EUCTR2020-000698-26-GB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 Subjects with Non-ambulatory 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
- 90
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
- •Double-blind phase:
- •Age, Consent, and Contraception
- •1. Males at least 12 years of age, non-ambulatory at screening initiation;
- •2. Written consent by patient and/or legal guardian as per regional/ country and/or IRB/IEC requirements;
- •3. Male subjects with partners of childbearing potential must use contraception during the conduct of the study, and for 3 months after the last dose of study drug.
- •DMD Diagnosis:
- •4. Medical history includes diagnosis of DMD and confirmed Duchenne mutation using a validated genetic test.
- •Performance criteria:
- •5. Brooke Score for Arms and Shoulders =5:
- •6. Able to undergo MRI test for the upper arm extremities (Biceps Brachii muscle) andcardiac muscle;
- •7. Able to perform spirometry.
- •Pulmonary and Cardiac criteria:
- •8. Reproducible (+/- 5% difference between screening and baseline) percent predicted FVC between 45 and 85, inclusive;
- •9. Left ventricular ejection fraction =50% as determined by cardiac MRI at screening or within 3 months prior to randomization (Day 0);
- •10. Prior diagnosis of cardiomyopathy, subjects must be on a stable regimen dose for cardiomyopathy/ heart failure medications (e.g., angiotensin converting enzyme inhibitors, aldosterone receptors blockers, angiotensin-receptor blockers, and beta-blockers) for at least 1 month prior to screening;
- •11. On a stable dose of systemic corticosteroids for a minimum of 6 months, with nos ubstantial 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.
- •Vaccination:
- •12. Received pneumococcal vaccine (PPSV23) (or any other pneumococcal polysaccharidevaccine as per national recommendations) and is receiving annual influenza vaccinations.
- •Laboratory criteria:
- •13. Adequate renal function: cystatin C =1.4 mg/L;
- •14. 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;
- •15. 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: 30
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 55
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 5
排除标准
- •Both phases:
- •General criteria:
- •1. Previous exposure to pamrevlumab;
- •2. BMI =40 kg/m2 or weight >117 kg;
- •3. History of allergic or anaphylactic reaction to human, humanized, chimeric or murine monoclonal antibodies;
- •4. 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 assessment:
- •5.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;
- •6. Arrhythmia requiring anti-arrhythmic therapy;
- •7. Requires =16 hours continuous ventilation;
- •8. Hospitalization due to respiratory failure within the 8 weeks prior to screening;
- •9. 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.
- •Clinical judgments:
- •10.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 or psychiatric conditions which could confound efficacy assessment and/or safety assessment.
研究者
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