EUCTR2020-000699-39-FR进行中（未招募）1 期
A Phase 3, Randomized, Double-Blind, Trial of Pamrevlumab (FG-3019) or Placebo in Combination with Systemic Corticosteroids in Ambulatory Subjects with Duchenne Muscular Dystrophy (DMD)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 70
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- Male
入选标准
- •Double-blind phase:
- •Age and Consent
- •1. Males at least 6 to < 12 years of age at screening initiation;
- •2. Written consent by legal guardian as per regional/ country and/or IRB/IEC requirements;
- •DMD Diagnosis:
- •3. Medical history includes diagnosis of DMD and confirmed Duchenne mutation using a validated genetic test.
- •Pulmonary Criteria:
- •4. Average (of Screening and Day 0) percent predicted FVC above 45%;
- •5. On a stable dose of systemic corticosteroids for a minimum of 6 months, with no substantial change in dosage for a minimum of 3 months (except for adjustments for changes in body weight) prior to screening. Corticosteroid dosage should be in compliance with the DMD Care Considerations Working Group recommendations (e.g. prednisone or prednisolone 0.75 mg/kg per day or deflazacort 0.9 mg/kg per day) orstable dose. A reasonable expectation is that dosage and dosing regimen would not change significantly for the duration of the study.
- •Performance Criteria:
- •6. Able to complte 6MWD test with a distance of at least 270M but no more than 450M on two occasions within 3 months prior to Randomisation with <=10% variation between these two tests.
- •7. Able to rise (TTSTAND) from floor in <10 seconds (without aids/orthoses) at screening visit.
- •8. Able to undergo MRI test for the lower extremities vastus lateralis muscle.
- •Vaccination:
- •9. Received pneumococcal vaccine (PPSV23) (or any other pneumococcal polysaccharidevaccine as per national recommendations) and is receiving annual influenza vaccinations.
- •Laboratory Criteria:
- •10. Adequate renal function: cystatin C =1.4 mg/L;
- •11. Adequate hematology and electrolytes parameters:
- •a. Platelets >100,000/mcL
- •b. Hemoglobin >12 g/dL
- •c. Absolute neutrophil count >1500 /µL
- •d. Serum calcium (Ca), potassium (K), sodium (Na), magnesium (Mg) and phosphorus(P) levels are within a clinically accepted range;
- •12. Adequate hepatic function:
- •a. No history or evidence of liver disease
- •b. Gamma glutamyl transferase (GGT) =3x upper limit of normal (ULN)
- •c. Total bilirubin =1.5xULN.
- •Open-label extension phase:
- •All patients must have completed treatment through the primary endpoint and completed the Week 52 visit on the double-blind phase. The study investigator must consider the subject medically stable for continued treatment. Written consent/assent by the patients and/or their legal guardian must be obtained prior to the patient’s participation in the open-label extension phase.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 70
- •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
- •Both phases:
- •General criteria:
- •1. Concurrent illness other than DMD that can cause muscle weakness and/or impairment of motor function.
- •2. Severe intellectual impairment (eg. severe autism, sever cognitive impairment, severe behavioral disturbance) preventing the ability to perform study assessments in the investigator´s judgement.
- •3. Previous exposure to pamrevlumab.
- •4. BMI =40 kg/m2 or weight >117 kg.
- •5. History of allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies.
- •6. Exposure to any investigational drug (for DMD or not), in the 30 days prior to screening initiation or use of approved DMD therapies (e.g., eteplirsen, ataluren) within 5 half-lives of screening, whichever is longer, with the exception of the systemic corticosteroids, including deflazacort.
- •Cardiac and Pulmonary Criteria:
- •7. Requires >= 16 hours continuous ventilation.
- •8. Poorly controlled asthma or underlying lung disease such as bronchitis, bronchiectasis, emphysema, recurrent pneumonia that in the opinion of the investigator might impact respiratory function.
- •9. Hospitalization due to respiratory failure within 8 weeks prior to screening.
- •10. Severe uncontrolled heart failure (NYHA Classes III-IV), including any of the following:
- •a. Need for intravenous diuretics or inotropic support within 8 weeks prior to screening
- •b. Hospitalization for a heart failure exacerbation or arrhythmia within 8 weeks prior to screening.
- •11. Arrhythmia requiring anti-arrhythmic therapy.
- •12. Any other evidence of clinically significant structural or functional heart abnormality.
- •Clinical Judgments:
- •13. The Investigator judges that the subject will be unable to fully participate in the study and complete it for any reason, including inability to comply with study procedures and treatment, or any other relevant medical, surgical or psychiatric conditions.
研究者
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