ErythromelalgiaPhase 4NCT02214615
Carbamazapine for Inherited Erythromelalgia Patients With NaV1.7 Mutations
A Phase 4 study testing Carbamazepine in people with Erythromelalgia, run by VA Connecticut Healthcare System at 2 sites in United States.
Trial summary
This research study is designed to investigate brain response using fMRI scan, and behavioral responses, to treatment with the drug carbamazepine (CBZ) in patients with the painful sodium channelopathy inherited Erythromelalgia (IEM). Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Erythromelalgia
- What is being tested?
- Carbamazepine
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 2 participants
- Where
- 2 sitesUnited States
- Study dates
- Apr 2014to Jul 2016
- Phase
- Phase 4
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- diagnosis/symptoms of EM
- specific NaV1.7 sodium channel mutations (including S241T)
Who cannot join
- patients with no identified NaV1.7 mutation
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Yale University
New Haven, Connecticut, 06520
VA Connecticut Healthcare System
West Haven, Connecticut, 06516
About this study
Official title: Pilot Study on the Response of Inherited Erythromelalgia Patients With NaV1.7 Mutations to Carbamazepine: Clinical Imaging Study
This research study is designed to investigate brain response using fMRI scan, and behavioral responses, to treatment with the drug carbamazepine (CBZ) in patients with the painful sodium channelopathy inherited Erythromelalgia (IEM). This study is designed to identify the central nervous system (CNS) regions that are activated during ongoing or evoked pain attacks, and the altered CNS response to CBZ treatment. This will advance our understanding of how IEM affects the brain. We also hope to validate a pharmacogenic approach to the study of IEM by use of an FDA approved drug. We hope, but cannot be sure, that subjects will directly benefit from this study.
- Conditions
- Erythromelalgia
- Being tested
- Carbamazepine, Placebo
- Medical subject headings
- Erythromelalgia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CarbamazepineDrug
Also called Tegretol
Day 1: Take 200 mg twice a day. Day 2: Take 200 mg twice a day. Day 3: Take 200 mg twice a day. Day 4: Take 200 mg twice a day. Day 5: Take 400 mg twice a day. Day 6: Take 400 mg twice a day. Day 7: Take 400 mg twice a day. Day 8: Take 400 mg twice a day. Day 9: Take 600 mg twice a day. Day 10: Take 600 mg twice a day. Day 11: Take 600 mg twice a day. Day 12: Take 600 mg twice a day. Day 13: Take 800 mg capsules twice a day. Day 14: Take 800 mg twice a day. Day 15: 800 mg twice a day. Day 16: Take 800 mg twice a day.
Taper Down (After Visit 4 and 7) If maximal dose of 800 mg/day has been achieved, tapering down will take 9 days Taper down for 600 mg/day will take 6 days, and for 400 mg/day will take 3 days.
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| Carbamazepine | Active comparator | Administered in gradual doses from 200 mg twice a day increasing to no more than 800 mg twice a day if symptoms do not reduce. After 2 weeks of steady dose drug will be tapered down every 3 days. Receives: Drug: Carbamazepine |
| Placebo | Placebo comparator | Administered in gradual doses from 200 mg twice a day increasing to no more than 800 mg twice a day if symptoms do not reduce to match dosing with carbamazepine. After 2 weeks of steady dose drug will be tapered down every 3 days. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Carbamazepine Affects Pain in Patients With S241T NaV1.7 IEM Mutation
Measured: 15 days
Secondary outcomes (1)
Carbamazepine Affects Mean Duration of Pain Episode
Measured: 15 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2014 (estimated)
- Study startApr 2014
- Primary completionJul 2016
- Study completionJul 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for ErythromelalgiaStudies 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind
- Who is masked
- Participant, Care provider
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- VA Connecticut Healthcare System
- Sponsor type
- US federal agency
- Responsible party
- Sponsor
- Investigators
- Stephen Waxman, MD, PhD, Principal investigator, VAMC West Haven and Yale University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02214615
- Sponsor's protocol ID
- SW0023
- Status last verified
- May 2017
- First submitted
- 10 Aug 2014
- Last updated
- 6 Jun 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jun 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study on the Response of Inherited Erythromelalgia Patients With NaV1.7 Mutations to Carbamazepine: Clinical Imaging Study
- ClinicalTrials.gov ID
- NCT02214615
- Acronym / sponsor ID
- SW0023
- Conditions
- Erythromelalgia
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- VA Connecticut Healthcare SystemUS federal agency
- Study start
- Apr 2014
- Primary completion
- Jul 2016 (actual)
- Study completion
- Jul 2016 (actual)
- First posted
- 12 Aug 2014 (estimated)
- Results
- Posted 6 Jun 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study is designed to investigate brain response using fMRI scan, and behavioral responses, to treatment with the drug carbamazepine (CBZ) in patients with the painful sodium channelopathy inherited Erythromelalgia (IEM). This study is designed to identify the central nervous system (CNS) regions that are activated during ongoing or evoked pain attacks, and the altered CNS response to CBZ treatment. This will advance our understanding of how IEM affects the brain. We also hope to validate a pharmacogenic approach to the study of IEM by use of an FDA approved drug. We hope, but cannot be sure, that subjects will directly benefit from this study.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Double blind (participant, care provider)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Carbamazepine | Active comparator | Drug: CarbamazepineAdministered in gradual doses from 200 mg twice a day increasing to no more than 800 mg twice a day if symptoms do not reduce. After 2 weeks of steady dose drug will be tapered down every 3 days. |
| Placebo | Placebo comparator | Drug: PlaceboAdministered in gradual doses from 200 mg twice a day increasing to no more than 800 mg twice a day if symptoms do not reduce to match dosing with carbamazepine. After 2 weeks of steady dose drug will be tapered down every 3 days. |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- diagnosis/symptoms of EM
- specific NaV1.7 sodium channel mutations (including S241T)
Key exclusion criteria (1 listed)
- patients with no identified NaV1.7 mutation
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 |
|---|---|---|
| CarbamazepineAlso: TegretolDay 1: Take 200 mg twice a day. Day 2: Take 200 mg twice a day. Day 3: Take 200 mg twice a day. Day 4: Take 200 mg twice a day. Day 5: Take 400 mg twice a day. Day 6: Take 400 mg twice a day. Day 7: Take 400 mg twice a day. Day 8: Take 400 mg twice a day. Day 9: Take 600 mg twice a day. Day 10: Take 600 mg twice a day. Day 11: Take 600 mg twice a day. Day 12: Take 600 mg twice a day. Day 13: Take 800 mg capsules twice a day. Day 14: Take 800 mg twice a day. Day 15: 800 mg twice a day. Day 16: Take 800 mg twice a day. Taper Down (After Visit 4 and 7) If maximal dose of 800 mg/day has been achieved, tapering down will take 9 days Taper down for 600 mg/day will take 6 days, and for 400 mg/day will take 3 days. | Drug | Carbamazepine |
| Placebo | Drug | Placebo |
Primary outcome
Carbamazepine Affects Pain in Patients With S241T NaV1.7 IEM Mutation
Time frame: 15 days
1 secondary outcome
Carbamazepine Affects Mean Duration of Pain Episode
Time frame: 15 days
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jun 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Stephen Waxman, MD, PhD principal investigator
VAMC West Haven and Yale University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Carbamazepine in people with Erythromelalgia, run by VA Connecticut Healthcare System at 2 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 2 inclusion and 1 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 Jun 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 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. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 6 Jun 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Carbamazepine Affects Pain in Patients With S241T NaV1.7 IEM Mutation (time frame: 15 days). 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 Jun 2017.