LymphangioleiomyomatosisPhase 1/2NCT05467397
Feasibility of [11C]Acetate-PET in LAM and TSC
A Phase 1/2 study testing [11C]acetate in people with Lymphangioleiomyomatosis and Tuberous Sclerosis Complex, run by Brigham and Women's Hospital at 1 site in United States.
Trial summary
This study aims to assess [11C]acetate positron emission tomography (PET)/computed tomography (CT) as a biomarker for renal angiomyolipomas and pulmonary lymphangioleiomyomatosis (LAM) and an early biomarker of response to rapamycin in LAM patients. Read more about this study
- Who is it for?
- Ages 18 years and older, women only
- Conditions the study is for
- Lymphangioleiomyomatosis, Tuberous Sclerosis Complex
- What is being tested?
- [11C]acetate
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- 30 Aug 2021to 30 Jun 2026
- 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
- Women only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- diagnosis of LAM (or TSC-LAM)
- age 18 or over
- at least one renal angiomyolipoma (at least 1 cm in each diameter) confirmed by CT or MRI
- no prior treatment with rapamycin/rapalogs OR candidate for initiating treatment with rapamycin/rapalogs OR under treatment with rapamycin/rapalogs for minimum 3 months and maximum of 1 year
Who cannot join
- under treatment with rapamycin or rapalogs for < 3 months or > 1 year
- participated in research studies involving radiation exposure (> 50 mSv/year) in the past 12 months
Trial locations and contacts
1 site in 1 country.
United States1 site
Brigham and Women's Hospital
Boston, Massachusetts, 02115
About this study
Official title: Feasibility Study of [11C]Acetate Positron Emission Tomography (PET) as an Indicator of Early Response to Rapamycin in Lymphangioleiomyomatosis (LAM) Patients
This study aims to assess [11C]acetate positron emission tomography (PET)/computed tomography (CT) as a biomarker for renal angiomyolipomas and pulmonary lymphangioleiomyomatosis (LAM) and an early biomarker of response to rapamycin in LAM patients.
[11C]Acetate is a radioactive form of acetate, a nutrient commonly processed in our body's cells to generate fat and energy. Preclinical studies support the hypothesis that TSC tumors enhance lipid synthesis compared to normal tissues, suggesting that quantification of [11C]acetate in these tumors by PET/CT may provide a metabolic biomarker of disease.
Participants in the study will undergo 1 or 2 PET/CT scans over 3 to 6 months at the Massachusetts General Hospital (Boston, MA). [11C]acetate is administered through an intravenous catheter. This small amount of radioactivity is short-lived and eliminated from the body within a few hours.
Read the detailed description
Lymphangioleiomyomatosis (LAM) is a rare, multisystem disease of women, consisting of a diffuse proliferation of smooth muscle actin-positive cells (LAM cells), which harbor inactivating mutations in either the TSC1 or TSC2 tumor suppressor gene. These inactivating mutations result in constitutive activation of mammalian/mechanistic TOR (target of rapamycin) complex 1 (mTORC1), which integrates growth factor and nutrient signaling to stimulate cell growth and metabolism. Pulmonary LAM is characterized by associated progressive cystic destruction of the lung parenchyma, recurrent pneumothorax, and chylous pleural effusions. Extrapulmonary proliferative lesions of LAM include renal angiomyolipomas and lymphangiomyomas. LAM can occur as a sporadic disorder where LAM cells harbor somatic inactivating mutations of the TSC1 or TSC2 gene, or in women with Tuberous Sclerosis Complex (TSC).
Rapamycin is an FKBP12-dependent allosteric inhibitor of mTORC1 approved by the FDA for the treatment of LAM and TSC-associated renal angiomyolipomas. Clinical trials of TSC and LAM have shown that 12 month-treatment with rapamycin induces response of renal angiomyolipomas and stabilization of pulmonary function. However, lung function decline and tumor growth resume when treatment is interrupted.
Sensitive and specific biomarkers of response to therapy and/or disease progression, including biomarkers of tumor metabolic activity, would facilitate clinical trials of novel therapeutic agents for LAM and enable optimization of current rapamycin-based regimens.
Our preclinical studies showed that lipogenic pathways can be detected in preclinical animal models of TSC and LAM using PET-based metabolic imaging.
The proposed study aims to quantitatively and non-invasively assess metabolic activity and response to rapamycin in LAM patients using [11C]acetate positron emission tomography (PET) imaging.
- Being tested
- [11C]acetate
- Medical subject headings
- Lymphangioleiomyomatosis, Tuberous Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
[11C]acetateDrug
Also called diagnostic test
Positron Emission Tomography (PET)
| Study group | Role | What they receive |
|---|---|---|
| Patients will undergo [11C]acetate PET/CT | Experimental | Patients will undergo a single [11c]acetate PET scan OR Patients will undergo an [11c]acetate PET scan, initiate treatment with rapamycin or rapalogs and receive a second [11c]acetate PET scan 3 or 4 months after starting the treatment. Receives: Drug: [11C]acetate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Quantitative Analysis of [11C]Acetate Uptake in LAM Lesions
Standardized uptake value (SUV) will be used to quantify uptake of [11C]acetate in kidney angiomyolipoma
Measured: Single assessment (day of imaging)
Secondary outcomes (1)
Quantitative Analysis of [11C]Acetate Uptake in LAM Lesions Following Treatment With mTORC1 Inhibitor
Standardized uptake value (SUV) will be used to quantify uptake of [11C]acetate in kidney angiomyolipoma following treatment with mTORC1 inhibitor
Measured: Single assessment conducted while participants were on treatment with mTORC1 inhibitor (day of imaging)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2022
- Study start30 Aug 2021
- Primary completion4 Jun 2024
- Study completion30 Jun 2026 (estimated)
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.
- More trials run by Brigham and Women's HospitalThe 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 1/2
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Brigham and Women's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Massachusetts General Hospital
- Responsible party
- Principal investigator, Carmen Priolo, M.D., Ph.D., Brigham and Women's Hospital
- Investigators
- Carmen P Priolo, MD PhD, Principal investigator, Brigham and Women's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05467397
- Sponsor's protocol ID
- 2019p003557
- Status last verified
- Jan 2026
- First submitted
- 18 Jul 2022
- Last updated
- 21 Jan 2026
- Results posted
- Yes, on ClinicalTrials.gov
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Jan 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feasibility Study of [11C]Acetate Positron Emission Tomography (PET) as an Indicator of Early Response to Rapamycin in Lymphangioleiomyomatosis (LAM) Patients
- ClinicalTrials.gov ID
- NCT05467397
- Acronym / sponsor ID
- 2019p003557
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Brigham and Women's HospitalUniversity, hospital or other
- Collaborators
- Massachusetts General Hospital
- Study start
- 30 Aug 2021 (actual)
- Primary completion
- 4 Jun 2024 (actual)
- Study completion
- 30 Jun 2026 (estimated)
- First posted
- 20 Jul 2022 (actual)
- Results
- Posted 21 Jan 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study aims to assess [11C]acetate positron emission tomography (PET)/computed tomography (CT) as a biomarker for renal angiomyolipomas and pulmonary lymphangioleiomyomatosis (LAM) and an early biomarker of response to rapamycin in LAM patients.
[11C]Acetate is a radioactive form of acetate, a nutrient commonly processed in our body's cells to generate fat and energy. Preclinical studies support the hypothesis that TSC tumors enhance lipid synthesis compared to normal tissues, suggesting that quantification of [11C]acetate in these tumors by PET/CT may provide a metabolic biomarker of disease.
Participants in the study will undergo 1 or 2 PET/CT scans over 3 to 6 months at the Massachusetts General Hospital (Boston, MA). [11C]acetate is administered through an intravenous catheter. This small amount of radioactivity is short-lived and eliminated from the body within a few hours.
Detailed description
Lymphangioleiomyomatosis (LAM) is a rare, multisystem disease of women, consisting of a diffuse proliferation of smooth muscle actin-positive cells (LAM cells), which harbor inactivating mutations in either the TSC1 or TSC2 tumor suppressor gene. These inactivating mutations result in constitutive activation of mammalian/mechanistic TOR (target of rapamycin) complex 1 (mTORC1), which integrates growth factor and nutrient signaling to stimulate cell growth and metabolism. Pulmonary LAM is characterized by associated progressive cystic destruction of the lung parenchyma, recurrent pneumothorax, and chylous pleural effusions. Extrapulmonary proliferative lesions of LAM include renal angiomyolipomas and lymphangiomyomas. LAM can occur as a sporadic disorder where LAM cells harbor somatic inactivating mutations of the TSC1 or TSC2 gene, or in women with Tuberous Sclerosis Complex (TSC).
Rapamycin is an FKBP12-dependent allosteric inhibitor of mTORC1 approved by the FDA for the treatment of LAM and TSC-associated renal angiomyolipomas. Clinical trials of TSC and LAM have shown that 12 month-treatment with rapamycin induces response of renal angiomyolipomas and stabilization of pulmonary function. However, lung function decline and tumor growth resume when treatment is interrupted.
Sensitive and specific biomarkers of response to therapy and/or disease progression, including biomarkers of tumor metabolic activity, would facilitate clinical trials of novel therapeutic agents for LAM and enable optimization of current rapamycin-based regimens.
Our preclinical studies showed that lipogenic pathways can be detected in preclinical animal models of TSC and LAM using PET-based metabolic imaging.
The proposed study aims to quantitatively and non-invasively assess metabolic activity and response to rapamycin in LAM patients using [11C]acetate positron emission tomography (PET) imaging.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients will undergo [11C]acetate PET/CT | Experimental | Drug: [11C]acetatePatients will undergo a single [11c]acetate PET scan OR Patients will undergo an [11c]acetate PET scan, initiate treatment with rapamycin or rapalogs and receive a second [11c]acetate PET scan 3 or 4 months after starting the treatment. |
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)
- diagnosis of LAM (or TSC-LAM)
- age 18 or over
- at least one renal angiomyolipoma (at least 1 cm in each diameter) confirmed by CT or MRI
- no prior treatment with rapamycin/rapalogs OR candidate for initiating treatment with rapamycin/rapalogs OR under treatment with rapamycin/rapalogs for minimum 3 months and maximum of 1 year
Key exclusion criteria (2 listed)
- under treatment with rapamycin or rapalogs for < 3 months or > 1 year
- participated in research studies involving radiation exposure (> 50 mSv/year) in the past 12 months
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 |
|---|---|---|
| [11C]acetateAlso: diagnostic testPositron Emission Tomography (PET) | Drug | Patients will undergo [11C]acetate PET/CT |
Primary outcome
Quantitative Analysis of [11C]Acetate Uptake in LAM Lesions
Time frame: Single assessment (day of imaging)
Standardized uptake value (SUV) will be used to quantify uptake of [11C]acetate in kidney angiomyolipoma
1 secondary outcome
Quantitative Analysis of [11C]Acetate Uptake in LAM Lesions Following Treatment With mTORC1 Inhibitor
Time frame: Single assessment conducted while participants were on treatment with mTORC1 inhibitor (day of imaging)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Jan 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Carmen P Priolo, MD PhD principal investigator
Brigham and Women's Hospital
1 site 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 [11C]acetate in people with Lymphangioleiomyomatosis and Tuberous Sclerosis Complex, run by Brigham and Women's Hospital 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 2 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: under way, not looking for participants, as of the sponsor's last update on 21 Jan 2026. 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 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 21 Jan 2026). 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?
Quantitative Analysis of [11C]Acetate Uptake in LAM Lesions (time frame: Single assessment (day of imaging)). 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 21 Jan 2026.