Infantile-onset Spinal Muscular AtrophyNCT02865109
Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
A study testing Nusinersen in people with Infantile-onset Spinal Muscular Atrophy, run by Biogen at 7 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Infantile-onset Spinal Muscular Atrophy
- What is being tested?
- Nusinersen
- Type of treatment
- Injection or infusion
- How many people
- Not given
- Where
- 7 sitesin 3 countries
- Study dates
- Start not givento not given
- Phase
- Expanded access
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
7 sites in 3 countries.
7 sites in 3 countries
Turkey (Türkiye)4 sites
Hacettepe University
Ankara, Central Anatolia, 06100
Medipol University Hospital
Istanbul, Marmara, 34214
Marmara Uni. Research & Educational Hospital
Kadıköy, Istanbul, 34722
Erciyes University Hospital
Kayseri, Anatolia, 38000
New Zealand2 sites
Auckland City Hospital
Grafton, Auckland, 1023
Auckland District Health Board ADHB
Grafton, Auckland, 1023
About this study
Official title: Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)
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.
Read the 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.
- Conditions
- Infantile-onset Spinal Muscular Atrophy
- Being tested
- Nusinersen
- 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
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NusinersenDrug
Also called ISIS 396443, BIIB058, Spinraza
Administered by intrathecal injection
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Infantile-onset Spinal Muscular AtrophyStudies of the same condition, open and under way.
- More trials run by BiogenThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Expanded access
Sponsor and investigators
- Lead sponsor
- Biogen
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02865109
- Sponsor's protocol ID
- 232-SM-901
- Status last verified
- Mar 2021
- First submitted
- 10 Aug 2016
- Last updated
- 5 Apr 2021
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Apr 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)
- ClinicalTrials.gov ID
- NCT02865109
- Acronym / sponsor ID
- 232-SM-901
- Conditions
- Infantile-onset Spinal Muscular Atrophy
- Keywords
- Spinal Muscular Atrophy, SMA, SMN, SMNRx, ISIS-SMNRx, ISIS-SMN Rx, ISIS 396443, ISIS-396443
- Study type
- Expanded access
- Recruitment status
- No Longer Available
- Lead sponsor
- BiogenIndustry (pharma, biotech, devices)
- First posted
- 12 Aug 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (3 listed)
- 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 (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NusinersenAlso: ISIS 396443, BIIB058, SpinrazaAdministered by intrathecal injection | Drug | – |
Primary outcome
Not reported in the source record.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Apr 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Biogen
7 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Nusinersen in people with Infantile-onset Spinal Muscular Atrophy, run by Biogen at 7 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 5 Apr 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in Turkey (Türkiye), New Zealand, Colombia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Apr 2021.