NCT07250737Available不适用
Managed Access to Investigational Use of AOC 1044 in Participants With DMD Mutations Amenable to Exon 44 Skipping
相关药物
试验速览
- 阶段
- 不适用
- 状态
- Available
- 试验地点
- 21
研究概览
简要总结
The purpose of this Managed Access Program is to allow access to delpacibart zotadirsen (AOC 1044) for eligible patients diagnosed with DMD mutations amenable to exon 44 skipping. The patient's Administering Physician should follow the suggested treatment guidelines and comply with all local health authority regulations.
研究设计
- 研究类型
- Expanded Access
入排标准
- 年龄范围
- 2 Years 至 —（Child, Adult, Older Adult）
- 性别
- Male
- 接受健康志愿者
- 否
入选标准
- •Rollover Participants
- •Completed Study EXPLORE44-OLE Treatment Period (through W102)
- •No significant tolerability issues with AOC 1044
- •New (Non-Rollover) Participants
- •Permanently residing in the US and have a US primary health care provider
- •Documented dystrophin gene mutation that is amenable to exon 44 skipping
- •Age 2 or older at the time of consent
- •If previously treated with gene therapy for DMD, treatment and associated immunosuppressive regimen was more than 12 months before consent and in the opinion of the prescriber, participant has had an unsatisfactory treatment response
排除标准
- •Rollover Participants
- •Prescence of any new condition or worsening of existing condition that could affect participant's safety or ability to comply with the program requirements
- •New (Non-Rollover) Participants
- •Recently treated with or on a clinical study for another investigation drug
- •Serious respiratory or cardiac dysfunction, or nearing end of life
- •Screening laboratory parameters do not meet protocol requirements
- •History of multiple drug allergies or to any component of AOC 1044
- •Participants who discontinued early from the treatment period of EXPLORE44 or EXPLORE44-OLE
研究者
研究点 (21)
Loading locations...
相似试验
Available
不适用
Global Managed Access Program Cohort for Remibrutinib in Adult Patients With Chronic Spontaneous UrticariaChronic Spontaneous UrticariaNCT05170724Novartis Pharmaceuticals
No Longer Available
不适用
Managed Access Programs for PKC412, MidostaurinMast Cell LeukemiaFMS-Like Tyrosine Kinase 3 (FLT3)-Mutated Acute Myeloid LeukemiaAggressive Systemic MastocytosisSystemic Mastocytosis With an Associated Hematologic NeoplasmAcute Myeloid LeukemiaNCT05219266Novartis Pharmaceuticals
Approved For Marketing
不适用
AveXis Managed Access Program Cohort for Access to AVXS-101Spinal Muscular AtrophyNCT03955679United BioSource, LLC
Available
不适用
Managed Access Programs for ETB115, EltrombopagThrombocytopeniaNCT04837703Novartis Pharmaceuticals
Available
不适用
Managed Access Programs for INC424, RuxolitinibNCT04745637Novartis Pharmaceuticals