NCT02865109No Longer Available不适用
Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)
适应症
相关药物
试验速览
- 阶段
- 不适用
- 状态
- No Longer Available
- 发起方
- Biogen
- 试验地点
- 7
研究概览
简要总结
To provide access to nusinersen to eligible patients with Infantile-onset Spinal Muscular Atrophy (SMA) (consistent with Type 1) to address a high-unmet medical need.
详细描述
The nusinersen expanded access program (EAP) is available at approved treatment centers in select territories.
A doctor must decide whether nusinersen treatment is appropriate for each patient, based on the patient's medical history and program eligibility criteria. A full list of participating treatment centers is provided in the 'Contacts and Locations' section of this listing, and is regularly updated.
Following local approval and official reimbursement of nusinersen in each territory, the EAP will close and patients will transfer to commercially available drug.
研究设计
- 研究类型
- Expanded Access
入排标准
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Genetic documentation of 5q SMA homozygous gene deletion, homozygous mutation, or compound heterozygote.
- •Onset of clinical signs and symptoms at ≤ 6 months (180 days) of age, consistent with infantile onset, Type I SMA
- •Patient whose care in the opinion of the treating physician meets, and is expected to continue to meet, the guidelines set out in the 2007 Consensus Statement for Standard of Care in SMA
排除标准
- •Patient is qualified to participate in an ongoing clinical trial with nusinersen
- •Participation in a prior nusinersen study
- •Previous exposure to nusinersen
- •History of brain or spinal cord disease that would interfere with the LP procedures or CSF circulation
- •Presence of implanted shunt for the drainage of CSF or implanted CNS catheter
- •Previous or current participation in a clinical trial with an investigational gene therapy for SMA
- •Participation in a study with an investigational therapy for SMA within 6 months or five half-lives of the investigational drug, whichever is the longer, prior to the first dose of nusinersen.
- •NOTE: Other protocol defined Inclusion/Exclusion criteria may apply
研究者
研究点 (7)
Loading locations...
相似试验
Temporarily Not Available
不适用
Expanded Access Program for Autologous Muscle Derived Cells (AMDCs)NCT04661293Cook MyoSite
No Longer Available
不适用
Individual Patient Expanded Access for AB-SA01, an Investigational Anti-Staphylococcus Aureus Bacteriophage TherapeuticNCT03395769Armata Pharmaceuticals, Inc.
No Longer Available
不适用
Expanded Access to Rovalpituzumab TesirineSmall Cell Lung CancerNCT03503890AbbVie
Approved For Marketing
不适用
Expanded Access of Pimavanserin for Patients With PD PsychosisParkinson's Disease PsychosisNCT02762591ACADIA Pharmaceuticals Inc.
No Longer Available
不适用
Expanded Access to ElezanumabAcute Spinal Cord Injury (SCI)NCT04278235AbbVie