EUCTR2019-000601-77-IE进行中（未招募）1 期
A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 24
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
- •A patient must meet all of the following criteria to be eligible to participate in this study:
- •1. Is male.
- •2. Has a genetic diagnosis of DMD and an out-of-frame deletion mutation of the DMD gene amenable to exon 51-skipping treatment.
- •3. Is 7 to 21 years of age, inclusive.
- •4. Has been on a stable dose of oral corticosteroids for at least 12 weeks prior to study drug administration, or has not received corticosteroids for at least 12 weeks prior to study drug administration.
- •5. If sexually active, agrees to use a male condom during such activity for the entire duration of the study and for 90 days after the last dose of study drug. The sexual partner must also use a highly effective form of contraceptive during this timeframe.
- •6. Is willing to provide informed consent or informed assent (if applicable) and has (a) parent(s) or legal guardian(s) who is (are) willing to provide written informed consent for the patient to participate in the study.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 24
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 4
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •A patient who meets any of the following criteria will be excluded from this study:
- •1. Has a LVEF < 40.0% based on an ECHO performed within 12 weeks prior to Screening or at the Screening visit.
- •2. Has a QT interval corrected with Fridericia’s formula = 450 msec on the Screening ECG.
- •3. Initiation or change of dosing (except for modifications to accommodate changes in weight) within 12 weeks prior to Screening for any of the following: angiotensin-converting enzyme inhibitors, angiotensin receptor-blocking agents, ß-blockers, or potassium.
- •4. Requires anti-arrhythmic and/or diuretic therapy for heart failure.
- •5. Has a FVC < 40.0% of predicted value (according to the American Thoracic Society/European Respiratory Society criteria) within 12 weeks prior to Screening or at Screening.
- •6. Has a current infection, or history of an infection within 12 weeks prior to Screening requiring treatment with an antibiotic.
- •7. Has a known kidney disease or had an acute kidney injury within 24 weeks prior to Screening.
- •8. Use of any herbal medication/supplement containing aristolochic acid.
- •9. Treatment with any exon 51-skipping therapy within 24 weeks prior to Screening, or with any experimental gene therapy for the treatment of DMD at any time.
- •10. Participation in an interventional clinical trial within 12 weeks prior to Screening.
- •11. Use of any aminoglycoside antibiotic or statin within 12 weeks prior to Screening.
- •12. Initiation or change of dosing within 12 weeks prior to Screening for over-the-counter preparations, such as herbal/nonherbal supplements, vitamins, minerals, and homeopathic preparations.
- •13. Major surgery within 12 weeks prior to Screening, or planned surgery or procedures that would interfere with the conduct of the study.
- •14. Presence of other clinically significant illness, including cardiac, pulmonary, hepatic, renal, hematologic, immunologic, or behavioral disease, or infection or malignancy.
- •15. Any other condition that, in the Investigator’s opinion, could interfere with the patient’s participation in the trial.
- •16. Inability to comply with the study protocol.
- •17. Is an employee of the Investigator or study center, with direct involvement in the proposed study or other studies under the direction of that Investigator or study center, as well as family members of the employees or the Investigator.
- •18. Any patient who is taking medications that increase the risk of bleeding, in the Investigator's opinion (eg, anticoagulants, antiplatelet agents, novel oral anticoagulants, selective norepinephrine reuptake inhibitors, etc.)
- •19. Platelet count < 150 × 103/µL.
研究者
相似试验
进行中（未招募）
1 期
A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsMedDRA version: 20.1Level: PTClassification code 10052655Term: Duchenne muscular dystrophy gene carrierSystem Organ Class: 10010331 - Congenital, familial and genetic disordersDuchenne Muscular DystrophyEUCTR2019-000601-77-NLSarepta Therapeutics, Inc.57
进行中（未招募）
1 期
A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsDuchenne Muscular DystrophyMedDRA version: 20.1Level: PTClassification code 10052655Term: Duchenne muscular dystrophy gene carrierSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2019-000601-77-GBSarepta Therapeutics, Inc.70
进行中（未招募）
1 期
A research study of a new investigational medicinal product for the treatment of Duchenne Muscular Dystrophy patientsMedDRA version: 20.1Level: PTClassification code 10052655Term: Duchenne muscular dystrophy gene carrierSystem Organ Class: 10010331 - Congenital, familial and genetic disordersDuchenne Muscular DystrophyEUCTR2019-000601-77-BESarepta Therapeutics, Inc.57
进行中（未招募）
1 期
Two-Part Study for Dose Determination of Vesleteplirsen (SRP-5051) (Part A), Then Dose Efficacy (Part B) in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (MOMENTUM)MedDRA version: 20.1Level: PTClassification code: 10052655Term: Duchenne muscular dystrophy gene carrier Class: 100000004850Duchenne Muscular DystrophyCTIS2023-509935-23-00Sarepta Therapeutics Inc.62
招募中
2 期
A Phase 2, Two-Part, Multiple-Ascending-Dose Study of SRP-5051 for Dose Determination, then Dose Expansion, in Patients with Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatmentmuscular dystrophy10028396DMDNL-OMON54626Sarepta Therapeutics, Inc.6