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Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)

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ClinicalTrials.gov Identifier: NCT02865109
Expanded Access Status : Available
First Posted : August 12, 2016
Last Update Posted : October 4, 2018
Sponsor:
Information provided by (Responsible Party):
Biogen

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.

Condition or disease Intervention/treatment
Infantile-onset Spinal Muscular Atrophy Drug: Nusinersen

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 Type : Expanded Access
Expanded Access Type : Treatment IND/Protocol
  See clinical trials of the intervention/treatment in this expanded access record.
Official Title: Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)



Intervention Details:
  • Drug: Nusinersen
    Administered by intrathecal injection
    Other Names:
    • ISIS 396443
    • BIIB058
    • Spinraza

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02865109


Contacts
Contact: US Biogen Clinical Trial Center 866-633-4636 clinicaltrials@biogen.com
Contact: Global Biogen Clinical Trial Center clinicaltrials@biogen.com

Locations
China, Taiwan, Province OF China
Kaohsiung Medical University Hospital
Kaohsiung City, Taiwan, Province OF China, China, 807
National Taiwan University Hospital, Taipei
Taipei City, Taiwan, Province OF China, China, 100
Korea, Republic of
Kyungpook National University Chilgok Hospital
Daegu Metropolital City, Korea, Republic of, 702-210
Samsung Medical Center
Seoul, Korea, Republic of, 06351
Gangnam Severance Hospital
Seoul, Korea, Republic of, 135-720
Mexico
Hospital Infantil de Mexico Federico Gomez
Mexico city, Mexico, 06720
Poland
Upper Silesian Child Health Centre
Katowice, Silesian, Poland, 40-752
University Clinical Center in Gdansk
Gdansk, Poland, 80-952
The Children's Memorial Health Institute
Warsaw, Poland, Al. Dzieci Polskich 20
Portugal
Doña Estefania Hospital
Lisbon, Lisboa, Portugal, 1169-045
Centro Hospitalar e Universitario de Coimbra
Coimbra, Portugal
Centro Hospitalar Lisboa Norte
Lisbon, Portugal, 1649-035
Centro Hospital do Porto
Porto, Portugal, 4099-001
Taiwan
Kaohsiung Medical University Hospital
Kaohsiung, Taiwan, 807
National Taiwan University Hospital
Taipei, Taiwan, 114
Turkey
Ege Universitesi Tip Fakultesi Cocuk Sagligi
Izmir, Anatolia, Turkey, 35100
Erciyes University Hospital
Kayseri, Anatolia, Turkey, 38000
Hacettepe University
Ankara, Central Anatolia, Turkey, 06100
Marmara Uni. Research & Educational Hospital
Kadıköy, Istanbul, Turkey, 34722
Medipol University Hospital
Istanbul, Marmara, Turkey, 34214
Karadeniz Technical University
Trabzon, Turkey, 61080
United Kingdom
Royal Victoria Hospital
Belfast City, Belfast, United Kingdom, BT12 6BA
Ninewells Hospital
Dundee, Scotland, United Kingdom, DD2 1SY
Sheffield Children's Hospital
Sheffield, South Yorkshire, United Kingdom, S10 2TH
Royal Stoke University Hospital
Stoke-on-Trent, Staffordshire, United Kingdom, ST4 6QG
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, Tyne And Wear, United Kingdom, NE7 7DN
The Leeds Teaching Hospitals
Leeds, Yorkshire, United Kingdom, LS10 2JQ
University Hospital of Wales
Cardiff, United Kingdom, CF144HY
Alder Hey Childrens Hospital
Liverpool, United Kingdom, L12 2AP
Guy's & St Thomas' NHS FT
London, United Kingdom, SE1 7EH
Great Ormond Street Hospital
London, United Kingdom, WC1N 3JH
Nottingham University Hospital
Nottingham, United Kingdom, NG5 1PB
Sponsors and Collaborators
Biogen
Investigators
Study Director: Medical Director Biogen

Responsible Party: Biogen
ClinicalTrials.gov Identifier: NCT02865109     History of Changes
Other Study ID Numbers: 232-SM-901
First Posted: August 12, 2016    Key Record Dates
Last Update Posted: October 4, 2018
Last Verified: September 2018

Keywords provided by Biogen:
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

Additional relevant MeSH terms:
Atrophy
Muscular Atrophy
Muscular Atrophy, Spinal
Pathological Conditions, Anatomical
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Spinal Cord Diseases
Central Nervous System Diseases
Motor Neuron Disease
Neurodegenerative Diseases
Neuromuscular Diseases