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

Last updated on October 6, 2018

FOR MORE INFORMATION
Study Location
David Geffen School of Medicine at UCLA/UCLA Neorology
Los Angeles, California, 90095 United States
Contact
1-800-718-1021
Eligibility criteria
Condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Duchenne Muscular Dystrophy
Sex
Male
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
6-18 years
Inclusion criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

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
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details

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.

NCT02907619
Pfizer
Recruiting
An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy

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An Open-label Extension Study To Evaluate Safety Of PF-06252616 In Boys With Duchenne Muscular Dystrophy
A Multicenter, Open-label Extension Study To Evaluate The Long Term Safety Of Pf-06252616 In Boys With Duchenne Muscular Dystrophy
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.
Not Provided
Interventional
Phase 2
Masking: None (Open Label)
Primary Purpose: Treatment
Duchenne Muscular Dystrophy
Biological: PF-06252616
Either 5mg/kg, 20mg/kg or 40mg/kg will be assigned to a subject based on their maximum tolerated dose from B5161002
Experimental: PF-06252616
Either 5mg/kg, 20mg/kg or 40mg/kg will be assigned to a subject based on their maximum tolerated dose from B5161002
Intervention: Biological: PF-06252616
Not Provided


*   Includes publications given by the data provider as well as publications
identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
105
March 13, 2023
March 13, 2023   (Final data collection date for primary outcome measure)

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.
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
6 Years to 18 Years   (Child, Adult)
No

Contact: Pfizer CT.gov Call Center 1-800-718-1021 [email protected]
Canada,   Japan,   United Kingdom,   United States
 
 
NCT02907619
B5161004
2016-001615-21 ( EudraCT Number )
Yes
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Not Provided
Pfizer
Pfizer
Not Provided
Study Director: Pfizer CT.gov Call Center Pfizer
Pfizer
November 2017

ICMJE     Data element required by the

International Committee of Medical Journal Editors
and the
World Health Organization ICTRP

FOR MORE INFORMATION

Contact a representative by phone, email, or visiting thte study website. To get updates and notications about this trail, sign up using the form below.

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1-800-718-1021

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