NCT00457808

Brief Summary

The purpose of this study was to determine if rapamycin reduced angiomyolipomata volume in patients with tuberous sclerosis complex or lam.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
25

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Dec 2002

Typical duration for phase_2

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

Study Start

First participant enrolled

December 1, 2002

Completed
3.2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2006

Completed
1.1 years until next milestone

First Submitted

Initial submission to the registry

April 6, 2007

Completed
3 days until next milestone

First Posted

Study publicly available on registry

April 9, 2007

Completed
Last Updated

April 9, 2007

Status Verified

April 1, 2007

First QC Date

April 6, 2007

Last Update Submit

April 6, 2007

Conditions

Tuberous SclerosisLymphangioleiomyomatosis

Keywords

TSC and LAMTuberous Sclerosis Complex and sporadic LAM

Outcome Measures

Primary Outcomes (1)

  • Volume of Angiomyolipomata

Interventions

Rapamycin, sirolimusDRUG

Eligibility Criteria

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

You may qualify if:

  • Diagnosed with angiomyolipomas and have either Tuberous Sclerosis complex or Lymphangioleiomyomatosis
  • Between the gaes of 18 and 65 years
  • Competency to voluntarily consent
  • Clinically definite diagnosis of tuberous sclerosis or S-LAM
  • Adequate contraception
  • At least one angiomyolipoma of 1 cm or greater in largest diameter

You may not qualify if:

  • Use of continuous supplemental oxygen
  • Concurrent infection
  • Recent surgery
  • Ongoing or planned pregnancy
  • Lactation
  • Use of an investigational drug within the last 30 days of study entrance

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, 45229-3039, United States

Location

Related Publications (1)

  • Bissler JJ, McCormack FX, Young LR, Elwing JM, Chuck G, Leonard JM, Schmithorst VJ, Laor T, Brody AS, Bean J, Salisbury S, Franz DN. Sirolimus for angiomyolipoma in tuberous sclerosis complex or lymphangioleiomyomatosis. N Engl J Med. 2008 Jan 10;358(2):140-51. doi: 10.1056/NEJMoa063564.

    PMID: 18184959DERIVED

MeSH Terms

Conditions

Tuberous SclerosisLymphangioleiomyomatosis

Interventions

Sirolimus

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, InbornLymphangiomyomaNeoplasm, Lymphatic TissueNeoplasms by Histologic TypePerivascular Epithelioid Cell NeoplasmsNeoplasms, Connective and Soft TissueLymphoproliferative DisordersLymphatic DiseasesHemic and Lymphatic DiseasesImmunoproliferative DisordersImmune System Diseases

Intervention Hierarchy (Ancestors)

MacrolidesLactonesOrganic Chemicals

Study Officials

  • John Bissler, MD

    Cincinnati Childrens Hospital Medical Center

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

April 6, 2007

First Posted

April 9, 2007

Study Start

December 1, 2002

Study Completion

March 1, 2006

Last Updated

April 9, 2007

Record last verified: 2007-04

Locations

US(1)