Extension Pfizer B5161004

Principal Investigator: Russell Butterfield
Keywords: Muscular Dystrophy , DMD , Duchenne , Duchenne Muscular Dystophy , PF-0625616 , Clinical Trial , Pfizer , Open-label , Neurology , Utah Program for Inherited Neuromuscular Disorders Department: Pediatric Administration
IRB Number: 00097619 Co Investigator: Nicholas Johnson
Specialty: Neurology, Pediatric Neurology, Neurology
Sub Specialties: Neuromuscular Diseases, Muscular Dystrophy
Recruitment Status: Active, not recruiting

Contact Information

Bryan Gardner
bryang@genetics.utah.edu
801-585-1499

Brief Summary

This study is an open-label extension (OLE) to Protocol B5161002 and will provide an
assessment of the long term safety, efficacy, pharmacodynamics (PD) and pharmacokinetics
(PK) of intravenous (IV) dosing of PF-06252616 in boys with Duchenne muscular dystrophy
(DMD).

Primary Objective

  • To evaluate the long-term safety of IV dosing of PF-06252616 in boys with DMD.

Secondary Objectives

  • To evaluate the long-term efficacy of PF-06252616 using functional assessments and strength.
  • To assess the PK and immunogenicity of PF-06252616.

Exploratory Objectives

  • To evaluate PD markers that may be informative in demonstrating the pharmacologic effect of PF-06252616.
  • To evaluate the long-term functional health effects of PF-06252616 on Parent-report and Adolescent self-report scores on the PODCI.
  • To assess the long-term effects of PF-06252616 on HRQL and healthcare resource utilization (HRU) in boys with DMD.
  • To evaluate the long-term effects of PF-06252616 on caregiver burden, HRQL and work productivity and activity impairment.
  • To collect exploratory biomarker samples for bio-banking.

Inclusion Criteria

  1. Subjects with Duchenne muscular dystrophy who enrolled and completed through Week 97 of Study B5161002.
  2. Evidence of a personally 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. Subjects may be required to provide assent in compliance with local regulations and IRB requirements.
  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. The following inclusion criteria will be assessed using data from the B5161002 study, and if data are unavailable, these assessments must be completed prior to enrollment:
  •  Adequate hepatic function on screening laboratory assessments from the Week 97 visit.
  • LDH £ 20 units/liter (2 x upper limit of normal [ULN]) from the Week 97 visit.
  • Iron content estimate on the liver MRI within the normal range as determined by R2* value (R2*£75 Hz at 1.5 T or R2*£139 Hz at 3.0 T) from the Week 93 visit.

Exclusion Criteria

1.    Unwilling or unable (eg, metal implants) to undergo examination with closed MRI. If subjects required sedation in the B5161002 study they will be permitted to enroll in the OLE. In the event that a subject becomes intolerant to MRI scanning, during the OLE, the subject may be separately consented to be administered sedation in order to complete the 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 with their female partner(s) a highly effective method of contraception consistently and correctly for the duration of the active treatment period and through the final study visit. 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, beginning with the first dose of investigational product and continuing through the final study visit.

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 or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator, would make the subject inappropriate for entry into this study.

5.    Participation in other studies involving investigational drug(s), with the exception of B5161002, 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.

6.    History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein or additives of this investigational product (histidine, sucrose, edetic acid [ethylenediaminetetraacetic acid], and polysorbate 80).