- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01910168
A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis
November 1, 2016 updated by: Hoffmann-La Roche
A Single Center Study to Collect Samples From SMA Patients for Biomarker Analysis
In this single center study blood samples for biomarker analysis will be collected from patients with spinal muscular atrophy.
Up to 21 mL blood will be drawn from eligible patients at a single visit.
Study Overview
Status
Completed
Conditions
Study Type
Observational
Enrollment (Actual)
36
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Michigan
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Kalamazoo, Michigan, United States, 49007
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Utah
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Salt Lake, Utah, United States, 84108
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Sampling Method
Probability Sample
Study Population
Patients with spinal muscular atrophy
Description
Inclusion Criteria:
- Self-identified as 5q-autosomal recessive spinal muscular atrophy (SMA) type I, II or III as judged by their neurologist upon diagnosis
- Ability and willingness to provide blood samples
- Willingness (by the patient or patient's parents or legal guardian) to complete to their best ability a questionnaire which requests specific clinical and genetic information
- Able to participate and willing to give written informed consent or assent. Informed consent will be obtained from the patient, or the patient's parent or legal guardian.
Exclusion Criteria:
- Any known genetic condition other than spinal muscular atrophy, unless it is not interfering with the purpose of this study based on the Sponsor's judgment
- Participation in a clinical trial (except observational studies) within the previous 14 days
- Donation of blood or significant blood loss within three months prior to screening
- Concomitant disease or condition that could interfere with, or treatment of which might interfere with, the conduct of this study, or that would, in the opinion of the investigator, pose an unacceptable risk to the patient in this study
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
Cohorts and Interventions
Group / Cohort |
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Cohort
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
SMN1/SMN2 detection in blood by mRNA assay
Time Frame: 1 day
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1 day
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
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SMN protein level in blood/lymphocytes
Time Frame: 1 day
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1 day
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start
August 1, 2013
Primary Completion (Actual)
February 1, 2014
Study Completion (Actual)
February 1, 2014
Study Registration Dates
First Submitted
July 25, 2013
First Submitted That Met QC Criteria
July 26, 2013
First Posted (Estimate)
July 29, 2013
Study Record Updates
Last Update Posted (Estimate)
November 2, 2016
Last Update Submitted That Met QC Criteria
November 1, 2016
Last Verified
November 1, 2016
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- BE29002
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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