Approval Probability
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Adjusted LOA
ML Risk
Zilucoplan · 2 trials · 1 indication
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...
Zilucoplan is an investigational small molecule being studied for the treatment of Amyotrophic Lateral Sclerosis (ALS). It is currently in Phase 2 clinical development, with trials evaluating its safety and efficacy in patients with this neurological condition.
Zilucoplan is being developed by Merit Medical Systems, Inc., a company traded on the NASDAQ under the ticker symbol MMSI. The company is conducting clinical trials to evaluate the drug's potential in treating Amyotrophic Lateral Sclerosis.
Zilucoplan is in Phase 2 clinical development for Amyotrophic Lateral Sclerosis. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still undergoing clinical trials to assess its safety and effectiveness.
Zilucoplan is being studied in two clinical trials. The first, NCT04297683, is the HEALEY ALS Platform Trial Master Protocol, which is currently recruiting with an enrollment of 1500 participants. The second, NCT04436497, is the HEALEY ALS Platform Trial Regimen A Zilucoplan, which has been completed with 162 participants.
Zilucoplan is a distinct investigational small molecule and is not known to be the same as any other approved or investigational drug. It is being evaluated specifically for its potential role in treating Amyotrophic Lateral Sclerosis.