Gene Therapy for Phenylketonuria

Phase 1 & 2

Recruiting

Research Sponsored by Sanofi

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Two historical plasma Phe values ≥ 600 μmol/L in the preceding 12 months while on Phe restricted diet therapy

Must not have

Screening laboratory testing demonstrating any of the following: HIV; 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; 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)

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

"This trial is testing a new gene therapy called SAR444836 for adults with phenylketonuria (PKU). The goal is to see if SAR444836 can reduce phenylalanine levels

Who is the study for?

Adults aged 18-65 with Phenylketonuria (PKU) due to PAH deficiency, who have been on a stable diet restricting phenylalanine. Participants must have had high plasma Phe levels in the past while on this diet and be willing to maintain their current diet for the trial's duration. They should not be able to bear children, have a BMI ≤ 35 kg/m2, and agree to use contraception.

What is being tested?

The study tests SAR444836, a gene therapy aiming to treat PKU by introducing a functional version of the PAH enzyme via an adeno-associated virus vector. It involves one IV administration followed by monitoring over approximately two years with up to 41 visits which may include remote assessments.

What are the potential side effects?

While specific side effects are not listed here, participants will be closely monitored for any adverse events following the intravenous administration of SAR444836. This includes tracking organ function and overall health throughout the study.