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



Status:Active, not recruiting
Conditions:Endocrine
Therapuetic Areas:Endocrinology
Healthy:No
Age Range:18 - 70
Updated:12/29/2017
Start Date:July 29, 2013
End Date:March 31, 2019

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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.

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: PK (plasma BMN 165) and PD (plasma Phe) assessment.

- Part 4: A long-term, open-label extension study.

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 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 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

- 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 ADHD-RS Investigator rated instrument and to complete the POMS-Subject
rated scale

- If applicable, maintained stable dose of medication for 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:

- 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

- 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

- ALT concentration at least 2x the upper limit of normal

- Creatinine at least 1.5x the upper limit of normal
We found this trial at
28
sites
9000 W Wisconsin Ave #270
Milwaukee, Wisconsin 53226
(414) 266-2000
Children's Hospital of Wisconsin Nothing matters more than our children. At Children's Hospital of Wisconsin,...
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5050 Anthony Wayne Dr
Detroit, Michigan 48201
(313) 577-2424
Wayne State University Founded in 1868, Wayne State University is a nationally recognized metropolitan research...
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Gainesville, Florida 32610
(352) 392-3261
University of Florida The University of Florida (UF) is a major, public, comprehensive, land-grant, research...
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Houston, TX
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1211 Medical Center Dr
Nashville, Tennessee 37232
(615) 322-5000
Vanderbilt Univ Med Ctr Vanderbilt University Medical Center (VUMC) is a comprehensive healthcare facility dedicated...
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1 Gustave L Levy Pl # 271
New York, New York 10029
 (212) 241-6500
Mount Sinai Med Ctr Founded in 1852, The Mount Sinai Hospital is a 1,171-bed, tertiary-care...
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Philadelphia, Pennsylvania 19134
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
503 494-8311
Oregon Health and Science University In 1887, the inaugural class of the University of Oregon...
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60 Crittenden Blvd # 70
Rochester, New York 14642
(585) 275-2121
University of Rochester The University of Rochester is one of the country's top-tier research universities....
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Rochester, NY
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4202 E Fowler Ave
Tampa, Florida 33620
(813) 974-2011
University of South Florida The University of South Florida is a high-impact, global research university...
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Albany, New York 12208
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Aurora, Colorado 80045
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Boston, Massachusetts 02115
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Camden, New Jersey 08103
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Chicago, IL
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Columbia, Missouri 65211
(573) 882-2121
University of Missouri T he University of Missouri was founded in 1839 in Columbia, Mo.,...
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Decatur, Georgia 30033
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Indianapolis, Indiana 46202
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Lexington, Kentucky
859) 257-9000
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Morristown, NJ
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Oakland, California 94609
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1100 N. Lindsay
Oklahoma City, Oklahoma 73104
(405) 271-4000
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4242 Dewey Ave
Omaha, Nebraska 68105
(800) 922-0000
Nebraska Medical Center Formed in 1997 by combining the operations of University Hospital, Bishop Clarkson...
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200 Lothrop St
Pittsburgh, Pennsylvania 15213
University of Pittsburgh Medical Center UPMC is one of the leading nonprofit health systems in...
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660 South Euclid Avenue
Saint Louis, Missouri 63110
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Salt Lake City, Utah 84132
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