Language: BG BG
← Back to results
Active (not recruiting) Phase 3 NCT05394116

A Study to Assess Safety, Tolerability and Efficacy of Garetosmab Versus Placebo Administered Intravenously (IV) in Adult Participants With Fibrodysplasia Ossificans Progressiva (FOP)

Phase 3 – large-scale trial before approval
Conditions: Fibrodysplasia Ossificans Progressiva

Sponsor: Regeneron Pharmaceuticals

trial.available_in: БГ
Overview
This study is researching an experimental drug called garetosmab. The study is focused on adult patients with fibrodysplasia ossificans progressiva (FOP). The aim of the study is to see how safe and effective the study drug is in patients with FOP. The study is looking at several other research questions, including: * What side effects may happen from receiving the study drug * How much study drug is in the blood at different times * Whether the body makes antibodies against the study drug (which could make the drug less effective or could lead to side effects)
Who can participate
Key Inclusion Criteria: 1. Clinical diagnosis of Fibrodysplasia Ossificans Progressiva (FOP) \[(based on findings of congenital malformation of the great toes, episodic soft tissue swelling, and/or progressive Heterotopic Ossification (HO)\] 2. Confirmation of FOP diagnosis with documentation of Type I activin A receptor (ACVR1) FOP causing mutation 3. FOP disease activity within 1 year of screening visit. FOP disease activity is defined as pain, swelling, stiffness, or other signs and symptoms associated with FOP flare-ups; or worsening of joint function, or radiographic progression of HO lesions (increase in size or number of HO lesions) with/without being associated with flare-up episodes 4. Willing and able to undergo CT imaging procedures and other procedures as defined in the protocol Key Exclusion Criteria: 1. Cumulative Analog Joint Involvement Scale (CAJIS) score at screening \>19 2. Participant has significant concomitant illness or history of significant illness such as but not limited to cardiac, renal, rheumatologic, neurologic, psychiatric, endocrine, metabolic, or lymphatic disease, that in the opinion of the study investigator might confound the results of the study or pose additional risk to the patient by their participation in the study 3. Previous history or diagnosis of cancer 4. Severely impaired renal function defined as estimated glomerular filtration rate \<30 milliliter per minute (mL/min) (/1.73 m\^2 calculated by the Modification of Diet in Renal Disease equation 5. Uncontrolled diabetes defined as hemoglobin A1C (HbA1c) \>9% at screening 6. History of poorly controlled hypertension, as defined by: 1. Systolic blood pressure ≥180 mm Hg or diastolic blood pressure ≥110 mm Hg at the screening visit 2. Systolic blood pressure of 160 mm Hg to 179 mm Hg or diastolic blood pressure of 100 mm Hg to 10\^9 mm Hg at the screening visit, AND a history of end-organ damage (including history of left-ventricular hypertrophy, heart failure, angina, myocardial infarction, stroke, transient ischemic attack, peripheral arterial disease, end-stage renal disease, and moderate-to-advanced retinopathy 7. Known history of cerebral vascular malformation 8. Cardiovascular conditions such as New York Heart Association class III or IV heart failure, cardiomyopathy, intermittent claudication, myocardial infarction, or acute coronary syndrome within 6 months prior to screening; symptomatic ventricular cardiac arrhythmia 9. History of severe respiratory compromise requiring oxygen, respiratory support (eg, bilevel positive airway pressure \[biPAP\] or continuous positive airway pressure \[CPAP\]), or a history of aspiration pneumonia requiring hospitalization 10. Prior use in the past year and concomitant use of bisphosphonates 11. Concurrent participation in another interventional clinical study or a non-interventional study with radiographic measures or invasive procedures (eg, collection of blood or tissue samples) 12. Treatment with another investigational drug, denosumab, imatinib or isotretinoin in the last 30 days or within 5 half-lives of the investigational drug, whichever is longer 13. Pregnant or breastfeeding women 14. Women of childbearing potential (WOCBP) who are unwilling to practice highly effective contraception, as defined in the protocol 15. Male patients with WOCBP partners who are not willing to use condoms with WOCBP partners to prevent potential fetal exposure, as defined in the protocol Note: Other protocol defined Inclusion/Exclusion Criteria apply
Locations 18
Australia (1)
Royal North Shore Hospital
St Leonards , New South Wales
Brazil (1)
Hospital Israelita Albert Einstein
São Paulo
Chile (1)
Universidad de Concepcion
Concepción , Biobio
China (1)
Tongji Hospital of Tongji University
Shanghai
Colombia (1)
Clinica Universidad de La Sabana
Chía , Cundinamarca
Finland (1)
HUS Children and Adolescents Park Hospital Clinical Trial Unit
Helsinki , Stenbäckinkatu 11
France (1)
Hôpital Lapeyronie
Montpellier
Hong Kong (1)
Queen Mary Hospital
Hong Kong
Italy (1)
IRCCS Istituto Giannina Gaslini
Genoa
Japan (1)
Nagoya University Hospital
Nagoya , Aichi-ken
Malaysia (1)
Hospital Kuala Lampur
Kuala Lumpur
Netherlands (1)
Amsterdam University Medical Center
Amsterdam , North Holland
Poland (1)
Szpital Centrum Medyczne Medyk
Rzeszów , Podkarpackie Voivodeship
South Africa (1)
University of Cape Town
Rondebosch , Cape Town
South Korea (1)
Seoul National University Hospital
Seoul
Spain (1)
Hospital Universitario Ramon y Cajal
Madrid
United Kingdom (1)
Royal National Orthropaedic Hospital NHS Trust
Middlesex , Greater London
United States (1)
University of California Los Angeles (UCLA) Medical Center
Los Angeles , California
Technical details
Status
Active (not recruiting)
Phase
Phase 3
Study type
INTERVENTIONAL
Sex
Male and female
Minimum age
18 Years
Healthy volunteers
No
Start date
21.11.2022
Completion date
27.02.2029
Registry ID
NCT05394116
Source
anzctr
trial.inquiry_btn

Information is automatically extracted from ClinicalTrials.gov. Consult your doctor before taking action.