Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
Primary Purpose
Infantile-onset Spinal Muscular Atrophy
Status
No longer available
Phase
Locations
International
Study Type
Expanded Access
Intervention
Nusinersen
Sponsored by
About this trial
This is an expanded access trial for Infantile-onset Spinal Muscular Atrophy focused on measuring Spinal Muscular Atrophy, SMA, SMN, SMNRx, ISIS-SMNRx, ISIS-SMN Rx, ISIS 396443, ISIS-396443, ENDEAR, IONIS-SMNRx, IONIS-SMN Rx, EMBRACE, NURTURE, IONIS, BIOGEN, SHINE, Nusinersen
Eligibility Criteria
Key Inclusion Criteria:
- 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
Key Exclusion Criteria:
- 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
Sites / Locations
- Hospital Pablo Tobon Uribe
- Auckland City Hospital
- Auckland District Health Board ADHB
- Erciyes University Hospital
- Hacettepe University
- Marmara Uni. Research & Educational Hospital
- Medipol University Hospital
Outcomes
Primary Outcome Measures
Secondary Outcome Measures
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT02865109
Brief Title
Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
Official Title
Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)
Study Type
Expanded Access
2. Study Status
Record Verification Date
March 2021
Overall Recruitment Status
No longer available
Study Start Date
undefined (undefined)
Primary Completion Date
undefined (undefined)
Study Completion Date
undefined (undefined)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Biogen
4. Oversight
5. Study Description
Brief Summary
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.
Detailed Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Infantile-onset Spinal Muscular Atrophy
Keywords
Spinal Muscular Atrophy, SMA, SMN, SMNRx, ISIS-SMNRx, ISIS-SMN Rx, ISIS 396443, ISIS-396443, ENDEAR, IONIS-SMNRx, IONIS-SMN Rx, EMBRACE, NURTURE, IONIS, BIOGEN, SHINE, Nusinersen
7. Study Design
8. Arms, Groups, and Interventions
Intervention Type
Drug
Intervention Name(s)
Nusinersen
Other Intervention Name(s)
ISIS 396443, BIIB058, Spinraza
Intervention Description
Administered by intrathecal injection
10. Eligibility
Sex
All
Eligibility Criteria
Key Inclusion Criteria:
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
Key Exclusion Criteria:
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Biogen
Official's Role
Study Director
Facility Information:
Facility Name
Hospital Pablo Tobon Uribe
City
Medellin
ZIP/Postal Code
050036
Country
Colombia
Facility Name
Auckland City Hospital
City
Grafton
State/Province
Auckland
ZIP/Postal Code
1023
Country
New Zealand
Facility Name
Auckland District Health Board ADHB
City
Grafton
State/Province
Auckland
ZIP/Postal Code
1023
Country
New Zealand
Facility Name
Erciyes University Hospital
City
Kayseri
State/Province
Anatolia
ZIP/Postal Code
38000
Country
Turkey
Facility Name
Hacettepe University
City
Ankara
State/Province
Central Anatolia
ZIP/Postal Code
06100
Country
Turkey
Facility Name
Marmara Uni. Research & Educational Hospital
City
Kadıköy
State/Province
Istanbul
ZIP/Postal Code
34722
Country
Turkey
Facility Name
Medipol University Hospital
City
Istanbul
State/Province
Marmara
ZIP/Postal Code
34214
Country
Turkey
12. IPD Sharing Statement
Learn more about this trial
Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
We'll reach out to this number within 24 hrs