Fabry DiseasePhase 2NCT01588314
Safety and Efficacy of Gabapentin for Neuropathic Pain in Fabry Disease
A Phase 2 study testing Gabapentin in people with Fabry Disease and Neuropathic Pain, run by University of Minnesota at 1 site in United States.
Trial summary
The purpose of this study is to determine efficacy of gabapentin vs. placebo at controlling peripheral neuropathic pain in patients with Fabry disease, and reducing their use of opioid analgesics. The investigators are conducting a randomized, double-blind, placebo controlled, single center, cross-over study. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fabry Disease, Neuropathic Pain
- What is being tested?
- Gabapentin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Apr 2012to Jun 2014
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- diagnosis of Fabry Disease
- age ≥ 18 years of age at study enrollment
- current neuropathic pain at any severity level
Who cannot join
- known sensitivity or allergy to study drug
- history of illicit drug use
- pregnancy
- suicidal thoughts at study enrollment as assess by the C-SSRS
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Minnesota, Fariview
Minneapolis, Minnesota, 55455
About this study
Official title: Placebo Controlled Trial Evaluating Gabapentin for the Treatment of Small Fiber Neuropathic Pain in Patients With Fabry Disease
The purpose of this study is to determine efficacy of gabapentin vs. placebo at controlling peripheral neuropathic pain in patients with Fabry disease, and reducing their use of opioid analgesics. The investigators are conducting a randomized, double-blind, placebo controlled, single center, cross-over study. The primary endpoint is percent reduction in patients' use of hydrocodone-acetaminophen.
Read the detailed description
Fabry disease is an X-linked lysosomal storage disorder which results from reduced activity of the enzyme α-galactosidase A. This reduced enzyme activity results in accumulation of globotriaosylceramide (GL-3), which causes disturbances in multiple organ systems. Patients often complain of acroparesthesia, caused by small-fiber neuropathy. Limited information is available regarding effective treatments for small-fiber neuropathic pain in Fabry disease, and no standard-of-care has yet been established. Opioid analgesics are often used because of their pharmacokinetic properties. While effective, the use of opioids has complications such as constipation, physical dependence and addiction. The purpose of this study is to determine efficacy of gabapentin vs. placebo at controlling peripheral neuropathic pain and reducing the use of opioid analgesics. The investigators are conducting a randomized, double-blind, placebo controlled, single center, cross-over study. The primary endpoint is percent reduction in hydrocodone-acetaminophen use.
- Conditions
- Fabry Disease, Neuropathic Pain
- Being tested
- Gabapentin, placebo
- Keywords
- Fabry disease, neuropathic pain, gabapentin
- Medical subject headings
- Fabry Disease, Neuralgia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GabapentinDrug
Also called Neurontin
gabapentin 100 mg capsules
placeboDrug
compounded placebo capsules
| Study group | Role | What they receive |
|---|---|---|
| gabapentin | Active comparator | Not described Receives: Drug: Gabapentin |
| placebo | Placebo comparator | Not described Receives: Drug: placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
average reduction in hydrocodone-acetaminophen use
Measured: assessed at the end of the study
Secondary outcomes (3)
Number and type of adverse events
Measured: assessed at the end of the study
Pain levels
Measured: assessed at the end of the study
Define therapeutic level for gabapentin
Measured: assessed at the end of the study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Apr 2012 (estimated)
- Study startApr 2012
- Primary completionJun 2014
- Study completionJun 2014
Why it stopped: No patients were enrolled. No data \& no study results to report.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- More trials run by University of MinnesotaThe 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
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Minnesota
- Sponsor type
- University, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Responsible party
- Sponsor
- Investigators
- Jeanine R. Jarnes, PharmD, Principal investigator, University of Minnesota, Fairview
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01588314
- Sponsor's protocol ID
- 1112M07943
- Status last verified
- May 2019
- First submitted
- 26 Apr 2012
- Last updated
- 29 May 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Placebo Controlled Trial Evaluating Gabapentin for the Treatment of Small Fiber Neuropathic Pain in Patients With Fabry Disease
- ClinicalTrials.gov ID
- NCT01588314
- Acronym / sponsor ID
- 1112M07943
- Conditions
- Fabry Disease, Neuropathic Pain
- Keywords
- Fabry disease, neuropathic pain, gabapentin
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: No patients were enrolled. No data \& no study results to report.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- University of MinnesotaUniversity, hospital or other
- Collaborators
- Genzyme, a Sanofi Company
- Study start
- Apr 2012
- Primary completion
- Jun 2014 (actual)
- Study completion
- Jun 2014 (actual)
- First posted
- 30 Apr 2012 (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 this study is to determine efficacy of gabapentin vs. placebo at controlling peripheral neuropathic pain in patients with Fabry disease, and reducing their use of opioid analgesics. The investigators are conducting a randomized, double-blind, placebo controlled, single center, cross-over study. The primary endpoint is percent reduction in patients' use of hydrocodone-acetaminophen.
Detailed description
Fabry disease is an X-linked lysosomal storage disorder which results from reduced activity of the enzyme α-galactosidase A. This reduced enzyme activity results in accumulation of globotriaosylceramide (GL-3), which causes disturbances in multiple organ systems. Patients often complain of acroparesthesia, caused by small-fiber neuropathy. Limited information is available regarding effective treatments for small-fiber neuropathic pain in Fabry disease, and no standard-of-care has yet been established. Opioid analgesics are often used because of their pharmacokinetic properties. While effective, the use of opioids has complications such as constipation, physical dependence and addiction. The purpose of this study is to determine efficacy of gabapentin vs. placebo at controlling peripheral neuropathic pain and reducing the use of opioid analgesics. The investigators are conducting a randomized, double-blind, placebo controlled, single center, cross-over study. The primary endpoint is percent reduction in hydrocodone-acetaminophen use.
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 |
|---|---|---|
| gabapentin | Active comparator | Drug: Gabapentin |
| placebo | Placebo comparator | Drug: placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- diagnosis of Fabry Disease
- age ≥ 18 years of age at study enrollment
- current neuropathic pain at any severity level
Key exclusion criteria (4 listed)
- known sensitivity or allergy to study drug
- history of illicit drug use
- pregnancy
- suicidal thoughts at study enrollment as assess by the C-SSRS
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 |
|---|---|---|
| GabapentinAlso: Neurontingabapentin 100 mg capsules | Drug | gabapentin |
| placebocompounded placebo capsules | Drug | placebo |
Primary outcome
average reduction in hydrocodone-acetaminophen use
Time frame: assessed at the end of the study
3 secondary outcomes
Number and type of adverse events
Time frame: assessed at the end of the study
Pain levels
Time frame: assessed at the end of the study
Define therapeutic level for gabapentin
Time frame: assessed at the end of the study
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 May 2019.
Related trials for Fabry Disease
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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.
Jeanine R. Jarnes, PharmD principal investigator
University of Minnesota, Fairview
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 Gabapentin in people with Fabry Disease and Neuropathic Pain, run by University of Minnesota 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 4 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: withdrawn, as of the sponsor's last update on 29 May 2019. 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 was withdrawn before enrolling participants, so there are no results.
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?
average reduction in hydrocodone-acetaminophen use (time frame: assessed at the end of the study). 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 29 May 2019.