Ongoing clinical trial to improve the treatment of myasthenia gravis.
| Trial ID | Phase/Status | Aim of the trial |
|---|---|---|
| NCT06463587MyClad | Phase 3, randomized, double-blind, placebo-controlled clinical studyRecruiting | To investigate the sustained efficacy, the need for retreatment, and the long-term safety of a new formulation of oral cladribine as compared with placebo in participants with gMG. |
| NCT06626919 | Phase 1 studyRecruiting | To evaluate the safety, tolerability, and preliminary efficacy of anitocabtagene autoleucel, a BCMA-directed CAR-T therapy, in subjects with gMG. |
| NCT06799247AURORA | Phase 3, randomized, double blind, placebo-controlled study.Recruiting | To evaluate the efficacy, safety, and tolerability of Descartes-08, mRNA CAR-T therapy, in patients with gMG. |
| NCT06359041RESET-MG | Phase 1/2, open-label, multicenter studyActive, not recruiting | To evaluate the effects of CABA-201, a CAR-T therapy, in patients with gMG. |
| NCT06704269 | A Phase I/II studyRecruiting | To assess the safety, efficacy, and cellular kinetics of YTB323, a biological CAR-T therapy, in participants with treatment-resistant gMG. |
| NCT06456580UPSTREAM MG | A randomized, double-blind, placebo-controlled Phase 3 studyRecruiting | To evaluate the efficacy and safety of telitacicept in the treatment of gMG. |
| NCT06193889KYSA-6 | A Phase 2/3, open-label, randomized, controlled, multicenter studyRecruiting | To investigate the effects of KYV-101, an autologous fully human anti-CD19 CAR-T therapy, vs. ongoing standard-of-care immunosuppressive therapy in patients with gMG. |
| NCT06414954SYNAPSE-MG | Phase 2 proof-of-concept, dose range finding studyRecruiting | 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. |
| NCT06558279ADAPT oculus | Randomized, double-blinded, placebo-controlled, Phase 3, parallel-group design studyActive, not recruiting | To evaluate the efficacy and safety of efgartigimod PH20 SC in adult patients with ocular MG. |
| NCT06106672 | A Phase 1b/2a First-in-Human (FIH) clinical trialRecruiting | To assess the safety, tolerability, pharmacodynamics, and efficacy of multiple ascending doses of CNP-106 in MG patients. |
| NCT06055959ziMyG | Phase 2/3, open-label studyRecruiting | To assess the pharmacokinetics, pharmacodynamics, safety, tolerability, immunogenicity, and activity of zilucoplan in pediatric study participants with gMG. |
| NCT06517758 | A randomized, double-blind, placebo-controlled, multicenter, Phase 3 studyRecruiting | To evaluate efficacy, safety, and tolerability of iptacopan in patients with AChR+ gMG who are on stable standard of care (SOC) treatment with symptom management and immunosuppression in participants aged 18 to 75 years with gMG. |
| NCT04833894 | Phase 2/3, open-label, uncontrolled trialRecruiting | To investigate the pharmacokinetics, pharmacodynamics, safety, and activity of efgartigimod IV in children and adolescents aged from 2 to less than 18 years of age with gMG. |
| NCT06607627 | An open-label, single-arm, Phase 3 studyRecruiting | To assess the pharmacokinetics and pharmacodynamics of gefurulimab in pediatric participants ages 6 to less than 18 with AChR+ gMG. |
| NCT05265273 | An open-label, uncontrolled multicenter Phase 2/3 studyRecruiting | To evaluate the safety and efficacy of an investigational medication, nipocalimab, for the treatment of gMG in children aged 2 to less than 18 years old. |
| NCT05644561 | A Phase 3, open-label, single-arm, multicenter studyActive, not recruiting | To characterize the pharmacokinetics and pharmacodynamics of treatment with ravulizumab intravenous infusion in pediatric participants with gMG. |
| NCT06392386 | A Phase 2/3, open-label, uncontrolled studyRecruiting | To measure the pharmacokinetics, pharmacodynamics, safety, tolerability, and immunogenicity of efgartigimod PH20 SC in pediatric participants with gMG aged 2 to < 18 years. |
| NCT04524273 | A Phase 3, multi-center, randomized, quadruple-blind, placebo-controlled studyActive, not recruiting | To assess the efficacy and safety of batoclimab as induction and maintenance therapy in adult participants with gMG. |
| NCT06436742 | Phase 1b studyRecruiting | To assess the safety and tolerability of ARGX-119 in adult participants with DOK7-congenital myasthenic syndromes. |
| NCT04951622 | Phase 3, multicenter, randomized, double-blind, placebo-controlled studyRecruiting | To evaluate the efficacy and safety of nipocalimab compared to placebo in participants with gMG. |
| NCT05403541 | A Phase 3, multi-center, randomized, quadruple-blind, placebo-controlled studyActive, not recruiting | To assess the efficacy and safety of batoclimab as induction and maintenance therapy in adult participants with gMG. |
| NCT05070858 | Phase 3Recruiting | To test how safe the pozelimab and cemdisiran combination therapy and cemdisiran alone are and how well they work in adult patients with gMG. |
| NCT06298552 | A randomized, double-blinded, placebo-controlled, Phase 3, parallel-group design studyActive, not recruiting | To measure the efficacy and safety of efgartigimod compared to placebo in participants with AChR binding antibody (AChR-Ab) seronegative gMG. |
gMG: generalized myasthenia gravis; BCMA: B-cell maturation antigen; CAR-T: chimeric antigen receptor T-cell; AChR: acetylcholine receptor; MuSK: muscle-specific kinase.