A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Patients With Cold Agglutinin Disease (CAD)

Conditions

• Cold Agglutinin Disease

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Pegcetacoplan — DRUG
  Pegcetacoplan taken twice weekly as subcutaneous injection
• Placebo matching Pegcetacoplan — DRUG
  Placebo matching pegcetacoplan taken twice weekly as subcutaneous injection

Study Details

The purpose of the study is to determine the efficacy of pegcetacoplan administration compared to placebo in increasing hemoglobin (Hgb) level from baseline and avoiding transfusion in participants with primary cold agglutinin disease (CAD).

Key Dates

Start date

Oct 20, 2022

Status verified

Oct 2025

Primary completion

May 27, 2024

Completion

Sep 11, 2024

Study Design

Enrollment

24 participants (actual)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Active Comparator: Pegcetacoplan Double Blind During Part A
  1080 mg, subcutaneous injection, twice weekly
• Placebo Comparator: Placebo Matching Pegcetacoplan-Double-blind During Part A
  Sodium acetate, subcutaneous injection, twice weekly
• Active Comparator: Open-label Pegcetacoplan During Parts B and C
  1080 mg, subcutaneous injection, twice weekly
• Placebo Comparator: Open-label Pegcetacoplan (Placebo Matching Pegcetacoplan During Part A) During Parts B&C
  Sodium acetate, subcutaneous injection, twice weekly

Primary Outcome Measure

Number of Patients Achieving a Response (R) at Week 24 [ Time Frame: Week 24 ]

Locations (5)

Related coverage on Hipa.ai

• Pegcetacoplan Phase 3 CAD Trial Results Posted: Increased Hemoglobin vs. Placebo
  Pegcetacoplan · Oct 30, 2025 · ClinicalTrials.gov

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