Myasthenia Gravis MedDRA version: 21.1 Level: PT Classification code 10028417 Term: Myasthenia gravis System Organ Class: 10029205 - Nervous system disorders
Conditions
Interventions
Product Name: ALXN2050
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: N/A
CAS Number: 208617-00-7
Current Sponsor code: ALXN2050
Other descriptive name: (1R,3S,5R)-2-(2-(3-acetyl-5-(2-me
Sponsors
Alexion Pharmaceuticals
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Key Inclusion Criteria: • Diagnosed with MG at least 3 months (90 days) prior to the date of the Screening Visit. • Myasthenia Gravis Foundation of America Clinical Classification Class II to IV at the Screening Visit. • MG-ADL total score must be = 5 (with at least 50% of the score attributed to non-ocular elements) at the Screening Visit and at randomization (Day 1). • Participants receiving treatment with protocol-specified immunosuppressive therapies, corticosteroids, or acetylcholinesterase inhibitors must have been receiving treatment and on a stable dose prior to the date of the Screening Visit, with no changes to the regimen expected during screening, the PEP, and/or the ETP. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 50 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 20
Exclusion criteria
Exclusion criteria: Key Exclusion Criteria: • Estimated glomerular filtration rate = 30 milliliters/minute/1.73 squared meters during Screening calculated by Chronic Kidney Disease Epidemiology Collaboration. • History of thymectomy, thymomectomy, or any other thymic surgery within 12 months prior to the Screening Visit. • Any untreated thymic malignancy, carcinoma, or thymoma. Participants with a history of treated thymic malignancy or carcinoma are eligible for enrollment if they meet pre-specified conditions outlined in the protocol. • Clinical features consistent with Clinical Deterioration at the time of the Screening Visit or at any time during the Screening Period prior to randomization (Day 1). • Use of the following within the time periods specified below: a. Intravenous immunoglobulin G or subcutaneous immunoglobulin within the 4 weeks (28 days) prior to the Screening Visit. b. Use of tacrolimus or cyclosporine within the 4 weeks (28 days) prior to the Screening Visit.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the efficacy of ALXN2050 compared with placebo in the treatment of generalized MG (gMG) based on improvement in the Myasthenia Gravis Activities of Daily Living (MG ADL) total score;Secondary Objective: - To assess the efficacy of ALXN2050 compared with placebo in the treatment of gMG based on improvement in the Quantitative Myasthenia Gravis (QMG) total score - To assess the efficacy of ALXN2050 compared with placebo in the treatment of gMG based on improvement in quality of life measures - To assess the efficacy of ALXN2050 compared with placebo in the treatment of gMG based on additional endpoints involving the MG ADL total score PK/PD -To characterize the PK/PD of ALXN2050 and to establish the PK/PD relationship in participants with gMG Biomarker: -To assess the effect of factor D inhibition on complement biomarkers Safety: -To characterize the overall safety of ALXN2050 compared with placebo in participants with gMG;Primary end point(s): 1. Proportion of participants with a Myasthenia Gravis Activities of Daily Living (MG-ADL) total score reduction of = 2 points in any 4 consecutive weeks during the first 8 weeks and who did not receive rescue therapy;Timepoint(s) of evaluation of this end point: any 4 consecutive weeks during first 8 weeks | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 2. Change from baseline in QMG total score at Week 8 3. Proportion Of Participants Meeting Prespecified Threshold In The QMG Total Score at Week 8 4. Proportion Of Participants Meeting Prespecified Threshold In The QMG Total Score In Any 4 Consecutive Weeks During The First 8 Weeks And Who Did Not Receive Rescue Therapy 5. Change From Baseline In MG-ADL Total Score At Week 8 6. Proportion Of Participants Meeting Prespecified Threshold In The MG-ADL Total Score In Any 4 Consecutive Weeks During The First 8 Weeks And Who Did Not Receive Rescue Therapy 7. Proportion Of Participants Meeting Prespecified Threshold In The MG-ADL Total Score At Week 8 8. Change From Baseline In Quality Of Life In Neurological Disorders Fatigue Questionnaire (Neuro-QoL) Fatigue Score At Week 8 9. Maximum Peak Plasma Concentration (Cmax) Of ALXN2050 Over Time 10. Pre-dose Concentration (Ctrough) Of ALXN2050 Over Time 11. Absolute Values And Change From Baseline In Plasma Concentration Of Bb Fragment Of Complement Factor B At Week 8 12. Absolute Values And Change From Baseline In Serum Alternative Pathway Activity At Week 8 13. Plasma Factor D Concentration Over Time 14. Serum Complement Component 3 Concentration Over Time 15. Serum Classical Pathway Activity Over Time;Timepoint(s) of evaluation of this end point: - week 8 - baseline through week 8 | — |
Countries
Canada, Germany, Italy, Korea, Democratic People's Republic of, Spain, Taiwan, United States
Contacts
Public ContactEuropean Clinical Trial Information
Alexion Europe SAS
Outcome results
None listed