Clinical trial · Interventional
A Study to Determine the Safety and Effectiveness of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis
An Exploratory, Open-label, Non-randomized, Within-patient Multiple Dose-escalation Safety, Tolerability, PK and Efficacy Trial of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis
NCT01059318CI-TRIAL-00048379completedPhase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This was an exploratory study to determine whether escalating doses of RAD001 (everolimus) were safe and effective in patients with Lymphangioleiomyomatosis
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Lymphangioleiomyomatosis | Lymphangioleiomyomatosis | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Everolimus | Drug | Everolimus | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Everolimus
- description
- All patients received a starting dose of everolimus 2.5mg/day for 4 weeks, followed by a dose of 5 mg/day for 4 weeks and finally a dose of 10mg/day for 18 weeks. The 26 week treatment period was followed by an optional extension period wherein patients continued therapy until the last patient had completed 26-weeks of treatment. The longest period a patient participated in the study was 62 weeks.
- interventionNames
- Drug: Everolimus
Primary outcomes (2)
- measure
- Change From Baseline in Vascular Endothelial Growth Factor-D (VEGF-D) Concentrations
- timeFrame
- Baseline, 26 weeks
- description
- Blood samples (1 mL) for determination of VEGF-D were collected from a forearm vein (direct venipuncture or from an indwelling cannula) and 2 aliquots of serum were collected. VEGF-D levels were determined from only 1 of the 2 serum aliquots, with the second acting as a back-up. A serum VEGF-D \>800 pg/mL level supports a diagnosis of Lymphangioleiomyomatosis (LAM)
Eligibility
Eligibility (as posted)
- Sex
- Female
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Female aged \>/= 18 years with a diagnosis of LAM * Pulmonary function abnormalities as follows: * FEV1 of ≤ 80% of the predicted value following administration of a standard dose of a short acting β2-agonist (\*200 µg Salbutamol, measured between 10 and 15 minutes of inhalation) OR * FEV1 \< 90% of the predicted value of bronchodilator following administration of a standard dose of a short acting β2-agonist (\*200 µg Salbutamol, measured between 10 and 15 minutes of inhalation) and DLco (uncorrected) \<80% predicted. * Female patients including those of childbearing potential will be included in this study. * Negative pregnancy test at screening and baseline Exclusion Criteria: * FEV1\<50% of predicted post-bronchodilator. * Change in FVC (ml) \> ± 15% of screening value at baseline visit (not less than 14d after screening visit). * Use of any medicine containing estrogen in the 4 months prior to the screening visit and for the duration of the study * Significant hematologic, renal, hepatic laboratory abnormality or amylase \> 1.5x the upper limit of the normal range at the screening or baseline visits * Fasting blood glucose \> 126mg/dl or random blood glucose \>200mg/dl at screening and/or baseline * Recent surgery (involving entry into a body cavity or requiring sutures) within 2 months of the screening visit or any evidence of unhealed surgical wound. * Uncontrolled hyperlipidemia (defined as persistent elevation of total cholesterol or triglycerides \>6.5nM/L) or a history of clinical atherosclerotic disease including heart attack, angina, peripheral vascular disease or stroke. * Previous organ transplantation * Inability to give informed consent * Inability to perform pulmonary function or 6 minute walk tests and imaging assessments Other protocol-defined inclusion/exclusion criteria may apply
References
Publications (2)
- RESULTMcCormack FX, Inoue Y, Moss J, Singer LG, Strange C, Nakata K, Barker AF, Chapman JT, Brantly ML, Stocks JM, Brown KK, Lynch JP 3rd, Goldberg HJ, Young LR, Kinder BW, Downey GP, Sullivan EJ, Colby TV, McKay RT, Cohen MM, Korbee L, Taveira-DaSilva AM, Lee HS, Krischer JP, Trapnell BC; National Institutes of Health Rare Lung Diseases Consortium; MILES Trial Group. Efficacy and safety of sirolimus in lymphangioleiomyomatosis. N Engl J Med. 2011 Apr 28;364(17):1595-606. doi: 10.1056/NEJMoa1100391. Epub 2011 Mar 16. PMID 21410393
- DERIVEDGoldberg HJ, Harari S, Cottin V, Rosas IO, Peters E, Biswal S, Cheng Y, Khindri S, Kovarik JM, Ma S, McCormack FX, Henske EP. Everolimus for the treatment of lymphangioleiomyomatosis: a phase II study. Eur Respir J. 2015 Sep;46(3):783-94. doi: 10.1183/09031936.00210714. Epub 2015 Jun 25. PMID 26113676