MAV-RAPA: Prospective evaluation of the efficacy of sirolimus (Rapamune) in the treatment of superficial arteriovenous malformations.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Centre Hospitalier Universitaire Amiens Picardie

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Not possible to specify

Decision date (initial)

2025-01-17

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

DGOS

External identifiers

EU CT number

2024-516001-23-00

EudraCT number

2011-000321-69

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy

Reduction in the "tumour" volume of the malformation on analysis of angioscanographic changes

Secondary objectives 2

1. understand how Rapamycin® plays a role in the therapeutic efficacy therapeutic efficacy in these malformations
2. understand and explain the pathophysiology of malformations

Conditions and MedDRA coding

superficial arteriovenous malformations

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. Patients (adults, adolescents and children over 2 years of age), of stage II+, III or IV of the Schöbinger classification (1994) whether their whether the lesion is progressive or quiescent, with or without haemorrhagic phenomena.
2. Patients (parents in the case of minors) must have signed a consent form consent form drawn up after clear information about the the expected risks and benefits of the study.
3. Patients should use effective contraception for the duration of the study for the duration of the study and for up to 12 weeks after the end of treatment.
4. Negative pregnancy test (blood β-HCG) for women of childbearing age.

Exclusion criteria 19

1. Acquired or chronic immunodepression
2. Patients with chronic active hepatitis B, hepatitis C or HIV infection
3. Pregnant or breast-feeding women.
4. Allergy to macrolides
5. Allergy to peanuts or soya
6. Hypersensitivity to 'Sirolimus' or to one of the excipients of the investigational product experimental product
7. Contraindication to the performance of an MRI scan
8. Leukopenia less than 1000 GB/mm3
9. Thrombocytopenia less than 80,000 PS/mm3
10. Anemia with Hb<9g/dl
11. Elevation of transaminases >2.5 N
12. History of cancer less than two years prior to inclusion
13. Surgery less than 2 months prior to inclusion
14. Active infection (viral and bacterial) at inclusion date
15. Hypercholesterolaemia > 7 mmol/l despite well-conducted medical treatment
16. Hyperlipidaemia > 2 mmol/l despite well-managed medical treatment
17. Uncontrolled diabetes
18. Patients unable to follow a clinical study
19. Adults under guardianship or trusteeship, persons deprived of their liberty

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. the proportion of patients with a reduction in tumour volume of the 30% reduction in AVM tumour volume by angioscannographic criteria during the first year of the study

Secondary endpoints 1

1. Assessment of treatment efficacy at three months, six months, nine months

Investigational products

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

Test 1

Auxiliary 2

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Centre Hospitalier Universitaire Amiens Picardie

Sponsor organisation

Centre Hospitalier Universitaire Amiens Picardie

Address

1 Rond Point Du Pr Christian Cabrol

City

Amiens Cedex 1

Postcode

80054

Country

France

Scientific contact point

Organisation

Centre Hospitalier Universitaire Amiens Picardie

Contact name

Professeur Sylvie TESTELIN

Public contact point

Organisation

Centre Hospitalier Universitaire Amiens Picardie

Contact name

Professeur Sylvie TESTELIN

Locations

1 EU/EEA country · 4 investigational sites

By country

Investigational sites

Results and documents

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

Documents 8 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