An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01819727
• Date of registration: 18/03/2013
• Prospective Registration: Yes
• Primary sponsor: BioMarin Pharmaceutical
• Public title: An Open-Label Phase 3 Study of BMN 165 for Adults With PKU Not Previously Treated w/ BMN 165 Prism301
• Scientific title: A Phase 3, Open-Label, Randomized, Multi-Center Study to Assess the Safety & Tolerability of an Induction, Titration, and Maintenance Dose Regimen of BMN 165 Self Administered by Adults With PKU Not Previously Treated With BMN 165
• Date of first enrolment: May 2013
• Target sample size: 261
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT01819727
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

United States

Contacts

• Name: Markus Merilainen, MD
• Affiliation: BioMarin Pharmaceutical

Key inclusion & exclusion 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 < 18 years of age but are already enrolled into the study may
continue to participate

- If taking Kuvan, have a treatment end date =14 days prior to Day 1 (ie, first dose of
BMN 165)

- 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

- Are willing and able to comply with all study procedures

- Has identified a person who is = 18 years of age who has the neurocognitive and
linguistic capacities to comprehend and complete the POMS-Observer-rated scale

- Has identified a competent person or persons who are = 18 years of age who can observe
the subject during study drug administration and for a minimum of 1 hour following
administration until dose titration has completed and if needed upon return to dosing
after an AE and per investigator determination.

- A home healthcare nurse may perform the study drug observations.

- For females of childbearing potential, must have a negative pregnancy test at
screening and be willing to have additional pregnancy tests during the study. (Females
are considered not of childbearing potential if they have been in menopause for at
least 2 years, have had a tubal ligation at least 1 year prior to screening, or have
had a total hysterectomy.)

- If sexually active, must be willing to use 2 acceptable methods of contraception while
participating in the study and 4 weeks after the study.

- Males post vasectomy 2 years with no known pregnancies for at least 2 years do
not need to use any other forms of birth control during the study.

- Females who have been in menopause for at least 2 years, have had a tubal
ligation at least 1 year prior to screening, or have had a total hysterectomy do
not need to use any other forms of contraception during the study.

- Have received documented approval from a study dietician confirming that the subject
is capable of maintaining their diet in accordance with dietary information presented
in the protocol.

- Have neurocognitive and linguistic capacities to comprehend and answer investigator's
prompts for the 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 for
=8 weeks prior to enrollment and willing to maintain stable dose throughout study
unless a change is medically indicated.

- Are in generally good health, as evidenced by physical examination, clinical
laboratory evaluations and ECG tests performed at screening

EXCLUSION CRITERIA

Individuals who meet any of the following exclusion criteria will not be eligible to
participate in the study:

- Use of any investigational product or investigational medical device within 30 days
prior to screening or requirement for any investigational agent prior to completion of
all scheduled study assessments.

- Use of any medication that is intended to treat PKU (except Kuvan), including the use
of large neutral amino acids, within 2 days prior to administration of study drug Day
1 (first dose of BMN 165). Note: Kuvan treatment must be stopped =14 days before Day 1

- Use or planned use of any injectable drugs containing PEG (other than BMN 165),
including medroxyprogesterone injection, within 3 months prior to screening and during
study participation

- Known hypersensitivity to any components of BMN 165

- Current use of levodopa

- A positive test for HIV antibody, hepatitis B surface antigen, or hepatitis C antibody

- A history of organ transplantation or on chronic immunosuppressive therapy

- A history of substance abuse (as defined by the Diagnostic and Statistical Manual of
Mental Disorders [DSM IV]) in the past 12 months or current alcohol or drug abuse

- Current participation in the Kuvan registry study (PKU Demographics, Outcomes and
Safety [PKUDOS]). Patients may discontinue the PKUDOS registry trial to allow
enrollment in this study

- Pregnant or breastfeeding at screening or planning to become pregnant (self or
partner) or breastfeed at any time during the study

- Concurrent disease or condition that would interfere with study participation or
safety (eg, history or presence of clinically significant cardiovascular, pulmonary,
hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic,
neurological, oncologic, or psychiatric disease)

- Major surgery planned during the study period

- Any condition that, in the view of the investigator, places the subject at high risk
of poor treatment compliance or terminating early from the study

- Alanine aminotransferase (ALT) concentration =2 times the upper limit of normal

- Creatinine >1.5 times the upper limit of normal.



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Phenylketonuria

Intervention(s)

Drug: BMN 165

Primary Outcome(s)

Number of Participants With Hypersensitivity Adverse Reaction [Time Frame: baseline and 36 weeks]

Secondary Outcome(s)

Blood Phenylalanine Concentration [Time Frame: baseline and 36 weeks]

Secondary ID(s)

165-301

Prism301

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 10/09/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01819727