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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: Active Phase 1 study of Phenylketonuria, sponsored by Sanofi.

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. According to ClinicalTrials.gov, the official US trial registry.

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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

Phenylketonuria

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

FieldValue
Enrollment Target 32 participants
Start Date 2023-08-07
Est. Completion 2030-04-02
Phase Phase 1
Sanofi

713 total trials

What the registry record for NCT05972629 still lists

NCT05972629 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 32 participants, a relatively small participant target, below the 96-participant average among 12 other Phenylketonuria trials with a reported enrollment target (67% lower).

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 lists 11 locations in 10 states (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.

Learn More About Clinical Trials

Similar trials for Phenylketonuria

Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.

Where NCT05972629's enrollment target sits among peer trials

32 10th of 12 higher than 3 of 12 other Phenylketonuria trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phenylketonuria trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Related registry views

Source: ClinicalTrials.gov NCT05972629, the US trial registry maintained by the National Library of Medicine. NCT05972629 (small enrollment · multi site footprint · active not recruiting) retrieved and formatted by PlainTrial, see methodology.