Familial Erythromelalgia
Conditions
Keywords
Erythomelalgia, IEM, Primary Erythomelalgia, SCN9A, NaV1.7, Channelopathy, Red Neuralgia, Neuropathic Pain, Inherited Erythomelalgia
Brief summary
The purpose of this study is to test the potential benefits of BHV-7000 in reducing chronic pain in participants with IEM with a previously demonstrated gain of function mutation in the SCN9A gene.
Interventions
DRUGBHV-7000
Participants will take blinded investigational product (IP) orally once daily
DRUGPlacebo
Matching placebo taken orally once daily
Sponsors
Biohaven Therapeutics Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
CROSSOVER
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
Key Inclusion Criteria: 1. Adult men and women between 18 to 75 years of age, inclusive, at time of consent with a diagnosis of inherited erythromelalgia with a previously characterized gain of function NaV1.7 mutation resulting in chronic pain. 2. Absence of concomitant mutation resulting in Kv7.2/7.3 gain of function. 3. Ability and willingness to adhere to the study procedures and complete accurate pain diaries 4. Stable background analgesic regimen for at least 30 days before screening and willingness to maintain the same analgesic regimen during the study period. Key
Exclusion criteria
1. Any clinically significant laboratory abnormalities or clinically significant abnormalities on screening physical examination, vital signs, or ECG that, in the judgment of the principal investigator, indicates a medical problem that would preclude study participation. 2. Any medical condition, based on the judgement of the Investigator, that would confound the ability to adequately assess safety and efficacy outcome measures
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Mean of the daily average maximum pain intensity scores collected every 2 hours. | The last 3 weeks of each 4-week crossover treatment period | Participants will be asked to record peak (worst) pain experienced in the previous 2 hours using an 11-point Likert scale (0-10) where 0=no pain and 10=worst possible pain |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| The average weekly frequency of pain attacks on treatment vs. placebo | The last 3 weeks of each 4-week crossover treatment period | Participants will be asked to record occurrence of pain attacks |
| The average duration of pain attacks on treatment vs. placebo | The last 3 weeks of each 4-week crossover treatment period | Participants will be asked to record the duration of their pain attacks |
| The average peak severity of pain attacks on treatment vs. placebo | The last 3 weeks of each 4-week crossover treatment period | Participants will be asked to record the maximum severity of their pain attacks using an 11-point Likert scale (0-10) where 0=no pain and 10=worst possible pain. |
| Safety and tolerability by reporting the frequency of unique participants with SAEs, severe AEs, AEs leading to discontinuation, deaths, and Grade 3-4 (CTCAE/DAIDS) laboratory abnormalities. | Up to 16 weeks | Measured by assessing the number of unique participants who experience treatment-emergent serious adverse events, adverse events leading to discontinuation, or moderate and severe adverse events. |
Countries
United States
Outcome results
None listed