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Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis


Phase 2
18 Years
65 Years
Not Enrolling
Both
Tuberous Sclerosis, Lymphangioleiomyomatosis

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Trial Information

Rapamycin Therapy of Angiomyolipomas in Patients With Tuberous Sclerosis Complex and Sporadic Lymphangioleiomyomatosis


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.


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

Type of Study:

Interventional

Study Design:

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Volume of Angiomyolipomata

Principal Investigator

John Bissler, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Cincinnati Childrens Hospital Medical Center

Authority:

United States: Food and Drug Administration

Study ID:

CCHMC IRB # 02-10-16

NCT ID:

NCT00457808

Start Date:

December 2002

Completion Date:

March 2006

Related Keywords:

  • Tuberous Sclerosis
  • Lymphangioleiomyomatosis
  • TSC and LAM
  • Tuberous Sclerosis Complex and sporadic LAM
  • Sclerosis
  • Tuberous Sclerosis
  • Lymphangioleiomyomatosis

Name

Location

Cincinnati Childrens Hospital Medical CenterCincinnati, Ohio  45229-3039