Safety and efficacy of intradermal mRNA SARS-CoV-2 vaccination in patients with Fibrodysplasia Ossificans Progressiva (IVY trial)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Amsterdam UMC Stichting

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Virus Diseases [C02], Diseases [C] - Musculoskeletal Diseases [C05]

Trial duration

27 Jan 2025 → ongoing

Decision date (initial)

2025-01-27

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

FOP Stichting Nederland · Rijksinstituut voor Volksgezondheid en Milieu (RIVM)

External identifiers

EU CT number

2024-518686-10-00

EudraCT number

2022-000692-39

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Dose response, Therapy

To assess the safety of fractional, intradermal mRNA SARS-CoV-2 vaccination in patients with Fibrodysplasia Ossificans Progressiva

Secondary objectives 1

1. To assess the efficacy of fractional, intradermal mRNA SARS-CoV-2 vaccination in patients with Fibrodysplasia Ossificans Progressiva

Conditions and MedDRA coding

Fibrodyplasia Ossificans Progressiva (FOP)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

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

Exclusion criteria 9

1. History of severe adverse reaction associated with a vaccine and/or severe allergic reaction (eg, anaphylaxis) to any component of the study intervention(s)
2. Receipt of medications intended to prevent SARS-CoV-2 infection
3. 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)
4. SARS-CoV-2 vaccination 6 months prior to participation
5. Immunosuppressed individuals with known or suspected immunodeficiency, as determined by history
6. Individuals with a history of autoimmune disease or an active autoimmune disease requiring therapeutic intervention
7. SARS-CoV-2 PCR-positive EMA approved lateral flow test at the screening before receipt of fist vaccine dose
8. Receipt of any other non-study vaccine within 28 days, before first study dose
9. Anticipated receipt of any other non-study vaccine within 28 days, after last study dose administration

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. 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.
2. 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

Secondary endpoints 3

1. 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
2. Secundary Safety: Use of corticosteroids, antipyretics and painkillers
3. Secundary Efficacy: B-cell and T-cell responses on day 1, day 29 and day 43

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Amsterdam UMC Stichting

Sponsor organisation

Amsterdam UMC Stichting

Address

De Boelelaan 1117

City

Amsterdam

Postcode

1081 HV

Country

Netherlands

Scientific contact point

Organisation

Amsterdam UMC Stichting

Contact name

E.M.W. Eekhoff

Public contact point

Organisation

Amsterdam UMC Stichting

Contact name

E.M.W. Eekhoff

Locations

1 EU/EEA country · 1 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 4 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications