RECRUITING

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

Description

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)

Conditions

Fibrodysplasia Ossificans Progressiva
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Related Conditions:

Fibrodysplasia Ossificans Progressiva

Study Overview

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Study Details

Study overview

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)

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)

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

Condition
Fibrodysplasia Ossificans Progressiva
Intervention / Treatment

-

Contacts and Locations

San Francisco

University of California San Francisco (UCSF), San Francisco, California, United States, 94143

Rochester

Mayo Clinic, Rochester, Minnesota, United States, 55905

Philadelphia

The Children's Hospital of Philadelphia (CHOP), Philadelphia, Pennsylvania, United States, 19104

Philadelphia

University of Pennsylvania - Perelman Center for Advanced Medicine, Philadelphia, Pennsylvania, United States, 19104

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility Criteria

  • 1. Body weight \<10kg
  • 2. Known non-healed fracture at time of Study Day 1.
  • 3. Planned surgery within the timeframe of the study duration or still recovering from recent surgery.
  • 4. Respiratory compromise that requires use of supplemental oxygen.
  • 5. Participant has
  • * Known monogenic disorder other than FOP.
  • * Bone or mineral disorder unrelated to FOP.
  • 6. Malignancy (within the past 5 years, except non-melanoma skin cancer, cervical carcinoma in situ, or ductal carcinoma in situ \[DCIS\]).
  • 7. Known active infection (including fungal, bacterial, mycobacterial, or viral infection including COVID19)
  • 8. Uncontrolled hypoparathyroidism or hyperparathyroidism.
  • 9. Per participant report or chart review (no testing required): Uncontrolled hyperthyroidism
  • 10. Use of the following medication:
  • * Systemic corticosteroids with a prednisone equivalent of \>10mg/day within 1 week of Study Day 1. If the participant requires chronic use of \>10mg/day prednisone equivalent of corticosteroids, eligibility will be discussed with the Sponsor.
  • * NSAIDs of higher than doses recommended by the May 2022 ICCFOP guidelines within 1 week of Study Day 1. If the participant requires chronic use of NSAIDs at doses higher than those recommended by the May 2022 ICCFOP guidelines doses, eligibility will be discussed with the Sponsor.
  • * Current or chronic use of tetracycline drugs (e.g., tetracycline, demeclocycline, doxycycline, or minocycline).
  • 11. Chronic use of any of unproven therapies for FOP.
  • 12. Palovarotene
  • * Within 1 month of Study Day 1 for all participants
  • * Within 2 years of Study Day 1 for female participants \<8 years of age Or male participants \<10 years of age
  • 13. Treatment with another investigational product within 5 half lives of last dose at the time of Study Day 1 or 1 month, whichever is longer.
  • 14. History of allergy or hypersensitivity to andecaliximab or its excipients.
  • 15. Significant current laboratory abnormalities
  • 16. Breastfeeding, pregnant, or planning pregnancy.
  • 17. Those of childbearing potential unwilling to agree to abstain from sexual activity that could result in pregnancy or unwillingness to use acceptable birth control during the study and for 90 days after the last dose.
  • 18. Simultaneous participation in another clinical trial involving another investigational product.
  • 19. Significant medical condition or disability or biochemical or hematologic abnormalities that in the opinion of the Investigator would expose the participant to undue risk, prevent the conduct of study procedures, or confound the study results.

Ages Eligible for Study

2 Years to

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Ashibio Inc,

Study Record Dates

2029-02-04