RAD001 Therapy of Angiomyolipomata in Patients With TS Complex and Sporadic LAM

NCT00457964 | Completed Phase 1 DMC

• 1 other identifier: CCHMC IRB #2008-0812(04-07-22)
• Study Type: interventional
• Target: 36
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this research study is to find out what effects RAD001 has on angiomyolipomas of a person with Tuberous Sclerosis Complex and to determine the safe dose of RAD001 without toxicity. The hypothesis is that the drug will inhibit the growth of the angiomyolipomas and possibly even cause regression.

Conditions

Tuberous Sclerosis; Lymphangioleiomyomatosis

Keywords

Tuberous Sclerosis Complex (TSC); Lymyphangioleiomyomatosis; Mammalian Target of Rapamycin (mTOR); RAD001; Angiomyolipmas

Outcome Measures

Primary Outcomes (1)

• Patients will be considered responders if their angiomyolipoma volume decreases by thirty percent or more from baseline

  12 months

Study Arms (1)

Administration of RAD001

EXPERIMENTAL

Drug: RAD001

Interventions

RAD001 DRUG

5 and 10 mg/day or 30, 50, 70mg/week

Administration of RAD001

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosed with angiomyolipomas and have either Tuberous Sclerosis Complex or lymphangioleiomyomatosis
• Angiomyolipoma one centimeter or greater in largest diameter
• Between the ages of 18 and 65 years old.
• If female, documentation of negative pregnancy test prior to enrollment and, where applicable, use of appropriate non-estrogen containing birth control contraceptive regimen while on study.
• Adequate renal function (creatinine \< 3 mg/dl)

You may not qualify if:

• Pregnant or lactating women
• Continuous requirement for supplemental oxygen
• Surgery within past 2 months
• Use of an investigational drug within last 30 days.

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• Novartis Pharmaceuticals: collaborator

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

MeSH Terms

Conditions

Tuberous Sclerosis; Lymphangioleiomyomatosis; Hereditary Sensory and Autonomic Neuropathies

Interventions

Everolimus

Condition Hierarchy (Ancestors)

Hamartoma; Neoplasms; Neoplasms, Multiple Primary; Neoplastic Syndromes, Hereditary; Malformations of Cortical Development, Group I; Malformations of Cortical Development; Nervous System Malformations; Nervous System Diseases; Neurocutaneous Syndromes; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Lymphangiomyoma; Neoplasm, Lymphatic Tissue; Neoplasms by Histologic Type; Perivascular Epithelioid Cell Neoplasms; Neoplasms, Connective and Soft Tissue; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases; Polyneuropathies; Peripheral Nervous System Diseases; Neuromuscular Diseases

Intervention Hierarchy (Ancestors)

Sirolimus; Macrolides; Lactones; Organic Chemicals

Study Officials

• John J. Bissler, M.D.

  LaBonheur Children's Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 6, 2007
• First Posted: April 9, 2007
• Study Start: August 1, 2005
• Primary Completion: July 1, 2013
• Study Completion: July 1, 2013
• Last Updated: September 25, 2013

Record last verified: 2013-09

Locations

US (1)