Clinical trial NCT06414954: Safety and Efficacy of 3 Dose Levels of NMD670 in Adult Patients With Myasthenia Gravis
Official title: A Phase 2b, Randomised, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of 3 Dose Levels of NMD670 Over 21 Days in Adult Patients With AChR/MuSK-Ab+ Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE2
Conditions: Myasthenia Gravis
Myasthenia Gravis, MuSK
Lead sponsor: NMD Pharma A/S
Sponsor class: INDUSTRY
First posted: 2024-05-16
Enrollment: 84
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.


Eligibility criteria: 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

Primary outcomes:
measure: Change from baseline to day 21 in QMG total score for NMD670 vs placebo
timeFrame: Baseline to day 21
Secondary outcomes:
measure: Change from baseline to day 21 in MG-ADL total score for NMD670 vs placebo
timeFrame: Baseline to day 21
measure: Change from baseline to day 21 in MGC total score for NMD670 vs placebo
timeFrame: Baseline to day 21
measure: Change from baseline to day 21 in MG-QOL15r for NMD670 vs placebo
timeFrame: Baseline to day 21
measure: Change from baseline to day 21 in Neuro-QoL Fatigue Short Form
timeFrame: Baseline to day 21
measure: Incidence of treatment emergent adverse event
timeFrame: Over 21 days of dosing
measure: Incidence of serious treatment emergent adverse events
timeFrame: Over 21 days of dosing
measure: Incidence of clinically significant abnormalities on physical examinations
timeFrame: Over 21 days of dosing
measure: Incidence of clinically significant abnormalities on safety laboratory parameters
timeFrame: Over 21 days of dosing
measure: Incidence of clinically significant vital signs abnormalities
timeFrame: Over 21 days of dosing
measure: Incidence of clinically significant ECG abnormalities
timeFrame: Over 21 days of dosing
measure: Incidence of Suicidal Ideation or Suicidal Behavior
timeFrame: Over 21 days of dosing
measure: Incidence of clinically significant abnormalities on opthalmological examinations
timeFrame: From screening (day -28 to day -1) until follow up (day 28)
Other outcomes:
Locations:
city: Carlsbad
country: United States
facility: Profound Research LLC
state: California
city: Irvine
country: United States
facility: University of California Irvine Medical Center
state: California
city: Aurora
country: United States
facility: University of Colorado Neuromuscular Division
state: Colorado
city: Boca Raton
country: United States
facility: SFM Clinical Research, LLC
state: Florida
city: Tampa
country: United States
facility: Neuromuscular Research Division | University of South Florida
state: Florida
city: Augusta
country: United States
facility: Augusta University, Neuroscience Center
state: Georgia
city: Columbia
country: United States
facility: NextGen Precision Health
state: Missouri
city: Chapel Hill
country: United States
facility: The University of North Carolina at Chapel Hill
state: North Carolina
city: Portland
country: United States
facility: University of Oregon
state: Oregon
city: Memphis
country: United States
facility: Semmes Murphey Clinic
state: Tennessee
city: Leuven
country: Belgium
facility: UZ Leuven
city: Aarhus
country: Denmark
facility: Aarhus University Hospital
city: Copenhagen
country: Denmark
facility: Rigshospitalet University of Copenhagen
city: Bordeaux
country: France
facility: Centre de référence des maladies neuromusculaires AOC-CHU de Bordeaux - Hôpital Pellegrin
city: Marseille
country: France
facility: Centre de Référence des Maladies Neuromusculaires et de la SLA
city: Nantes
country: France
facility: Centre Hospitalier Universitaire de Nantes - Hôtel Dieu Centre de Référence des Maladies Neuromusculaires Rares -
city: Nice
country: France
facility: Unité de Recherche Clinique NeuroSciences
city: Tbilisi
country: Georgia
facility: JSC Curatio
city: Tbilisi
country: Georgia
facility: LTD David Tatishvili Health Center
city: Tbilisi
country: Georgia
facility: Ltd New Hospitals
city: Bergamo
country: Italy
facility: ASST Papa Giovanni XXIII (Azienda Ospedaliera Papa Giovanni XXIII)
city: Bologna
country: Italy
facility: IRCCS Istituo delle Scienze neurologiche di Bologna, UOC Clinica neurologica Ospedale
city: Genova
country: Italy
facility: IRCCS Ospedale Policlinico San Martino
city: Milano
country: Italy
facility: IRCCS Fondazione Istituto Neurologico Carlo Besta, Dipartimento di Ricerca e Sviluppo clinico
city: Milano
country: Italy
facility: DIMER, IRCCS, Ospedale San Raffaele
city: Napoli
country: Italy
facility: Centro Sclerosi Multipla Napoli - AOU Vanvitelli
city: Pisa
country: Italy
facility: AOU Pisana
city: Roma
country: Italy
facility: Azienda Ospedaliera Sant'Andrea - Universita di Roma La Sapienza
city: Leiden
country: Netherlands
facility: Leiden Universitair Medisch Centrum (LUMC)
city: Katowice
country: Poland
facility: Neurologia Śląska Centrum Medyczne
city: Kraków
country: Poland
facility: Szpital Specjalistyczny im. Ludwika Rydygiera w Krakowie sp.z o.o
city: Lublin
country: Poland
facility: Centrum Medyczne Hope Clinic
city: Lublin
country: Poland
facility: Galen Clinic
city: Warsaw
country: Poland
facility: Centrum Medyczne Neuro Protect
city: Belgrad
country: Serbia
facility: University Clinical Center of Serbia, Neurology Clinic
city: Niš
country: Serbia
facility: University Clinical Center Nis, Neurology Clinic
city: Barcelona
country: Spain
facility: Hospìtal Universitari Vall d'Hebrón
Study officials:
References: