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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT01910168
Other study ID # BE29002
Secondary ID
Status Completed
Phase N/A
First received July 25, 2013
Last updated November 1, 2016
Start date August 2013
Est. completion date February 2014

Study information

Verified date November 2016
Source Hoffmann-La Roche
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug Administration
Study type Observational

Clinical Trial Summary

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.


Recruitment information / eligibility

Status Completed
Enrollment 36
Est. completion date February 2014
Est. primary completion date February 2014
Accepts healthy volunteers No
Gender Both
Age group N/A and older
Eligibility 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 Design

Observational Model: Case Control, Time Perspective: Prospective


Related Conditions & MeSH terms


Locations

Country Name City State
n/a

Sponsors (2)

Lead Sponsor Collaborator
Hoffmann-La Roche PTC Therapeutics

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary SMN1/SMN2 detection in blood by mRNA assay 1 day No
Secondary SMN protein level in blood/lymphocytes 1 day No
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