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NCT01910168 Clinical Trial

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

Muscular Atrophy, Spinal Clinical Trial

Official title:
A Single Center Study to Collect Samples From SMA Patients for Biomarker Analysis

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
See also
  Status Clinical Trial Phase
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Completed NCT02908685 - A Study to Investigate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Efficacy of Risdiplam (RO7034067) in Type 2 and 3 Spinal Muscular Atrophy (SMA) Participants Phase 2
Recruiting NCT05575011 - A Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115 Phase 1
Completed NCT05073133 - Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (OFELIA) Phase 4
Terminated NCT02240355 - A Study of RO6885247 in Adult and Pediatric Patients With Spinal Muscular Atrophy (MOONFISH) Phase 1
Recruiting NCT05042921 - Pediatric Spinal Muscular Atrophy (SMA) China Registry
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Recruiting NCT05861986 - A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy Phase 4
Completed NCT00466349 - International SMA Patient Registry
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Recruiting NCT05481164 - Newborn Screening for Spinal Muscular Atrophy
Completed NCT04089566 - Study of Nusinersen (BIIB058) in Participants With Spinal Muscular Atrophy Phase 3
Recruiting NCT05861999 - A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy Phase 4
Recruiting NCT05755451 - Natural History of SMA
Active, not recruiting NCT01233817 - Progressive Strength Training in Spinal Muscular Atrophy N/A
Recruiting NCT04317794 - Observational, Postmarketing Surveillance Study of Spinraza Injection (Nusinersen Sodium)
Active, not recruiting NCT05618379 - Adult Spinal Muscular Atrophy (SMA) China Registry
Active, not recruiting NCT04488133 - A Study of Nusinersen Among Participants With Spinal Muscular Atrophy Who Received Onasemnogene Abeparvovec Phase 4
Completed NCT03781479 - Controlled Trial to Evaluate Amifampridine Phosphate in Spinal Muscular Atrophy Type 3 Patients Phase 2
Completed NCT00568698 - A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients Phase 1/Phase 2