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Safety and Efficacy of Everolimus (Afinitor®) in Chinese Adult Patients With Angiomyolipoma Associated With Tuberous Sclerosis Complex.

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ClinicalTrials.gov Identifier: NCT03525834
Recruitment Status : Recruiting
First Posted : May 16, 2018
Last Update Posted : January 23, 2019
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
The purpose of this study is to assess the safety and efficacy of Afinitor® in Chinese patients with renal angiomyolipoma associated with tuberous sclerosis complex (TSC). The study will evaluate the safety and anti-tumor activity of Afinitor® given at a dose of 10 mg per day.

Condition or disease Intervention/treatment Phase
Renal Angiomyolipoma Drug: everolimus Phase 4

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 40 participants
Intervention Model: Single Group Assignment
Intervention Model Description: Single arm, open label
Masking: None (Open Label)
Primary Purpose: Treatment
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
Actual Study Start Date : November 9, 2018
Estimated Primary Completion Date : January 6, 2022
Estimated Study Completion Date : January 6, 2022


Arm Intervention/treatment
Experimental: everolimus
Everolimus oral tablets, 10 mg per day
Drug: everolimus
Everolimus 10 mg oral tablets per day
Other Name: RAD001




Primary Outcome Measures :
  1. Number of Adverse Events as a Measure of Safety and TolerabilitySafety [ Time Frame: Baseline up approximately 48 weeks ]
    Incidence of treatment-emergent AEs, SAEs and laboratory abnormalities.


Secondary Outcome Measures :
  1. Percentage of patients with angiomyolipoma response up to 48 weeks [ Time Frame: Baseline up to approximately 48 weeks ]

    At screening via MRI/CT up to five measurable lesions (≥1 cm in longest diameter) in each kidney will be defined as target angiomyolipoma. The volume of these lesions will be measured at each assessment during the trial. For non-target angiomyolipoma, the volume of each kidney will be measured. Increases in the volume will be evidence of worsening angiomyolipoma.

    Angiomyolipoma response will be defined as: a reduction in angiomyolipoma volume of at least 50% relative to screening, no new angiomyolipoma ≥ 1.0 cm in longest diameter are identified, neither kidney increases in volume by more than 20% from nadir, the patient does not have any angiomyolipoma-related bleeding of grade ≥2


  2. Percentage of patients with angiomyolipoma progression up to 48 weeks [ Time Frame: Baseline up to approximately 48 weeks ]

    At screening via MRI/CT up to five measurable lesions (≥1 cm in longest diameter) in each kidney will be defined as target angiomyolipoma. The volume of these lesions will be measured at each assessment during the trial. For non-target angiomyolipoma, the volume of each kidney will be measured. Increases in the volume will be evidence of disease progression.

    Angiomyolipoma progression will be defined as one or more of the following: an increase from nadir of 25% or more in angiomyolipoma volume to a value greater than screening, the appearance of a new angiomyolipoma ≥ 1.0 cm in longest diameter, an increase from nadir of 20% or more in the volume of either kidney to a value greater than screening, angiomyolipoma-related bleeding grade ≥2


  3. Change of renal function from baseline (change of creatinine clearance) up to 48 weeks [ Time Frame: Baseline up to 48 weeks ]
    Percentage of patients with change in renal function (CrCl < 30 mL/min) will be determined. Creatinine clearance will be calculated from the formula of Cockcroft and Gault (1976).

  4. Change of renal function from baseline (patients with serum creatinine abnormalities) [ Time Frame: Baseline up to 48 weeks ]
    Percentage of patients with NCI CTCAE grade 3/4 serum creatinine increase will be determined.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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 Other protocol-defined inclusion/exclusion criteria may apply

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03525834


Contacts
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Contact: Novartis Pharmaceuticals +41613241111 Novartis.email@novartis.com
Contact: Novartis Pharmaceuticals

Locations
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China, Hubei
Novartis Investigative Site Recruiting
Wuhan, Hubei, China, 430030
China, Shanghai
Novartis Investigative Site Recruiting
Shanghai, Shanghai, China, 200032
China, Sichuan
Novartis Investigative Site Recruiting
Chengdu, Sichuan, China, 610041
China
Novartis Investigative Site Recruiting
Beijing, China, 100028
Novartis Investigative Site Recruiting
Beijing, China, 100730
Sponsors and Collaborators
Novartis Pharmaceuticals

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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03525834     History of Changes
Other Study ID Numbers: CRAD001M2401
First Posted: May 16, 2018    Key Record Dates
Last Update Posted: January 23, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
TSC
tuberous sclerosis complex
AML
afinitor
everolimus
RAD001
mTOR
kidney
lymphangioleiomyomatosis
renal angiomyolipoma

Additional relevant MeSH terms:
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Tuberous Sclerosis
Angiomyolipoma
Sclerosis
Pathologic Processes
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
Genetic Diseases, Inborn
Neoplasms, Adipose Tissue
Neoplasms, Connective and Soft Tissue
Neoplasms by Histologic Type
Perivascular Epithelioid Cell Neoplasms
Sirolimus
Everolimus
Antineoplastic Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic