Study to evaluate the efficacy and safety of enoxaparin in children with slow-flow vascular problems undergoing interventional procedures

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Fundacio Sant Joan De Deu

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Hemic and Lymphatic Diseases [C15]

Trial duration

12 Sep 2025 → ongoing

Decision date (initial)

2025-07-22

Transition trial

No

Low-intervention

Yes

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Instituto de Salud Carlos III (ISCIII)

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Efficacy

To evaluate the efficacy and safety of enoxaparin in the prevention of localized intravascular coagulopathy and its complications during percutaneous interventional procedures in pediatric patients with slow-flow vascular malformations.

Secondary objectives 3

1. 1. To describe baseline parameters of slow-flow intravascular coagulopathy and markers of endothelial damage in a pediatric population of patients with slow-flow vascular malformations undergoing percutaneous interventional procedures.
2. 2. To identify risk factors associated with severe baseline localized intravascular coagulopathy parameters.
3. 3. To evaluate whether the use of prophylactic doses of enoxaparin is useful in improving slow-flow intravascular coagulopathy parameters, endothelial damage markers, reducing episodes of pain, and deep vein thrombosis during percutaneous interventional procedures.

Conditions and MedDRA coding

Venous malformation or low-flow venous/lymphatic malformation

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. 1. Patients aged 0–18 years with a diagnosis of venous or low-flow venous/lymphatic malformation.
2. 2. Scheduled percutaneous invasive procedure during the study period: sclerotherapy, cryoablation, or electroporation.
3. 3. Signed informed consent from the patient, parents, or legal guardians (and assent for patients ≥12 years).

Exclusion criteria 17

1. 1. Patients on active anticoagulant treatment.
2. 2. Known allergy or hypersensitivity to enoxaparin.
3. 3. History of heparin-induced thrombocytopenia.
4. 4. Presence of severe unrelated disease that prevents the procedure or administration of enoxaparin.
5. 5. Thrombocytopenia < 50,000/mm³.
6. 6. Active or recent bleeding (severe gastrointestinal bleeding with hemodynamic compromise or urgent need for intervention, or cerebral hemorrhage in the last 3 months).
7. 7. Spinal or epidural anesthesia or locoregional anesthesia within 24 hours prior to study treatment.
8. 8. Major surgery or severe trauma in the last 4 weeks.
9. 9. Scheduled lumbar puncture during the study.
10. 10. Glomerular filtration rate < 30 ml/m²/min calculated using the Schwartz formula.
11. 11. Alanine aminotransferase: • 0 - 1 day of life > 165 U/L • 1 day - 1 month of life > 210 U/L • 1 - 3 months of life > 185 U/L • 3 - 9 months of life > 165 U/L • 9 months - 12 years of life > 155 U/L • 12 - 18 years of life > 150 U/L
12. 12. Aspartate aminotransferase: • 0 - 1 day of life > 475 U/L • 1 day - 1 month of life > 385 U/L • 1 - 2 months of life > 315 U/L • 2 - 9 months of life > 280 U/L • 9 months - 6 years of life > 250 U/L • 6 - 18 years of life > 190 U/L
13. 13. Total bilirubin: • 0 - 1 day of life > 17.91 mg/dL • 1 - 2 days of life > 20.88 mg/dL • 2 - 5 days of life > 17.91 mg/dL • 5 days - 1 year of life > 2.97 mg/dL • 1 - 18 years of life > 2.97 mg/dL
14. 14. Uncontrolled arterial hypertension.
15. 15. Pregnancy or breastfeeding.
16. 16. Psychological or cognitive disorders that prevent treatment administration or inability to attend scheduled study visits.
17. 17. Simultaneous participation in another clinical trial with medication.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. 1. Values of localized intravascular coagulopathy and endothelial damage in patients receiving/not receiving prophylactic treatment with enoxaparin.
2. 2. Hemorrhagic clinical signs related to treatment through: • Subjective assessment of bleeding • Hemoglobin levels • Classification of haemorrhage

Secondary endpoints 3

1. 1. Baseline analytical parameters of localized intravascular coagulopathy and endothelial damage.
2. 2. Values of localized intravascular coagulopathy (D-dimer, fibrinogen, platelets) in relation to possible risk factors: age, characteristics and extent of the malformation, genetic diagnosis, and bone/muscular/visceral involvement.
3. 3. Analytical parameters of localized intravascular coagulopathy and endothelial damage in relation to the degree of inflammation, pain presented by patients after the intervention, appearance of new phleboliths, or development of deep vein thrombosis during the study period.

Investigational products

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

Test 5

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Fundacio Sant Joan De Deu

Sponsor organisation

Fundacio Sant Joan De Deu

Address

Calle Santa Rosa 39-57 3a Planta

City

Esplugues De Llobregat

Postcode

08950

Country

Spain

Scientific contact point

Organisation

Fundacio Sant Joan De Deu

Contact name

Dr. Rubén Berrueco

Public contact point

Organisation

Fundacio Sant Joan De Deu

Contact name

Dr. Rubén Berrueco

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 12 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