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Monoclonal Antibodies

Andecaliximab for Stone Man Syndrome (ANDECAL Trial)

Phase 2 & 3

Recruiting

Research Sponsored by Ashibio Inc

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Serum creatinine ≤ upper limit of normal for age

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

Must not have

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

Timeline

Screening 3 weeks

Treatment Varies

Follow Up week 14, 27 and 53

Summary

This trial is testing a new drug called andecaliximab on both children and adults with fibrodysplasia ossificans progressiva (FOP). The goal is to see if the

Who is the study for?

This trial is for pediatric and adult patients with fibrodysplasia ossificans progressiva (FOP), also known as Stone Man Syndrome. Participants must meet specific health criteria to join, but these details are not provided here.

What is being tested?

The study tests the safety and effectiveness of an experimental drug called andecaliximab in reducing new bone lesions and flare-ups in FOP patients. It includes a comparison between the drug and a placebo, monitoring blood levels, effects on biomarkers, and potential antibody development against the drug.

What are the potential side effects?

Possible side effects may include reactions to the drug such as immune responses where the body makes antibodies against it. This could reduce its effectiveness or cause other side effects which are not detailed here.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My kidney function, measured by creatinine, is within the normal range.

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My disease has worsened or flared up in the last year.

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I can receive IV treatments and have never had a reaction to them.

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I have at least one active bone lesion detectable by a specific scan.

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I have been diagnosed with FOP, showing signs like flare-ups or bone growth in muscles.

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My flare-ups include at least two symptoms like pain, swelling, or redness.

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I am 12 years old or older.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a fracture that has not yet healed.

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I am scheduled for surgery during the study or am recovering from recent surgery.

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My body weight is less than 10kg.

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I need extra oxygen to breathe properly.

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My thyroid is overactive and not controlled by medication.

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I haven't had cancer in the last 5 years, except for certain skin, cervical, or breast cancers.

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I do not have any active infections, including COVID-19.

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My parathyroid condition is not under control.

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I am currently taking or have been taking tetracycline antibiotics.

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I am not allergic to andecaliximab or its ingredients.

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I have a genetic disorder that is not FOP or related to bone/mineral issues.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ week 14, 27 and 53

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~week 14, 27 and 53

This trial's timeline: 3 weeks for screening, Varies for treatment, and week 14, 27 and 53 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Number of New HO Lesions as Assessed by WBCT-LH [Whole body, Computerized Tomography (CT), not including the head (less head)]

Secondary study objectives

Change in HO volume over time as assessed by WBCT-LH [Whole body, Computerized Tomography (CT), not including the head (less head)] (Part 1a)

Change in patient joint involvement as assessed by Investigator using Cumulative Analog Joint Involvement Scale (CAJIS)

Change in patient quality of life as assessed by the EuroQol 5 dimensions questionnaire with a 5-level scale (EQ-5D-5L)

+5 more

Side effects data

From 2019 Phase 3 trial • 432 Patients • NCT02545504

49%

Nausea

40%

Diarrhoea

37%

Fatigue

35%

Neutropenia

34%

Peripheral sensory neuropathy

29%

Vomiting

29%

Asthenia

29%

Decreased appetite

28%

Anaemia

28%

Constipation

19%

Abdominal pain

18%

Thrombocytopenia

14%

Neutrophil count decreased

13%

Hypokalaemia

13%

Weight decreased

13%

Neuropathy peripheral

13%

Paraesthesia

13%

Insomnia

12%

Mucosal inflammation

12%

Back pain

12%

Pyrexia

12%

Cough

11%

Stomatitis

10%

Dysgeusia

10%

Dysphagia

Abdominal pain upper

Dyspepsia

Platelet count decreased

Dizziness

Dyspnoea

Oedema peripheral

Headache

Urinary tract infection

Dysaesthesia

Upper respiratory tract infection

Blood alkaline phosphatase increased

Dehydration

Epistaxis

Rash

Arthralgia

Alopecia

Abdominal distension

Temperature intolerance

Aspartate aminotransferase increased

Hyperglycaemia

Hypoalbuminaemia

Musculoskeletal pain

Pulmonary embolism

Hiccups

Ascites

Flatulence

Alanine aminotransferase increased

Myalgia

Neurotoxicity

Hypertension

Dry mouth

Gastrooesophageal reflux disease

Pneumonia

Chest pain

Febrile neutropenia

Gastrointestinal haemorrhage

Taste disorder

Death

General physical health deterioration

Pancreatitis acute

Cardio-respiratory arrest

Melaena

Obstruction gastric

Sepsis

Influenza

Syncope

Acute kidney injury

Pneumonitis

Respiratory failure

Deep vein thrombosis

Poor venous access

Small intestinal obstruction

Colitis

100%

80%

60%

40%

20%

Study treatment Arm

Placebo + mFOLFOX6

Andecaliximab + mFOLFOX6

Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: Part 1b: Flare-up StudyExperimental Treatment1 Intervention

a 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.

Group II: Part 1a: PET/CT StudyExperimental Treatment1 Intervention

a 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).

Group III: Part 2: Main StudyPlacebo Group2 Interventions

1-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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Andecaliximab

2013

Completed Phase 3

~900

0 / 18Eligibility criteria met