Carbamazapine for Inherited Erythromelalgia Patients With NaV1.7 Mutations

Overview

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.

Full Title of Study: “Pilot Study on the Response of Inherited Erythromelalgia Patients With NaV1.7 Mutations to Carbamazepine: Clinical Imaging Study”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Crossover Assignment
    • Primary Purpose: Treatment
    • Masking: Double (Participant, Care Provider)
  • Study Primary Completion Date: July 2016

Interventions

  • Drug: Carbamazepine
    • 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.
  • Drug: Placebo

Arms, Groups and Cohorts

  • Active Comparator: Carbamazepine
    • 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.
  • Placebo Comparator: Placebo
    • 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.

Clinical Trial Outcome Measures

Primary Measures

  • Carbamazepine Affects Pain in Patients With S241T NaV1.7 IEM Mutation
    • Time Frame: 15 days

Secondary Measures

  • Carbamazepine Affects Mean Duration of Pain Episode
    • Time Frame: 15 days

Participating in This Clinical Trial

Inclusion Criteria

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

  • patients with no identified NaV1.7 mutation

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: 80 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • VA Connecticut Healthcare System
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Stephen Waxman, MD, PhD, Principal Investigator, VAMC West Haven and Yale University

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