Efficacy and Safety of the Mammalian Target of Rapamycin... | NCT01811667

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

NCT01811667

Sponsor

Cliniques universitaires Saint-Luc- Université Catholique de Louvain

Status

Completed

Last Update Posted

Apr 14, 2016Estimated

Enrollment

19Actual

Phase

Phase 3

Conditions

Cardiovascular Abnormalities

Interventions

Sirolimus

Countries

Belgium

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Apr 2016

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

May 2012

Primary Completion Date

Jan 2016Actual

Completion Date

Jan 2016Actual

First Submitted Date

Oct 5, 2012

First Submission Date that Met QC Criteria

Mar 12, 2013

First Posted Date

Mar 14, 2013Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Apr 13, 2016

Last Update Posted Date

Apr 14, 2016Estimated

Oversight Module

Has Data Monitoring Committee (DMC)

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Detailed Description

The complex vascular malformations induce chronical pains and organic dysfunctions causing significant morbidity and mortality. Therefore, the investigators need to establish guidelines in order to treat these pathologies. Standard treatments such as surgery or interventional radiology are of limited efficacy and related to a high level of recurrences as well as complications. Recent preclinical studies have shown the important role of the PI3Kinase/AKT/mTor pathway on the development and the lymphatic-vascular organisations suggesting an appealing therapeutic target to treat patients with complex vascular malformations.

The aim of this clinical study is to prospectively evaluate the efficacy and the safety of the Rapamycin, an mTOR inhibitor, to treat children and adults with microcystic lymphatic malformations, general lymphatics abnormalities (GLA) or complex vascular malformations for which conventional therapies as surgery or sclerotherapy are ineffective or associated with high risk of important complications.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

19Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
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

Name	Type	Description	Arm Group Labels	Other Names
Sirolimus	Drug		Sirolimus	rapamycin

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Time of duration of the treatment.(Efficacy)		up to 12 months

Secondary Outcomes

Measure	Description	Time Frame
The number of adverse events observed	With Common Toxicity Criteria for Adverse Effects version 3	up to 12 months

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

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

Exclusion Criteria:

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

70 Years

Standard Ages

ChildAdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Laurence Boon, MD, PhD	Cliniques universitaires Saint-Luc- Université Catholique de Louvain	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Cliniques universitaires Saint-Luc		Brussels	Brussels Capital	1200	Belgium

Condition Browse Module

MeSH Terms

ID	Term
D018376	Cardiovascular Abnormalities

Ancestor Terms

ID	Term
D002318	Cardiovascular Diseases
D000013	Congenital Abnormalities
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Browse Leaves

Not provided

Browse Branches

Not provided

ID	Term
D018942	Macrolides
D007783	Lactones
D009930	Organic Chemicals