A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for Biomarker Analysis

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

36 participants

Study Classification

OBSERVATIONAL

Study Start Date

2013-08-31

Study Completion Date

2014-02-28

Brief Summary

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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.

Detailed Description

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Conditions

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Muscular Atrophy, Spinal

Study Design

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Observational Model Type

CASE_CONTROL

Study Time Perspective

PROSPECTIVE

Study Groups

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Cohort

No interventions assigned to this group

Eligibility Criteria

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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

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Clinical Trials

Role: STUDY_DIRECTOR

Hoffmann-La Roche

Locations

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Kalamazoo, Michigan, United States

Site Status

Salt Lake City, Utah, United States

Site Status

Countries

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United States

Other Identifiers

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BE29002

Identifier Type: -

Identifier Source: org_study_id