Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.
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
1 other identifier
interventional
40
1 country
5
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_4
Started Nov 2018
5 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 3, 2018
CompletedFirst Posted
Study publicly available on registry
May 16, 2018
CompletedStudy Start
First participant enrolled
November 9, 2018
CompletedPrimary Completion
Last participant's last visit for primary outcome
September 25, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
September 25, 2020
CompletedResults Posted
Study results publicly available
September 9, 2021
CompletedSeptember 9, 2021
September 1, 2021
1.9 years
May 3, 2018
July 28, 2021
September 3, 2021
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Percentage of participants with AEs and SAEs including significant changes from baseline in vital signs, electrocardiograms and laboratory parameters (laboratory assessments included hematology, biochemistry, coagulation and urinalysis) qualifying and reported as AEs. The percentage of participants in each category is reported in the table.
From the start of treatment to 30 days after end of treatment, assessed up to maximum duration of 52 weeks
Secondary Outcomes (4)
Percentage of Participants With Best Overall Response (BOR) Status of Angiomyolipoma (AML) Response During Maximum Treatment Duration of 48 Weeks
Baseline, 48 weeks
Percentage of Participants With Best Overall Response Status of Angiomyolipoma (AML) Progression During Maximum Treatment Duration of 48 Weeks
Baseline, 48 weeks
Percentage of Participants With Severe Renal Impairment
Baseline, 48 weeks
Percentage of Participants With NCI CTCA Grade 3/4 Serum Creatinine
Baseline, 48 weeks
Study Arms (1)
Everolimus
EXPERIMENTALParticipants targeted to receive Everolimus tablets 10 mg orally once daily for 48 weeks.
Interventions
Everolimus 2.5 mg and 5 mg tablets with dosage regimen of 10 mg orally once daily.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (5)
Novartis Investigative Site
Wuhan, Hubei, 430030, China
Novartis Investigative Site
Shanghai, Shanghai Municipality, 200032, China
Novartis Investigative Site
Chengdu, Sichuan, 610041, China
Novartis Investigative Site
Beijing, 100028, China
Novartis Investigative Site
Beijing, 100730, China
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Novartis Pharmaceuticals
Study Officials
- STUDY DIRECTOR
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 4
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 3, 2018
First Posted
May 16, 2018
Study Start
November 9, 2018
Primary Completion
September 25, 2020
Study Completion
September 25, 2020
Last Updated
September 9, 2021
Results First Posted
September 9, 2021
Record last verified: 2021-09
Data Sharing
- IPD Sharing
- Will share
Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on https://www.clinicalstudydatarequest.com/.