Phase 3
N=261
An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165
Phenylketonuria
Bottom Line
View on ClinicalTrials.gov: NCT01819727 ↗Enrolled (actual)
261
Serious AEs
10.0%
Results posted
Sep 2018
Primary outcome: Primary: Number of Participants With Hypersensitivity Adverse Reaction — 111; 119 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- BMN 165 (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- BioMarin Pharmaceutical
- Primary completion
- Nov 2015
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Hypersensitivity Adverse Reaction |
111; 119 | — |
| SECONDARY Blood Phenylalanine Concentration |
1241.0; 1224.4; 868.4; 624.4 | — |
Summary
The BMN 165 clinical development program has been designed to demonstrate the safety and efficacy of BMN 165 in reducing blood Phe concentrations in patients 18 to 70 years old with hyperphenylalaninemia due to PKU. Study BMN 165-301 is a Phase 3, open-label, randomized study designed to further characterize the safety of BMN 165 during two induction, titration, and maintenance dose regimens in adults with PKU who have not had previous exposure to BMN 165 (naive). Subjects will be randomized (1:1) to titrate up to one of two dose regimens. Other key features of this study are the dose regimens chosen for induction and titration; the study duration; self administration of study drug; and the chosen tertiary objectives.
Eligibility Criteria
INCLUSION CRITERIA
Individuals eligible to participate in this study must meet all of the following criteria:
- A current diagnosis of PKU with the following:
- Current blood Phe concentration >600 µmol/L at screening and
- Average blood Phe concentration of >600 µmol/L over the past 6 months (per available data)
- Have no previous exposure to BMN 165
- Are ≥18 and ≤70 years of age at the time of screening
- Subjects who are 1.5 times the upper limit of normal.
Data sourced from ClinicalTrials.gov (NCT01819727). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.