Spinal Muscular AtrophyPhase 2VALIANTSMANCT00481013
Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy
A Phase 2 study testing Valproic Acid (VPA) in people with Spinal Muscular Atrophy, run by University of Utah at 1 site in United States.
Trial summary
The primary objective of this proposal is to determine whether oral VPA is effective in treating SMA in adult patients. Read more about this study
- Who is it for?
- Ages 18 to 60 years
- Conditions the study is for
- Spinal Muscular Atrophy
- What is being tested?
- Valproic Acid (VPA)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 33 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2007to Nov 2010
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 60 years
- All sexes
- No healthy volunteers
- Adult
Who may be able to join
- 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).
- Interest in participating and the ability to meet the study requirements.
- Women of child bearing age are required to be on birth control or abstain while participating in the study.
Who cannot join
- Non-ambulatory type 3 adults and all type 2 adults.
- Patients with co-morbid conditions that preclude travel, testing or study medications.
- 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.
- 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.
- Patients with a need for non-invasive ventilatory support (e.g. BiPAP) for > 12 hours/day
- 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
- 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.
- Women who are pregnant or who intend to become pregnant while participating in the research study or who are breastfeeding.
Trial locations and contacts
1 site in 1 country.
United States1 site
Ohio State University Medical Center, Dept. of Neurology
Columbus, Ohio, 43210
About this study
Official title: Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study
The primary objective of this proposal is to determine whether oral VPA is effective in treating SMA in adult patients.
Read the 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
- Being tested
- Valproic Acid (VPA), Placebo
- Keywords
- Spinal Muscular Atrophy, Adult
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Valproic Acid (VPA)Drug
Also called Depakote, Carnitor
Drug: Valproic Acid and Levocarnitine; capsules
PlaceboDrug
For six months, pts are randomized into placebo or treatment. After 6 months, all pts are on treatment
| Study group | Role | What they receive |
|---|---|---|
| 1a | Placebo comparator | For six months, half of patients are randomized into placebo . After 6 months, all patients are on treatment. Receives: Drug: Placebo |
| 1b | Active comparator | Cohort 1b patients are randomized onto treatment. After 6 months, all patients are on drug. Receives: Drug: Valproic Acid (VPA) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 13 months
Secondary outcomes (9)
Change in SMAFRS
Measured: 13 months
Change in strength assessed by hand-held dynamometer
Measured: 13 months
Change in MUNE and CMAP
Measured: 13 months
SMN2 copy number
Measured: 13 months
Change in PFTs, including forced vital capacity (FVC) and negative inspiratory force (NIF)
Measured: 13 months
Change in lean body mass through DEXA scanning
Measured: 13 months
Change in distance walked in 6 minutes
Measured: 13 months
Change in time to climb four standard stairs
Measured: 13 months
Change in health-related QOL assessed through the modified sickness impact profile (SIP)
Measured: 13 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2007 (estimated)
- Study startJul 2007
- Primary completionDec 2009
- Study completionNov 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular AtrophyStudies of the same condition, open and under way.
- More trials run by University of UtahThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 33 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Utah
- Sponsor type
- University, hospital or other
- Collaborators
- Families of Spinal Muscular Atrophy, Abbott
- Responsible party
- Principal investigator, Kathryn Swoboda, University of Utah
- Investigators
- John T Kissel, Principal investigator, Ohio State UniversitySandra P Reyna, M.D., Study director, Families of Spinal Muscular AtrophyKathryn J Swoboda, M.D., Principal investigator, University of Utah
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00481013
- Sponsor's protocol ID
- 2006H0249
- Acronym
- VALIANTSMA
- Status last verified
- Dec 2016
- First submitted
- 30 May 2007
- Last updated
- 6 Dec 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Dec 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Controlled Trial of Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (VALIANTSMA) Study
- ClinicalTrials.gov ID
- NCT00481013
- Acronym / sponsor ID
- VALIANTSMA / 2006H0249
- Conditions
- Spinal Muscular Atrophy
- Keywords
- Spinal Muscular Atrophy, Adult
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 33 participants (actual)
- Lead sponsor
- University of UtahUniversity, hospital or other
- Collaborators
- Families of Spinal Muscular Atrophy, Abbott
- Study start
- Jul 2007
- Primary completion
- Dec 2009 (actual)
- Study completion
- Nov 2010 (actual)
- First posted
- 1 Jun 2007 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 1a | Placebo comparator | Drug: PlaceboFor six months, half of patients are randomized into placebo . After 6 months, all patients are on treatment. |
| 1b | Active comparator | Drug: Valproic Acid (VPA)Cohort 1b patients are randomized onto treatment. After 6 months, all patients are on drug. |
Who may qualify
- Ages
- 18 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (3 listed)
- 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).
- Interest in participating and the ability to meet the study requirements.
- Women of child bearing age are required to be on birth control or abstain while participating in the study.
Key exclusion criteria (8 listed)
- Non-ambulatory type 3 adults and all type 2 adults.
- Patients with co-morbid conditions that preclude travel, testing or study medications.
- 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.
- 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.
- Patients with a need for non-invasive ventilatory support (e.g. BiPAP) for > 12 hours/day
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Valproic Acid (VPA)Also: Depakote, CarnitorDrug: Valproic Acid and Levocarnitine; capsules | Drug | 1b |
| PlaceboFor six months, pts are randomized into placebo or treatment. After 6 months, all pts are on treatment | Drug | 1a |
Primary outcome
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.
Time frame: 13 months
9 secondary outcomes
Change in SMAFRS
Time frame: 13 months
Change in strength assessed by hand-held dynamometer
Time frame: 13 months
Change in MUNE and CMAP
Time frame: 13 months
SMN2 copy number
Time frame: 13 months
Change in PFTs, including forced vital capacity (FVC) and negative inspiratory force (NIF)
Time frame: 13 months
Change in lean body mass through DEXA scanning
Time frame: 13 months
Change in distance walked in 6 minutes
Time frame: 13 months
Change in time to climb four standard stairs
Time frame: 13 months
Change in health-related QOL assessed through the modified sickness impact profile (SIP)
Time frame: 13 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Dec 2016.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
John T Kissel principal investigator
Ohio State University
Sandra P Reyna, M.D. study director
Families of Spinal Muscular Atrophy
Kathryn J Swoboda, M.D. principal investigator
University of Utah
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Valproic Acid (VPA) in people with Spinal Muscular Atrophy, run by University of Utah at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Dec 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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. (time frame: 13 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Dec 2016.