Muscular Atrophy, SpinalNCT00466349
International SMA Patient Registry
An observational study of Muscular Atrophy, Spinal, run by Indiana University at 1 site in United States.
Trial summary
This is a registry of individuals affected by Spinal Muscular Atrophy (SMA) and/or family members of individuals affected by SMA. The purpose of the registry is to allow researchers studying the biological basis of SMA and potential therapies in SMA access to individuals interested in participating in research and/or… Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Muscular Atrophy, Spinal
- How many people
- 3,000 participants
- Where
- 1 siteUnited States
- Study dates
- May 1986to Jan 2018
- How long will I take part?
- 50 Years
- Phase
- Observational
- 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
- No healthy volunteers
- Child, Adult, Older adult
Study population: Individuals or family members of individuals who have been diagnosed with SMA.
Who may be able to join
- Individuals or family members of individuals who have been diagnosed with SMA.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Indiana University School of Medicine
Indianapolis, Indiana, 46202
About this study
Official title: International Spinal Muscular Atrophy Patient Registry
This is a registry of individuals affected by Spinal Muscular Atrophy (SMA) and/or family members of individuals affected by SMA. The purpose of the registry is to allow researchers studying the biological basis of SMA and potential therapies in SMA access to individuals interested in participating in research and/or experimental therapies. The International SMA Patient Registry is supported by CureSMA.
Read the detailed description
This project is supported by CureSMA (http://curesma.org/).
The Registry functions as a liaison between patients and families interested in participating in research and researchers interested in studying SMA. The Registry contains information from 2,500 families and over 3,000 individuals with SMA from all over the world and continues to grow.
The Registry has helped recruit participants for clinical trials and has provided data for important SMA research studies. The Registry helps centralize information on this rare genetic disease, provides families a way to learn about research studies and provides researchers a way to find research participants.
Individuals and families affected by SMA are invited to join the Registry. Participants are asked to complete questionnaires about the symptoms, treatment, medications, and other experiences with SMA. Visit the Participant Portal for more information on how to sign up for the Registry.
Participant information is stored in a secure database. Researchers who are interested in studying SMA can request two types of data from the Registry, de-identified information and identifiable information. De-identified information does not contain any names or personal identifiers, and can be given to researchers without having to contact Registry families. Identifiable information includes information that can identify you and will never be released without getting your written permission to do so. Identifiable information includes data such as names and dates of birth. Some researchers may also request contact with families to obtain specific information or to request participation in a research study. In these instances, the Registry will contact each potential participant to ask if they are willing to share their identifiable information for a research project.
The Registry recognizes the importance of global collaboration. Working together with researchers from all over the world is very important in the investigation of SMA. In 2008 the Registry joined the group, Translational Research in Europe for the Assessment and Treatment of Neuromuscular Diseases (TREAT-NMD) in a global collaboration to further the research goals of the neuromuscular disease community. TREAT-NMD has developed a global database that compiles deidentified data transferred from participating registries around the world. The main objective of the TREAT-NMD database is to assess the feasibility of clinical trials, to facilitate the planning of clinical trials and to support the enrollment of patients in clinical trials. This international database will also be used to answer questions regarding the prevalence of particular neuromuscular disorders and assessing the support of other activities such as assessing standards of care and diagnosis.
- Conditions
- Muscular Atrophy, Spinal
- Keywords
- SMA Type I, SMA Type II, SMA Type III, SMN, Werdnig-Hoffman Disease, Spinal Muscular Atrophy, Registry, SMA Type IV
- Medical subject headings
- Muscular Atrophy, Spinal, Spinal Muscular Atrophies of Childhood
What is being measured
The primary outcome is the main question the study is designed to answer.
Study is a patient registry
Measured: unlimited
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2007 (estimated)
- Study startMay 1986
- Primary completionJan 2018
- Study completionJan 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Atrophy, SpinalStudies of the same condition, open and under way.
- More trials run by Indiana UniversityThe 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
- Observational
- Observational model
- Family based
- Time perspective
- Other
- Enrolment
- 3,000 participants (actual)
- Target duration
- 50 Years
Sponsor and investigators
- Lead sponsor
- Indiana University
- Sponsor type
- University, hospital or other
- Collaborators
- Cure SMA
- Responsible party
- Principal investigator, Tatiana Foroud, Indiana University
- Investigators
- Tatiana Foroud, PhD, Principal investigator, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00466349
- Sponsor's protocol ID
- 0202-03
- Status last verified
- Aug 2018
- First submitted
- 24 Apr 2007
- Last updated
- 7 Aug 2018
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 7 Aug 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- International Spinal Muscular Atrophy Patient Registry
- ClinicalTrials.gov ID
- NCT00466349
- Acronym / sponsor ID
- 0202-03
- Conditions
- Muscular Atrophy, Spinal
- Keywords
- SMA Type I, SMA Type II, SMA Type III, SMN, Werdnig-Hoffman Disease, Spinal Muscular Atrophy, Registry, SMA Type IV
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 3,000 participants (actual)
- Lead sponsor
- Indiana UniversityUniversity, hospital or other
- Collaborators
- Cure SMA
- Study start
- May 1986
- Primary completion
- Jan 2018 (actual)
- Study completion
- Jan 2018 (actual)
- First posted
- 27 Apr 2007 (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.
This is a registry of individuals affected by Spinal Muscular Atrophy (SMA) and/or family members of individuals affected by SMA. The purpose of the registry is to allow researchers studying the biological basis of SMA and potential therapies in SMA access to individuals interested in participating in research and/or experimental therapies. The International SMA Patient Registry is supported by CureSMA.
Detailed description
This project is supported by CureSMA (http://curesma.org/).
The Registry functions as a liaison between patients and families interested in participating in research and researchers interested in studying SMA. The Registry contains information from 2,500 families and over 3,000 individuals with SMA from all over the world and continues to grow.
The Registry has helped recruit participants for clinical trials and has provided data for important SMA research studies. The Registry helps centralize information on this rare genetic disease, provides families a way to learn about research studies and provides researchers a way to find research participants.
Individuals and families affected by SMA are invited to join the Registry. Participants are asked to complete questionnaires about the symptoms, treatment, medications, and other experiences with SMA. Visit the Participant Portal for more information on how to sign up for the Registry.
Participant information is stored in a secure database. Researchers who are interested in studying SMA can request two types of data from the Registry, de-identified information and identifiable information. De-identified information does not contain any names or personal identifiers, and can be given to researchers without having to contact Registry families. Identifiable information includes information that can identify you and will never be released without getting your written permission to do so. Identifiable information includes data such as names and dates of birth. Some researchers may also request contact with families to obtain specific information or to request participation in a research study. In these instances, the Registry will contact each potential participant to ask if they are willing to share their identifiable information for a research project.
The Registry recognizes the importance of global collaboration. Working together with researchers from all over the world is very important in the investigation of SMA. In 2008 the Registry joined the group, Translational Research in Europe for the Assessment and Treatment of Neuromuscular Diseases (TREAT-NMD) in a global collaboration to further the research goals of the neuromuscular disease community. TREAT-NMD has developed a global database that compiles deidentified data transferred from participating registries around the world. The main objective of the TREAT-NMD database is to assess the feasibility of clinical trials, to facilitate the planning of clinical trials and to support the enrollment of patients in clinical trials. This international database will also be used to answer questions regarding the prevalence of particular neuromuscular disorders and assessing the support of other activities such as assessing standards of care and diagnosis.
Study design
- Observational model
- Family based
- Time perspective
- Other
- Target follow-up
- 50 Years
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Individuals or family members of individuals who have been diagnosed with SMA.
Key inclusion criteria (1 listed)
- Individuals or family members of individuals who have been diagnosed with SMA.
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Study is a patient registry
Time frame: unlimited
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Aug 2018.
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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.
Tatiana Foroud, PhD principal investigator
Indiana University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Muscular Atrophy, Spinal, run by Indiana University at 1 site in United States.
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 1 inclusion and 0 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: finished, as of the sponsor's last update on 7 Aug 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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.
What does the primary outcome measure?
Study is a patient registry (time frame: unlimited). It is what the study was designed to measure, not a finding.
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 7 Aug 2018.