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| Name | Class |
|---|---|
| Columbia University | OTHER |
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This is a phase 1 clinical trial comparing imatinib mesylate to placebo for individuals with lymphangioleiomyomatosis (LAM).
This is a double blind, adjusted parallel design, randomized clinical trial comparing imatinib mesylate 400 mg daily or matching placebo on the primary outcome of log transformed serum VEGF-D level in patients with LAM.
Sirolimus using patients will have co-administration of Imatinib mesylate or placebo for 28 days prior to sirolimus discontinuation.
The duration of 400 mg imatinib mesylate or placebo will be 56 days, a dose reduction is allowed for toxicity.
The primary endpoint will be the change in the log transformed VEGF-D one month after monotherapy imatinib mesylate or placebo.
Total trial duration is 2 months of drug administration.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Imatinib Mesylate 400mg capsule | Experimental | 56 days of Imatinib mesylate 400 mg oral daily with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction to 200 mg daily is allowed for toxicity. |
|
| Placebo Capsule | Placebo Comparator | 56 days of Placebo with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction is allowed for toxicity. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Imatinib Mesylate 400Mg Capsule | Drug | Sirolimus or everolimus will be withdrawn after 28 days if used at baseline |
|
| Measure | Description | Time Frame |
|---|---|---|
| Serum VEGF-D | Change in the square root of the intrasubject plasma VEGF-D | Before and 1 month after initiation of monotherapy imatinib mesylate or placebo |
| Measure | Description | Time Frame |
|---|---|---|
| Adverse Events | Adverse event and Serious Adverse Event numbers using the CTCAE Version 4.03 definitions | 3 months |
| Measure | Description | Time Frame |
|---|---|---|
| Lung Function | FEV1 % predicted change | 2 months |
| SGRQ | Saint Georges Respiratory Questionnaire change | 2 months |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Christopher Meinberg | Congressionally Directed Medical Research Programs | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Columbia University | New York | New York | 10032 | United States | ||
| Medical University of South Carolina |
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18 Subjects signed consent, 2 subjects failed baseline evaluation and were not randomized. 1 subject was randomized, took 4 placebo pills and stopped (without side effects) and is deemed not evaluable.
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| ID | Title | Description |
|---|---|---|
| FG000 | Imatinib Mesylate 400mg Capsule | 56 days of Imatinib mesylate 400 mg oral daily with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction to 200 mg daily is allowed for toxicity. Imatinib Mesylate 400Mg Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline |
| FG001 | Placebo Capsule | 56 days of Placebo with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction is allowed for toxicity. Placebo - Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
Withdrawal after 4 pills of placebo is not included in baseline population
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| ID | Title | Description |
|---|---|---|
| BG000 | Imatinib Mesylate 400mg Capsule | 56 days of Imatinib mesylate 400 mg oral daily with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction to 200 mg daily is allowed for toxicity. Imatinib Mesylate 400Mg Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline |
| BG001 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Serum VEGF-D | Change in the square root of the intrasubject plasma VEGF-D | Posted | Mean | Standard Deviation | Log transformed VEGF-D change pg/dL | Before and 1 month after initiation of monotherapy imatinib mesylate or placebo |
|
3 months
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Imatinib Mesylate 400mg Capsule | 56 days of Imatinib mesylate 400 mg oral daily with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction to 200 mg daily is allowed for toxicity. Imatinib Mesylate 400Mg Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea and/or vomitting | Gastrointestinal disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Charlton Strange, MD | Medical University of South Carolina | 843-792-3174 | strangec@musc.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Mar 28, 2018 | Mar 13, 2020 | Prot_SAP_001.pdf |
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| ID | Term |
|---|---|
| D018192 | Lymphangioleiomyomatosis |
| ID | Term |
|---|---|
| D008203 | Lymphangiomyoma |
| D018190 | Neoplasm, Lymphatic Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
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| ID | Term |
|---|---|
| D000068877 | Imatinib Mesylate |
| ID | Term |
|---|---|
| D001549 | Benzamides |
| D000577 | Amides |
| D009930 | Organic Chemicals |
| D001565 | Benzoates |
| D000146 |
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| Placebo - Capsule | Drug | Sirolimus or everolimus will be withdrawn after 28 days if used at baseline |
|
|
| Charleston |
| South Carolina |
| 29425 |
| United States |
| Placebo Capsule |
56 days of Placebo with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction is allowed for toxicity. Placebo - Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Forced Expiratory Volume in 1 Second | Geometric Mean | Standard Deviation | Percent predicted |
|
|
|
| Secondary | Adverse Events | Adverse event and Serious Adverse Event numbers using the CTCAE Version 4.03 definitions | Posted | Number | number of events | 3 months |
|
|
|
| Other Pre-specified | Lung Function | FEV1 % predicted change | Not Posted | 2 months | Participants |
| Other Pre-specified | SGRQ | Saint Georges Respiratory Questionnaire change | Not Posted | 2 months | Participants |
| 0 |
| 8 |
| 0 |
| 8 |
| 8 |
| 8 |
| EG001 | Placebo Capsule | 56 days of Placebo with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction is allowed for toxicity. Placebo - Capsule: Sirolimus or everolimus will be withdrawn after 28 days if used at baseline | 0 | 7 | 0 | 7 | 5 | 7 |
| Fatigue | General disorders | Systematic Assessment |
|
| Insomnia | General disorders | Systematic Assessment |
|
| Headache | Nervous system disorders | Systematic Assessment |
|
| Elevated Trough Sirolimus | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
|
| Hypoxemia | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
|
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| D054973 |
| Perivascular Epithelioid Cell Neoplasms |
| D018204 | Neoplasms, Connective and Soft Tissue |
| D008232 | Lymphoproliferative Disorders |
| D008206 | Lymphatic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D007160 | Immunoproliferative Disorders |
| D007154 | Immune System Diseases |
| Acids, Carbocyclic |
| D002264 | Carboxylic Acids |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D010879 | Piperazines |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D011743 | Pyrimidines |