Myasthenia Gravis, Myasthenia Gravis, MuSK
Conditions
Brief summary
This Phase 2 proof-of-concept, dose range finding study aims to evaluate the safety and efficacy of 3 dose levels of NMD670 vs placebo in adult patients with MG with antibodies against AChR or MuSK, administered twice a day (BID) for 21 days.
Interventions
DRUGNMD670
Tablets taken twice a day for 21 days
DRUGPlacebo
Tablets taken twice a day for 21 days
Sponsors
NMD Pharma A/S
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Participant must be a male or female being 18 or more, at the time of signing the informed consent * Diagnosis of MG, MGFA class II, III or IV * Documented positive AChR or MuSK antibody test. * Participant must be able to swallow tablets * Body mass index between 18 and 35 kg/m2, inclusive, at screening, and with a minimum weight of 40 kg * Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies * Participant is capable of and has given signed informed consent
Exclusion criteria
* Known medical or psychological condition(s) or risk factor that, in the opinion of the Investigator, might interfere with the patient's full participation in the study, pose any additional risk for the patient, or confound the assessment of the patient or outcome of the study * Participants with other significant clinical and/or laboratory safety findings that may interfere with the conduction or interpretation of the study * Participants that received treatment with an investigational medical product within 30 days or 5 half-lives of the medication, whichever is longer prior to Day 1 * Participants with history of poor compliance with relevant MG therapy * Female patients who plan to become pregnant during the study or are currently pregnant or breastfeeding
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline to day 21 in QMG total score for NMD670 vs placebo | Baseline to day 21 | Scale goes from 0-39 and higher score indicates worse symptomatology |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from baseline to day 21 in MG-ADL total score for NMD670 vs placebo | Baseline to day 21 | Scale goes from 0-24 and higher score indicates worse symptomatology |
| Change from baseline to day 21 in MGC total score for NMD670 vs placebo | Baseline to day 21 | Scale goes from 0-50 and higher score indicate worse symptomatology |
| Change from baseline to day 21 in MG-QOL15r for NMD670 vs placebo | Baseline to day 21 | Scale goes from 0-30 and higher score indicate worse quality of life |
| Change from baseline to day 21 in Neuro-QoL Fatigue Short Form | Baseline to day 21 | Scale goes from 8-40 and higher score indicate worse symptomalogy |
| Incidence of treatment emergent adverse event | Over 21 days of dosing | Summarised per treatment |
| Incidence of serious treatment emergent adverse events | Over 21 days of dosing | Summarised per treatment |
| Incidence of clinically significant abnormalities on physical examinations | Over 21 days of dosing | Summarised per treatment |
| Incidence of clinically significant abnormalities on safety laboratory parameters | Over 21 days of dosing | Summarised per treatment |
| Incidence of clinically significant vital signs abnormalities | Over 21 days of dosing | Summarised per treatment |
| Incidence of clinically significant ECG abnormalities | Over 21 days of dosing | Summarised per treatment |
| Incidence of Suicidal Ideation or Suicidal Behavior | Over 21 days of dosing | Summarised per treatment |
| Incidence of clinically significant abnormalities on opthalmological examinations | From screening (day -28 to day -1) until follow up (day 28) | Summarised per treatment |
Countries
Belgium, Denmark, France, Georgia, Italy, Netherlands, Poland, Serbia, Spain, United States
Contacts
CONTACTNMD Pharma A/S
Outcome results
None listed