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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/show/NCT02865109




Registration number
NCT02865109
Ethics application status
Date submitted
10/08/2016
Date registered
12/08/2016
Date last updated
21/06/2019

Titles & IDs
Public title
Expanded Access Program (EAP) for Nusinersen in Participants With Infantile-onset (Consistent With Type 1) Spinal Muscular Atrophy (SMA)
Scientific title
Expanded Access Program (EAP) to Provide Nusinersen to Patients With Infantile-onset Spinal Muscular Atrophy (SMA)
Secondary ID [1] 0 0
232-SM-901
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Infantile-onset Spinal Muscular Atrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Neurological 0 0 0 0
Other neurological disorders
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Expanded Access
Description of intervention(s) / exposure
Treatment: Drugs - Nusinersen

Treatment: Drugs: Nusinersen
Administered by intrathecal injection

Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes

Eligibility
Key inclusion criteria
Key

- 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
Minimum age
No limit
Maximum age
No limit
Gender
Both males and females
Can healthy volunteers participate?
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

Study design
Purpose of the study
Allocation to intervention
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?



Intervention assignment
Other design features
Phase
Type of endpoint(s)
Statistical methods / analysis

Recruitment
Recruitment status
Available
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
China
State/province [1] 0 0
Taiwan, Province OF China
Country [2] 0 0
Colombia
State/province [2] 0 0
Antioquia
Country [3] 0 0
Colombia
State/province [3] 0 0
Medellin
Country [4] 0 0
Mexico
State/province [4] 0 0
Mexico city
Country [5] 0 0
New Zealand
State/province [5] 0 0
Auckland
Country [6] 0 0
Taiwan
State/province [6] 0 0
Beitou District
Country [7] 0 0
Taiwan
State/province [7] 0 0
Kaohsiung
Country [8] 0 0
Taiwan
State/province [8] 0 0
Taipei
Country [9] 0 0
Turkey
State/province [9] 0 0
Anatolia
Country [10] 0 0
Turkey
State/province [10] 0 0
Central Anatolia
Country [11] 0 0
Turkey
State/province [11] 0 0
Istanbul
Country [12] 0 0
Turkey
State/province [12] 0 0
Marmara
Country [13] 0 0
Turkey
State/province [13] 0 0
Trabzon
Country [14] 0 0
United Kingdom
State/province [14] 0 0
Belfast
Country [15] 0 0
United Kingdom
State/province [15] 0 0
South Yorkshire
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Staffordshire
Country [17] 0 0
United Kingdom
State/province [17] 0 0
The Northwest
Country [18] 0 0
United Kingdom
State/province [18] 0 0
Tyne And Wear
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Yorkshire
Country [20] 0 0
United Kingdom
State/province [20] 0 0
Cardiff
Country [21] 0 0
United Kingdom
State/province [21] 0 0
Liverpool
Country [22] 0 0
United Kingdom
State/province [22] 0 0
London
Country [23] 0 0
United Kingdom
State/province [23] 0 0
Nottingham

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Biogen
Address
Country

Ethics approval
Ethics application status

Summary
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.
Trial website
https://clinicaltrials.gov/show/NCT02865109
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medical Director
Address 0 0
Biogen
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
US Biogen Clinical Trial Center
Address 0 0
Country 0 0
Phone 0 0
866-633-4636
Fax 0 0
Email 0 0
clinicaltrials@biogen.com
Contact person for scientific queries

Summary results
For IPD and results data, please see https://clinicaltrials.gov/show/NCT02865109