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NCT05972629 · ClinicalTrials.gov registry record · Phase 1
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
A Phase 1 study of Phenylketonuria, sponsored by Sanofi.
- Active
- Registry status
- Phase 1
- Development phase
- 32
- Enrollment target
- 11
- Study locations
NCT05972629 is a Phase 1 study of Phenylketonuria that is active but no longer recruiting, run by Sanofi. The registered enrollment target is 32 participants, below the 96-participant average among 12 other Phenylketonuria trials with a reported enrollment target (67% lower). The trial reports 11 study locations across 10 states.
The verdict
NCT05972629, a Phase 1 study of Phenylketonuria, is active but no longer recruiting, sponsored by Sanofi.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 32 participants
- Enrollment target
- 11
- Study locations
Study Summary
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.
Conditions Studied
Interventions
- DRUG SAR444836
Study Locations (11)
Other
- Investigational Site Number : 3760001 - Tel Litwinsky
- Investigational Site Number : 7920001 - Ankara
California
- University of California San Francisco- Site Number : 8400007 - San Francisco
Colorado
- Children's Hospital IMD Clinic- Site Number : 8400015 - Aurora
Florida
- University of Florida-Genetics- Site Number : 8400010 - Gainesville
Ohio
- UHCMC- Site Number : 8400014 - Cleveland
Pennsylvania
- UPMC Children's Hospital of Pittsburgh-4401 Penn Ave- Site Number : 8400018 - Pittsburgh
South Carolina
- Medical University of SC- Site Number : 8400004 - Charleston
Texas
- University of Texas- Site Number : 8400002 - Houston
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 32 participants |
| Start Date | 2023-08-07 |
| Est. Completion | 2030-04-02 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05972629
The ClinicalTrials.gov registry entry for NCT05972629 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 32 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 96-participant average among 12 other Phenylketonuria trials with a reported enrollment target (67% lower). The listed sponsor is Sanofi, which has 713 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Phenylketonuria appearing as the primary indexed condition, and to 1 intervention - of which SAR444836 is the first listed.
NCT05972629 reports 11 study locations spanning 10 distinct geographic areas - top geographies include Other, California, Colorado.
Frequently Asked Questions
What is clinical trial NCT05972629 about?
NCT05972629 is a clinical study titled "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". 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 stu...
What is the current status of trial NCT05972629?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 32 participants. The study started on 2023-08-07. Estimated completion is 2030-04-02.
What conditions does trial NCT05972629 study?
This clinical trial studies the following conditions: Phenylketonuria.
What interventions are being tested in trial NCT05972629?
The interventions under investigation include: SAR444836 (DRUG).
Who is sponsoring clinical trial NCT05972629?
This trial is sponsored by Sanofi, which has 713 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05972629 being conducted?
This trial has 11 study locations across California, Colorado, Florida, Ohio, Pennsylvania. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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