A randomized double-blind, placebo-controlled study of RAD001 in the treatment of Angiomyolipoma in patients with either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 99

Inclusion Criteria

1. Male or female = 18 years.
2. Clinically definite diagnosis of tuberous sclerosis according to the modified Gomez criteria (Roach et al, 1998; Hyman and Whittemore, 2000) or sporadic LAM (biopsy-proven or compatible chest CT).
3. Clinically definite diagnosis of renal angiomyolipoma.
4. Presence of at least one angiomyolipoma = 3 cm in its longest diameter using MRI.
5. If female and of child bearing potential, documentation of negative pregnancy test prior to enrollment. Sexually active pre-menopausal female patients (and female partners of male patients) must use adequate contraceptive measures, excluding estrogen containing contraceptives, while on study.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

1. Angiomyolipoma-related bleeding or embolization during the 6 months prior to randomization.
2. History of myocardial infarction, angina or stroke related to atherosclerosis.
3. Impaired lung function, defined as any of the following:
• FEV1 = 70% of predicted, or
• DLCO = 70% of predicted, or
• = 88% O2 saturation at rest in room air.
4. Chylous ascites sufficient to affect diaphragmatic function or pulmonary function testing.
5. Significant hematological or hepatic abnormality (i.e., transaminase levels > 2.5 × the upper limit of normal (ULN), serum bilirubin > 1.5 × ULN, hemoglobin < 9g/dL, platelets < 80,000/mm3, or absolute neutrophil count < 1,000/mm3).
6. Pregnancy or breast feeding.
7. Use of estrogen containing medications.
8. Intercurrent infection at date of randomization.
9. Prior history of organ transplant.
10. Recent surgery (involving entry into a body cavity or requiring sutures) within the 2 months prior to randomization.
11. Prior therapy with mTOR inhibitors (sirolimus, temsirolimus, everolimus).
12. Use of an investigational drug within the 30 days prior to randomization.
13. Uncontrolled hyperlipidemia: Fasting serum cholesterol > 300 mg/dL OR > 7.75 mmol/L AND Fasting triglycerides > 2.5 × ULN.
14. Uncontrolled diabetes mellitus as defined by fasting serum glucose > 1.5 × ULN.
15. Patients with bleeding diathesis or on oral anti-vitamin K medication (except low dose warfarin).
16. Patients with a known history of HIV seropositivity.
17. Inability to attend scheduled clinic visits.
18. For the purpose of MRI assessments:
• Ferromagnetic metal implants other than those approved as safe for use in MR scanner (e.g., braces, some types of aneurysm clips, shrapnel).
• Patients suffering from uncontrollable claustrophobia or physically unable to fit into the machine (e.g., obesity, etc).
19. Serum creatinine > 1.5 × ULN.
20. History of malignancy in the past two years, other than squamous or basal cell skin cancer.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method

Secondary Outcome Measures

Name	Time	Method

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Updated 10/8/2021