A Phase 2 Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of PF-06252616 in Duchenne Muscular Dystrophy



Status:Completed
Conditions:Neurology, Orthopedic
Therapuetic Areas:Neurology, Orthopedics / Podiatry
Healthy:No
Age Range:6 - 15
Updated:1/6/2019
Start Date:November 24, 2014
End Date:November 23, 2018

Use our guide to learn which trials are right for you!

A PHASE 2 RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTIPLE ASCENDING DOSE STUDY TO EVALUATE THE SAFETY, EFFICACY, PHARMACOKINETICS AND PHARMACODYNAMICS OF PF-06252616 IN AMBULATORY BOYS WITH DUCHENNE MUSCULAR DYSTROPHY

This is a Phase 2 randomized, 2-period, double-blind, placebo-controlled, multiple ascending
dose study to evaluate the safety, efficacy, PK and PD of PF-06252616 administered to
ambulatory boys diagnosed with Duchenne Muscular Dystrophy. Three intravenous (IV) dose
levels will be investigated in a within subject dose escalating fashion. Subjects will be
randomly assigned to 1 of 3 sequence groups for approximately 96 weeks (2 periods of 48 weeks
each). In period 1, two of the sequence groups will receive PF-06252616 and one sequence
group will receive placebo. In period 2, the placebo group will switch to PF-06252616 and the
two remaining sequence groups will either receive placebo or PF-06252616. Efficacy will be
based on an observed mean change from baseline on function (4 stair climb) of PF-06252616 as
compared to the placebo at the end of period 1. Period 2 provides an opportunity to evaluate
PK. Subjects will receive monthly IV infused doses of either PF-06252616 or placebo and will
undergo safety evaluations (Laboratory, cardiac monitoring, physical exams, x-ray, MRI),
functional evaluations (pulmonary function testing, 4 stair climb, range of motion, strength
testing, Northstar Ambulatory Assessment, upper limb functional testing and the six minute
walk test), pharmacokinetic testing and pharmacodynamic testing to evaluate changes in muscle
volume (MRI).


Inclusion Criteria:

1. Ambulatory boys age 6 to <16 years old (at the time of randomization), diagnosed with
DMD. Diagnosis must be confirmed in subject's medical history and by genetic testing
obtained during routine clinical care for diagnostic purposes as reported from an
appropriate regulated laboratory using a clinically validated genetic test (genetic
testing is not provided by the sponsor).

2. Subjects who are able to perform the 4 stair climb in > or = 0.33 but < or =1.6
stairs/second.

3. Subjects must be receiving glucocorticosteroids for a minimum of 6 months prior to
signing informed consent. There should be no significant change (>0.2 mg/kg) in dosage
or dose regimen (not related to body weight change) for at least 3 months immediately
prior to signing the informed consent and a reasonable expectation that dosage and
dosing regimen will not change significantly for the duration of the study.

4. Adequate hepatic and renal function on screening laboratory assessments.

5. No underlying disposition for iron accumulation on screening laboratory assessments.

6. Iron content estimate on the screening liver MRI is within the normal range.

Exclusion Criteria:

1. Subjects with known cognitive impairment or behavioral issues that would impede the
ability to follow instructions.

2. History of major surgical procedure within 6 weeks of signing the informed consent or
planned surgery during the study.

3. Any injury which may impact functional testing. Previous injuries must be fully healed
prior to consenting. Prior lower limb fractures must be fully healed and at least 3
months from injury date.

4. Presence or history of other musculoskeletal or neurologic disease or somatic disorder
not related to DMD including pulmonary and cardiac disease.

5. Compromised cardiac function (left ventricular ejection fraction <55% as determined on
a screening cardiac MRI or echocardiogram). Subjects may be receiving ACE (angiotensin
converting enzyme) inhibitors or beta blockers, ARB (angiotensin II receptor
antagonist) or aldosterone blocker/thiazide diuretic; however they must have initiated
treatment more than 3 months prior to screening to ensure stable therapy.

6. Evidence or history of clinically significant hematological, renal, endocrine,
pulmonary, gastrointestinal, cardiovascular (including uncontrolled hypertension),
hepatic, neurologic, or allergic disease (including drug allergies, but excluding
untreated, asymptomatic, seasonal allergies at time of dosing).

7. Documented history of iron overload including hemochromatosis, beta thalassemia major,
beta thalassemia intermedia or hemolytic anemia.

8. Unwilling or unable (eg, metal implants, requires sedation) to undergo examination
with closed MRI without sedation.

9. Participation in other studies involving investigational drug(s) for a minimum of 30
days or within 5 half lives (whichever is longer) prior to signing the informed
consent and/or during study participation.

10. Current or prior treatment with anti-myostatin, exon skipping, nonsense mutation
targeted therapies ever or more than 30 days of treatment with utrophin modifiers and
treatment with utrophin modifiers within 30 days prior ot signing the informed consent
and/or during study participation.

11. Current or prior treatment within the past 3 months with androgens or human growth
hormone.

12. Current treatment with immunosuppressant therapies (other than glucocorticoid
steroids), aminoglycosides (eg, gentamicin), multi vitamins with iron and iron
supplements and other investigational therapies (including idebenone).
We found this trial at
31
sites
Salt Lake City, Utah 84132
?
mi
from
Salt Lake City, UT
Click here to add this to my saved trials
707 North Broadway
Baltimore, Maryland 21205
443-923-9200
Kennedy Krieger Institute While not officially part of Johns Hopkins Medicine, Kennedy Krieger Institute is...
?
mi
from
Baltimore, MD
Click here to add this to my saved trials
185 Cambridge Street
Boston, Massachusetts 02114
617-724-5200
?
mi
from
Boston, MA
Click here to add this to my saved trials
3333 Burnet Avenue # Mlc3008
Cincinnati, Ohio 45229
 1-513-636-4200 
Cincinnati Children's Hospital Medical Center Patients and families from across the region and around the...
?
mi
from
Cincinnati, OH
Click here to add this to my saved trials
Durham, North Carolina 27710
(919) 684-8111
Duke University Younger than most other prestigious U.S. research universities, Duke University consistently ranks among...
?
mi
from
Durham, NC
Click here to add this to my saved trials
South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
?
mi
from
Philadelphia, PA
Click here to add this to my saved trials
4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
?
mi
from
Pittsburgh, PA
Click here to add this to my saved trials
13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
?
mi
from
Aurora, CO
Click here to add this to my saved trials
1800 Orleans St.
Baltimore, Maryland 21287
410-955-5000
Johns Hopkins Hospital Patients are the focus of everything we do at The Johns Hopkins...
?
mi
from
Baltimore, MD
Click here to add this to my saved trials
Baltimore, Maryland 21287
?
mi
from
Baltimore, MD
Click here to add this to my saved trials
?
mi
from
Baltimore, MD
Click here to add this to my saved trials
225 E Chicago Ave
Chicago, Illinois 60611
(312) 227-4000
Ann & Robert H. Lurie Children's Hospital of Chicago Ann & Robert H. Lurie Children
?
mi
from
Chicago, IL
Click here to add this to my saved trials
3000 Erwin Road
Durham, North Carolina 27705
?
mi
from
Durham, NC
Click here to add this to my saved trials
?
mi
from
Fairway, KS
Click here to add this to my saved trials
?
mi
from
Fairway, KS
Click here to add this to my saved trials
Iowa City, Iowa 52242
?
mi
from
Iowa City, IA
Click here to add this to my saved trials
?
mi
from
Kansas City, KS
Click here to add this to my saved trials
3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
?
mi
from
Kansas City, KS
Click here to add this to my saved trials
Los Angeles, California 90024
?
mi
from
Los Angeles, CA
Click here to add this to my saved trials
Los Angeles, California 90095
?
mi
from
Los Angeles, CA
Click here to add this to my saved trials
Los Angeles, California 90095
?
mi
from
Los Angeles, CA
Click here to add this to my saved trials
2450 Riverside Avenue
Minneapolis, Minnesota 55454
?
mi
from
Minneapolis, MN
Click here to add this to my saved trials
2315 Stockton Blvd.
Sacramento, California 95817
(916) 734-2011
University of California, Davis Medical Center UC Davis Medical Center serves a 65,000-square-mile area that...
?
mi
from
Sacramento, CA
Click here to add this to my saved trials
Saint Louis, Missouri 63110
?
mi
from
Saint Louis, MO
Click here to add this to my saved trials
?
mi
from
Salt Lake City, UT
Click here to add this to my saved trials
?
mi
from
Salt Lake City, UT
Click here to add this to my saved trials
Salt Lake City, Utah 84112
?
mi
from
Salt Lake City, UT
Click here to add this to my saved trials
Salt Lake City, Utah 84132
?
mi
from
Salt Lake City, UT
Click here to add this to my saved trials
Salt Lake City, Utah 84132
?
mi
from
Salt Lake City, UT
Click here to add this to my saved trials
South Brisbane, Queensland 4101
?
mi
from
South Brisbane,
Click here to add this to my saved trials
?
mi
from
Tampa, FL
Click here to add this to my saved trials