Anticoagulation with apixaban in venous malformations

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Oslo University Hospital HF

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14], Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

15 Apr 2025 → ongoing

Decision date (initial)

2024-07-22

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

South-Eastern Norway Regional Health authority

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy, Efficacy

To investigate the efficacy of apixaban on pain intensity

Secondary objectives 6

1. To investigate the efficacy of apixaban on coagulation parameters (Part 1)
2. To investigate the efficacy of apixaban on quality of life (Part 1)
3. To find the minimum effective dose apixaban in reducing pain in venous malformations (Part 2)
4. To investigate safety of apixaban in patients with venous malformations (Part 1 and 2)
5. To investigate long term effect of apixaban in venous malformations (3 months) (Part 2)
6. To investigate the efficacy of apixaban on amount of thrombi in venous malformations (Part 1)

Conditions and MedDRA coding

Venous malformation

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. 1. Participant must be 18–85 years of age at the time of signing the consent form
2. The participant must have a simple venous malformation confirmed by MRi
3. Localized intravascular coagulation (definition: d-dimer > 2 times upper reference area)
4. Localized pain in venous malformation (NRS >/=4) that inhibits daily activity and/or interferes with sleep

Exclusion criteria 10

1. History of major bleeding
2. Contraindications for MRi (cochlear implant, cardiac pacemaker, intracranial clips, claustrophobia)
3. Current treatment with platelet inhibitor (prasugrel, ticagrelor, clopidogrel, aspirin), other anticoagulation treatment e.g. unfractionated heparin, low molecular weight heparin (dalteparin, enoxaparin), heparin derivates (fondaparinux), oral anticoagulants (warfarin, dabigatran, rivaroxaban, edoxaban), NSAIDs, sirolimus, azole-antimycotics (e.g., ketoconazole, itraconazole, voriconazole and posaconazole), HIV protease inhibitors (e.g., ritonavir)
4. Under cancer therapy
5. Impaired kidney function (eGFR < 50 ml/min)
6. Impaired liver function (INR > 1,3 or aminotrasferases > 3 times upper limit)
7. Pregnancy or breast feeding
8. Known hypersensitivity to the active substance or to any of the excipients listed in the SmPC
9. Low platelet count (<100 x 10e9/mL)
10. Lesion or condition if considered a significant risk factor for major bleeding.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Difference between apixaban and placebo in change of self-reported pain intensity before and 8 weeks after starting treatment

Secondary endpoints 6

1. Difference between apixaban and placebo in change in coagulation parameters before and 8 weeks after starting treatment (Part 1)
2. Difference between apixaban and placebo in change of quality of life before and 8 weeks after starting treatment (Part 1)
3. Difference between apixaban and placebo in change of amount of thrombi in venous malformation before and after 8 weeks of treatment (Part 1)
4. Change in pain intensity and quality of life three months after reducing dose (Part 2)
5. Registering number and severity of bleeding episodes under treatment with apixaban and placebo (Part 1 and 2)
6. Change in pain intensity 3 months after continuing apixaban 5 mg twice daily (Part 2)

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Oslo University Hospital HF

Sponsor organisation

Oslo University Hospital HF

Address

Taarnbygget, Kirkeveien 166 Kirkeveien 166

City

Oslo

Postcode

0450

Country

Norway

Scientific contact point

Organisation

Oslo University Hospital HF

Contact name

Nina Haagenrud Schultz

Public contact point

Organisation

Oslo University Hospital HF

Contact name

Nina Haagenrud Schultz

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 6 files

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

Application history

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