Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT00481013
Other study ID # 2006H0249
Secondary ID
Status Completed
Phase Phase 2
First received May 30, 2007
Last updated December 5, 2016
Start date July 2007
Est. completion date November 2010

Study information

Verified date December 2016
Source University of Utah
Contact n/a
Is FDA regulated No
Health authority United States: Institutional Review Board
Study type Interventional

Clinical Trial Summary

The primary objective of this proposal is to determine whether oral VPA is effective in treating SMA in adult patients.


Description:

Participation in this study entails six visits and seven to eight blood draws over 13 months. Each visit entails a stay of two days and one night at the General Clinical Research Center (GCRC).

Subjects who live within driving distance will be allowed to participate in the study without an overnight stay through two consecutive outpatient visits. All subjects will be evaluated at two screening visits 2-4 weeks apart to determine eligibility for participation. Eligible subjects will be randomized to receive VPA or placebo for the first six months. At the six-month visit, patients will be evaluated and crossed over to the other regimen.


Recruitment information / eligibility

Status Completed
Enrollment 33
Est. completion date November 2010
Est. primary completion date December 2009
Accepts healthy volunteers No
Gender Both
Age group 18 Years to 60 Years
Eligibility Inclusion Criteria:

1. Ambulatory adults with SMA 3 ages 18-60. The diagnosis of SMA must be documented by the homozygous deletion of both SMN1 genes on standard genetic tests for the disorder. Patients must be able to walk thirty feet without assistance (i.e. no canes, walkers).

2. Interest in participating and the ability to meet the study requirements.

3. Women of child bearing age are required to be on birth control or abstain while participating in the study.

Exclusion Criteria:

1. Non-ambulatory type 3 adults and all type 2 adults.

2. Patients with co-morbid conditions that preclude travel, testing or study medications.

3. Patients who have participated in a treatment trial for SMA in the 3 months prior to this trial, or plan on enrolling in any other treatment trial during the duration of this trial.

4. Patients who are, in the investigator's opinion, mentally or legally incapacitated from providing informed consent for the study, or are otherwise unable to meet study requirements or cooperate reliably with study procedures, especially strength testing.

5. Patients with a need for non-invasive ventilatory support (e.g. BiPAP) for > 12 hours/day

6. Transaminases, amylase or lipase > 3.0 x normal values, WBC < 3.0 or neutropenia < 1.0, platelet count < 100 K, or hematocrit < 30 persisting over a 30 day period

7. Use of medications or supplements which interfere with VPA metabolism and increase the potential risks of the medications, or are hypothesized to have a beneficial effect in SMA animal models or human neuromuscular disorders within 3 months of study enrollment. These agents include riluzole, creatine, butyrate derivatives, growth hormone, anabolic steroids, daily albuterol use, anticonvulsants, or other HDAC inhibitors.

8. Women who are pregnant or who intend to become pregnant while participating in the research study or who are breastfeeding.

Study Design

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Crossover Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment


Intervention

Drug:
Valproic Acid (VPA)
Drug: Valproic Acid and Levocarnitine; capsules
Placebo
For six months, pts are randomized into placebo or treatment. After 6 months, all pts are on treatment

Locations

Country Name City State
United States Ohio State University Medical Center, Dept. of Neurology Columbus Ohio

Sponsors (3)

Lead Sponsor Collaborator
University of Utah Abbott, Families of Spinal Muscular Atrophy

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary The primary outcome for the study is change in muscle strength from baseline to six months in muscle strength as assessed by MVICT using a fixed testing system. 13 months No
Secondary Change in SMAFRS 13 months No
Secondary Change in strength assessed by hand-held dynamometer 13 months No
Secondary Change in MUNE and CMAP 13 months No
Secondary SMN2 copy number 13 months No
Secondary Change in PFTs, including forced vital capacity (FVC) and negative inspiratory force (NIF) 13 months No
Secondary Change in lean body mass through DEXA scanning 13 months No
Secondary Change in distance walked in 6 minutes 13 months No
Secondary Change in time to climb four standard stairs 13 months No
Secondary Change in health-related QOL assessed through the modified sickness impact profile (SIP) 13 months No
See also
  Status Clinical Trial Phase
Completed NCT04851873 - Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) Phase 3
Completed NCT03223051 - Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy N/A
Completed NCT04335942 - Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor N/A
Recruiting NCT05794139 - Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy Phase 2
Not yet recruiting NCT06300996 - Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Limb N/A
Not yet recruiting NCT00961103 - Motor Development and Orthoses in Spinal Muscular Atrophy (SMA) N/A
Completed NCT02003937 - Aerobic Training in Patients With Spinal Muscular Atrophy Type III N/A
Completed NCT00227266 - Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy Phase 2
Completed NCT00374075 - Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy Phase 1
Enrolling by invitation NCT05539456 - Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old
Recruiting NCT05779956 - Personalized Medicine for SMA: a Translational Project
Recruiting NCT03217578 - Neonatal Spinal Muscular Atrophy (SMA) Screening
Recruiting NCT03300869 - Natural History of Types 2 and 3 SMA in Taiwan
Completed NCT01703988 - An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Phase 1/Phase 2
Withdrawn NCT02235090 - Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting N/A
Completed NCT02123186 - Newborn Screening for Spinal Muscular Atrophy N/A
Completed NCT00756821 - A Pilot Study of Biomarkers for Spinal Muscular Atrophy N/A
Completed NCT00004771 - Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease Phase 2
Recruiting NCT05366465 - Quality of Life and Participation of the Adult With Spinal Muscular Atrophy in France
Recruiting NCT06310421 - Spinal Muscular Atrophy Neonatal Screening Program