Phenylketonuria MedDRA version: 20.0 Level: PT Classification code 10034872 Term: Phenylketonuria System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Product Code: BMN 307
Pharmaceutical Form: Solution for infusion
Current Sponsor code: BMN 307
Concentration unit: Other
Concentration type: equal
Concentration number: 6E13-
Sponsors
BioMarin Pharmaceutical Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. age 15 or 18 years and older at Screening 2. Diagnosis of PKU which is a condition characterized by PAH deficiency 3. Average of two plasma Phe levels > 600 µmol/L during the Screening period 4. Ability and willingness to maintain dietary protein intake consistent with baseline intake for the duration of the study unless otherwise directed. Are the trial subjects under 18? yes Number of subjects for this age range: 8 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 100 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria
Exclusion criteria: 1. Subjects with primary BH4 deficiency or other forms of BH4 metabolism deficiency. 2. Clinically significant liver disease as assessed by ultrasound at Screening. 3. Prior treatment with gene therapy. 4. Detectable antibodies to the AAV5 capsid at Screening. 5. Contraindication to use of corticosteroids (CS) or history of condition that could worsen with CS therapy. 6. Hemoglobin A1C = 8.0% or glucose = 250 mg/dL at Screening.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: - Determine safe, effective, and tolerable dose of BMN 307 - Determine the efficacy of a single administration of BMN 307 ;Secondary Objective: - Determine the efficacy of a single administration of BMN 307 on changing plasma Phe level over time - Assess the change in dietary protein intake post-infusion following a single administration of BMN 307 - Assess changes in dietary Phe and protein intake over time following a single administration of BMN 307 ;Primary end point(s): - Change in mean plasma Phe levels ;Timepoint(s) of evaluation of this end point: Week 10 - 12 | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Change in mean plasma Phe levels - Proportion of subjects achieving plasma Phe milestones - Change in dietary protein intake from intact food - Proportion of subjects consuming protein from intact food while maintaining plasma Phe ;Timepoint(s) of evaluation of this end point: Week 22 - 24, Week 24, Week 48, Week 96 | — |
Countries
Australia, Germany, Italy, Spain, Taiwan, Turkey, United Kingdom, United States
Contacts
Public ContactClinical Trials Information
BioMarin Pharmaceutical Inc.
Outcome results
None listed