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Trial record 32 of 134 for:    SRT501

Resveratrol and Sirolimus in Lymphangioleiomyomatosis Trial (RESULT)

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ClinicalTrials.gov Identifier: NCT03253913
Recruitment Status : Not yet recruiting
First Posted : August 18, 2017
Last Update Posted : October 26, 2017
Sponsor:
Collaborator:
National Heart, Lung, and Blood Institute (NHLBI)
Information provided by (Responsible Party):
Nishant Gupta, University of Cincinnati

Brief Summary:
RESULT is a phase II dose-escalating, open-label, safety and efficacy study to determine if there is a potential benefit of resveratrol in combination with sirolimus in patients with lymphangioleiomyomatosis (LAM). The primary study objective is to assess the change in serum vascular endothelial growth factor-D (VEGF-D) level after 24 weeks of treatment with a combination of resveratrol and sirolimus as compared to the VEGF-D level in patients on a stable dose of sirolimus alone. The secondary objectives of this study include an assessment of the safety and adverse effect profile of combined resveratrol and sirolimus in adult patients with LAM, and to determine the effect of treatment with a combination of resveratrol and sirolimus on changes in lung function and quality of life.

Condition or disease Intervention/treatment Phase
Lymphangioleiomyomatosis Drug: Sirolimus Drug: Resveratrol Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 25 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Resveratrol and Sirolimus in Lymphangioleiomyomatosis Trial (RESULT)
Anticipated Study Start Date : January 15, 2018
Estimated Primary Completion Date : January 30, 2020
Estimated Study Completion Date : June 30, 2020


Arm Intervention/treatment
Experimental: Treatment Arm

Resveratrol 250mg daily for the first 8 weeks, followed by 250mg twice daily for the next 8 weeks, and then 500mg twice daily for the last 8 weeks.

Patients will be on a stable background therapy of sirolimus prior to enrolling and will continue on that regimen throughout the study.

Drug: Sirolimus
Patients will be on a stable dose of sirolimus prior to enrolling and will continue that throughout the study.
Other Name: Rapamycin
Drug: Resveratrol
Escalating doses of resveratrol will be given throughout the study. Patients will start at 250mg daily for 8 weeks, followed by 500mg daily for 8 weeks, and 500mg BID for the last 8 weeks.



Primary Outcome Measures :
  1. Change in serum VEGF-D level after treatment with a combination of resveratrol and sirolimus [ Time Frame: 24 weeks ]
    Change in serum VEGF-D level in pg/ml after treatment with a combination of resveratrol and sirolimus as compared to the VEGF-D level in patients on a stable dose of sirolimus alone.


Secondary Outcome Measures :
  1. Incidence of Treatment-Emergent Adverse Events [ Time Frame: 24 weeks ]
    Safety and adverse effect profile of combined resveratrol and sirolimus in adult patients with LAM

  2. Rate of change in Pulmonary function tests (PFTs) before and after treatment [ Time Frame: 24 weeks ]
    Difference in the rate of change as well as absolute change in PFT parameters (Forced expiratory volume in one-second (FEV1), forced vital capacity (FVC), and diffusion capacity for carbon monoxide (DLCO)) will be measured after 24 weeks of treatment.

  3. Difference in the St. George's Respiratory Questionnaire (SGRQ) quality of life assessment scores before and after treatment [ Time Frame: 24 weeks ]
    Quality of life scores will be calculated by using SGRQ to determine if the combination of resveratrol and sirolimus can improve the quality of life in patients with LAM.

  4. Difference in the A Tool to Assess Quality of Life in LAM (ATAQ-LAM) quality of life assessment scores before and after treatment [ Time Frame: 24 weeks ]
    Quality of life scores will be calculated by using ATAQ-LAM to determine if the combination of resveratrol and sirolimus can improve the quality of life in patients with LAM.

  5. Difference in the San Diego Shortness of Breath Score (SD-SOB) quality of life assessment scores before and after treatment [ Time Frame: 24 weeks ]
    Quality of life scores will be calculated by using SD-SOB to determine if the combination of resveratrol and sirolimus can improve the quality of life in patients with LAM.

  6. Difference in the EuroQOL visual analogue scale (EQ-5D) before and after treatment. [ Time Frame: 24 weeks ]
    Quality of life scores will be calculated by using EQ-5D to determine if the combination of resveratrol and sirolimus can improve the quality of life in patients with LAM.



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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   Female
Gender Based Eligibility:   Yes
Gender Eligibility Description:   LAM is a disease that almost exclusively involves females. Thus trial participants will be females only.
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Subjects enrolled in the trial must meet all of the following criteria.

  1. Definitive diagnosis LAM based on the presence of characteristic cystic change on high-resolution computed tomography (HRCT) of the chest. The diagnosis must be confirmed by one of the following:

    A) Histopathological confirmation by biopsy (lung, abdominal mass, lymph node or kidney or cytology from thoracic or abdominal sources revealing human melanoma black-45 (HMB45)+ staining of spindled/epithelioid cells) B) Compatible chest CT scan findings in the setting of tuberous sclerosis, angiomyolipomas (diagnosed by CT, magnetic resonance imaging (MRI) by the site radiologist or biopsy) or chylous pleural effusion (verified by tap) C) Chest CT scan findings compatible with LAM and a VEGF-D level ≥ 800pg/ml.

  2. Age 18 years or greater.
  3. Signed and dated informed consent
  4. Currently on sirolimus for treatment of LAM for at least 20 weeks
  5. Evidence of disease stabilization on sirolimus as demonstrated by two stable values of serum VEGF-D post initiation of sirolimus drawn at least 12 weeks apart from each other. For the purpose of this study, a variation in serum VEGF-D of less than or equal to 15% is considered stable.

Exclusion Criteria:

  • Subjects who meet any of the following criteria are not eligible for enrollment as study participants:

    1. Known allergy or hypersensitivity to Resveratrol
    2. Inability to provide informed consent
    3. Active enrollment in other clinical drug trials for LAM
    4. Pregnant or plan to become pregnant in the next 6 months
    5. Breast feeding
    6. Inability to comply with pulmonary function tests or follow up visits
    7. Inadequate contraception
    8. Use of estrogen containing medications within the 30 days prior to randomization
    9. History of organ transplant
    10. Actively listed for lung transplantation
    11. Inability to comply with study procedures or attend scheduled study visits
    12. Any clinically significant medical disease (other than LAM) that is associated with an expected survival of less than 2 years, or likely to impact the ability of the patient to participate in the study in the opinion of the investigator, or impact the study efficacy or safety assessments.

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): NCT03253913


Contacts
Contact: Nishant Gupta, MD 513-558-4831 guptans@ucmail.uc.edu
Contact: Elizabeth Kopras 513-558-4831 koprasej@ucmail.uc.edu

Locations
United States, Ohio
University of Cincinnati Active, not recruiting
Cincinnati, Ohio, United States, 45267
Sponsors and Collaborators
University of Cincinnati
National Heart, Lung, and Blood Institute (NHLBI)
Investigators
Principal Investigator: Nishant Gupta, MD University of Cincinnati

Responsible Party: Nishant Gupta, Adjunct Assistant Professor, University of Cincinnati
ClinicalTrials.gov Identifier: NCT03253913     History of Changes
Other Study ID Numbers: 2016-4904
1R34HL138235-01 ( U.S. NIH Grant/Contract )
First Posted: August 18, 2017    Key Record Dates
Last Update Posted: October 26, 2017
Last Verified: October 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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

Additional relevant MeSH terms:
Resveratrol
Lymphangioleiomyomatosis
Lymphangiomyoma
Lymphatic Vessel Tumors
Neoplasms by Histologic Type
Neoplasms
Perivascular Epithelioid Cell Neoplasms
Neoplasms, Connective and Soft Tissue
Lymphoproliferative Disorders
Lymphatic Diseases
Immunoproliferative Disorders
Immune System Diseases
Sirolimus
Everolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Anti-Inflammatory Agents, Non-Steroidal
Analgesics, Non-Narcotic
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Anti-Inflammatory Agents
Antirheumatic Agents
Antineoplastic Agents, Phytogenic