EUCTR2014-002072-92-IT进行中（未招募）1 期
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
Pfizer Inc. 235 East 42nd Street, New York, NY 100170 个研究点目标入组 105 人开始时间: 2015年1月12日最近更新:
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 发起方
- 入组人数
- 105
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- Male
入选标准
- •1. Ambulatory boys age 6 to <10 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). Results must confirm the presence of a mutation in the dystrophin gene(s) which is clinically consistent with the diagnosis of DMD.
- •2. Subjects who are able to perform the 4 stair climb in =0.33 but <1.6 stairs/second.*
- •3. Evidence of a personally signed and dated informed consent and assent (where appropriate) document indicating that the subject and a legally acceptable representative/parent(s)/legal guardian have been informed of all pertinent aspects of the study.
- •4. Subjects and their legal guardians who are willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures. Subjects will be required to provide assent in compliance with local regulations and IRB requirements.
- •5. 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.
- •6. Adequate hepatic and renal function on screening laboratory assessments:
- •GGT =upper limit of normal (ULN).
- •Alkaline phosphatase =ULN.
- •Total Bilirubin =ULN.
- •Serum Albumin=LLN.
- •Serum creatinine =ULN.
- •7. No underlying disposition for iron accumulation on screening laboratory assessments:
- •Serum Iron=ULN.
- •Serum Ferritin =ULN.
- •% Transferrin Saturation =ULN.
- •8. No underlying disposition for bleeding disorder on screening laboratory assessments:
- •PT/INR =1.25 x ULN.
- •aPTT =1.25x ULN.
- •9. Iron content estimate on the screening liver MRI is within the normal range as determined by R2* value (R2*=75 Hz at 1.5 T or R2*=139 Hz at 3.0 T).
- •*Note: The 4 stair climb is expressed as a velocity in the inclusion criteria rather than a time in seconds (>2.5s but =12s), in an attempt to reduce the risk of subjects purposefully manipulating their performance at screening in order to enroll.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 105
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •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 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 <60% as determined on a screening cardiac MRI or echocardiogram). Subjects may be receiving ACE (angiotensin-converting-enzyme) inhibitors or beta blockers; 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) (Phases 1-4) 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 Exon skipping and nonsense mutation targeted therapies.
- •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.
- •13. History of allergic or anaphylactic reaction to a therapeutic or diagnostic protein.
- •14. Have suicidal ideation and behavior associated with actual intent and/or method and/or plan and/or action (eg, self-harming behaviors) in the past 6 months based on the Columbia-Suicide Severity Rating Scale (C-SSRS Children’s Baseline/Screening Appendix 1) or at baseline (C-SSRS Children’s Since Last Visit Appendix 2).
- •15. Subjects who, in the opinion of the investigator, are biologically capable of having children and are sexually active who are unwilling or unable to use a condom to prevent potential transfer of exposure to drug through semen; male subjects of childbearing potential, with their female partners at risk for pregnancy, who are unwilling or unable to use a highly effective method of contraception as outlined in this protocol (in addition to the condom to prevent potential transfer of drug through semen) for the duration of the study and through completion on final study visit.
- •16. 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.
- •17. Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase t
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