Clinical trial NCT03525834: Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.
Official title: Phase IV, Single Arm Study of Safety and Efficacy of Everolimus in Chinese Adults With Tuberous Sclerosis Complex Who Have Renal Angiomyolipoma Not Requiring Immediate Surgery
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Renal Angiomyolipoma
Keywords: TSC
tuberous sclerosis complex
AML
afinitor
everolimus
RAD001
mTOR
kidney
lymphangioleiomyomatosis
renal angiomyolipoma
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2018-05-16
Enrollment: 40
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: Single arm, open label
Primary purpose: TREATMENT

Brief summary: The purpose of this study was to assess the safety and efficacy of everolimus (Afinitor®) in Chinese patients with renal angiomyolipoma (AML) associated with tuberous sclerosis complex (TSC).

Detailed description: This was an open label, single arm, multi-center, Phase IV Post-Approval Commitment (PAC) study with once daily oral dose of 10 mg everolimus in participants with renal AML associated with TSC. There were three separate phases in this study: a Screening phase, an Open-label treatment phase where participants received everolimus for 48 weeks or until disease progression, and a Treatment discontinuation follow-up phase for patients who discontinue study drug for reasons other than disease progression. Every participant had an End of Treatment visit within 28 days after last dose and a safety follow-up visit 30 days after last dose.

Eligibility criteria: Inclusion Criteria:

* Eligible for treatment with everolimus as per the locally approved label.
* Presence of at least one AML ≥ 3 cm in its longest diameter using CT or MRI.

Exclusion Criteria:

* AML related bleeding or embolization during the 6 months prior to enrollment.
* History of myocardial infarction, angina or stroke related to atherosclerosis.
* Impaired lung function.
* Significant hematological or hepatic abnormality.
* Any severe and/or uncontrolled medical conditions.

Primary outcomes:
measure: Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: From the start of treatment to 30 days after end of treatment, assessed up to maximum duration of 52 weeks
Secondary outcomes:
measure: Percentage of Participants With Best Overall Response (BOR) Status of Angiomyolipoma (AML) Response During Maximum Treatment Duration of 48 Weeks
timeFrame: Baseline, 48 weeks
measure: Percentage of Participants With Best Overall Response Status of Angiomyolipoma (AML) Progression During Maximum Treatment Duration of 48 Weeks
timeFrame: Baseline, 48 weeks
measure: Percentage of Participants With Severe Renal Impairment
timeFrame: Baseline, 48 weeks
measure: Percentage of Participants With NCI CTCA Grade 3/4 Serum Creatinine
timeFrame: Baseline, 48 weeks
Other outcomes:
Locations:
city: Wuhan
country: China
facility: Novartis Investigative Site
state: Hubei
city: Shanghai
country: China
facility: Novartis Investigative Site
state: Shanghai
city: Chengdu
country: China
facility: Novartis Investigative Site
state: Sichuan
city: Beijing
country: China
facility: Novartis Investigative Site
city: Beijing
country: China
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: