NCT06508021

A Study of Andecaliximab in Participants With Fibrodysplasia Ossificans Progressiva (FOP)

A Phase 2/3, Global, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Two-Part Study With Open-Label Extension (OLE) to Assess the Efficacy and Safety of Andecaliximab in Participants With Fibrodysplasia Ossificans Progressiva (FOP)

• Status: Active Not Recruiting
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 92 (estimated)
• Sponsor: Ashibio Inc · Industry
• Sex: All
• Age: 2 Years
• Healthy volunteers: Not accepted

Summary

This study is researching an experimental drug called andecaliximab. The study will include pediatric and adult patients with fibrodysplasia ossificans progressiva (FOP). The study will evaluate how safe and effective andecaliximab is in patients with FOP. The study is looking at several research questions, including: * Safety of andecaliximab in participants with FOP * Whether andecaliximab reduces the number of new heterotopic bone lesions (Heterotopic Ossification; HO) * Whether andecaliximab reduces the number or severity of flare-ups * Pharmacokinetics/pharmacodynamics (PK/PD): How much study drug is in your blood at different times and its impact on blood biomarker(s) * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)

Detailed description

The ASH-FOP-201 Study consists of 2 parts: Part 1 is a Lead-in Study to assess safety, PK/PD and preliminary efficacy; Part 2 is the Main Study, a Phase 2/3 randomized, double-blind, placebo-controlled trial. Part 1 is composed of Part 1a, a Na18F positron emission tomography (PET)/computed tomography, less head (CT) Study in up to 6 participants age ≥ 15 years, and Part 1b, a flare-up Study in up to 6 participants ≥ 12 years of age. Participants enrolled in Part 1 will be randomized to one of two dose levels for 13 weeks. Participants in Part 2 will be randomized to one of two dose levels of drug vs. placebo during the 52 week trial. All participants in Part 1 or Part2 will receive study drug in the extension period of the trial.

Conditions

• Fibrodysplasia Ossificans Progressiva

Interventions

Type	Name	Description
DRUG	Andecaliximab	Dose level A or B
DRUG	Placebo	Blinded
DRUG	Andecaliximab	Dose level A or B (or age adjusted dose)

Timeline

• Start date: 2024-10-25
• Primary completion: 2029-02-04
• Completion: 2029-02-04
• First posted: 2024-07-18
• Last updated: 2025-10-14

Locations

3 sites across 1 country: United States

Regulatory

• FDA-regulated drug study