Research requests for studies taking place in North America can be submitted to the Registry using the steps below. Please note the International SMA Patient Registry is a member of the TREAT-NMD network of national registries. If your research request involves multinational study sites you will need to submit your request directly to TREAT-NMD. They will assist you in utilizing all the relevant national registries. Please click here to be taken to TREAT-NMD’s website.

If you are interested in conducting research using data from the Registry, you will need to submit your request for use of the Registry through the following steps.

Step #1: Submit your proposal and all supporting documents to the International SMA Patient Registry
In order to request use of the Registry you will need to submit the Request to Use the International SMA Patient Registry form along with the following documents:

• Study proposal which should describe the study design, study purpose, method of data collection with complete example provided including any proposed questionnaires, phone screens, etc.
• Informed consent
• Institutional Review Board (IRB) approval from your institution
• Inclusion/exclusion criteria
• Number of participants needed for study and any other information which may be important such as “within a 50 mile radius of a particular University”, “no funds are available for travel”, etc.

The Request to Use the International SMA Patient Registry form will also alert you to your obligations as a user of the Registry. Specifically, the Registry requires the following from researchers:  

• The International SMA Patient Registry must be cited in any publications, conferences, or any other presentations of material resulting from the International SMA Patient Registry use. Use the acknowledgement language below:
  
  

  “This study acknowledges the collaboration of the International Spinal Muscular Atrophy Patient Registry (Indiana University) which is supported by the Patient Advisory Group of the International Coordinating Committee for SMA clinical trialswhich includes Families of SMA, FightSMA, the Muscular Dystrophy Association, and the Spinal Muscular Atrophy Foundation, and other SMA advocacy groups."

• The International SMA Patient Registry should receive a copy of any cited materials for our files.

Your request form and documents may be submitted to the SMA Registry via mail or email, though it is preferred that the submission be made electronically through the Request to Use Registry Form link to the right.

Step #2: Evaluation by the International SMA Patient Registry Oversight Committee
If you are seeking identifiable data such as for recruitment of participants for study or clinical trial, the Registry will forward your proposal on to the International SMA Patient Registry Oversight Committee (Oversight Committee). This committee is responsible for evaluating proposals and determining if they are appropriate and ethical. Oversight Committee members are given the opportunity to excuse themselves from reviews due to any conflict of interest. A majority vote is necessary for research requests to be approved.

If you are requesting de-identified data, the International SMA Patient Registry Oversight Committee will be informed of the request but will not be required to review and approve the request.

Step #3: Submission of Request to IUPUI Institutional Review Board
Upon approval by the Oversight Committee, your proposal will be submitted to the Institutional Review Board at Indiana University as a minor amendment. The review by IU is not specific to your research study but rather to review and approve the recruitment documents that will go to Registry participants in regards to your study. It is estimated that review will take up to 2 weeks.

Step #4: Execution of Approved Requests
Once your proposal has been approved by the International SMA Patient Registry Oversight Committee and the Indiana University IRB, Registry staff will work with you to fulfill your request.

• Statistical/Demographic Data: If you are requesting data that is statistical or demographic in nature and you do not need any identifying information, the Registry will compile the requested data. If you are requesting that participants be informed of an ‘anonymous’ research opportunity such as an online survey, an informational letter will be sent to qualifying participants telling them how to take part in your study.
  
  
• Research with Identifiable Information: If you are requesting identified information or if your proposal involves identifying individuals in the database that fit your requested criteria for study recruitment, the Registry will identify the appropriate participants and send an informational letter and information release form to Registry participants. This letter will explain to those identified individuals what your study is about and ask them if they would like to have their personal information forwarded to your study site. When the Registry contacts these individuals, they are not obligated to take part in the offered study.