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AAV Gene Therapy Study for Subjects with PKU

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Trial Status Active Not Recruiting

Trial Identifier

NCT04480567

Condition

Phenylketonuria, Phenylketonuria (PKU)

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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.

Eligibility Criteria

Birth Sex

All

Age

From 15 Years

Healthy Volunteers

No

Drug/Treatment:

BMN 307

Phase:

Phase 1/Phase 2

Study Type:

Interventional

Number of Participants:

100

Study Started:

2020-09-24

Study Updated:

2024-12-12

Trial Locations

  • University of South Florida

    Tampa, Florida, United States

  • Morristown Medical Center

    Morristown, New Jersey, United States

  • University Hospital Birmingham NHS Foundation Trust

    Birmingham, , United Kingdom

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 malignanc

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