Phase 3 N=167 Randomized Quadruple-blind Treatment

HEALEY ALS Platform Trial - Regimen B Verdiperstat

Amyotrophic Lateral Sclerosis

Bottom Line

View on ClinicalTrials.gov: NCT04436510 ↗

Enrolled (actual)

167

Serious AEs

13.2%

Results posted

Jun 2023

Primary outcome: Primary: Disease Progression as Assessed by the ALSFRS-R-Slope — -1.02; -1.05 ALSFRS-R total score points per month

Study Design & Population

Study type: Interventional
Phase: Phase 3
Interventions: Matching Placebo (Drug); Verdiperstat (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Merit E. Cudkowicz, MD
Primary completion: Apr 2022

Outcome Measures

Outcome	Result	p-value
PRIMARY Disease Progression as Assessed by the ALSFRS-R-Slope	-1.02; -1.05	—
PRIMARY Mortality Even Rate	0.011; 0.012	—
SECONDARY Respiratory Function	-7.76; -8.05	0.8875
SECONDARY Muscle Strength	-23.24; -26.81	0.3538
SECONDARY Number of Participants That Experienced Death or Death Equivalent	7; 5	0.318

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 B will evaluate the safety and efficacy of a single study drug, verdiperstat, in participants with ALS.

Eligibility Criteria

Inclusion Criteria

No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).

Exclusion Criteria

The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).
Participants who are taking strong inhibitors of CYP1A2 (i.e., ciprofloxacin, enoxacin, fluvoxamine, zafirlukast) for chronic/long-term use defined as more than two weeks.
Participants who are taking strong inhibitors of CYP3A4 (i.e., conivaptan, itraconazole, ketoconazole, posaconazole, troleandomycin, voriconazole, clarithromycin, diltiazem, idelalisib, nefazodone, and certain antiviral agents [cobicistat, danoprevir, ritonavir, elvitegravir, indinavir, lopinavir, paritaprevir, ombitasavir, dasabuvir, saquinavir, tipranavir, nelfinavir]) for chronic/long-term use defined as more than two weeks. Note: Topical antifungal use is not exclusionary. Participants should not consume large quantities of grapefruit juice (more than 8oz per day) on a regular basis.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04436510). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.