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Clinical Trials in Turkey / NCT05972629
Active, not recruiting Phase 1/2

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 · tracked via the Priya Life Science Turkey tracker
Sponsor
Phase
Phase 1/2
Started
2023-08-07
Last updated
2025-09-23

Condition(s) studied

Phenylketonuria

Investigational drug(s) / intervention(s)

SAR444836

SAR444836: Infusion pump, intravenous infusion (IV)

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.

Eligibility

Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
Inclusion Criteria: * Adult males, and females of non-childbearing potential, 18-65 years of age at the time of informed consent. * Participants must have uncontrolled classical PKU due to PAH deficiency (despite Phe-restricted dietary management or Palynziq) in the judgement of the Investigator. * Two historical plasma Phe values ≥ 600 μmol/L in the preceding 12 months while on Phe restricted diet therapy. Two plasma Phe values ≥ 600 μmol/L drawn at least 72 hours apart during the screening period while on Phe restricted diet therapy in the absence of an acute illness. * Participant has the ability and willingness to maintain their present diet for the duration of the Post-treatment Follow-up Phase (through Week 96), unless otherwise directed as per protocol * Body mass index (BMI) ≤ 35 kg/m2 * Willingness to use effective methods of contraception. * Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol. Exclusion Criteria: * Presence of neutralizing antibodies against the AAV SNY001 capsid * Abnormal liver function laboratory testing evidenced by alanine aminotransferase (ALT)\>1.5X upper limit normal (ULN), aspartate transaminase (AST)\>1.5X ULN, alkaline phosphatase \>1.5X ULN, Total and direct bilirubin \>1.5X ULN (bilirubin levels above the laboratory's normal range are acceptable in individuals with a documented history or laboratory evidence of Gilbert's Disease) * Any significant underlying liver disease or any of the following documented diagnoses, indicative of significant underlying liver disease: * Portal hypertension; or * Splenomegaly; or * Hepatic encephalopathy * Serum albumin measurement below the lower limit of normal of the laboratory OR AST-to-Platelet Ratio Index \> 1.0 * Serum creatinine \>1.5X ULN * Hemoglobin A1c \>6.5% or fasting glucose \>126 mg/dL * Screening laboratory testing demonstrating any of the following: * HIV; or * active or prior hepatitis B virus (HBV) infection defined as positive test for hepatitis B surface antigen (HBsAg) or positive test for hepatitis B core antibody (total HBcAb) or detectable HBV DNA; or * active hepatitis C virus (HCV) infection defined as positive test for hepatitis C antibody followed by detectable HCV RNA or if a participant is presently receiving (or has received within 6 months prior to screening) anti-viral therapy for hepatitis C * Clinically significant, active bacterial, viral, fungal, or parasitic infection (based on Investigator's judgement) The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial.

Primary outcome measure(s)

Trial sites (11)

FacilityCityRegionStatus
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 Israel
Investigational Site Number : 7920001 Ankara Turkey (Türkiye)

More Sanofi trials in Turkey

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05972629 on ClinicalTrials.gov ↗ ← All trials in Turkey