BRETOVAMERAN for Fibrodysplasia ossificans progressiva (FOP)

Inclusion criteria

• {"criterion_text":"- Aged 18 years or older"}
• {"criterion_text":"- A clinical diagnosis of Fibrodysplasia Ossificans Progressiva (FOP) as determined by confirmation of any causative genetic mutation in the ACVR1 gene"}
• {"criterion_text":"- Willing and able to comply with all scheduled visits, vaccination tests and other study procedure"}
• {"criterion_text":"- Capable of giving personal signed consent, which includes compliance with the requirements and restrictions"}

Exclusion criteria

• {"criterion_text":"- History of severe adverse reaction associated with a vaccine and/or severe allergic reaction (eg, anaphylaxis) to any component of the study intervention(s)"}
• {"criterion_text":"- Receipt of medications intended to prevent SARS-CoV-2 infection"}
• {"criterion_text":"- Current clinical complaints consistent with SARS-CoV-2 infection (three or more of the following complaints: headache, loss of smell, sore throat, hoarseness, cough, chest pain, shortness of breath, fatigue, diarrhea, fever)"}
• {"criterion_text":"- SARS-CoV-2 vaccination 6 months prior to participation"}
• {"criterion_text":"- Immunosuppressed individuals with known or suspected immunodeficiency, as determined by history"}
• {"criterion_text":"- Individuals with a history of autoimmune disease or an active autoimmune disease requiring therapeutic intervention"}
• {"criterion_text":"- SARS-CoV-2 PCR-positive EMA approved lateral flow test at the screening before receipt of fist vaccine dose"}
• {"criterion_text":"- Receipt of any other non-study vaccine within 28 days, before first study dose"}
• {"criterion_text":"- Anticipated receipt of any other non-study vaccine within 28 days, after last study dose administration"}

Primary endpoints

• {"endpoint_text":"- Primary Safety: Nature, frequency and severity of systemic events, including flare-ups, fever, fatigue, headache, chills, vomiting, diarrhea, new or worsened muscle pain, and new or worsened joint pain.","definition_or_measurement_approach":""}
• {"endpoint_text":"- Primary Efficacy: SARS-CoV-2 WT neutralising antibody titres rate and SARS-CoV-2-spike protein–specific binding IgG antibody titres rate on day 1, day 29 and day 43","definition_or_measurement_approach":"Antibody titres measured on day 1, day 29 and day 43."}

Secondary endpoints

• {"endpoint_text":"- Secundary Safety: Nature, frequency and severity of local reactions. Solicited adverse events include: pain, redness and swelling at the injection site and pain and swelling at the regional lymph nodes","definition_or_measurement_approach":"Solicited local adverse event reporting (pain, redness, swelling at injection site; pain and swelling at regional lymph nodes)."}
• {"endpoint_text":"- Secundary Safety: Use of corticosteroids, antipyretics and painkillers","definition_or_measurement_approach":""}
• {"endpoint_text":"- Secundary Efficacy: B-cell and T-cell responses on day 1, day 29 and day 43","definition_or_measurement_approach":"B-cell and T-cell responses measured on day 1, day 29 and day 43."}

Netherlands

Earliest CTIS Part Ii Submission Date

22-01-2025

Latest Decision Or Authorization Date

27-01-2025

Processing Time Days

5

Number Of Sites

1

Number Of Participants

10

Primary sponsor

Full Name

Amsterdam UMC Stichting

Organisation Type

Patient organisation/association

Country Of Registered Address

Netherlands