AAV Gene Therapy Study for Subjects With PKU

  • STATUS
    Recruiting
  • End date
    Dec 5, 2026
  • participants needed
    100
  • sponsor
    BioMarin Pharmaceutical
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Updated on 19 February 2024
See if I qualify
deficiency
gene therapy

Summary

This is a Phase 1/2, open-label, dose escalation study to evaluate the safety, efficacy and tolerability of BMN 307 in adult PKU subjects with PAH deficiency. Participants will receive a single administration of BMN 307 and will be followed for safety and efficacy.

Details
Condition Phenylketonuria, Phenylketonuria
Age 15years - 100years
Treatment BMN 307
Clinical Study IdentifierNCT04480567
SponsorBioMarin Pharmaceutical
Last Modified on19 February 2024

Eligibility

 Yes No Not Sure

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 mol/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
Clear my responses
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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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