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AAV Gene Therapy Study for Subjects with PKU

NCT ID: NCT04480567

Last Updated: 2024-12-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

100 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-09-24

Study Completion Date

2027-12-31

Brief Summary

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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.

Detailed Description

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Conditions

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Phenylketonuria (PKU)

Keywords

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PKU PAH Deficiency

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Dose 1 of BMN 307

Group Type EXPERIMENTAL

BMN 307

Intervention Type DRUG

AAV Gene Therapy Infusion

Dose 2 of BMN 307

Group Type EXPERIMENTAL

BMN 307

Intervention Type DRUG

AAV Gene Therapy Infusion

Dose 3 of BMN 307

Group Type EXPERIMENTAL

BMN 307

Intervention Type DRUG

AAV Gene Therapy Infusion

Interventions

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BMN 307

AAV Gene Therapy Infusion

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

15 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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BioMarin Pharmaceutical

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director, MD

Role: STUDY_DIRECTOR

BioMarin Pharmaceutical

Locations

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University of South Florida

Tampa, Florida, United States

Site Status

Morristown Medical Center

Morristown, New Jersey, United States

Site Status

University Hospital Birmingham NHS Foundation Trust

Birmingham, , United Kingdom

Site Status

Countries

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United States United Kingdom

Other Identifiers

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307-201

Identifier Type: -

Identifier Source: org_study_id