Rapamycin Therapy for Patients With Tuberous Sclerosis Co... | NCT00457808

Studies
Rapamycin Therapy for Patients With Tuberous Sclerosis Complex and Sporadic LAM

NCT00457808

Sponsor

Children's Hospital Medical Center, Cincinnati

Status

Completed

Last Update Posted

Apr 9, 2007Estimated

Enrollment

Phase

Phase 2

Conditions

Tuberous Sclerosis
Lymphangioleiomyomatosis

Interventions

Rapamycin, sirolimus

Countries

United States

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

ID	Type	Description	Link
R21CA103486	U.S. NIH Grant/Contract		View source

Status Module

Record Verification Date

Apr 2007

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

Dec 2002

Primary Completion Date

Not provided

Completion Date

Mar 2006Actual

First Submitted Date

Apr 6, 2007

First Submission Date that Met QC Criteria

Apr 6, 2007

First Posted Date

Apr 9, 2007Estimated

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 6, 2007

Last Update Posted Date

Apr 9, 2007Estimated

Name	Class
The LAM Foundation	OTHER
Tuberous Sclerosis Alliance	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

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

Detailed Description

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.

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 2

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

Name	Type	Description	Arm Group Labels	Other Names
Rapamycin, sirolimus	Drug

Measure	Description	Time Frame
Volume of Angiomyolipomata

Eligibility Module

Eligibility Criteria

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

65 Years

Standard Ages

AdultOlder Adult

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
John Bissler, MD	Cincinnati Childrens Hospital Medical Center	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Cincinnati Childrens Hospital Medical Center		Cincinnati	Ohio	45229-3039	United States

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
18184959	Derived	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.

ID	Term
D006222	Hamartoma
D009369	Neoplasms
D009378	Neoplasms, Multiple Primary
D009386	Neoplastic Syndromes, Hereditary

ID	Term
D018942	Macrolides
D007783	Lactones
D009930	Organic Chemicals

ID	Term
D014402	Tuberous Sclerosis
D018192	Lymphangioleiomyomatosis