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Monoclonal Antibodies

Part 1a: PET/CT Study for Stone Man Syndrome (ANDECAL Trial)

Phase 2 & 3
Waitlist Available
Research Sponsored by Ashibio Inc
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
1. 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.
2. 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.
Timeline
Screening 3 weeks
Treatment Varies
Follow Up week 14, 27 and 53
Awards & highlights

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)

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

Timeline

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)]
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)
+8 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%
Constipation
28%
Anaemia
19%
Abdominal pain
18%
Thrombocytopenia
14%
Neutrophil count decreased
13%
Weight decreased
13%
Hypokalaemia
13%
Neuropathy peripheral
13%
Paraesthesia
13%
Insomnia
12%
Mucosal inflammation
12%
Back pain
12%
Pyrexia
12%
Cough
11%
Stomatitis
10%
Dysgeusia
10%
Dysphagia
9%
Dizziness
9%
Dyspepsia
9%
Abdominal pain upper
9%
Platelet count decreased
9%
Dyspnoea
8%
Oedema peripheral
8%
Headache
7%
Dysaesthesia
7%
Upper respiratory tract infection
7%
Urinary tract infection
7%
Blood alkaline phosphatase increased
7%
Dehydration
7%
Epistaxis
6%
Rash
6%
Musculoskeletal pain
6%
Alopecia
6%
Arthralgia
6%
Abdominal distension
6%
Temperature intolerance
6%
Aspartate aminotransferase increased
6%
Hyperglycaemia
6%
Hypoalbuminaemia
5%
Hiccups
5%
Pulmonary embolism
5%
Alanine aminotransferase increased
5%
Myalgia
5%
Ascites
5%
Flatulence
5%
Neurotoxicity
4%
Hypertension
4%
Dry mouth
4%
Gastrooesophageal reflux disease
3%
Chest pain
3%
Pneumonia
3%
Febrile neutropenia
3%
Gastrointestinal haemorrhage
3%
Taste disorder
2%
Death
2%
General physical health deterioration
1%
Poor venous access
1%
Acute kidney injury
1%
Pneumonitis
1%
Obstruction gastric
1%
Syncope
1%
Cardio-respiratory arrest
1%
Melaena
1%
Pancreatitis acute
1%
Sepsis
1%
Influenza
1%
Respiratory failure
1%
Deep vein thrombosis
1%
Small intestinal obstruction
1%
Colitis
100%
80%
60%
40%
20%
0%
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

Find a Location

Who is running the clinical trial?

Ashibio IncLead Sponsor
~61 spots leftby Feb 2029
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