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
Active, not recruiting · Phase 1, Phase 2 · NCT05972629
Conditions: Phenylketonuria
At a glance
- Research payments reported, 2025
- $206,065
- U.S. investigators named
- 5
- Sites paid
- 5
- Planned or actual enrollment
- 32
About the trial
- Sponsor
- Sanofi
- Tested
- SAR444836 (drug)
- Dates
- Started 2023-08-07; completion 2030-04-02
- Results posted
- Not yet
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 dos…
From ClinicalTrials.gov, last updated 2025-09-23. Full record, eligibility and contacts (opens in a new tab)
Industry funding
Genzyme Corporation reported $206,065 in research payments for this study in 2025 (11 payments).
Research payments usually go to hospitals, universities and research sites to run a study, not to the investigators personally. A payment is not evidence of wrongdoing.
U.S. investigators named on this study
- Irene J. Chang, M.D. Medical Genetics · San Francisco, CA
- Janet Thomas, MD Medical Genetics · Aurora, CO
- Laura L. Konczal, M.D. Medical Genetics · Cleveland, OH
- Gerard Vockley, MD, PHD Specialist · Pittsburgh, PA
- Neena L. Champaigne, M.D. Medical Genetics · Charleston, SC
Clinicians the company named as principal investigators in its Open Payments reports.
Sites paid to run it
- UCSF Langley Porter Psychiatric Hospital and Clinics, CA $75,659
- Medical University of South Carolina $49,625
- University of Pittsburgh $33,266
- Uh Cleveland Medical Center, OH $31,400
- Children's Hospital Colorado, CO $16,115
Sources: CMS Open Payments research payments (program year 2025) and ClinicalTrials.gov, U.S. National Library of Medicine.