FOP, caused by an activating mutation in the ACVR1 gene/ALK2 protein, can be associated with skeletal malformations (e.g., of the big toes (hallux valgus)) at birth. After birth, flare-ups and bone formation outside the normal skeleton (heterotopic ossification) occur.
Andecaliximab is an experimental drug that specifically blocks Matrix Metalloproteinase-9 (MMP9). Andecaliximab is being studied for the treatment of FOP. The ANDECAL study will evaluate the safety and efficacy of andecaliximab in participants living with FOP to block excess bone formation (heterotopic bone) and flare-ups. Andecaliximab was previously found to be safe and well tolerated in approximately 1,000 clinical trial participants in other disorders, including the use of andecaliximab at doses higher than the doses planned for the ANDECAL study. It has not, however, been previously tested in anyone <18 years of age, nor in patients living with FOP.
About the Clinical Trial
The ANDECAL Phase 2/3 trial is a global, multi-center, placebo-controlled trial. Approximately 92 patients, starting with those age 12 years or older living with FOP will be enrolled. The trial is designed to evaluate the safety and efficacy of andecaliximab compared with placebo in pediatric and adult participants living with FOP.
The study will start with a Part 1 study (US only) of up to 12 participants to evaluate safety and Pharmacokinetics/Pharmacodynamics and exploratory efficacy endpoints. Part 2, the Main Study, will evaluate safety and efficacy in approximately 80 participants.
Eligibility Criteria*
- AGE: ≥ 12 in Part 1. Age will be lowered during Part 2 following protocol-specified criteria. If found to be safe and well tolerated, age step-down will allow inclusion of participants 6-12 years and then 2-6 years in the Main Study.
- DISEASE ACTIVITY: Participants need to have had evidence of FOP disease activity within one year of the screening visit.
- MUTATIONS: Any FOP-causing mutation (variant) in the ACVR1 gene.
Study Design*
- STUDY TYPE: Interventional
- RANDOMIZED STUDY: Yes
- PLACEBO CONTROLLED:
- No placebo for Part 1: All participants will receive andecaliximab (one of two dose levels) for the duration of the Part 1 study and its extension.
- Yes, some participants will receive placebo in Part 2: All participants in the Main Study will be randomized to andecaliximab or placebo for 52 weeks. After 52 weeks, participants receiving placebo will be transitioned to the open-label extension where all participants will receive andecaliximab.
- LENGTH OF PARTICIPATION:
- Part 1: 13 weeks (followed by a 91-week open-label extension and 4-week follow-up)
- Part 2: 52 weeks (followed by a 52-week open-label extension and 4-week follow-up)
- NUMBER OF STUDY VISITS: Participants enrolled in either Part 1 or Part 2 will have 8 to 9 on-site study visits over two years (including the extension). Both Part 1 and Part 2 have additional visits that can be conducted locally (e.g., with a visiting nurse or at a local facility) or remotely (via video conference with the site).
Status
Phase 2 Recruiting
Therapy Approach
Anti-MMP9 antibody
Study Sponsor
āshibio, Inc
Publications
*For additional details regarding the eligibility criteria and details on this study, visit ClinicalTrials.gov and enter Identifier NCT06508021.
Participating Clinical Sites (as of November 10, 2024)
Mayo Clinic - Not yet recruiting
Rochester, Minnesota, United States
The Children's Hospital of Philadelphia (CHOP) - Not yet recruiting
Philadelphia, Pennsylvania, United States
University of Pennsylvania - Perelman Center for Advanced Medicine - Not yet recruiting
Philadelphia, Pennsylvania, United States
University of California, San Francisco (UCSF) - Recruiting
San Francisco, California, United States
The IFOPA does not endorse nor recommend specific clinical trials. Please speak with your doctor if you are interested in participating in a clinical trial.
