Current trials in SMA
TREAT-NMD is working closely with the pharmaceutical companies planning trials in neuromuscular diseases. The resources and expertise available through the network can make it much easier for companies to do trials, and many of the doctors involved in the network are principal investigators for the trials that are currently ongoing.
Nevertheless, we cannot ourselves keep an up-to-date overview of all the trials that are currently happening across the world. The most comprehensive online listing of trials is at clinicaltrials.gov - companies are obliged to provide details of their trials there.
The table below lists all SMA trials registered or updated on clinicaltrials.gov in the last 1000 days. Because it links directly to clinicaltrials.gov it is automatically updated whenever the information on clinicaltrials.gov changes.
Please note that TREAT-NMD can take no responsibility for the accuracy of the information below, which is pulled directly from clinicaltrials.gov.
Trials for SMA listed on clinicaltrials.gov
A Study to Assess the Safety and Pharmacokinetics of ISIS SMNRx in Infants With Spinal Muscular Atrophy
22nd April 2013
Condition: Spinal Muscular Atrophy
Intervention: Drug: ISIS-SMNRx
Sponsors: Isis Pharmaceuticals; Isis Pharmaceuticals
Recruiting - verified May 2013
An Open-label Safety and Tolerability Study of ISIS SMNRx in Patients With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1
28th January 2013
Condition: Spinal Muscular Atrophy
Intervention: Drug: ISIS-SMNRx
Sponsors: Isis Pharmaceuticals; Isis Pharmaceuticals
Recruiting - verified April 2013
Mechanisms of Cell Death in Spinal Muscular Atrophy
13th December 2012
Condition: Spinal Muscular Atrophy
Intervention:
Sponsors: Nemours Children's Clinic; Nemours Children's Clinic
Enrolling by invitation - verified December 2012
Spinal Muscular Atrophy (SMA) Biomarkers Study in the Immediate Postnatal Period of Development
19th November 2012
Condition: Spinal Muscular Atrophy (SMA)
Intervention:
Sponsors: Ohio State University; Ohio State University; National Institute of Neurological Disorders and Stroke (NINDS); Families of SMA; Massachusetts General Hospital; University of Iowa
Recruiting - verified March 2013
An Open-label Safety, Tolerability and Dose-range Finding Study of Multiple Doses of ISIS SMNRx in Patient With Spinal Muscular Atrophy
8th October 2012
Condition: Spinal Muscular Atrophy
Intervention: Drug: ISIS-SMNRx
Sponsors: Isis Pharmaceuticals; Isis Pharmaceuticals
Recruiting - verified April 2013
Valproate and Levocarnitine in Children With Spinal Muscular Atrophy
13th August 2012
Condition: Spinal Muscular Atrophy
Interventions: Drug: Valproate, Levocarnitine; Drug: Placebo
Sponsors: All India Institute of Medical Sciences, New Delhi; All India Institute of Medical Sciences, New Delhi
Not yet recruiting - verified August 2012
Multi-disease Carrier Screening Test Validation
9th August 2012
Conditions: Spinal Muscular Atrophy (SMA); Carrier Screening; Genetic Testing
Intervention: Procedure: Blood draw
Sponsors: Natera, Inc.; Natera, Inc.
Recruiting - verified August 2012
Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients
5th July 2012
Condition: Spinal Muscular Atrophy
Interventions: Drug: 4-aminopyridine (dalfampridine, Ampyra); Drug: Placebo
Sponsors: Columbia University; Columbia University
Recruiting - verified July 2012
Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe)
30th May 2012
Condition: Spinal Muscular Atrophy
Interventions: Other: Functional tests; Other: Functional tests
Sponsors: Institut de Myologie, France; Institut de Myologie, France
Recruiting - verified June 2012
Continuous Measurement of the Activity for Clinical Evaluation at Home, for Non-ambulant Neuromuscular Patients
30th May 2012
Condition: Neuromuscular Disease
Intervention: Other: Different standardized tasks with an accelerometer device
Sponsors: Institut de Myologie, France; Institut de Myologie, France
Completed - verified April 2013
Infants With Spinal Muscular Atrophy Type I
6th March 2012
Condition: Spinal Muscular Atrophy
Intervention:
Sponsors: National Institutes of Health Clinical Center (CC); National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting - verified November 2012
Spinal Muscular Atrophy and Cardiac Autonomic Function
24th January 2012
Condition: Spinal Muscular Atrophy
Intervention: Procedure: Air stacking manuever
Sponsors: Centro Universitário Augusto Motta; Centro Universitário Augusto Motta
Completed - verified January 2012
An Open-label Safety, Tolerability, and Dose-range Finding Study of ISIS SMNRx in Patients With Spinal Muscular Atrophy
13th December 2011
Condition: Spinal Muscular Atrophy
Intervention: Drug: ISIS-SMNRx
Sponsors: Isis Pharmaceuticals; Isis Pharmaceuticals
Completed - verified April 2013
Flu Vaccine Study in Neuromuscular Patients 2011
22nd August 2011
Conditions: Duchenne Muscular Dystrophy; Spinal Muscular Atrophy; Congenital Muscular Dystrophy
Interventions: Biological: 2011-2012 seasonal flu vaccine; Biological: 2011-2012 seasonal flu vaccine
Sponsors: Children's Hospital Medical Center, Cincinnati; Children's Hospital Medical Center, Cincinnati
Recruiting - verified August 2011
Safety and Efficacy of Olesoxime (TRO19622) in 3-25 Years SMA Patients.
18th February 2011
Conditions: Spinal Muscular Atrophy Type II; Spinal Muscular Atrophy Type III Non Ambulant
Interventions: Drug: Olesoxime; Drug: Placebo
Sponsors: Trophos; Trophos; Association Française contre les Myopathies (AFM), Paris
Active, not recruiting - verified December 2012
Safety and Tolerability of Anakinra in Combination With Riluzol in Amyotrophic Lateral Sclerosis
13th January 2011
Condition: Amyotrophic Lateral Sclerosis (ALS)
Intervention: Drug: Anakinra
Sponsors: Charite University, Berlin, Germany; Charite University, Berlin, Germany; Max Planck Institute for Infection Biology
Recruiting - verified January 2011
Progressive Strength Training in Spinal Muscular Atrophy
19th October 2010
Condition: Muscular Atrophy, Spinal
Intervention: Other: Progressive strength training
Sponsors: University of Utah; University of Utah
Active, not recruiting - verified February 2013
Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA)
14th July 2010
Conditions: Spinal Muscular Atrophy; Neuromuscular Disease
Intervention: Other: Exercise
Sponsors: Columbia University; Columbia University
Recruiting - verified October 2011
SMN Copy Number Distribution in Mali, West Africa
28th January 2010
Condition: Spinal Muscular Atrophy
Intervention:
Sponsors: National Institutes of Health Clinical Center (CC); National Institute of Neurological Disorders and Stroke (NINDS)
Recruiting - verified August 2012
Effects of Power Mobility on Young Children With Severe Motor Impairments
7th December 2009
Conditions: Cerebral Palsy; Arthrogryposis; Spinal Muscular Atrophy; Other Central Nervous System or Musculoskeletal Disorders
Interventions: Other: Power mobility; Other: No intervention
Sponsors: University of Oklahoma; University of Oklahoma
Active, not recruiting - verified February 2012
Development of iPS From Donated Somatic Cells of Patients With Neurological Diseases
2nd April 2009
Condition: Neurodegenerative Disorders
Intervention:
Sponsors: Hadassah Medical Organization; Hadassah Medical Organization
Active, not recruiting - verified November 2012
Study to Evaluate the Efficacy of Riluzole in Children and Young Adults With Spinal Muscular Atrophy (SMA)
16th October 2008
Condition: SMA
Intervention: Drug: Riluzole
Sponsors: Assistance Publique - Hôpitaux de Paris; Assistance Publique - Hôpitaux de Paris
Completed - verified September 2011
A Pilot Study of Biomarkers for Spinal Muscular Atrophy
18th September 2008
Condition: Spinal Muscular Atrophy
Intervention:
Sponsors: New England Research Institutes; New England Research Institutes; The Spinal Muscular Atrophy Foundation
Completed - verified October 2012
CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I
14th April 2008
Condition: Spinal Muscular Atrophy Type I
Intervention: Drug: Valproic Acid and Levocarnitine
Sponsors: University of Utah; University of Utah; Families of Spinal Muscular Atrophy; Sigma Tau Pharmaceuticals, Inc.
Completed - verified November 2012
Pilot Study of Growth Hormon to Treat SMA Typ II and III
12th September 2007
Condition: Muscular Atrophy, Spinal
Interventions: Drug: somatotropin; Drug: Placebo
Sponsors: University Hospital Freiburg; University Hospital Freiburg; Novo Nordisk
Completed - verified February 2013
Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy
10th September 2007
Condition: Spinal Muscular Atrophy
Intervention: Drug: Sodium phenylbutyrate (NaPB)
Sponsors: University of Utah; University of Utah; Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
Active, not recruiting - verified April 2013
Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy
30th May 2007
Condition: Spinal Muscular Atrophy
Interventions: Drug: Valproic Acid (VPA); Drug: Placebo
Sponsors: University of Utah; University of Utah; Families of Spinal Muscular Atrophy; Abbott
Completed - verified May 2012
International SMA Patient Registry
24th April 2007
Condition: Muscular Atrophy, Spinal
Intervention:
Sponsors: Indiana University; Indiana University School of Medicine; Families of Spinal Muscular Atrophy
Recruiting - verified April 2013
Clinical Study of Spinal Muscular Atrophy
1st March 2007
Condition: Spinal Muscular Atrophy
Intervention:
Sponsors: Columbia University; Columbia University; The Spinal Muscular Atrophy Foundation
Active, not recruiting - verified February 2013
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Type I
22nd February 2007
Condition: Spinal Muscular Atrophy Type I
Intervention: Drug: sodium phenylbutyrate
Sponsors: Westat; Westat; National Institute of Neurological Disorders and Stroke (NINDS)
Terminated - verified May 2009
Clinical Trial of Sodium Phenylbutyrate in Children With Spinal Muscular Atrophy Types II or III
21st February 2007
Conditions: Spinal Muscular Atrophy Type II; Spinal Muscular Atrophy Type III
Intervention: Drug: sodium phenylbutyrate
Sponsors: Westat; Westat; National Institute of Neurological Disorders and Stroke (NINDS)
Terminated - verified August 2009
Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy
23rd September 2005
Condition: Spinal Muscular Atrophy
Interventions: Drug: Valproic Acid and Levocarnitine; Drug: Placebo
Sponsors: University of Utah; University of Utah; Families of Spinal Muscular Atrophy; Sigma Tau Pharmaceuticals, Inc.; Abbott
Completed - verified September 2011
Measuring Levels of SMN in Blood Samples of SMA Patients
29th May 2003
Condition: Spinal Muscular Atrophy
Intervention:
Sponsors: National Institutes of Health Clinical Center (CC); National Institute of Neurological Disorders and Stroke (NINDS)
Completed - verified April 2013
