Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04436497 | HEALEY ALS Platform Trial - Regimen A Zilucoplan | PHASE2 | COMPLETED | 162 | — | — | Jul 29, 2020 | May 4, 2022 | Jul 25, 2023 | 1 | United States |
| NCT04297683 | HEALEY ALS Platform Trial - Master Protocol | PHASE2 | RECRUITING | 1,500 | — | — | Jun 14, 2020 | Aug 1, 2028 | May 14, 2026 | 78 | United States |
Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. Each of 12 questions assessing distinct functional ability is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function.
Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.
Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score and survival.
| Arm | Type | Description |
|---|---|---|
| Zilucoplan | EXPERIMENTAL | Drug: Zilucoplan Administration: Subcutaneous injection Dosage: 0.3mg/kg administered daily |
| Matching Placebo | PLACEBO_COMPARATOR | Administration: Subcutaneous injection Dosage: Daily subcutaneous injection |
| Regimen A - Zilucoplan | EXPERIMENTAL | Participants are randomized to receive either active zilucoplan or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen B - Verdiperstat | EXPERIMENTAL | Participants are randomized to receive either active verdiperstat or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen C - CNM-Au8 | EXPERIMENTAL | Participants are randomized to receive either active CNM-Au8 or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen D - Pridopidine | EXPERIMENTAL | Participants are randomized to receive either active Pridopidine or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen E - SLS-005 Trehalose | EXPERIMENTAL | Participants are randomized to receive either active SLS-005 Trehalose or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen F- ABBV-CLS-7262 | EXPERIMENTAL | Participants are randomized to receive either active ABBV-CLS-7262 or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen G - DNL343 | EXPERIMENTAL | Participants are randomized to receive either active DNL343 or matching placebo. NO LONGER RECRUITING; RESULTS REPORTED. |
| Regimen I - NUZ-001 | EXPERIMENTAL | Participants are randomized to receive either active NUZ-001 or matching placebo. |
| Name | Type | Description |
|---|---|---|
| Zilucoplan | DRUG | Drug: Zilucoplan Administration: Subcutaneous injection Dosage: 0.3mg/kg administered daily |
| Matching Placebo | DRUG | Drug: Matching Placebo Administration: Subcutaneous injection Dosage: Daily subcutaneous injection |
| Verdiperstat | DRUG | Drug: Verdiperstat Administration: Oral Dose: 600mg twice daily |
| CNM-Au8 | DRUG | Drug: CNM-Au8 Administration: Oral Dose: 30 mg or 60 mg daily |
| Pridopidine | DRUG | Drug: Pridopidine Administration: Oral Dose: 45mg twice daily |
| SLS-005 Trehalose | DRUG | Drug: SLS-005 Trehalose Administration: Infusion Dose: 0.75 g/kg weekly |
| ABBV-CLS-7262 | DRUG | Drug: ABBV-CLS-7162 Administration: Oral Dose: Dose 1 or Dose 2 |
| DNL343 | DRUG | Drug: DNL343 Administration: Oral Dose: Once per day |
| NUZ-001 | DRUG | Drug: NUZ-001. Administration: Oral. Dose: Once per day. |
Inclusion Criteria: * The following inclusion criterion is in addition to the inclusion criteria specified in the Master Protocol (NCT NCT04297683). 1. Vaccination with a quadrivalent meningococcal vaccine and meningococcal serotype B vaccine at least 14 days prior to the first dose of study dru...