Phenylketonuria (PKU)Phase 3Prism-2NCT01889862

Phase 3 Study to Evaluate the Efficacy & Safety of Self-Administered Injections of BMN165 by Adults With PKU

A Phase 3 study testing BMN165 20mg/day and BMN165 40mg/day in people with Phenylketonuria (PKU), run by BioMarin Pharmaceutical at 29 sites in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial 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 adults with PKU. Read more about this study

Who is it for?: Ages 18 to 70 years
Conditions the study is for: Phenylketonuria (PKU)
What is being tested?: BMN165 20mg/day and BMN165 40mg/day
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 215 participants
Where: 29 sitesUnited States
Study dates: 29 Jul 2013to 5 Feb 2019
Phase: Phase 3
Are there results?: Yes, posted in the registrySummary in the doctors' view

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 70 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

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
Subjects who are < 18 y/o and are already enrolled into Study 165-301 under Amendment #1 (10JAN2014) may enroll into this study
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

Show all 18Show fewer

Has identified a competent person(s) > 18 y/o who can observe the subject during study drug administration at certain points in the study
A home healthcare nurse may perform the study drug observations
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
Males post vasectomy for 2 years with no known pregnancies do not need to use any other forms of contraception 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 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

Who cannot join

Individuals who meet any of the following exclusion criteria will not be eligible to participate in the study:
Use of any investigational product (except BMN 165) 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 (except BMN 165) intended to treat PKU, including the use of large neutral amino acids, within 2 days prior to the administration of study drug
Have known hypersensitivity to Dextran® or components of Dextran
Use or planned use of any injectable drugs containing PEG (except for BMN 165), including medroxyprogesterone injection, within 3 months prior to screening and during study participation
Current use of levodopa

Show all 16Show fewer

A positive test for HIV antibody, hepatitis B surface antigen, or hepatitis C antibody
A history of organ transplantation or taking chronic immunosuppressive therapy
A history of substance abuse in the past 12 months or current alcohol or drug abuse
Current participation in the Kuvan registry study (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.
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 at least 2x the upper limit of normal
Creatinine at least 1.5x the upper limit of normal

Trial locations and contacts

29 sites in 1 country.

City, postcode or hospital

29 sites in 1 country

United States29 sites

Albany Medical Center
Albany, New York, 12208
Emory Universty, Department of Human Genetics, Division of Medical Genetics
Atlanta, Georgia, 30322
Children's Hospital Colorado
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
Cooper Health Systems
Camden, New Jersey, 08103
Ann and Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
University Hospitals Case Medical Center
Cleveland, Ohio, 44106
University of Missouri Health Center
Columbia, Missouri, 65212
Wayne State University Clinical Research Center at the Mott Center
Detroit, Michigan, 48202
University of Florida Clinical Research Center
Gainesville, Florida, 32610
University of Texas Houston Medical School
Houston, Texas, 77030
Indiana CTSI Clinical Research Center
Indianapolis, Indiana, 46202
University of California, San Diego Clinical and Translational Research Institute
La Jolla, California, 92093
University of Kentucky Medical Center
Lexington, Kentucky, 40536
University of Louisville, Kosair Charities Pediatric Clinical Research Unit
Louisville, Kentucky, 40202
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Atlantic Health System - Morristown Medical Center
Morristown, New Jersey, 07960
Vanderbilt Children's Hospital
Nashville, Tennessee, 37232
Icahn School of Medicine at Mount Sinai Medical Center
New York, New York, 10029
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
University of Nebraska Medical Center
Omaha, Nebraska, 68198
St. Christopher's Hospital for Children
Philadelphia, Pennsylvania, 19134
Children's Hospital of Pittsburgh, University of Pittsburgh Medical Center
Pittsburgh, Pennsylvania, 15224
Oregon Health and Science University
Portland, Oregon, 97239
University of Utah Hospital
Salt Lake City, Utah, 84108
University of Washinton
Seattle, Washington, 98195
Washington University Center for Applied Research Sciences
St Louis, Missouri, 63110
University of South Florida
Tampa, Florida, 33606

Other trials in United States

About this study

Official title: A Four-Part, Phase 3, Randomized, Double-Blind, Placebo- Controlled, Four-Arm, Discontinuation Study to Evaluate the Efficacy and Safety of Subcutaneous Injections of BMN 165 Self-Administered by Adults With Phenylketonuria (PKU)

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.

Read the detailed 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.

Part 1: Open-label period
Part 2: A Randomized, double-blind, placebo-controlled period of 8 weeks
Part 3: Two sub-Parts to Part 3:- Part 3A - BMN 165 administered using vial and syringe during this open label period. Part 3B - BMN 165 administered using prefilled syringe during this open-label period.
Part 4: A long-term, open-label extension period.

Conditions: Phenylketonuria (PKU)
Being tested: BMN165 20mg/day, BMN165 40mg/day, Placebo
Keywords: PKU, PEG-PAL, Prism, BioMarin, rAvPAL-PEG, BMN165
Medical subject headings: Phenylketonurias

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

BMN165 20mg/dayDrug
Also called PEG-PAL
BMN165 20mg/day self-administered daily
BMN165 40mg/dayDrug
Also called PEG-PAL
BMN165 40mg/day self-administered daily
PlaceboDrug
Also called Dextran 40
Non-drug placebo, self-administered daily

Study groups in NCT01889862
Study group	Role	What they receive
BMN165 20mg/day	Active comparator	BMN165 20mg/day self-administered daily Receives: Drug: BMN165 20mg/day
20 mg/day Placebo	Placebo comparator	20 mg/day Placebo self-administered daily Receives: Drug: Placebo
BMN165 40mg/day	Active comparator	BMN165 40mg/day self-administered daily Receives: Drug: BMN165 40mg/day
40 mg/day Placebo	Placebo comparator	40 mg/day Placebo self-administered daily Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2
Blood phe concentration in subjects previously exposed to BMN165 who were administered BMN165 20 or 40 mg/day compared with those who were administered a matching placebo.
Measured: At Part 2 baseline and change from baseline at Part 2 week 8

Secondary outcomes (5)

Change From Baseline in the Cognitive and Mood Symptoms Measured by ADHD Rating Scale (RS)-IV Inattention Subscale at Part 2
Attention deficit hyperactivity disorder rating scale IV (ADHD RS-IV) is investigator-rated, 9-item, inattention subscale score ranges from 0 to 27, with higher scores indicative of a greater degree of impairment and a baseline score >9 indicative of a presence of symptoms.
Measured: At Part 2 baseline and change from baseline at Part 2 week 8
Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 4
The long term effect of multiple dose levels of BMN165 on blood phe concentration in subjects who are administered BMN165 using pre-filled syringes.
Measured: At Part 4 week 57, 105, 161, 209 and 249
Change From Baseline in the Cognitive and Mood Symptoms Measured by PKU POMS TMD at Part 2
Phenylketonuria (PKU) Total Mood Disturbance (TMD) Profile of Mood States (POMS)
PKU POMS TMD is self-rated, each item in the tool is rated using a five-point Likert scale: 0 (not at all), 1 (a little), 2 (moderately), 3 (quite a bit), and 4 (extremely).
Measured: At Part 2 baseline and change from baseline at Part 2 week 8
Change From Baseline in the Cognitive and Mood Symptoms Measured by PKU POMS Confusion Subscale at Part 2
PKU POMS confusion mood domain (referred to as the confusion subscale) is self-rated, each item in the tool is rated using a five-point Likert scale: 0 (not at all), 1 (a little), 2 (moderately), 3 (quite a bit), and 4 (extremely).
Measured: At Part 2 baseline and change from baseline at Part 2 week 8
Change From Baseline in the Cognitive and Mood Symptoms Measured by POMS TMD at Part 2
Full-length POMS TMD is self-rated, each item in the tool is rated using a five-point Likert scale: 0 (not at all), 1 (a little), 2 (moderately), 3 (quite a bit), and 4 (extremely).
Measured: At Part 2 baseline and change from baseline at Part 2 week 8

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

29 Jul 20135 Feb 2019

First posted1 Jul 2013 (estimated)
Study start29 Jul 2013
Primary completion13 Jan 2016
Study completion5 Feb 2019

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Publications (2)Papers the sponsor has linked to this study.
Other trials for Phenylketonuria (PKU)Studies of the same condition, open and under way.
More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind
Who is masked: Participant, Care provider, Investigator
Enrolment: 215 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01889862
Sponsor's protocol ID: 165-302
Acronym: Prism-2
Status last verified: Apr 2021
First submitted: 18 Jun 2013
Last updated: 21 May 2021
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
Individual data sharing: No

Publications

Harding CO, Whitehall KB, Lilienstein J, Sazova O, Lindstrom K, Levy DG, Burton BK. Long-term management strategies for pegvaliase use in phenylketonuria: Lessons learned from the phase-3 PRISM open-label extension study. Genet Med. 2025 Aug;27(8):101459. doi: 10.1016/j.gim.2025.101459. Epub 2025 May 20.
PubMed 40411344
Bilder DA, Arnold GL, Dimmock D, Grant ML, Janzen D, Longo N, Nguyen-Driver M, Jurecki E, Merilainen M, Amato G, Waisbren S. Improved attention linked to sustained phenylalanine reduction in adults with early-treated phenylketonuria. Am J Med Genet A. 2022 Mar;188(3):768-778. doi: 10.1002/ajmg.a.62574. Epub 2021 Nov 26.
PubMed 34826353

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Phenylketonuria (PKU) trials

Study at a glance

From the registry record, last updated by the sponsor on 21 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Four-Part, Phase 3, Randomized, Double-Blind, Placebo- Controlled, Four-Arm, Discontinuation Study to Evaluate the Efficacy and Safety of Subcutaneous Injections of BMN 165 Self-Administered by Adults With Phenylketonuria (PKU)
ClinicalTrials.gov ID: NCT01889862
Acronym / sponsor ID: Prism-2 / 165-302
Conditions: Phenylketonuria (PKU)
Keywords: PKU, PEG-PAL, Prism, BioMarin, rAvPAL-PEG, BMN165
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 215 participants (actual)
Lead sponsor: BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
Study start: 29 Jul 2013 (actual)
Primary completion: 13 Jan 2016 (actual)
Study completion: 5 Feb 2019 (actual)
First posted: 1 Jul 2013 (estimated)
Results: Posted 12 Jul 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

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.

Detailed description

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.

Part 1: Open-label period
Part 2: A Randomized, double-blind, placebo-controlled period of 8 weeks
Part 3: Two sub-Parts to Part 3:- Part 3A - BMN 165 administered using vial and syringe during this open label period. Part 3B - BMN 165 administered using prefilled syringe during this open-label period.
Part 4: A long-term, open-label extension period.

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Triple blind (participant, care provider, investigator)
Number of arms: 4

Arm	Type	Receives
BMN165 20mg/day	Active comparator	Drug: BMN165 20mg/dayBMN165 20mg/day self-administered daily
20 mg/day Placebo	Placebo comparator	Drug: Placebo20 mg/day Placebo self-administered daily
BMN165 40mg/day	Active comparator	Drug: BMN165 40mg/dayBMN165 40mg/day self-administered daily
40 mg/day Placebo	Placebo comparator	Drug: Placebo40 mg/day Placebo self-administered daily

Who may qualify

Ages: 18 to 70 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (18 listed)

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

Key exclusion criteria (16 listed)

Individuals who meet any of the following exclusion criteria will not be eligible to participate in the study:
Use of any investigational product (except BMN 165) 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 (except BMN 165) intended to treat PKU, including the use of large neutral amino acids, within 2 days prior to the administration of study drug
Have known hypersensitivity to Dextran® or components of Dextran
Use or planned use of any injectable drugs containing PEG (except for BMN 165), including medroxyprogesterone injection, within 3 months prior to screening and during study participation

Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.

Interventions

Intervention	Type	Arms
BMN165 20mg/dayAlso: PEG-PALBMN165 20mg/day self-administered daily	Drug	BMN165 20mg/day
BMN165 40mg/dayAlso: PEG-PALBMN165 40mg/day self-administered daily	Drug	BMN165 40mg/day
PlaceboAlso: Dextran 40Non-drug placebo, self-administered daily	Drug	20 mg/day Placebo, 40 mg/day Placebo

Primary outcome

Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2
Time frame: At Part 2 baseline and change from baseline at Part 2 week 8
Blood phe concentration in subjects previously exposed to BMN165 who were administered BMN165 20 or 40 mg/day compared with those who were administered a matching placebo.

5 secondary outcomes

Change From Baseline in the Cognitive and Mood Symptoms Measured by ADHD Rating Scale (RS)-IV Inattention Subscale at Part 2
Time frame: At Part 2 baseline and change from baseline at Part 2 week 8
Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 4
Time frame: At Part 4 week 57, 105, 161, 209 and 249
Change From Baseline in the Cognitive and Mood Symptoms Measured by PKU POMS TMD at Part 2
Time frame: At Part 2 baseline and change from baseline at Part 2 week 8
Change From Baseline in the Cognitive and Mood Symptoms Measured by PKU POMS Confusion Subscale at Part 2
Time frame: At Part 2 baseline and change from baseline at Part 2 week 8
Change From Baseline in the Cognitive and Mood Symptoms Measured by POMS TMD at Part 2
Time frame: At Part 2 baseline and change from baseline at Part 2 week 8

Results and safety

Results posted to the registry on 12 Jul 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
BMN165 20mg/Day	86	74
Placebo 20mg/Day	0	0
BMN165 40mg/Day	78	78
Placebo 40mg/Day	0	0
BMN165	0	0

Primary outcome: Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2

Time frame: At Part 2 baseline and change from baseline at Part 2 week 8. Mean (Standard Deviation). Unit: µmol/L

Measure	BMN165 20mg/Day n = 29	Placebo 20mg/Day n = 14	BMN165 40mg/Day n = 29	Placebo 40mg/Day n = 14
Part 2 Baseline	596.8 (582.75)	563.9 (504.62)	410.9 (439.95)	508.2 (363.68)
Change from Baseline Part 2 Week 8	-65.9 (192.02)	996.4 (555.00)	114.1 (332.40)	599.0 (507.40)

BMN165 20mg/Day vs Placebo 20mg/Day: Mean Difference (Net): -973.02 (95% CI -1204.19 to -741.85), p <0.0001, ANCOVA
BMN165 40mg/Day vs Placebo 40mg/Day: Mean Difference (Net): -588.50 (95% CI -830.07 to -346.94), p <0.0001, ANCOVA

Population analysed: Modified Intent-to-Treat (mITT) population consists of all subjects who reached the randomized dose of 20mg/day or 40mg/day and were randomized into Part 2 with a mean blood Phe reduction of ≥20% (using the last two consecutive blood Phe assessments of Part 1) from the baseline of 165-301 (or the Phase 2 study in which they initiated BMN165).

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to 301 weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Part 1 - 20 mg/Day	6 of 86 (7%)	70 of 86 (81.4%)	0 of 86
Part 1 - 40 mg/Day	2 of 78 (2.6%)	59 of 78 (75.6%)	0 of 78
Part 2 - 20 mg/Day Active	0 of 34 (0%)	27 of 34 (79.4%)	0 of 34
Part 2 - 40 mg/Day Active	2 of 32 (6.3%)	28 of 32 (87.5%)	0 of 32
Part 2 - 20 mg/Day Placebo	1 of 15 (6.7%)	14 of 15 (93.3%)	0 of 15
Part 2 - 40 mg/Day Placebo	0 of 14 (0%)	13 of 14 (92.9%)	0 of 14
Part 3 - Stay on Active 20 mg/Day	1 of 23 (4.3%)	19 of 23 (82.6%)	0 of 23
Part 3 - Stay on Active 40 mg/Day	0 of 18 (0%)	15 of 18 (83.3%)	0 of 18
Part 3 - Switch From Placebo to Active 20 mg/Day	0 of 11 (0%)	8 of 11 (72.7%)	0 of 11
Part 3 - Switch From Placebo to Active 40 mg/Day	0 of 8 (0%)	7 of 8 (87.5%)	0 of 8
Part 4 - < 20 mg/Day	7 of 67 (10.4%)	62 of 67 (92.5%)	0 of 67
Part 4 - 20 to < 40 mg/Day	6 of 152 (3.9%)	105 of 152 (69.1%)	0 of 152
Part 4 - 40 to < 60 mg/Day	16 of 186 (8.6%)	178 of 186 (95.7%)	0 of 186
Part 4 - >= 60 mg/Day	5 of 89 (5.6%)	59 of 89 (66.3%)	0 of 89

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 21 May 2021.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Derived from this study: PubMed 40411344Harding CO, Whitehall KB, Lilienstein J, Sazova O, Lindstrom K, Levy DG, Burton BK. Long-term management strategies for pegvaliase use in phenylketonuria: Lessons learned from the phase-3 PRISM open-label extension study. Genet Med. 2025 Aug;27(8):101459. doi: 10.1016/j.gim.2025.101459. Epub 2025 May 20.
Derived from this study: PubMed 34826353Bilder DA, Arnold GL, Dimmock D, Grant ML, Janzen D, Longo N, Nguyen-Driver M, Jurecki E, Merilainen M, Amato G, Waisbren S. Improved attention linked to sustained phenylalanine reduction in adults with early-treated phenylketonuria. Am J Med Genet A. 2022 Mar;188(3):768-778. doi: 10.1002/ajmg.a.62574. Epub 2021 Nov 26.
BioMarin sponsored PKU educational/community websiteLink provided by the sponsor.
Individual participant dataSharing planned: No

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Referral and contact

AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.

Clinical Trial Specialist study director

BioMarin Pharmaceutical

29 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing BMN165 20mg/day and BMN165 40mg/day in people with Phenylketonuria (PKU), run by BioMarin Pharmaceutical at 29 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 18 inclusion and 16 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 21 May 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 29 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 12 Jul 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Change From Baseline in the Blood Plasma Phenylalanine (Phe) Concentration at Part 2 (time frame: At Part 2 baseline and change from baseline at Part 2 week 8). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01889862, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 21 May 2021.