Disease · MONDO_0011705

lymphangioleiomyomatosis

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. lymphangioleiomyomatosis. Diseases, record MONDO_0011705. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0011705

On this page

Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: lymphangioleiomyomatosis; Open Targets
Open Targets ID: MONDO_0011705; Open Targets
Other names: lymphangioleiomyomatosis, somatic, lymphangiomyomatosis; Open Targets
MeSH: D018192; MeSH
NCI Thesaurus: C3725; NCI Thesaurus
Highest stage of its drugs: Approval, for 1 of its 13 drugs, from PMDA and TTD; Open Targets

Drugs13

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
SIROLIMUS	CHEMBL413	Small molecule	Approval	PMDA and TTD	10	7	7	Drugs and targets
EVEROLIMUS	CHEMBL1908360	Small molecule	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
SARACATINIB	CHEMBL217092	Small molecule	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
CELECOXIB	CHEMBL118	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
LETROZOLE	CHEMBL1444	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
LORATADINE	CHEMBL998	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
NINTEDANIB	CHEMBL502835	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
OCTREOTIDE	CHEMBL1680	Protein	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
RESVERATROL	CHEMBL165	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
ALBUTEROL	CHEMBL714	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets

1 to 10 of 13

See all 13 drugs

Targets25

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
ABL1	Bcr/Abl fusion protein	3	Bcr/Abl fusion protein inhibitor; Tyrosine-protein kinase ABL inhibitor	Phase 2	https://conductscience.org/library/genes/25	Drugs and targets
PDGFRB	Platelet-derived growth factor receptor	3	Platelet-derived growth factor receptor beta inhibitor; Platelet-derived growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/5159	Drugs and targets
BCR	Bcr/Abl fusion protein	2	Bcr/Abl fusion protein inhibitor	Phase 1/2	https://conductscience.org/library/genes/613	Drugs and targets
FKBP1A	Peptidyl-prolyl cis-trans isomerase FKBP1A	2	FK506-binding protein 1A inhibitor	Approval	https://conductscience.org/library/genes/2280	Drugs and targets
KIT	Mast/stem cell growth factor receptor Kit	2	Stem cell growth factor receptor inhibitor	Phase 1/2	https://conductscience.org/library/genes/3815	Drugs and targets
ADRB2	Beta-2 adrenergic receptor	1	Beta-2 adrenergic receptor agonist	Phase 1/2	https://conductscience.org/library/genes/154	Drugs and targets
CYP19A1	Aromatase	1	Cytochrome P450 19A1 inhibitor	Phase 2	https://conductscience.org/library/genes/1588	Drugs and targets
FGFR1	Fibroblast growth factor receptor	1	Fibroblast growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/2260	Drugs and targets
FGFR2	Fibroblast growth factor receptor	1	Fibroblast growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/2263	Drugs and targets
FGFR3	Fibroblast growth factor receptor	1	Fibroblast growth factor receptor inhibitor	Phase 2	https://conductscience.org/library/genes/2261	Drugs and targets

1 to 10 of 25

See all 25 targets

Trials18

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00414648	Efficacy and Safety of Sirolimus in LAM	Phase 3	89	2023-11-02	Lymphangioleiomyomatosis	SIROLIMUS	Its record names the disease	ClinicalTrials.gov
NCT00126672	RAPAMYCIN FOR KIDNEY ANGIOMYOLIPOMAS	Phase 2	36	2022-04-25	Nonmalignant Neoplasm, Tuberous Sclerosis, Lymphangioleimyomatosis, Kidney Angiomyolipoma	SIROLIMUS	Its record names the disease	ClinicalTrials.gov
NCT03304678	Discovery of Sirolimus Sensitive Biomarkers in Blood	Phase 2	33	2026-02-18	Lymphangioleiomyomatosis	SIROLIMUS	Its record names the disease	ClinicalTrials.gov
NCT03253913	Resveratrol and Sirolimus in Lymphangioleiomyomatosis Trial	Phase 2	25	2023-02-01	Lymphangioleiomyomatosis	RESVERATROL, SIROLIMUS	Its record names the disease	ClinicalTrials.gov
NCT01059318	A Study to Determine the Safety and Effectiveness of RAD001 (Everolimus) in Patients With Lymphangioleiomyomatosis	Phase 2	24	2020-11-19	Lymphangioleiomyomatosis	EVEROLIMUS	Its record names the disease	ClinicalTrials.gov
NCT03131999	LAM Pilot Study With Imatinib Mesylate	Phase 1/2	18	2020-03-26	Lymphangioleiomyomatosis	IMATINIB MESYLATE	Its record names the disease	ClinicalTrials.gov
NCT01353209	Letrozole for Lymphangioleiomyomatosis	Phase 2	17	2024-04-17	Lymphangioleiomyomatosis	LETROZOLE	Its record names the disease	ClinicalTrials.gov
NCT01687179	Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis	Phase 1	14	2018-10-11	Lymphangioleiomyomatosis	HYDROXYCHLOROQUINE, SIROLIMUS	Its record names the disease	ClinicalTrials.gov
NCT02484664	COLA: A Pilot Clinical Trial of COX-2 Inhibition in LAM and TSC	Phase 2	12	2022-01-20	Lymphangioleiomyomatosis (LAM)	CELECOXIB	Its record names the disease	ClinicalTrials.gov
NCT00005906	Treatment With Octreotide in Patients With Lymphangioleiomyomatosis	Phase 2	4	2010-04-30	Lymphangioleiomyomatosis, Lymphangiomyomas, Pleural Effusions, Ascites	OCTREOTIDE	Its record names the disease	ClinicalTrials.gov

1 to 10 of 18

See all 18 trials

Comparisons in its trials7

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial	Source
Sirolimus Treatment Arm vs Placebo Treatment Arm: deaths from any cause	0 of 46	1 of 43	89	NCT00414648	Trial comparisons
Sirolimus Treatment Arm vs Placebo Treatment Arm: serious adverse events	23 of 46	18 of 43	89	NCT00414648	Trial comparisons
Letrozole vs Placebo: serious adverse events	0 of 9	1 of 8	17	NCT01353209	Trial comparisons
Imatinib Mesylate 400mg Capsule vs Placebo Capsule: deaths from any cause	0 of 8	0 of 7	15	NCT03131999	Trial comparisons
Imatinib Mesylate 400mg Capsule vs Placebo Capsule: serious adverse events	0 of 8	0 of 7	15	NCT03131999	Trial comparisons
Sirolimus and Hydroxychloroquine 200 mg vs Sirolimus and Hydroxychloroquine 400 mg: deaths from any cause	0 of 3	0 of 10	13	NCT01687179	Trial comparisons
Sirolimus and Hydroxychloroquine 200 mg vs Sirolimus and Hydroxychloroquine 400 mg: serious adverse events	0 of 3	1 of 10	13	NCT01687179	Trial comparisons

Papers from its trials21

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
30846465	RESULT	1	PubMed
28570161	RESULT	1	PubMed
26799509	RESULT	1	PubMed
25979023	RESULT	1	PubMed
24591665	RESULT	1	PubMed
24556663	RESULT	1	PubMed
24159565	RESULT	1	PubMed
21410393	RESULT	2	PubMed
12172553	RESULT	1	PubMed
39489895	DERIVED	1	PubMed

1 to 10 of 21

See all 21 papers from its trials

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0011705

GET https://conductscience.org/api/diseases/v1/MONDO_0011705/targets?page=2

API reference For AI assistants