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This study examines the AAV-based gene therapy called BMN 307 in people with phenylketonuria (PKU). It can enroll male and female patients diagnosed with PKU (a condition caused by PAH deficiency) who have plasma phenylalanine (Phe) levels above 600 µmol/L and can comply with the study requirements. It is being conducted in Phase 1/Phase 2 and is currently not recruiting but still ongoing (active, not recruiting).
The summary above is a plain-language rendering of the official record. The original English title is shown for reference.
Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.
Inclusion Criteria: * Male and female subjects with diagnosis of PKU which is a condition characterized by PAH deficiency * Ability and willingness to maintain dietary protein intake consistent with baseline intake * Willingness to abstain from hepatotoxic substances post-BMN 307 administration * Willingness and capable per investigator opinion to comply with study procedures and requirements * Willingness to use effective methods of contraception * Plasma Phe levels \> 600 µmol/L Exclusion Criteria: * Subjects with primary BH4 deficiency or other forms of BH4 metabolism deficiency * Clinically significant liver dysfunction or disease * Prior treatment with gene therapy * Any condition that, in the opinion of the investigator or Sponsor, would prevent the subject from fully complying with the requirements of the study * History of malignancy