Efficacy and Safety of the Mammalian Target of Rapamycin (mTor Rapamycin) Inhibitor in Vascular Malformations

NCT01811667 | Completed Phase 3

• 1 other identifier: vasca-LM
• Study Type: interventional
• Target: 19
• Locations: 1 country
• Sites: 1

Brief Summary

The phosphatidylinositol 3-kinase (PI3Kinase)/Protein Kinase B (AKT)/mammalian target of rapamycin (mTor) pathway plays a role on the development and the lymphatic-vascular organisations. The investigators want to study the efficacy and the safety of Rapamycin, an mTor inhibitor.

Conditions

Cardiovascular Abnormalities

Keywords

Vascular Abnormalities

Outcome Measures

Primary Outcomes (1)

• Time of duration of the treatment.(Efficacy)

  up to 12 months

Secondary Outcomes (1)

• The number of adverse events observed

  up to 12 months

Study Arms (1)

Sirolimus

EXPERIMENTAL

Seric level between 10 to 15 ng/ml Pills for the adults and liquid for the children. Twice a day.

Drug: Sirolimus

Interventions

Sirolimus DRUG

Also known as: rapamycin

Sirolimus

Eligibility Criteria

• Age: Up to 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients with complex vascular abnormalities to be threat by a systemic therapy
• Patients must have adequate liver function (LDL-cholesterol, triglycerides,…)
• Patients must have adequate organ function: neutrophils \>1500/mm³, Hb \> 8,0 g et platelets\> 50.000/mm³ (no platelets limits for the Kasabach Merritt syndrome)
• Patients must have adequate renal function(normal creatinin depending on the age), clearance \> 70 ml/min/1.73m² and Urin Protein Creatinine ratio \<0.3 g.
• Karnofsky or Landry \> 50

You may not qualify if:

• Dental equipments or prosthesis interfering onto a radiological examen
• Other uncontrolled medical condition (uncontrolled diabetes, hypertension…)
• Concomitant drugs such as inhibitors/inducers of cytochrome P450 3A4 (CYP3A4)
• Immunocompromised patients, including known seropositivity for HIV
• Digestive problems modifying the absorption of Rapamycin (gastric tube feeding accepted)
• Pregnant or nursing (lactating) women
• Prior treatment with phosphatidylinositol 3-kinase (PI3K) and/or mTOR inhibitors

Sponsors & Collaborators

• Cliniques universitaires Saint-Luc- Université Catholique de Louvain: lead

Study Sites (1)

Cliniques universitaires Saint-Luc

Brussels, Brussels Capital, 1200, Belgium

Location

MeSH Terms

Conditions

Cardiovascular Abnormalities

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Cardiovascular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Laurence Boon, MD, PhD

  Cliniques universitaires Saint-Luc- Université Catholique de Louvain

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 5, 2012
• First Posted: March 14, 2013
• Study Start: May 1, 2012
• Primary Completion: January 1, 2016
• Study Completion: January 1, 2016
• Last Updated: April 14, 2016

Record last verified: 2016-04

Locations

BE (1)