Last updated 10 days ago
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
32 patients around the world
Available in Brazil
Sanofi
1Research sites
32Patients around the world
This study is for people with
Rare diseases
Phenylketonuria
Requirements for the patient
To 65 Years
All Gender
Medical requirements
- Adult males, and females of non-child bearing potential, 18-65 years of age at the time of informed consent.Participants must have uncontrolled classical PKU due to PAH deficiency 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 trial, 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 and in this protocol.
- 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.Any significant underlying liver disease or any of the following documented diagnoses, indicative of significant underlying liver disease.Portal hypertension.Splenomegaly.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.Active or prior hepatitis B virus infection defined as positive test for hepatitis B surface antigen or positive test for hepatitis B core antibody or detectable HBV DNA.Active hepatitis C virus infection defined as positive test for hepatitis C antibody followed by detectable HCV RNA or if a participant is presently receiving anti-viral therapy for hepatitis C.Clinically significant, active bacterial, viral, fungal, or parasitic infection based on Investigator's judgement.
Sites
Hospital de Clínicas de Porto Alegre - HCPA/UFRGS
Recruiting
SponsorSanofi
Study typeInterventional
Conditions
Phenylketonuria
Requirements
To 65 YearsAll Gender
Unique study IDclinicaltrials.gov
NCT05972629
