NCT03433807No Longer Available不适用
Expanded Access Protocol (EAP) of Idebenone in Patients With Duchenne Muscular Dystrophy
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- No Longer Available
- 试验地点
- 12
研究概览
简要总结
The primary objective of this Expanded Access Program is to provide idebenone as a treatment for eligible participants with Duchenne Muscular Dystrophy before it is commercially available in the United States (U.S.) for the indication of DMD.
研究设计
- 研究类型
- Expanded Access
入排标准
- 年龄范围
- 8 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documented diagnosis of DMD (severe dystrophinopathy) and clinical features consistent of typical DMD at diagnosis (i.e., documented delayed motor skills and muscle weakness by age 5 years) and who in the opinion of the Treating physician would benefit from treatment with idebenone. DMD should be confirmed by mutation analysis in the dystrophin gene or by substantially reduced levels of dystrophin protein (i.e., absent or <5% of normal) on Western blot or immunostaining.
- •Minimum 8 years old at Prescreening.
- •PEF or FVC ≤80% and >25% of predicted value based on most recent assessment noted in the patient's medical record and subsequently confirmed at the Enrollment Visit.
- •Able to understand program requirements and swallow program medication.
- •Signed and dated Informed Consent Form (to be obtained at the Enrollment Visit from patient or parent/legal guardian (if applicable) prior to performing any program-specific procedures and dispensing idebenone to the patient).
排除标准
- •Eligible for and able to participate in an ongoing clinical trial of idebenone.
- •Is at high-risk of a fatal outcome from lung infection and/or advanced cardiomyopathy in the opinion of the Treating physician.
- •Known moderate or severe impairment of hepatic function or severe impairment of renal function.
- •Prior or ongoing medical condition or laboratory abnormality which in the Treating physician's opinion may put the patient at significant risk or may interfere significantly with the patient's participation in the program.
- •Abuse of drugs or alcohol, which in Treating physician's opinion would interfere with the compliance to treatment.
- •Known individual hypersensitivity to idebenone or to any of the ingredients/excipients of the program medication.
研究者
研究点 (12)
Loading locations...
相似试验
Approved For Marketing
不适用
Expanded Access for Treatment With ImetelstatMyelodysplastic SyndromesNCT05937568Geron Corporation
Available
不适用
Expanded Access to UpadacitinibSpondyloarthritisAxial SpondyloarthritisNon-radiographic AxialNon Segmental VitiligoHidradenitis SuppurativaCrohn DiseaseUlcerative ColitisAtopic DermatitisRheumatoid ArthritisPsoriatic ArthritisGiant Cell ArteritisSystemic Lupus ErythematosusAlopecia AreataNCT04159597AbbVie
Approved For Marketing
不适用
Expanded Access Program (EAP) for Participants With Advanced Cancers and Fibroblast Growth Factor Receptor (FGFR) Genetic Alterations Who Have Exhausted All Treatment OptionsAdvanced Cancers and FGFR Genetic AlterationsNCT03825484Janssen Scientific Affairs, LLC
Approved For Marketing
不适用
Pre-Approval Access With Amivantamab (JNJ-61186372) in Participants With Metastatic Non-Small Cell Lung CancerMetastatic Non-Small Cell Lung CancerNCT04599712Janssen Research & Development, LLC
No Longer Available
不适用
Individual Patient Expanded Access for AB-PA01, an Investigational Anti-Pseudomonas Aeruginosa Bacteriophage TherapeuticNCT03395743Armata Pharmaceuticals, Inc.