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Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

NCT ID: NCT00457808

Last Updated: 2007-04-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2002-12-31

Study Completion Date

2006-03-31

Brief Summary

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

Detailed Description

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The study design was an open label, phase I/II trial of sirolimus for one year followed by one year off therapy.Patients were seen at baseline,at two weeks to four weeks, and at 2,4,6,9,12,18 and 24 months. Angiomyolipomata imaging was performed at all but the two to four week visit. Complete pulmonary function tests and six-minute walk were obtained at baseline,6 or 9 month, 12 and 24 month visits, while simple spirometry only was performed at all other visits.

Conditions

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Tuberous Sclerosis Lymphangioleiomyomatosis

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Interventions

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Rapamycin, sirolimus

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

* 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
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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The LAM Foundation

OTHER

Sponsor Role collaborator

Tuberous Sclerosis Alliance

OTHER

Sponsor Role collaborator

Children's Hospital Medical Center, Cincinnati

OTHER

Sponsor Role lead

Principal Investigators

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John Bissler, MD

Role: PRINCIPAL_INVESTIGATOR

Cincinnati Childrens Hospital Medical Center

Locations

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Cincinnati Childrens Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Countries

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United States

References

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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.

Reference Type DERIVED
PMID: 18184959 (View on PubMed)

Other Identifiers

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R21CA103486

Identifier Type: NIH

Identifier Source: secondary_id

View Link

CCHMC IRB # 02-10-16

Identifier Type: -

Identifier Source: org_study_id