LymphangioleiomyomatosisPhase 1/2LAMP-1NCT03131999
LAM Pilot Study With Imatinib Mesylate
A Phase 1/2 study testing Imatinib Mesylate 400Mg Capsule in people with Lymphangioleiomyomatosis, run by Medical University of South Carolina at 2 sites in United States.
Trial summary
This is a phase 1 clinical trial comparing imatinib mesylate to placebo for individuals with lymphangioleiomyomatosis (LAM). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Lymphangioleiomyomatosis
- What is being tested?
- Imatinib Mesylate 400Mg Capsule
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 18 participants
- Where
- 2 sitesUnited States
- Study dates
- 23 Jan 2018to 7 Mar 2019
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Definite or Probable LAM
- FVC or Postbronchodilator FEV1 <90% predicted
Who cannot join
- Current or planned pregnancy or lactation
- Unwillingness to discontinue sirolimus
- Change in the dose or use of sirolimus within the past month
- Inability to perform spirometry
- Allergy or intolerance of albuterol and/or ipratropium
- Other serious illness that would impact the outcome of the study including cancer that has not received curative therapy, Grade III/IV cardiac problems as defined by the New York Heart Association Criteria. (i.e., congestive heart failure, myocardial infarction within 6 months of study), uncontrolled diabetes, chronic renal disease, chronic liver disease, or active uncontrolled infection
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- Current lung transplant
- Known diagnosis of human immunodeficiency virus (HIV) infection
- Current cigarette smoking
- Required use of warfarin, ketoconazole, itraconazole, clarithromycin, or rifampin during the 2 months of the study.
- Unwillingness to avoid grapefruit juice or St. Johns Wort during the study.
- Planned surgery during the 2 months of the study.
- Patient with any significant history of non-compliance to medical regimens or with inability to grant reliable informed consent.
- Patient has received and other investigational agents within 28 days of first day of study drug dosing.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Medical University of South Carolina
Charleston, South Carolina, 29425
Columbia University
New York, New York, 10032
About this study
This is a phase 1 clinical trial comparing imatinib mesylate to placebo for individuals with lymphangioleiomyomatosis (LAM).
Read the detailed description
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.
- Conditions
- Lymphangioleiomyomatosis
- Being tested
- Imatinib Mesylate 400Mg Capsule, Placebo - Capsule
- Keywords
- VEGF-D, Imatinib mesylate, Sirolimus
- Medical subject headings
- Lymphangioleiomyomatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Imatinib Mesylate 400Mg CapsuleDrug
Also called Gleevec
Sirolimus or everolimus will be withdrawn after 28 days if used at baseline
Placebo - CapsuleDrug
Also called Placebo
Sirolimus or everolimus will be withdrawn after 28 days if used at baseline
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Imatinib Mesylate 400Mg Capsule |
| 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. Receives: Drug: Placebo - Capsule |
What is being measured
The primary outcome is the main question the study is designed to answer.
Serum VEGF-D
Change in the square root of the intrasubject plasma VEGF-D
Measured: Before and 1 month after initiation of monotherapy imatinib mesylate or placebo
Secondary outcomes (1)
Adverse Events
Adverse event and Serious Adverse Event numbers using the CTCAE Version 4.03 definitions
Measured: 3 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Apr 2017
- Study start23 Jan 2018
- Primary completion7 Mar 2019
- Study completion7 Mar 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for LymphangioleiomyomatosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Medical University of South Carolina
- Sponsor type
- University, hospital or other
- Collaborators
- Columbia University
- Responsible party
- Principal investigator, Charlie Strange, Medical University of South Carolina
- Investigators
- Christopher Meinberg, Study director, Congressionally Directed Medical Research Programs
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03131999
- Sponsor's protocol ID
- PRO00044389
- Acronym
- LAMP-1
- Status last verified
- Jun 2020
- First submitted
- 24 Apr 2017
- Last updated
- 16 Jun 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- LAM Pilot Study With Imatinib Mesylate
- ClinicalTrials.gov ID
- NCT03131999
- Acronym / sponsor ID
- LAMP-1 / PRO00044389
- Conditions
- Lymphangioleiomyomatosis
- Keywords
- VEGF-D, Imatinib mesylate, Sirolimus
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Medical University of South CarolinaUniversity, hospital or other
- Collaborators
- Columbia University
- Study start
- 23 Jan 2018 (actual)
- Primary completion
- 7 Mar 2019 (actual)
- Study completion
- 7 Mar 2019 (actual)
- First posted
- 27 Apr 2017 (actual)
- Results
- Posted 26 Mar 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a phase 1 clinical trial comparing imatinib mesylate to placebo for individuals with lymphangioleiomyomatosis (LAM).
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Imatinib Mesylate 400mg capsule | Experimental | Drug: Imatinib Mesylate 400Mg Capsule56 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 | Drug: Placebo - Capsule56 days of Placebo with or without co-administration of an mTOR inhibitor for 28 days. A dose reduction is allowed for toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Definite or Probable LAM
- FVC or Postbronchodilator FEV1 <90% predicted
Key exclusion criteria (14 listed)
- Current or planned pregnancy or lactation
- Unwillingness to discontinue sirolimus
- Change in the dose or use of sirolimus within the past month
- Inability to perform spirometry
- Allergy or intolerance of albuterol and/or ipratropium
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Imatinib Mesylate 400Mg CapsuleAlso: GleevecSirolimus or everolimus will be withdrawn after 28 days if used at baseline | Drug | Imatinib Mesylate 400mg capsule |
| Placebo - CapsuleAlso: PlaceboSirolimus or everolimus will be withdrawn after 28 days if used at baseline | Drug | Placebo Capsule |
Primary outcome
Serum VEGF-D
Time frame: Before and 1 month after initiation of monotherapy imatinib mesylate or placebo
Change in the square root of the intrasubject plasma VEGF-D
1 secondary outcome
Adverse Events
Time frame: 3 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jun 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Christopher Meinberg study director
Congressionally Directed Medical Research Programs
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Imatinib Mesylate 400Mg Capsule in people with Lymphangioleiomyomatosis, run by Medical University of South Carolina at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Jun 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 26 Mar 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Serum VEGF-D (time frame: Before and 1 month after initiation of monotherapy imatinib mesylate or placebo). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jun 2020.