Clinical trial • Phase II • Neurology|Rare Disease

NMD670 for Myasthenia gravis (AChR/MuSK antibody positive)

Phase II trial of NMD670 for Myasthenia gravis (AChR/MuSK antibody positive).

Overview

Trial Therapeutic Area
Neurology|Rare Disease
Trial Disease
Myasthenia gravis (AChR/MuSK antibody positive)
Trial Stage
Phase II
Drug Modality
Small molecule

Key dates

Initial CTIS Submission Date
29-02-2024
First CTIS Authorization Date
17-06-2024

Trial design

Randomised, placebo; nmd670 in 3 dose levels over 21 days (specific dose amounts per arm not stated in summary)-controlled Phase II trial in Spain, France, Belgium and others.

Randomised
Yes
Comparator
Placebo; NMD670 in 3 dose levels over 21 days (specific dose amounts per arm not stated in summary)
Target Sample Size
42
Trial Duration For Participant
21

Eligibility

Recruits 42 Vulnerable population selected in study metadata. Participants must be capable of and have given signed informed consent (no assent/minor consent procedures described)..

Pregnancy Exclusion
8. Female patients who plan to become pregnant during the study or are currently pregnant or breastfeeding
Vulnerable Population
Vulnerable population selected in study metadata. Participants must be capable of and have given signed informed consent (no assent/minor consent procedures described).

Inclusion criteria

  • {"criterion_text":"- 1. Participant must be a male or female being ≥18 years, at the time of signing the informed consent.\n- 2. Diagnosis of MG, MGFA class II, III or IV\n- 3. Documented positive AChR or MuSK antibody test.\n- 4. Participant must be able to swallow tablets\n- 5. Body mass index between 18 and 35 kg/m2, inclusive, at screening, and with a minimum weight of 40 kg\n- 6. Female participants who are WOCBP and male participants with partners who are WOCBP must agree to use a highly effective contraception method during the study. Contraceptive use by men and women must be consistent with local regulations regarding the methods of contraception for those participating in clinical studies.\n- 7. Participant is capable of and has given signed informed consent."}

Exclusion criteria

  • {"criterion_text":"- 1. 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.\n- 2. Active or untreated thymoma, a history of thymic carcinoma or thymic malignancy (unless deemed cured by adequate treatment with no evidence of recurrence for 5 years or more before screening), a history of thymectomy in the 6 months before screening.\n- 3. Participant with a clinical diagnosis of gout, or with serum uric acid >upper limit of normal (ULN) at screening.\n- 4. Participant with any of the following: a. Abnormal liver function test defined as total bilirubin >1.5× ULN (except for participants with known Gilbert’s syndrome). b. Abnormal liver transaminase levels at baseline and confirmed current or chronic history of liver disease including (but is not limited to) hepatitis virus infections, drug- or alcohol-related liver disease, non alcoholic steatohepatitis, autoimmune hepatitis, hemochromatosis, Wilson’s disease, α 1 antitrypsin deficiency, primary biliary cholangitis, primary sclerosing cholangitis, or any other liver disease considered clinically significant by the Investigator. c. Known hepatic or biliary abnormalities (with the exception of Gilbert’s syndrome or asymptomatic gallstones). d. Any renal impairment or kidney abnormalities considered clinically significant by the Investigator at screening.\n- 5. Participant with laboratory test abnormalities at screening considered clinically significant by the Investigator.\n- 6. Participants received treatment with IMP within 30 days (or 5 half-lives of the medication, whichever is longer) prior to day 1.\n- 7. Participants with history of poor compliance with relevant MG therapy.\n- 8. Female patients who plan to become pregnant during the study or are currently pregnant or breastfeeding"}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Change from baseline to day 21 in quantitative myasthenia gravis (QMG) total score for NMD670 vs placebo","definition_or_measurement_approach":"Change from baseline to day 21 in quantitative myasthenia gravis (QMG) total score"}

Recruitment

Registry Or Advocacy Recruitment
True; materials reference PAG (patient advocacy groups) engagement (documents titled e.g. "Advocacy PAG Email", "Advocacy PAG Patient FAQ sheet").
Digital Remote Recruitment
True; digital methods include Social Media Ads, Patient Email Blasts, eNewsletters, PatientGO app materials and online clinical trial listings.
Planned Sample Size
42
Recruitment Window Months
29
Consent Approach
Participants must be capable of and provide signed informed consent. Subject information and informed consent forms (SIS and ICF) are provided in multiple country/language versions (EN, FR, NL, PL, IT, DK and regional variants). Specific ICFs for pregnant partners and pregnancy-related information are available.

Methods

  • Patient advocacy engagement (PAG) materials (PAG emails, PAG letters, PAG FAQ sheets) targeted to patient groups (country-specific PAG materials available).
  • Social media advertisements (country-specific versions listed).
  • Posters and printed patient brochures (country-specific versions).
  • Patient email blasts / eNewsletter content (targeted to patients).
  • Dr-to-Dr letters and clinical referral materials (targeted to clinicians/GPs).
  • Clinical trial listing documents (for distribution to healthcare providers and patients).
  • Flipcharts and flowcharts for site/clinic use.
  • Study visit guides and patient-facing materials (including PatientGO application materials in some countries).

Geography

Total Number Of Sites
27
Total Number Of Participants
59

Spain

Earliest CTIS Part Ii Submission Date
19-04-2024
Latest Decision Or Authorization Date
17-06-2024
Processing Time Days
59
Number Of Sites
1
Number Of Participants
5

Sites

Site Name
Hospital Universitari Vall D Hebron
Department Name
Neurology
Contact Person Name
Raul Juntas Morales
Contact Person Email
raul.juntas@vallhebron.cat

France

Earliest CTIS Part Ii Submission Date
03-05-2024
Latest Decision Or Authorization Date
20-06-2024
Processing Time Days
48
Number Of Sites
5
Number Of Participants
10

Sites

Site Name
Centre Hospitalier Universitaire De Bordeaux
Department Name
Neurologie et Maladies neuromusculaires
Contact Person Name
Guilhem Sole
Contact Person Email
guilhem.sole@chu-bordeaux.fr
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Neurologie
Contact Person Name
Saskia Bresch
Contact Person Email
bresch.s@chu-nice.fr
Site Name
Centre Hospitalier Universitaire De Nantes
Department Name
Centre de reference des maladies neuromusculaires
Contact Person Name
Yann Pereon
Contact Person Email
yann.pereon@univ-nantes.fr
Site Name
Centre Hospitalier Universitaire De Nice
Department Name
Service de neurologie - Systeme Nerveux Peripherique
Contact Person Name
Sabrina Sacconi
Contact Person Email
sacconi.s@chu-nice.fr
Site Name
Centre Hospitalier Regional De Marseille
Department Name
Centre de reference des maladies neuromusculaires et de la SLA
Contact Person Name
Shahram Attarian
Contact Person Email
shahram.attarian@ap-hm.fr

Belgium

Earliest CTIS Part Ii Submission Date
21-05-2024
Latest Decision Or Authorization Date
19-06-2024
Processing Time Days
29
Number Of Sites
1
Number Of Participants
5

Sites

Site Name
UZ Leuven
Department Name
Neurology
Contact Person Name
Kristl Claeys
Contact Person Email
Kristl.claeys@uzleuven.be

Poland

Earliest CTIS Part Ii Submission Date
20-05-2024
Latest Decision Or Authorization Date
24-06-2024
Processing Time Days
35
Number Of Sites
9
Number Of Participants
24

Sites

Site Name
Indywidualna Praktyka Lekarska Prof. dr hab. n. med. Konrad Rejdak
Contact Person Name
Konrad Rejdak
Contact Person Email
rejdakk@gmail.com
Site Name
Neurologia Slaska Centrum Medyczne
Contact Person Name
Marek Śmiłowski
Contact Person Email
marek.smilowski2@gmail.com
Site Name
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie
Department Name
Zespół Poradni Specjalistycznych – Botaniczna 3, Poradnia Neurologiczna
Contact Person Name
Agnieszka Slowik
Contact Person Email
neurologiabk@su.krakow.pl
Site Name
Neurocor Banaszkiewicz Tomaszewski Lekarze sp.p.
Contact Person Name
Krzysztof Banaszkiewicz
Contact Person Email
rejestracja@neurocor.pl
Site Name
Neuroprotect Sp. z o.o.
Department Name
ND
Contact Person Name
Mariusz Grudniak
Site Name
Szpital Specjalistyczny Im. Ludwika Rydygiera W Krakowie Sp. z o.o.
Contact Person Name
Ryszard Nowak
Contact Person Email
nowak.medical@wp.pl
Site Name
Clinirem Sp. z o.o.
Contact Person Name
Urszula Chyrchel-Paszkiewicz
Contact Person Email
pgluchyrchel@gmail.com
Site Name
Miejskie Centrum Medyczne Im. Dr. Karola Jonschera W Lodzi
Department Name
Oddział Neurologiczny i Udarowy
Contact Person Name
Tomasz Berkowicz
Site Name
Centrum Medyczne Hope Clinic
Department Name
Poradnia Parkinsonizmu, Poradnia chorób nerwowo-mięśniowych, Poradnia leczenia bólów głowy
Contact Person Name
Sebastian Szklener
Contact Person Email
sebastianszklener@gmail.com

Netherlands

Earliest CTIS Part Ii Submission Date
21-05-2024
Latest Decision Or Authorization Date
20-06-2024
Processing Time Days
30
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Leids Universitair Medisch Centrum (LUMC)
Department Name
Neurology
Contact Person Name
Jan Verschuuren
Contact Person Email
j.j.g.m.verschuuren@lumc.nl

Italy

Earliest CTIS Part Ii Submission Date
14-05-2024
Latest Decision Or Authorization Date
20-06-2024
Processing Time Days
37
Number Of Sites
8
Number Of Participants
8

Sites

Site Name
Instituto Di Ricovero E Cura A Carattere Scientifico (Ospedale Bellaria)
Department Name
UOC Neurological Clinic
Contact Person Name
Rocco Liguori
Contact Person Email
rocco.liguori@unibo.it
Site Name
IRCCS Foundation Istituto Neurologico Carlo Besta
Department Name
Clinical Neurology – Immunotherapy and Apheresis Unit
Contact Person Name
Carlo Antozzi
Site Name
Azienda Ospedaliero Universitaria Pisana
Department Name
Department of Medical Specialties – Unit of Neurology
Contact Person Name
Michelangelo Maestri Tassoni
Contact Person Email
m.maestri@ao-pisa.toscana.it
Site Name
Ospedale San Raffaele S.r.l.
Department Name
Neurology
Contact Person Name
Massimo Filippi
Contact Person Email
filippi.massimo@hsr.it
Site Name
Azienda Ospedaliera Universita' Degli Studi Della Campania Luigi Vanvitelli
Department Name
I Division of Neurology, Department of Advanced Medical and Surgical Sciences
Contact Person Name
Alvino Bisecco
Contact Person Email
alvino.bisecco@unicampania.it
Site Name
Azienda Ospedagiera Papa Giovanni XXIII
Department Name
Neurology
Contact Person Name
Manlio Sgarzi
Contact Person Email
msgarzi@asst-pg23.it
Site Name
IRCCS Ospedale Policlinico San Martino
Department Name
DINOGMI
Contact Person Name
Marina Grandis
Contact Person Email
mgrandis@neurologia.unige.it
Site Name
Azienda Ospedaliero-Universitaria Sant Andre
Department Name
Neurology Unit
Contact Person Name
Matteo Garibaldi
Contact Person Email
matteo.garibaldi@uniroma1.it

Denmark

Earliest CTIS Part Ii Submission Date
21-05-2024
Latest Decision Or Authorization Date
17-06-2024
Processing Time Days
27
Number Of Sites
2
Number Of Participants
4

Sites

Site Name
Aarhus Universitetshospital
Department Name
Department of Neurology
Contact Person Name
Henning Andersen
Contact Person Email
hennande@rm.dk
Site Name
Rigshospitalet
Department Name
Neuromuscular Clinic and Research Unit Dep. 8077
Contact Person Name
Nicolai Preisler

Sponsor

Primary sponsor

Full Name
NMD Pharma A/S
Organisation Type
Pharmaceutical company
Country Of Registered Address
Denmark

Contract research organisations

Name
Syneos Health Inc.
Responsibilities
codes:1,10,12,13,2,5,6,8
Name
PPD Global Central Labs
Name
Labcorp Early Development Laboratories Limited
Responsibilities
PK Samples Resulting and probably Skeletal Troponin testing
Name
Illingworth Research Group Limited
Responsibilities
Patient reimbursement and concierge services
Name
Medidata Solutions Inc.
Responsibilities
RTSM Services

Third parties

  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"RTSM Services","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"Central Cardiac Services","organisation_type":"Pharmaceutical company"}
  • {"country":"United Kingdom","full_name":"Illingworth Research Group Limited","duties_or_roles":"Patient reimbursement and concierge services","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United Kingdom","full_name":"Labcorp Early Development Laboratories Limited","duties_or_roles":"PK Samples Resulting and probably Skeletal Troponin testing","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"PPD Global Central Labs","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Syneos Health Inc.","duties_or_roles":"codes:1,10,12,13,2,5,6,8","organisation_type":"Pharmaceutical company"}

Investigational products

Investigational Product Name
NMD670
Active Substance
NMD670
Modality
Small molecule
Routes Of Administration
ORAL USE
Route
oral
Authorisation Status
prodAuthStatus: 1
Dose Levels
3 dose levels (exact dosing not specified in summary)
Maximum Dose
max daily dose 800 mg; max total dose 16800 mg
Investigational Product Name
Placebo
Modality
Other

Related trials

Other published trials that may interest you.