NCT06463587Recrutement en cours
Efficacy and Safety of a New Formulation of Oral Cladribine Compared With Placebo in Participants With Generalized Myasthenia Gravis (MyClad)
Résumé
The purpose of this clinical study is to determine the efficacy and safety of a new oral cladribine formulation in participants with Generalized Myasthenia Gravis (gMG) in comparison to placebo. It will also investigate the sustained efficacy, the need for retreatment, and the long-term safety of oral cladribine in gMG. An additional component is included to characterize the Pharmacokinetics (PK) of the new cladribine formulation in gMG participants. This study is divided into 3 periods: the double-blind placebo control (DBPC) pivotal period, and 2 extensions, the blinded extension (BE) and the retreatment (RT) period. Furthermore, in trial interviews will be conducted as a sub-study to MyClad with a sub-set of participants to gain an in depth understanding of the participant cladribine treatment and study experience.
Critères d'inclusion
• Adults of ≥ 18 years of age at the time of signing the informed consent. • Diagnosis of Myasthenia Gravis with generalized muscle weakness, meeting clinical criteria for Myasthenia Gravis Foundation of America Class II to IVa classification. • In participants positive for Acetylcholine receptor antibody (anti-AChR) or muscle-specific kinase antibody(anti-MuSK) • In participants that are autoantibody seronegative i.e. not positive for anti-AChR and anti-MuSK antibodies and participants who are positive for anti-low-density lipoprotein receptor-related protein 4 antibodies (anti-LRP4) • Has a Screening and Baseline MG-ADL score more than or equal to (\>=) 6 with \>= 50 percentage (%) of the total score due to non-ocular symptoms. Screening and Baseline MG-ADL scores must be stable. The difference between the Screening and Baseline scores should not be more than 2 and there should be no reported MG exacerbation during the Screening period • If treated with oral corticosteroids: should be on a stable daily dose for at least 3 months prior to and during screening. In such case, the daily dose of oral steroids should not exceed 20 milligrams(mg)/day for prednisone/ prednisolone, 16 mg/day for methylprednisolone, 3 mg/day for dexamethasone, or 80 mg for hydrocortisone or equivalent doses for other corticosteroids. • If treated with acetylcholinesterase inhibitor should be on a stable daily dose (pyridostigmine dose ≤ 480 mg/day or neostigmine ≤ 300 mg/day) for at least 3 months prior to and during screening • Have a body weight \>= 40 kilograms • Other protocol defined inclusion criteria could apply
Critères d'exclusion
• Immunologic disorder other than MG or any other condition requiring chronic oral, intravenous, intramuscular, or intraarticular corticosteroid therapy. Well-controlled thyroid disease, as per the Treating Investigator or the participants regular treating physician recorded in the source documents, is not exclusionary • Molecularly characterized or suspected congenital myasthenic syndrome, Lambert-Eaton myasthenic syndrome, inherited myopathy, muscular dystrophy, acquired myopathy or any other neurologic or systematic disease that mimics MG muscular weakness • Active, clinically significant viral, bacterial, or fungal infection, including brain MRI or chest X-ray findings consistent with signs of infection such as PML or TB, or any major episode of infection requiring hospitalization or treatment with parenteral anti-infectives within 8 weeks prior or during Screening, or completion of oral anti-infectives within 8 weeks prior or during Screening. Vaginal candidiasis, onychomycosis, and genital or oral herpes simplex virus considered by the Investigator to be sufficiently controlled would not be exclusionary • Has a history of or current diagnosis of active tuberculosis (TB) or is currently undergoing treatment for latent TB infection or has an untreated latent TB infection as determined by documented results within 3 months of the Screening Visit of a positive TB skin test . • Active malignancy, or history of cancer or signs of malignancy in any Screening assessment • Treatment with nonsteroidal immunosuppressants, used in gMG, such as azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, cyclophosphamide, tacrolimus within 4 weeks prior to randomization • Treatment with FcRn or complement inhibitors (such as eculizumab, rozanolixizumab efgartigimod, ravulizumab, zilucoplan or nipocalimab) within 8 weeks prior to randomization • History of thymectomy within 6 months prior to Screening. • History of generalized seizures (except for history of febrile seizures during the participant's childhood). • Negative or indeterminate for Varicella Zoster Virus antibodies at screening • History of myasthenic crisis in the last 12 months prior to and during screening • History of recurrent infections (that is 3 or more infections per year documented in available source data) within the last 2 years • Discontinuation of treatment with any non-steroidal immunosuppressants used in gMG, such as azathioprine, mycophenolate mofetil, methotrexate, cyclosporine, cyclophosphamide, tacrolimus within the last 6 months prior to Screening • If treated with non-steroidal immunosuppressants for gMG, the dose at Screening higher than 50 mg/day for azathioprine, 500 mg/day for mycophenolate mofetil, 1 mg/day for tacrolimus, 50 mg/day for cyclosporine, 25 mg/day for cyclophosphamide, or 7.5 mg/week for methotrexate • Participation in clinical study of any investigational drug within 6 months, or 5 half-lives of the investigational drug used in the previous clinical study prior to randomization, whichever is longer. However, participants with any prior exposure to cladribine may not enter the study regardless of timing of exposure • Other protocol defined exclusion criteria could apply
Contacts
Les coordonnées de l'équipe de l'essai s'affichent sur demande.
Voir la fiche officielle de l'essai- Statut
- Recrutement en cours
- Phase
- Phase 3
- Type d'étude
- Interventionnelle
- Pays
- Suisse, France, Allemagne, Italie, États-Unis, Argentine, Australie, Belgique, Bulgarie, Canada, Chine, Géorgie, Grèce, Hongrie, Inde, Japon, Pologne, Roumanie, Serbie, Corée du Sud, Espagne, Suède, Taïwan, Turquie, Royaume-Uni
- Promoteur
- Merck Healthcare KGaA, Darmstadt, Germany, an affiliate of Merck KGaA, Darmstadt, Germany(Industrie)
- N° de protocole
- MS700568_0183
- Pathologies
- Generalized Myasthenia Gravis
- Interventions
- Autre, Médicament
Traitements évalués
- Placebo
- Cladribine Low Dose
- Cladribine High Dose
Collaborateurs
EMD Serono Research & Development Institute, Inc.
Lieux · 150
- Bern, SuisseInselspital - Universitaetsspital Bern - Neuropsychologische Rehabilitation, NeurologieRecrute dans ce centre
- Nice, FranceCHU Nice - Hôpital Pasteur - CardiologyRecrute dans ce centre
- Strasbourg, FranceCHU Strasbourg - Hopital Hautepierre - Service Anesthesie-Reanimation&Medecine Peri-OperaRecrute dans ce centre
- La Tronche, FranceCHU de Grenoble - Hôpital Albert Michallon - Centre de Références des Maladies NeuromusculairesRecrute dans ce centre
- Paris, FranceFondation Ophtalmologique Adolphe de Rothschild - Service de neurologieRecrute dans ce centre
- Clermont-Ferrand, FranceCHU Clermont Ferrand - Hopital Gabriel Montpied - service de neurologie BRecrute dans ce centre
- Bron, FranceHopital Neurologique Pierre Wertheimer - Pathologies neuro-musculaires. ElectromyographieRecrute dans ce centre
- Créteil, FranceHôpital Henri Mondor - Service de NeurologieRecrute dans ce centre
- Paris, FranceGroupe Hospitalier Pitie-Salpetriere - Centre Référent Maladies Rares SLA - NeurologieRecrute dans ce centre
- Ulm, AllemagneUniversitaetsklinikum Ulm - Klinik fuer NeurologieRecrute dans ce centre
- Göttingen, AllemagneUniversitaetsmedizin Goettingen - Abteilung fuer Nephrologie und RheumatologieRecrute dans ce centre
- Hanover, AllemagneMedizinische Hochschule Hannover - Klinik fur Neurologie mit Klinischer NeurophysiolRecrute dans ce centre
- Bochum, AllemagneSt. Joseph Hospital - St. Joseph Hospital HaanRecrute dans ce centre
- Düsseldorf, AllemagneUniversitaetsklinikum Duesseldorf AoeR - Klinik fuer NeurologieRecrute dans ce centre
- Dresden, AllemagneUniversitaetsklinikum Carl Gustav Carus TU Dresden - Medizinische Klinik IRecrute dans ce centre
- Leipzig, AllemagneUniversitaetsklinikum Leipzig AoeR - Klinik und Poliklinik fuer NeurologieRecrute dans ce centre
- Lübeck, AllemagneUniversitaetsklinikum Schleswig-Holstein - Campus Luebeck - NeurologieRecrute dans ce centre
- Berlin, AllemagneCharité Universitaetsmedizin Berlin - Campus Charité Mitte - Klinik fuer NeurologieRecrute dans ce centre
- Bergamo, ItalieAzienda Socio Sanitaria Territoriale Papa GiovanniRecrute dans ce centre
- Bologna, ItalieIRCCS Istituto delle Scienze Neurologiche di Bologna - Unità Operativa Complessa Clinica NeurologicaRecrute dans ce centre
- Genova, ItalieIRCCS Ospedale Policlinico San Martino - NeuroradiologiaRecrute dans ce centre
- Milan, ItalieFondazione IRCCS Istituto Neurologico Carlo BestaRecrute dans ce centre
- Pavia, ItalieFondazione Istituto Neurologico Casimiro Mondino - NeurologyRecrute dans ce centre
- Rome, ItalieAzienda Ospedaliera Universitaria Policlinico Tor Vergata - U.O.C. NeurologiaRecrute dans ce centre
- Rome, ItaliePoliclinico Universitario Agostino Gemelli - UOC NeurologiaRecrute dans ce centre
- Treviso, ItalieOspedale ca' Foncello - NeurologyRecrute dans ce centre
- Phoenix, États-UnisArizona Neuroscience Research, LLCRecrute dans ce centre
- Farmington, États-UnisUniversity of Connecticut Health Center - Department of MedicineRecrute dans ce centre
- Washington D.C., États-UnisThe George Washington University Medical Faculty Associates Foggy Bottom South PavilionRecrute dans ce centre
- Altamonte Springs, États-UnisNeurology of Central Florida Research Center, LLCRecrute dans ce centre
Au total, dans le monde, 150 des 154 centres listés au registre recrutent ; les 4 autres ne recrutent pas actuellement.
Statut par centre selon le registre ClinicalTrials.gov (dernière mise à jour publiée : 21 octobre 2026).
Ce statut peut avoir changé et ne dit rien de votre éligibilité : avant toute démarche, vérifiez avec votre oncologue ou le centre.
+ 120 autres lieux
Autres identifiants
- 2023-507746-83-00 · autre registre
- CTR20243857 · autre registre
- jRCT2031240175 · autre registre
- SNCTP000006603 · autre identifiant
- 2023 · financement NIH
Voir aussi
Autres versions
Ces résultats sont indicatifs et ne remplacent pas un avis médical — à discuter avec votre médecin.