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Fibrodysplasia Ossificans ProgressivaPhase 2/3ANDECALNCT06508021

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

A Phase 2/3 study testing Andecaliximab in people with Fibrodysplasia Ossificans Progressiva, run by Ashibio Inc at 3 sites in United States.

Active, not recruiting

Under way, not looking for participants. Everyone needed has enrolled. The study is still running.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 2 years and older
Conditions the study is for
Fibrodysplasia Ossificans Progressiva
What is being tested?
Andecaliximab
Type of treatment
Injection or infusion
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
92 participants
Where
3 sitesUnited States
Study dates
25 Oct 2024to 4 Feb 2029
Phase
Phase 2/3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 2 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Participant and/or guardian able and willing to give informed consent and/or assent as applicable, and willing to adhere to the visits schedule and study procedures.
  • Clinical diagnosis of FOP including congenital malformation(s) consistent with FOP (e.g., of the great toes), and either episodic soft tissue swelling consistent with an FOP flare-up and/or progressive HO.
  • 4. CAJIS score of ≤19. 5. Disease activity within 1 year of screening visit. Disease activity is defined as physician confirmed flare-up like symptoms or clinical progression including newly identified HO or worsening joint function.
  • 6. Able to understand, undergo, and perform all protocol related procedures, including low-dose WBCT-LH scan without sedation. Assistance from a caregiver is allowed.
  • 7. Agree to provide access to all relevant current and historical medical records (including radiographs or radiographic records) and growth records.
  • Male or female ≥ 15 years of age.
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  • Serum creatinine ≤ upper limit of normal for age.
  • No open growth plates on bilateral PA hand/wrist or AP knee films at baseline
  • Able to receive IV radiotracer [both IV access and no history of a reaction to radiotracer].
  • No use of bisphosphonates or bone active agent within the past year.
  • At least 1 active HO lesion at baseline per Na18F PET/CT
  • Additional inclusion criteria apply including those listed above for all parts of the study.
  • Male or female ≥12 years of age.
  • History of multiple flare-up episodes within the past 6 months (to be reviewed and confirmed as qualifying by the PI together with the Sponsor). Qualifying flare-up episodes include any of the following:
  • At least 3 qualifying flare-ups in the past 6 months each with continuous symptoms for at least 1 week
  • Migratory flare-up swellings across the back
  • Multiple flare-up episodes the sum of which lasted at least 21 days The timing of the individual flare-ups can be overlapping; they may be ongoing at the time of enrollment or resolved.
  • The qualifying flare-ups must involve at least 2 of the following flare-up symptoms:
  • Pain
  • Soft tissue swelling
  • Warmth
  • Redness
  • Joint stiffness
  • Decreased range of motion
  • Additional inclusion criteria apply including those listed above for all parts of the study.
  • Initial enrollment age requirement is ≥12 years
  • Enrollment may be extended to participants ≥6 years of age after safety in adult and participants age 12-17 years has been established.
  • Enrollment of participants ≥2 to <6 years of age will commence after safety in adult and participants ≥6 years of age has been established.
  • Additional inclusion criteria apply including those listed above for all parts of the study.

Who cannot join

  • Body weight <10kg
  • Known non-healed fracture at time of Study Day 1.
  • Planned surgery within the timeframe of the study duration or still recovering from recent surgery.
  • Respiratory compromise that requires use of supplemental oxygen.
  • Participant has
  • Known monogenic disorder other than FOP.
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  • Bone or mineral disorder unrelated to FOP.
  • Malignancy (within the past 5 years, except non-melanoma skin cancer, cervical carcinoma in situ, or ductal carcinoma in situ [DCIS]).
  • Known active infection (including fungal, bacterial, mycobacterial, or viral infection including COVID19)
  • Uncontrolled hypoparathyroidism or hyperparathyroidism.
  • Per participant report or chart review (no testing required): Uncontrolled hyperthyroidism
  • 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).
  • Chronic use of any of unproven therapies for FOP.
  • 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
  • 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.
  • History of allergy or hypersensitivity to andecaliximab or its excipients.
  • Significant current laboratory abnormalities
  • Breastfeeding, pregnant, or planning pregnancy.
  • 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.
  • Simultaneous participation in another clinical trial involving another investigational product.
  • 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.
  • Note: Other protocol defined Inclusion/Exclusion Criteria apply

Trial locations and contacts

3 sites in 1 country.

United States3 sites

  • University of Pennsylvania - Perelman Center for Advanced Medicine

    Philadelphia, Pennsylvania, 19104

  • Mayo Clinic

    Rochester, Minnesota, 55905

  • University of California San Francisco (UCSF)

    San Francisco, California, 94143

Other trials in United States

About this study

Official title: 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)

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)
Read the 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.

Being tested
Andecaliximab, Placebo
Keywords
Myositis Ossificans, Ossification, Heterotopic, Hallux Valgus, Matrix Metalloproteinase Inhibitors, Matrix Metalloproteinase 9, Inflammation, Activin A receptor, type I protein, human, ALK-2 protein, human, ACVR1 protein, human, Muscle, Skeletal, Myositis, Muscular diseases, Musculoskeletal diseases, Flare, Flare-ups, ANDECAL, Heterotopic bone, Ectopic bone, Munchmeyer's Disease
Medical subject headings
Myositis Ossificans, Ossification, Heterotopic, Hallux Valgus, Inflammation, Myositis, Muscular Diseases, Musculoskeletal Diseases

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • AndecaliximabDrug

    Dose level A or B

  • PlaceboDrug

    Blinded

  • AndecaliximabDrug

    Dose level A or B (or age adjusted dose)

Study groups in NCT06508021
Study groupRoleWhat they receive
Part 1a: PET/CT StudyExperimentala 13-week double-blind (Investigator and Participant blinded; Sponsor unblinded) Study to assess the impact of two dose levels of andecaliximab administered subcutaneously (SC), once-a-week (QW) in participants age ≥ 15 years, with FOP on a number of outcomes including Safety, Pharmacokinetic (PK) and pharmacodynamic (PD) and the change from baseline of Na18F uptake in HO lesions by PET/CT scan, and Patient Reported Outcomes (PROs).
Receives: Drug: Andecaliximab
Part 1b: Flare-up StudyExperimentala 13-week double-blind (Investigator and Participant blinded; Sponsor unblinded) study to assess the impact of two dose levels of andecaliximab administered SC QW in participants ≥12 years of age with a recent history of frequent flare-up episodes on a number of outcomes including safety, PK/PD, and flare-up incidence and symptoms and PROs.
Receives: Drug: Andecaliximab
Part 2: Main StudyPlacebo comparator1-year (52-week) double-blind (Investigator, participant, and sponsor all blinded), placebo-controlled study of andecaliximab Dose level A or B (or age adjusted) SC QW or placebo in pediatric and adult patients with FOP. The Main Study will enroll approximately 80 participants, randomized in a 1:1:1 ratio to andecaliximab Dose level A or B (or age adjusted) SC QW or placebo.
Receives: Drug: Placebo; Drug: Andecaliximab

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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.

    Measured: Week 27 and 53

Secondary outcomes (11)

  1. 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.

    Measured: Week 14

  2. 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.

    Measured: Week 14

  3. Change in HO volume over time as assessed by WBCT-LH [Whole body, Computerized Tomography (CT), not including the head (less head)]

    Measured: Week 27 and 53

  4. 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.

    Measured: Week 14, 27 and 53

  5. Number of flare-ups as reported by the participant

    Measured: Week 14, 27 and 53

  6. Number of flare-ups as reported by the participant and confirmed by the Principal Investigator

    Measured: Week 14, 27 and 53

  7. 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.

    Measured: Week 27 and 53

  8. 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.

    Measured: Week 27 and 53

  9. 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.

    Measured: Week 27 and 53

  10. 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.

    Measured: Week 27 and 53

  11. 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.

    Measured: Week 27 and 53

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Jul 2024
  2. Study start25 Oct 2024
  3. Primary completion4 Feb 2029 (estimated)
  4. Study completion4 Feb 2029 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
92 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06508021
Sponsor's protocol ID
ASH-FOP-201
Acronym
ANDECAL
Status last verified
Nov 2026
First submitted
17 Jun 2024
Last updated
5 Nov 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
Yes: Redacted Protocol and SAP will be made available.

Publications

  • Lounev V, Groppe JC, Brewer N, Wentworth KL, Smith V, Xu M, Schomburg L, Bhargava P, Al Mukaddam M, Hsiao EC, Shore EM, Pignolo RJ, Kaplan FS. Matrix metalloproteinase-9 deficiency confers resilience in fibrodysplasia ossificans progressiva in a man and mice. J Bone Miner Res. 2024 May 2;39(4):382-398. doi: 10.1093/jbmr/zjae029.

    PubMed 38477818

  • Wein MN, Yang Y. Actionable disease insights from bedside-to-bench investigation in fibrodysplasia ossificans progressiva. J Bone Miner Res. 2024 May 2;39(4):375-376. doi: 10.1093/jbmr/zjae044. No abstract available.

    PubMed 38644656

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study testing Andecaliximab in people with Fibrodysplasia Ossificans Progressiva, run by Ashibio Inc at 3 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 29 inclusion and 27 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: under way, not looking for participants, as of the sponsor's last update on 5 Nov 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of New HO Lesions as Assessed by WBCT-LH [Whole body, Computerized Tomography (CT), not including the head (less head)] (time frame: Week 27 and 53). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06508021, which also lists 2 linked publications.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Nov 2026.

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