An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

Pfizer

Status and phase: Terminated; Phase 2

Conditions: Duchenne Muscular Dystrophy

Treatments: Biological: PF-06252616

Study type: Interventional

Funder types: Industry

Identifiers

NCT02907619

B5161004

2016-001615-21 (EudraCT Number)

Details and patient eligibility

About

This study is an open-label extension to protocol B5161002 and will provide an assessment of the long term safety, efficacy, pharmacodynamics and pharmacokinetics of intravenous dosing of PF 06252616 in boys with Duchenne muscular dystrophy. Approximately 105 eligible subjects will be assigned to receive a monthly individualized maximum tolerated dose based on their tolerability profile/data from B5161002. This study will not contain a placebo comparator. Subjects will undergo safety evaluations (Laboratory, cardiac monitoring, physical exams, x-ray, MRI), functional capacity evaluations (4 stair climb, range of motion, strength testing, Northstar Ambulatory Assessment, upper limb functional testing, six minute walk test and pulmonary function tests) and pharmacokinetic testing.

Enrollment: 59 patients

Sex: Male

Ages: 6 to 18 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Subjects with Duchenne muscular dystrophy who enrolled and completed study B5161002.

2. Signed and dated informed consent document (ICD) indicating that the subject's parent or legal guardian/caregiver has been informed of all pertinent aspects of the study.

3. Subjects and their legal guardians/caregivers who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures.

4. Subject have;

   1. Adequate hepatic function on screening laboratory assessments
   2. GLDH less than 20 units/liter (2 x upper limit of normal [ULN])
   3. Iron content estimate on the liver MRI within the normal range.

Exclusion criteria

1. Unwilling or unable (eg, metal implants) to undergo examination with closed MRI.
2. All male subjects who are able to father children and are sexually active and at risk for impregnating a female partner, who are unwilling or unable to use a highly effective method of contraception. In addition, all sexually active male subjects who are unwilling or unable to prevent potential transfer of and exposure to drug through semen to their partners by using a condom consistently and correctly. .
3. Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, or subjects who are related to Pfizer employees directly involved in the conduct of the study.
4. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation.
5. Participation in other studies involving investigational drug(s), with the exception of B5161002.
6. History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein or additives of this investigational product.

Trial design

59 participants in 1 patient group

PF-06252616

Experimental group

Description:

Either 5mg/kg, 20mg/kg or 40mg/kg will be assigned to a subject based on their maximum tolerated dose from B5161002

Treatment:

Biological: PF-06252616