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The BMN 165 clinical development program has been designed to demonstrate the safety and efficacy of BMN 165 in reducing blood Phe concentrations in adults with PKU.
Full description
165-302 is a Phase 3, 4 Part, Randomized, Double-Blind, Placebo-Controlled, Discontinuation Study to Evaluate the Efficacy and Safety of Self Administered Subcutaneous Injections of BMN 165 by Adults With PKU.
This study is open only to adults who have been exposed to BMN165 in a previous study.
Study BMN 165-302 is a four-Part, Phase 3 study.
Enrollment
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Inclusion and exclusion criteria
INCLUSION CRITERIA
Individuals eligible to participate in this study must meet all of the following criteria:
Have completed a prior BMN 165 study (PAL-003, 165-205, or 165-301) prior to screening
Have had a stable BMN 165 dose regimen for at least 14 days prior to screening
Are at least 18 y/o and no older than 70 y/o at screening
Has identified a person who is > 18 y/o who has the neurocognitive and linguistic capacities to comprehend and complete the Profile of Mood States (POMS)-Observer rated scale
Has identified a competent person(s) > 18 y/o who can observe the subject during study drug administration at certain points in the study
Are willing and able to provide written, signed informed consent after the nature of the study has been explained and prior to any research-related procedures; for minors, parent or guardian provides written consent and assent may be requested
Are willing and able to comply with all study procedures
For females of childbearing potential, a negative pregnancy test at screening and willing to have additional pregnancy tests during the study
If sexually active, willing to use two acceptable methods of contraception during and for 4 weeks after the study
Have received documented approval from a study dietitian confirming that the subject is capable of maintaining their diet
Have neurocognitive and linguistic capacities to comprehend and answer investigator's prompts for the Attention Deficit Hyperactivity Disorder Rating Scale (ADHD-RS) Investigator rated instrument and to complete the POMS-Subject rated scale
If applicable, maintained stable dose of medication for Attention Deficit Hyperactivity Disorder (ADHD), depression, anxiety, or other psychiatric disorder ≥8 weeks prior to enrollment and willing to maintain stable dose throughout study unless a change is medically indicated
General good health, as evidenced by physical examination, clinical laboratory evaluations, and ECG tests at screening
Exclusion Criteria
Individuals who meet any of the following exclusion criteria will not be eligible to participate in the study:
Primary purpose
Allocation
Interventional model
Masking
215 participants in 4 patient groups, including a placebo group
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Data sourced from clinicaltrials.gov
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