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Gene Therapy Trial Report

Summary

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


NCTID NCT05972629 (View at clinicaltrials.gov)
Description
Development Status Inactive
Indication Phenylketonuria (PKU)
Disease Ontology Term DOID:9281
Compound Name SAR444836
Sponsor Sanofi
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 32 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant PAH
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Functional gene replacement
Route of Administration Intravenous
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV SNY001
Editor Type none
Dose 1 Undisclosed dose 1

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2023-06-23
Completion Date 2030-04-02
Last Update 2025-09-23

Participation Criteria


Eligible Age 18 Years - 65 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
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.
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 11
Locations Argentina,United States,Turkey (Türkiye),Brazil,Israel

Regulatory Information


Has US IND True
FDA Designations
Recent Updates Deprioritized by Sponsor

Resources/Links