Lymphangioleiomyomatosis (LAM)Phase 1NCT04388371
Glutamine PET Imaging in LAM
A Phase 1 study testing Glutamine in people with Lymphangioleiomyomatosis (LAM), run by Vanderbilt University Medical Center at 1 site in United States.
Trial summary
In this study, subjects with spontaneous or tuberous sclerosis complex associated lymphangioleiomyomatosis (LAM) who have not been started on therapy with mTOR inhibitors such as sirolimus or everolimus to undergo a PET/CT scan using an novel PET tracer that may better evaluate disease activity in LAM subjects both… Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Lymphangioleiomyomatosis (LAM)
- What is being tested?
- Glutamine
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 5 participants
- Where
- 1 siteUnited States
- Study dates
- 18 Oct 2019to 10 Aug 2021
- Phase
- Phase 1
- Are there results?
- None posted in the registry
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
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Female subjects
- ≥ 18 years of age
- Diagnosis of LAM via either a. Histopathologic diagnosis b. Compatible CT chest and one of the following i. Tuberous Sclerosis Complex* ii. Angiomyolipoma or lymphangioma iii. Chylous Effusion iv. Serum VEGF-D level >800 pg/mL
- The diagnosis of TSC will be based on the presence of at least two major criteria or one major and one or more minor features per published guidelines.(30)
Who cannot join
- Patients with any known intrathoracic cancer (primary lung cancer or metastatic disease) or undergoing active treatment for malignancy
- Patients with use of investigational therapies for LAM either currently or in the prior 3 months
- Patients with body weight ≥400 pounds or body habitus or disability that will not permit the imaging protocol to be performed
- Patients known to be pregnant or breastfeeding
- Patients with clinically active known or suspected pulmonary infection of any type
- Patients known or suspected to have any inborn error of metabolism
Show all 11Show fewer
- Patients with known type I diabetes mellitus
- Patients who cannot have a peripheral IV for any reason
- Patients who cannot lie flat for the duration of the PET scan
- Patients who are claustrophobic
- Patients with a prior allergy to contrast agents or to PET tracers
Trial locations and contacts
1 site in 1 country.
United States1 site
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
About this study
In this study, subjects with spontaneous or tuberous sclerosis complex associated lymphangioleiomyomatosis (LAM) who have not been started on therapy with mTOR inhibitors such as sirolimus or everolimus to undergo a PET/CT scan using an novel PET tracer that may better evaluate disease activity in LAM subjects both before and after the initiation of mTOR inhibitor therapy will be enrolled. The procedure for each scan will be similar, involving one administration of the novel tracer C11-glutamine followed by a whole body PET/CT scan.
Read the detailed description
Objectives This is a hypothesis-driven prospective pilot study of the targeted PET reagent 11C-Glutamine in LAM.
The objective is to test the hypothesis that 11C-Glutamine PET/CT will demonstrate uptake within the lungs and/or associated neoplasm of patients with LAM and that this effect will be modified by treatment with mTOR inhibitors.
Rationale Our rationale is that 11C-Glutamine PET/CT may provide an improved ability to diagnose LAM, as well as predict and monitor treatment response to mTOR inhibitors.
Aims Test the hypothesis that 11C-Gln PET imaging of the lungs in humans will reflect the known "glutamine addiction" seen in mechanistic preclinical studies of LAM. As a result, PET imaging will show increased tracer uptake in affected areas of diseased lungs and will show reduced uptake after initiating treatment with mTOR inhibitors.
Approach: We will evaluate 11C-Glutamine PET/CT uptake in patients with known LAM, and if possible, we will test subjects again after 8 weeks of mTOR inhibitor therapy (either sirolimus or everolimus).
- Conditions
- Lymphangioleiomyomatosis (LAM)
- Being tested
- Glutamine
- Medical subject headings
- Lymphangioleiomyomatosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
GlutamineDrug
Glutamine will be administered by IV injection prior to PET imaging.
| Study group | Role | What they receive |
|---|---|---|
| Prior to subject taking sirolimus or everolimus | Experimental | To compare images from subjects prior to use of sirolimus or everolimus to images produced after use of sirolimus or everolimus. Receives: Drug: Glutamine |
| subjects taking sirolimus or everolimus | Experimental | To compare images from subjects prior to use of sirolimus or everolimus to images produced after use of sirolimus or everolimus. Receives: Drug: Glutamine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Evaluate the uptake of the PET tracer throughout the entire lung and any associated neoplasms (AML, Lymphangiomas) in patients with LAM
While these imaging techniques have not been used in normal populations, the pulmonary uptake of patients with known intraabdominal malignancy will serve as control for evaluation of any potential uptake. When comparing treatment effects, each patient can serve as their own control as they will have already had imaging completed prior to the initiation of therapy. VEGF-D levels will be collected from clinical laboratory assessment or will be collected at time of enrollment, and relative elevation of VEGF-D will be compared to the relative uptake of tracer within the pulmonary parenchyma of each individual patient.
Measured: 8 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 May 2020
- Study start18 Oct 2019
- Primary completion30 Jun 2021
- Study completion10 Aug 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Lymphangioleiomyomatosis (LAM)Studies of the same condition, open and under way.
- More trials run by Vanderbilt University Medical CenterThe sponsor's other registered studies.
- 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
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Test subjects with spontaneous or tuberous sclerosis complex associated lymphangioleiomyomatosis (LAM) who have not been started on therapy with mTOR inhibitors such as sirolimus or everolimus to undergo a PET/CT scan using an novel PET tracer that may better evaluate disease activity in LAM subjects both before and after the initiation of mTOR inhibitor therapy.
- Masking
- None (open label)
- Enrolment
- 5 participants (actual)
Sponsor and investigators
- Lead sponsor
- Vanderbilt University Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- The LAM Foundation
- Responsible party
- Principal investigator, Timothy Blackwell, Vanderbilt University Medical Center
- Investigators
- Timothy S Blackwell, MD, Principal investigator, Vanderbilt University Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04388371
- Sponsor's protocol ID
- 190481
- Status last verified
- Aug 2021
- First submitted
- 11 May 2020
- Last updated
- 11 Aug 2021
- Results posted
- No
- 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 11 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Glutamine PET Imaging in LAM
- ClinicalTrials.gov ID
- NCT04388371
- Acronym / sponsor ID
- 190481
- Conditions
- Lymphangioleiomyomatosis (LAM)
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 5 participants (actual)
- Lead sponsor
- Vanderbilt University Medical CenterUniversity, hospital or other
- Collaborators
- The LAM Foundation
- Study start
- 18 Oct 2019 (actual)
- Primary completion
- 30 Jun 2021 (actual)
- Study completion
- 10 Aug 2021 (actual)
- First posted
- 14 May 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
In this study, subjects with spontaneous or tuberous sclerosis complex associated lymphangioleiomyomatosis (LAM) who have not been started on therapy with mTOR inhibitors such as sirolimus or everolimus to undergo a PET/CT scan using an novel PET tracer that may better evaluate disease activity in LAM subjects both before and after the initiation of mTOR inhibitor therapy will be enrolled. The procedure for each scan will be similar, involving one administration of the novel tracer C11-glutamine followed by a whole body PET/CT scan.
Detailed description
Objectives This is a hypothesis-driven prospective pilot study of the targeted PET reagent 11C-Glutamine in LAM.
The objective is to test the hypothesis that 11C-Glutamine PET/CT will demonstrate uptake within the lungs and/or associated neoplasm of patients with LAM and that this effect will be modified by treatment with mTOR inhibitors.
Rationale Our rationale is that 11C-Glutamine PET/CT may provide an improved ability to diagnose LAM, as well as predict and monitor treatment response to mTOR inhibitors.
Aims Test the hypothesis that 11C-Gln PET imaging of the lungs in humans will reflect the known "glutamine addiction" seen in mechanistic preclinical studies of LAM. As a result, PET imaging will show increased tracer uptake in affected areas of diseased lungs and will show reduced uptake after initiating treatment with mTOR inhibitors.
Approach: We will evaluate 11C-Glutamine PET/CT uptake in patients with known LAM, and if possible, we will test subjects again after 8 weeks of mTOR inhibitor therapy (either sirolimus or everolimus).
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Test subjects with spontaneous or tuberous sclerosis complex associated lymphangioleiomyomatosis (LAM) who have not been started on therapy with mTOR inhibitors such as sirolimus or everolimus to undergo a PET/CT scan using an novel PET tracer that may better evaluate disease activity in LAM subjects both before and after the initiation of mTOR inhibitor therapy.
| Arm | Type | Receives |
|---|---|---|
| Prior to subject taking sirolimus or everolimus | Experimental | Drug: GlutamineTo compare images from subjects prior to use of sirolimus or everolimus to images produced after use of sirolimus or everolimus. |
| subjects taking sirolimus or everolimus | Experimental | Drug: GlutamineTo compare images from subjects prior to use of sirolimus or everolimus to images produced after use of sirolimus or everolimus. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Female subjects
- ≥ 18 years of age
- Diagnosis of LAM via either a. Histopathologic diagnosis b. Compatible CT chest and one of the following i. Tuberous Sclerosis Complex* ii. Angiomyolipoma or lymphangioma iii. Chylous Effusion iv. Serum VEGF-D level >800 pg/mL
Key exclusion criteria (11 listed)
- Patients with any known intrathoracic cancer (primary lung cancer or metastatic disease) or undergoing active treatment for malignancy
- Patients with use of investigational therapies for LAM either currently or in the prior 3 months
- Patients with body weight ≥400 pounds or body habitus or disability that will not permit the imaging protocol to be performed
- Patients known to be pregnant or breastfeeding
- Patients with clinically active known or suspected pulmonary infection of any type
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 |
|---|---|---|
| GlutamineGlutamine will be administered by IV injection prior to PET imaging. | Drug | Prior to subject taking sirolimus or everolimus, subjects taking sirolimus or everolimus |
Primary outcome
Evaluate the uptake of the PET tracer throughout the entire lung and any associated neoplasms (AML, Lymphangiomas) in patients with LAM
Time frame: 8 weeks
While these imaging techniques have not been used in normal populations, the pulmonary uptake of patients with known intraabdominal malignancy will serve as control for evaluation of any potential uptake. When comparing treatment effects, each patient can serve as their own control as they will have already had imaging completed prior to the initiation of therapy. VEGF-D levels will be collected from clinical laboratory assessment or will be collected at time of enrollment, and relative elevation of VEGF-D will be compared to the relative uptake of tracer within the pulmonary parenchyma of each individual patient.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2021.
- 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.
Timothy S Blackwell, MD principal investigator
Vanderbilt University Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Glutamine in people with Lymphangioleiomyomatosis (LAM), run by Vanderbilt University Medical Center at 1 site 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 4 inclusion and 11 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 11 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Evaluate the uptake of the PET tracer throughout the entire lung and any associated neoplasms (AML, Lymphangiomas) in patients with LAM (time frame: 8 weeks). 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 11 Aug 2021.