Status:
ACTIVE_NOT_RECRUITING
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
Lead Sponsor:
Sanofi
Conditions:
Phenylketonuria
Eligibility:
All Genders
18-65 years
Phase:
PHASE1
PHASE2
Brief 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 treatmen...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
August 7 2023
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
April 2 2030
Estimated Enrollment :
32 Patients enrolled
Trial Details
Trial ID
NCT05972629
Start Date
August 7 2023
End Date
April 2 2030
Last Update
September 23 2025
Active Locations (11)
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1
University of California San Francisco- Site Number : 8400007
San Francisco, California, United States, 94143
2
Children's Hospital IMD Clinic- Site Number : 8400015
Aurora, Colorado, United States, 80045
3
University of Florida-Genetics- Site Number : 8400010
Gainesville, Florida, United States, 32610
4
UHCMC- Site Number : 8400014
Cleveland, Ohio, United States, 44106