HEALEY ALS Platform Trial - Regimen A Zilucoplan

NCT04436497 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: 2019P003518A
• Study Type: interventional
• Target: 162
• Locations: 1 country
• Sites: 1

Brief Summary

The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. Regimen A will evaluate the safety and efficacy of a single study drug, zilucoplan, in participants with ALS.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS; Placebo-Controlled; Double-Blind; Regimen-Specific Appendix; Lou Gehrig's Disease; Zilucoplan; Ra Pharmaceuticals

Outcome Measures

Primary Outcomes (2)

• Disease Progression as Assessed by the ALSFRS-R Total Score

  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.

  Baseline through 24 Weeks

• Mortality Event Rate

  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.

  Baseline through 24 Weeks

Secondary Outcomes (3)

• Respiratory Function

  Baseline and 24 Weeks

• Muscle Strength

  Baseline and 24 Weeks

• Number of Participants That Experienced Death or Death Equivalent

  Baseline through 24 Weeks

Study Arms (2)

Zilucoplan

EXPERIMENTAL

Drug: Zilucoplan Administration: Subcutaneous injection Dosage: 0.3mg/kg administered daily

Drug: Zilucoplan

Matching Placebo

PLACEBO COMPARATOR

Administration: Subcutaneous injection Dosage: Daily subcutaneous injection

Drug: Matching Placebo

Interventions

Zilucoplan DRUG

Drug: Zilucoplan Administration: Subcutaneous injection Dosage: 0.3mg/kg administered daily

Zilucoplan

Matching Placebo DRUG

Drug: Matching Placebo Administration: Subcutaneous injection Dosage: Daily subcutaneous injection

Matching Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Vaccination with a quadrivalent meningococcal vaccine and meningococcal serotype B vaccine at least 14 days prior to the first dose of study drug at the Baseline (Day 0) visit. Meningococcal vaccines (including boosters) should be administered in accordance with the study's vaccination worksheet.

You may not qualify if:

• History of meningococcal disease.
• Prior treatment with a complement inhibitor.

Sponsors & Collaborators

• Merit E. Cudkowicz, MD: lead
• Ra Pharmaceuticals: collaborator

Study Sites (1)

Healey Center for ALS at Mass General

Boston, Massachusetts, 02114, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Interventions

zilucoplan

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Healey Center for ALS Project Management
• Organization: Healey Center for ALS at Massachusetts General Hospital

healeyalsplatform@mgh.harvard.edu

833-425-8257 (HALT ALS)

Study Officials

• Merit Cudkowicz

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief, Neurology Department

Study Record Dates

• First Submitted: June 1, 2020
• First Posted: June 18, 2020
• Study Start: July 29, 2020
• Primary Completion: April 13, 2022
• Study Completion: May 4, 2022
• Last Updated: July 25, 2023
• Results First Posted: July 25, 2023

Record last verified: 2023-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)