Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis
- Conditions
- Myasthenia GravisMyasthenia Gravis, MuSK
- Interventions
- Drug: Placebo
- Registration Number
- NCT06414954
- Lead Sponsor
- NMD Pharma A/S
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 84
- 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
- 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
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description NMD670 high dose NMD670 - NMD670 mid dose NMD670 - Placebo Placebo - NMD670 low dose NMD670 -
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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
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
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 Neuro-QoL Fatigue Short Form Baseline to day 21 Scale goes from 8-40 and higher score indicate worse symptomalogy
Incidence of clinically significant abnormalities on safety laboratory parameters 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
Incidence of clinically significant vital signs abnormalities Over 21 days of dosing Summarised per treatment
Trial Locations
- Locations (37)
Galen Clinic
🇵🇱Lublin, Poland
Centrum Medyczne Neuro Protect
🇵🇱Warsaw, Poland
Profound Research LLC
🇺🇸Carlsbad, California, United States
University of Colorado Neuromuscular Division
🇺🇸Aurora, Colorado, United States
University of California Irvine Medical Center
🇺🇸Irvine, California, United States
SFM Clinical Research, LLC
🇺🇸Boca Raton, Florida, United States
NextGen Precision Health
🇺🇸Columbia, Missouri, United States
University of Oregon
🇺🇸Portland, Oregon, United States
Neuromuscular Research Division | University of South Florida
🇺🇸Tampa, Florida, United States
Augusta University, Neuroscience Center
🇺🇸Augusta, Georgia, United States
The University of North Carolina at Chapel Hill
🇺🇸Chapel Hill, North Carolina, United States
Semmes Murphey Clinic
🇺🇸Memphis, Tennessee, United States
UZ Leuven
🇧🇪Leuven, Belgium
Centre de Référence des Maladies Neuromusculaires et de la SLA
🇫🇷Marseille, France
LTD David Tatishvili Health Center
🇬🇪Tbilisi, Georgia
Ltd New Hospitals
🇬🇪Tbilisi, Georgia
Aarhus University Hospital
🇩🇰Aarhus, Denmark
Rigshospitalet University of Copenhagen
🇩🇰Copenhagen, Denmark
Centre de référence des maladies neuromusculaires AOC-CHU de Bordeaux - Hôpital Pellegrin
🇫🇷Bordeaux, France
ASST Papa Giovanni XXIII (Azienda Ospedaliera Papa Giovanni XXIII)
🇮🇹Bergamo, Italy
Centre Hospitalier Universitaire de Nantes - Hôtel Dieu Centre de Référence des Maladies Neuromusculaires Rares -
🇫🇷Nantes, France
Unité de Recherche Clinique NeuroSciences
🇫🇷Nice, France
JSC Curatio
🇬🇪Tbilisi, Georgia
IRCCS Istituo delle Scienze neurologiche di Bologna, UOC Clinica neurologica Ospedale
🇮🇹Bologna, Italy
IRCCS Ospedale Policlinico San Martino
🇮🇹Genova, Italy
IRCCS Fondazione Istituto Neurologico Carlo Besta, Dipartimento di Ricerca e Sviluppo clinico
🇮🇹Milano, Italy
DIMER, IRCCS, Ospedale San Raffaele
🇮🇹Milano, Italy
Azienda Ospedaliera Sant'Andrea - Universita di Roma La Sapienza
🇮🇹Roma, Italy
Leiden Universitair Medisch Centrum (LUMC)
🇳🇱Leiden, Netherlands
Neurologia Śląska Centrum Medyczne
🇵🇱Katowice, Poland
Centro Sclerosi Multipla Napoli - AOU Vanvitelli
🇮🇹Napoli, Italy
AOU Pisana
🇮🇹Pisa, Italy
Szpital Specjalistyczny im. Ludwika Rydygiera w Krakowie sp.z o.o
🇵🇱Kraków, Poland
Centrum Medyczne Hope Clinic
🇵🇱Lublin, Poland
University Clinical Center of Serbia, Neurology Clinic
🇷🇸Belgrad, Serbia
University Clinical Center Nis, Neurology Clinic
🇷🇸Niš, Serbia
Hospìtal Universitari Vall d'Hebrón
🇪🇸Barcelona, Spain