NCT02214615

Brief 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). 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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
2

participants targeted

Target at below P25 for phase_4

Timeline
Completed

Started Apr 2014

Typical duration for phase_4

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2014

Completed
4 months until next milestone

First Submitted

Initial submission to the registry

August 10, 2014

Completed
2 days until next milestone

First Posted

Study publicly available on registry

August 12, 2014

Completed
1.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2016

Completed
11 months until next milestone

Results Posted

Study results publicly available

June 6, 2017

Completed
Last Updated

June 6, 2017

Status Verified

May 1, 2017

Enrollment Period

2.3 years

First QC Date

August 10, 2014

Results QC Date

June 15, 2016

Last Update Submit

May 8, 2017

Conditions

Erythromelalgia

Outcome Measures

Primary Outcomes (1)

  • Carbamazepine Affects Pain in Patients With S241T NaV1.7 IEM Mutation

    15 days

Secondary Outcomes (1)

  • Carbamazepine Affects Mean Duration of Pain Episode

    15 days

Study Arms (2)

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.

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.

Drug: Placebo

Interventions

CarbamazepineDRUG

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.

Also known as: Tegretol
Carbamazepine
PlaceboDRUG
Placebo

Eligibility Criteria

Age18 Years - 80 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • diagnosis/symptoms of EM
  • specific NaV1.7 sodium channel mutations (including S241T)

You may not qualify if:

  • patients with no identified NaV1.7 mutation

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Yale University

New Haven, Connecticut, 06520, United States

Location

VA Connecticut Healthcare System

West Haven, Connecticut, 06516, United States

Location

MeSH Terms

Conditions

Erythromelalgia

Interventions

Carbamazepine

Condition Hierarchy (Ancestors)

Peripheral Vascular DiseasesVascular DiseasesCardiovascular Diseases

Intervention Hierarchy (Ancestors)

DibenzazepinesHeterocyclic Compounds, 3-RingHeterocyclic Compounds, Fused-RingHeterocyclic Compounds

Limitations and Caveats

all subjects had to have erythromelalgia SCN9A mutation S241T

Results Point of Contact

Title
Stephen Waxman
Organization
VAMC and Yale University
stephen.waxman@yale.edu
203-937-3802

Study Officials

  • Stephen Waxman, MD, PhD

    VAMC West Haven and Yale University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
No
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 4
Allocation
RANDOMIZED
Masking
DOUBLE
Who Masked
PARTICIPANT, CARE PROVIDER
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
FED
Responsible Party
SPONSOR

Study Record Dates

First Submitted

August 10, 2014

First Posted

August 12, 2014

Study Start

April 1, 2014

Primary Completion

July 1, 2016

Study Completion

July 1, 2016

Last Updated

June 6, 2017

Results First Posted

June 6, 2017

Record last verified: 2017-05

Locations

US(2)