Label Study of Long-Term Neurocognitive Outcomes in Children With Phenylketonuria Treated With Sepiapterin
🩺 Plain-English Summary (8th-Grade Level)
The main purpose of this trial is to evaluate the long-term efficacy of sepiapterin on preserving neurocognitive functioning in children with PKU when treatment is initiated in early childhood.
Who can join: Review the eligibility criteria below.
Location: Indiana University School of Medicine — Indianapolis, Indiana
Age range: Not specified to 9 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 (10 Sites)
- Indiana University School of Medicine — Indianapolis, Indiana
- Women and Children Hospital — North Adelaide,
- The Royal Children's Hospital — Parkville,
- Centre Hospitalier Universitaire (CHU) de Lille - Hôpital Jeanne de Flandre — Lille,
- Assistance Publique - Hôpitaux de Paris (AP-HP) - Hôpital Necker-Enfants Malades — Paris,
- Centre Hospitalier Régional Universitaire (CHRU) de Tours - Hôpital Clocheville — Tours,
- Children's Health Ireland (CHI) — Dublin,
- Pomorski Uniwersytet Medyczny w Szczecinie — Szczecin,
- Instytut Matki i Dziecka — Warsaw,
- Birmingham Women's and Children's NHS Foundation Trust — Birmingham,
📞 Contact the Study Coordinator
Phone: (866) 562-4620
Email: medinfo@ptcbio.com
Coordinator details come directly from the registered NIH protocol. Confirm the study is still recruiting before traveling.
💰 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
- 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 — San Francisco, California
- 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.
- Contact the study coordinator using the phone/email above.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.