NCT02104011

Brief Summary

Treatment of angiomyolipomas is based on invasive techniques such as surgery or embolization. Development of anti-angiogenic therapies is a major and growing field of research in hypervascularized tumors. Angiomyolipomas have been shown to regress after prolonged treatment with mTOR inhibitors (Sirolimus), but with a large proportion of secondary effects. We showed recently that beta-blockers were able to induce regression of infantile hemagiomas. Consequently, we looked for and found, histologically, in a few cases of angiomyolipomas the presence of beta2 receptors. The aim of the study is to estimate if beta-blockers could induce regression or stabilization of renal angiomyolipomas in tuberous sclerosis in a pilot study.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
2

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started May 2015

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

April 1, 2014

Completed
3 days until next milestone

First Posted

Study publicly available on registry

April 4, 2014

Completed
1.1 years until next milestone

Study Start

First participant enrolled

May 22, 2015

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

November 22, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

November 22, 2017

Completed
Last Updated

May 19, 2026

Status Verified

January 1, 2018

Enrollment Period

2.5 years

First QC Date

April 1, 2014

Last Update Submit

May 18, 2026

Conditions

Renal AngiomyolipomasTuberous Sclerosis

Keywords

Angiomyolipomas in tuberous sclerosisBeta-blockersAnti-angiogenic therapies

Outcome Measures

Primary Outcomes (1)

  • Evolution of angiomyolipomas volume

    Stabilization or even regression of angiomyolipomas volume after 6 months and 1 year of treatment with a quantification of the vascular component.

    6 months and 1 year after inclusion

Secondary Outcomes (4)

  • Renal function evolution

    6 months and 1 year after inclusion

  • Effect on the potential haemorraghic transformation

    6 months and 1 year after inclusion

  • Improvement of the quality of life

    6 months and 1 year after inclusion.

  • Effect on face angiofibromas

    6 months and 1 year after inclusion

Study Arms (1)

Patient

EXPERIMENTAL
Drug: Propranolol

Interventions

PropranololDRUG
Patient

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Tuberous sclerosis patients with one or several angiomyolipomas of a size of at least 4 cms.

You may not qualify if:

  • Patients whom CT or MR scan shows one or several intra-lesional aneurisms requiring a preventive embolization.
  • Patients with a retroperitoneal hemorragic complication requiring a preventive embolization.
  • Patients whom biopsy will show an adenocarcinoma, hypertension non controlled, renal failure and severe liver.
  • Diabetic subjects insufficiently controlled.
  • Beta-blockers contra-indication.
  • Psychosis, severe mental disorder.
  • Patient already treated with beta-blockers or mTOR inhibitors.
  • Pregnant or nursing women.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Nephrology department

Bordeaux, France

Location

MeSH Terms

Conditions

Tuberous Sclerosis

Interventions

Propranolol

Condition Hierarchy (Ancestors)

HamartomaNeoplasmsNeoplasms, Multiple PrimaryNeoplastic Syndromes, HereditaryMalformations of Cortical Development, Group IMalformations of Cortical DevelopmentNervous System MalformationsNervous System DiseasesNeurocutaneous SyndromesHeredodegenerative Disorders, Nervous SystemNeurodegenerative DiseasesCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, Inborn

Intervention Hierarchy (Ancestors)

PhenoxypropanolaminesPropanolaminesAmino AlcoholsAlcoholsOrganic ChemicalsPropanolsAminesNaphthalenesPolycyclic Aromatic HydrocarbonsHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsPolycyclic Compounds

Study Officials

  • Claire RIGOTHIER, Dr

    University Hospital, Bordeaux

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

April 1, 2014

First Posted

April 4, 2014

Study Start

May 22, 2015

Primary Completion

November 22, 2017

Study Completion

November 22, 2017

Last Updated

May 19, 2026

Record last verified: 2018-01

Locations

FR(1)