Amyotrophic Lateral SclerosisPhase 1/2NCT00107770
Safety Study of Oral Sodium Phenylbutyrate in Subjects With ALS (Amyotrophic Lateral Sclerosis)
A Phase 1/2 study testing sodium phenylbutyrate in people with Amyotrophic Lateral Sclerosis, run by US Department of Veterans Affairs at 10 sites in United States.
Trial summary
The purpose of the study is to evaluate the safety of sodium phenylbutyrate (NaPB) treatment in subjects with amyotrophic lateral sclerosis (ALS) and the ability to take this medication without major side effects. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis
- What is being tested?
- sodium phenylbutyrate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 40 participants
- Where
- 10 sitesUnited States
- Study dates
- Apr 2005to Sep 2007
- Phase
- Phase 1/2
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Diagnosed with ALS
- At least 18 years of age
- Women, who can become pregnant, must actively use effective birth control measures
Who cannot join
- Must not have any other neurological (nervous system) disease
- Veterans only are eligible to participate at VA sites.
Trial locations and contacts
10 sites in 1 country.
10 sites in 1 country
United States10 sites
VA Maryland Health Care System, Baltimore
Baltimore, Maryland, 21201
Edith Nourse Rogers Memorial Veterans Hospital, Bedford
Bedford, Massachusetts, 01730
VA Medical Center, Jamaica Plain Campus
Boston, Massachusetts, 02130
VA Medical Center, Cincinnati
Cincinnati, Ohio, 45220
VA Medical Center, Durham
Durham, North Carolina, 27705
Michael E. DeBakey VA Medical Center (152)
Houston, Texas, 77030
VA Medical Center, Iowa City
Iowa City, Iowa, 52246-2208
VA Medical Center, Lexington
Lexington, Kentucky, 40502
VA Pittsburgh Health Care System
Pittsburgh, Pennsylvania, 15240
VA Medical Center, Syracuse
Syracuse, New York, 13210
About this study
Official title: Safety and Dose Escalating Study of Oral Sodium Phenylbutyrate in Subjects With Amyotrophic Lateral Sclerosis
The purpose of the study is to evaluate the safety of sodium phenylbutyrate (NaPB) treatment in subjects with amyotrophic lateral sclerosis (ALS) and the ability to take this medication without major side effects.
Read the detailed description
Although it is known that nerve cells die in the brains and spinal cords of patients who have ALS, the cause of the cell death is unknown. There is evidence that this cell death may be caused by changes in DNA, the body's genetic material. Drugs such as sodium phenylbutyrate (NaPB) can increase the expression of genes, block how the motor nerve cells in ALS die, and may prove to be an effective therapy for ALS. NaPB has shown an improvement in survival in mice with conditions similar to ALS.
STUDY DESIGN:
All research participants will take sodium phenylbutyrate for a total of 20 weeks. The dose of medication will be increased every 2 to 4 weeks until a maximum, easily tolerated dose is achieved (study maximum is 21 g/day).
- Conditions
- Amyotrophic Lateral Sclerosis
- Being tested
- sodium phenylbutyrate
- Keywords
- ALS, histone deacetylase inhibition, open label, safety study, sodium phenylbutyrate
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
sodium phenylbutyrateDrug
histone deacteylase inhibitor
| Study group | Role | What they receive |
|---|---|---|
| 1 | Other | ALS patient Receives: Drug: sodium phenylbutyrate |
What is being measured
The primary outcome is the main question the study is designed to answer.
safety and tolerability
Measured: 20 weeks
Secondary outcomes (2)
The number of side effects at each dosage, including abnormalities in vital signs, physical examination, blood tests and EKGs, change in vital capacity (breathing function) and ALS functional rating scale
Relationship between blood levels and sodium phenylbutyrate dosage
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Apr 2005 (estimated)
- Study startApr 2005
- Primary completionNov 2006
- Study completionSep 2007
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- 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 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 40 participants (actual)
Sponsor and investigators
- Lead sponsor
- US Department of Veterans Affairs
- Sponsor type
- US federal agency
- Collaborators
- Muscular Dystrophy Association
- Investigators
- Robert Ferrante, PhD MSc, Principal investigator, Edith Nourse Rogers Memorial Veterans Hospital, Bedford
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00107770
- Sponsor's protocol ID
- 0015
- Status last verified
- Oct 2008
- First submitted
- 7 Apr 2005
- Last updated
- 11 Jan 2010
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 11 Jan 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Dose Escalating Study of Oral Sodium Phenylbutyrate in Subjects With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT00107770
- Acronym / sponsor ID
- 0015
- Conditions
- Amyotrophic Lateral Sclerosis
- Keywords
- ALS, histone deacetylase inhibition, open label, safety study, sodium phenylbutyrate
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (actual)
- Lead sponsor
- US Department of Veterans AffairsUS federal agency
- Collaborators
- Muscular Dystrophy Association
- Study start
- Apr 2005
- Primary completion
- Nov 2006 (actual)
- Study completion
- Sep 2007 (actual)
- First posted
- 8 Apr 2005 (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 purpose of the study is to evaluate the safety of sodium phenylbutyrate (NaPB) treatment in subjects with amyotrophic lateral sclerosis (ALS) and the ability to take this medication without major side effects.
Detailed description
Although it is known that nerve cells die in the brains and spinal cords of patients who have ALS, the cause of the cell death is unknown. There is evidence that this cell death may be caused by changes in DNA, the body's genetic material. Drugs such as sodium phenylbutyrate (NaPB) can increase the expression of genes, block how the motor nerve cells in ALS die, and may prove to be an effective therapy for ALS. NaPB has shown an improvement in survival in mice with conditions similar to ALS.
STUDY DESIGN:
All research participants will take sodium phenylbutyrate for a total of 20 weeks. The dose of medication will be increased every 2 to 4 weeks until a maximum, easily tolerated dose is achieved (study maximum is 21 g/day).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Other | Drug: sodium phenylbutyrateALS patient |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Diagnosed with ALS
- At least 18 years of age
- Women, who can become pregnant, must actively use effective birth control measures
Key exclusion criteria (2 listed)
- Must not have any other neurological (nervous system) disease
- Veterans only are eligible to participate at VA sites.
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 |
|---|---|---|
| sodium phenylbutyratehistone deacteylase inhibitor | Drug | 1 |
Primary outcome
safety and tolerability
Time frame: 20 weeks
2 secondary outcomes
The number of side effects at each dosage, including abnormalities in vital signs, physical examination, blood tests and EKGs, change in vital capacity (breathing function) and ALS functional rating scale
Relationship between blood levels and sodium phenylbutyrate dosage
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 11 Jan 2010.
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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.
Robert Ferrante, PhD MSc principal investigator
Edith Nourse Rogers Memorial Veterans Hospital, Bedford
10 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing sodium phenylbutyrate in people with Amyotrophic Lateral Sclerosis, run by US Department of Veterans Affairs at 10 sites 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 2 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 11 Jan 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
safety and tolerability (time frame: 20 weeks). 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 11 Jan 2010.