MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)

Apellis Pharmaceuticals

Status and phase: Terminated; Phase 2

Conditions: Motor Neuron Disease; Amyotrophic Lateral Sclerosis

Treatments: Drug: Pegcetacoplan (APL-2); Other: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT04579666

APL2-ALS-206

Details and patient eligibility

About

This is a 24-month, Phase 2, multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of pegcetacoplan in subjects with amyotrophic lateral sclerosis (ALS)

Enrollment: 249 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• At least 18 years of age
• Sporadic ALS diagnosed as definite, probable, or laboratory-supported probable as defined by the revised El Escorial criteria
• Slow vital capacity (SVC) ≥60% of the predicted value at screening
• Onset of ALS symptoms within 72 weeks (18 months) prior to screening
• Total ALSFRS-R score of ≥30 at screening
• Have vaccination within 5 years against Streptococcus pneumoniae, Neisseria meningitidis (types A, C, W, Y, and B), and Haemophilus influenzae (type B) or agree to receive vaccination

Exclusion criteria

• Confirmed or suspected other causes of neuromuscular weakness
• Diagnosed with another neurodegenerative disease (examples include Parkinson's disease and Huntington's disease)
• Significant pulmonary disorder not attributed to ALS (eg, chronic obstructive pulmonary disease, pulmonary fibrosis, cystic fibrosis, pulmonary arterial hypertension)
• If taking riluzole, participant must be on a stable dose for 30 days prior to the start of the screening period. Use of riluzole is not required for participation.
• If taking edaravone, participant must be on a stable dose for 60 days prior to the start of the screening period. Use of edaravone is not required for participation.
• Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or within 5-half lives of the treatment (whichever is longer) prior to the start of the screening period or during study participation
• Use of any other complement inhibitor within 30 days or within 5-half lives of the treatment (whichever is longer) prior to the start of the screening period or during study participation

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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249 participants in 2 patient groups, including a placebo group

1,080 mg pegcetacoplan (APL-2)

Experimental group

Description:

administered subcutaneously twice weekly

Treatment:

Drug: Pegcetacoplan (APL-2)

Placebo administered subcutaneously twice weekly

Placebo Comparator group

Treatment:

Other: Placebo