Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy

NCT ID: NCT00481013

Last Updated: 2016-12-06

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 33 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2007-07-31
• Study Completion Date: 2010-11-30

Brief Summary

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

Detailed 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.

Conditions

Spinal Muscular Atrophy

Keywords

Spinal Muscular Atrophy; Adult

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

1a

For six months, half of patients are randomized into placebo . After 6 months, all patients are on treatment.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

For six months, pts are randomized into placebo or treatment. After 6 months, all pts are on treatment

1b

Cohort 1b patients are randomized onto treatment. After 6 months, all patients are on drug.

Group Type: ACTIVE_COMPARATOR

Valproic Acid (VPA)

Intervention Type: DRUG

Drug: Valproic Acid and Levocarnitine; capsules

Interventions

Valproic Acid (VPA)

Drug: Valproic Acid and Levocarnitine; capsules

Intervention Type: DRUG

Placebo

For six months, pts are randomized into placebo or treatment. After 6 months, all pts are on treatment

Intervention Type: DRUG

Other Intervention Names

Depakote; Carnitor

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 60 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Families of Spinal Muscular Atrophy

OTHER

Sponsor Role: collaborator

Abbott

INDUSTRY

Sponsor Role: collaborator

University of Utah

OTHER

Sponsor Role: lead

Responsible Party

Kathryn Swoboda

Associate Professor, Neurology and Pediatrics

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

John T Kissel

Role: PRINCIPAL_INVESTIGATOR

Ohio State University

Sandra P Reyna, M.D.

Role: STUDY_DIRECTOR

Families of Spinal Muscular Atrophy

Kathryn J Swoboda, M.D.

Role: PRINCIPAL_INVESTIGATOR

University of Utah

Locations

Ohio State University Medical Center, Dept. of Neurology

Columbus, Ohio, United States

Countries

United States

Other Identifiers

2006H0249

Identifier Type: -

Identifier Source: org_study_id