A Study of Andecaliximab in Participants With Fibrodysplasia Ossificans Progressiva (FOP)
NCT Number: NCT06508021
Acronym: ANDECAL
Status: NOT_YET_RECRUITING
Study Type: INTERVENTIONAL
Phases: PHASE2|PHASE3
Conditions: Fibrodysplasia Ossificans Progressiva
Interventions: DRUG: Andecaliximab|DRUG: Placebo|DRUG: Andecaliximab
Sponsor: Ashibio Inc
Sex: ALL
Age: CHILD, ADULT, OLDER_ADULT
Enrollment: 92
Study Design: Allocation: RANDOMIZED|Intervention Model: PARALLEL|Masking: QUADRUPLE (PARTICIPANT, CARE_PROVIDER, INVESTIGATOR, OUTCOMES_ASSESSOR)|Primary Purpose: TREATMENT
Start Date: 2024-08-01
Completion Date: 2029-02-04
Locations: University of California San Francisco (UCSF), San Francisco, California, 94143, United States|Mayo Clinic, Rochester, Minnesota, 55905, United States|The Children's Hospital of Philadelphia (CHOP), Philadelphia, Pennsylvania, 19104, United States|University of Pennsylvania - Perelman Center for Advanced Medicine, Philadelphia, Pennsylvania, 19104, United States

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)

Primary Outcome Measures: Number of New HO Lesions as Assessed by WBCT-LH [Whole body, Computerized Tomography (CT), not including the head (less head)], Low dose WBCT-LH (whole body CT less head) is used to create detailed images of soft tissues and bones., Week 27 and 53
Secondary Outcome Measures: Percent change from baseline in Na18F standardized uptake value maximum (SUVmax) of up to 7 individual HO lesion(s) per participant active at baseline as assessed by Na18F PET/CT (Part 1a)., SUV max is the SUV of the most intense voxel within a region of interest., Week 14|Change in HO volume over time as assessed by WBCT-LH [Whole body, Computerized Tomography (CT), not including the head (less head)] (Part 1a), Low dose WBCT-LH (whole body CT less head) is used to create detailed images of soft tissues and bones. In Part 1a, the WBCT-LH scan will be acquired as part of the PET/CT scan at baseline and Week 14., Week 14|Change in HO volume over time as assessed by WBCT-LH [Whole body, Computerized Tomography (CT), not including the head (less head)], Week 27 and 53|Number of days during which a flare-up is experienced by the participant as reported by the participant, Flare-up defined as having at least two of the following symptoms: pain, soft tissue swelling, warmth, redness, joint stiffness, or decreased range of motion., Week 14, 27 and 53|Number of flare-ups as reported by the participant, Week 14, 27 and 53|Number of flare-ups as reported by the participant and confirmed by the Principal Investigator, Week 14, 27 and 53|Change in patient joint involvement as assessed by Investigator using Cumulative Analog Joint Involvement Scale (CAJIS), CAJIS is a clinician assessment of 15 major joints; each major joint rated normal unaffected (0), affected (1), or completely functionally ankylosed (2). The total score ranges from 0 to 30., Week 27 and 53|Change in patient quality of life as assessed by the EuroQol 5 dimensions questionnaire with a 5-level scale (EQ-5D-5L), EQ-5D-5L measures: mobility, self-care, usual activities, pain/discomfort and anxiety/depression. Each dimension has 5 levels: no problems, slight problems, moderate problems, severe problems and extreme problems. The patient is asked to indicate his/her health state by ticking the box next to the most appropriate statement in each of the five dimensions., Week 27 and 53|Number of Participants With Treatment-Emergent Adverse Events (TEAEs), Number of participants with Treatment-Emergent Adverse Events (AE) (TEAEs), i.e., adverse events not present at baseline or exacerbation of a pre-existing condition during the on-treatment period will be reported., Week 27 and 53|Number of Participants With Serious Treatment-Emergent Adverse Events (Serious TEAEs), Number of participants with Serious Treatment-Emergent Adverse Events (AE) (TEAEs), i.e, required initial/prolonged in-patient hospitalization, death, life-threatening, persistent/significant disability/incapacity, congenital anomaly/birth defect, considered as a medically important event., Week 27 and 53|Number of Participants With TEAEs by Severity, Severity of TEAEs will be graded as follows: Mild: Does not interfere in a significantly with functioning. Moderate: Causes some impairment of functioning but is not hazardous to health. Severe: Significant impairment or incapacitation; hazardous to one's health. Number of participants with TEAEs by severity will be reported., Week 27 and 53