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Rapamycin

Sirolimus · Rapamune · mTOR inhibitor

Metabolic & Circadian HormonesApproved
MW
914.2g/mol
Formula
C51H79NO13

Rapamycin, also known as sirolimus, is a macrocyclic lactone originally derived from the bacterium Streptomyces hygroscopicus, found in soil samples from Easter Island. Researchers primarily study rapamycin for its immunosuppressive properties and its potential role in extending lifespan and healthspan through the inhibition of the mechanistic target of rapamycin (mTOR) pathway. Key findings indicate that rapamycin can effectively inhibit T cell proliferation and has shown promise in preclinical models for promoting longevity and reducing age-related diseases. Clinical evidence suggests that rapamycin is currently approved for preventing organ transplant rejection and has garnered interest for off-label use in aging research. Ongoing studies continue to explore its applications and mechanisms, highlighting its significance in both transplantation medicine and the field of gerontology.

Overview

Übersicht

Rapamycin, also known as sirolimus or Rapamune, is a synthetic compound derived from a natural product produced by the bacterium Streptomyces hygroscopicus, originally isolated from a soil sample from Easter Island (Rapa Nui). It belongs to the class of macrocyclic immunosuppressive drugs and is recognized for its potent immunosuppressive, antifungal, and antiproliferative properties. Researchers have found rapamycin to play a significant role in immunosuppressive therapy, particularly in the prevention of organ transplant rejection. It has also gained attention for its potential in anti-aging therapies and cancer treatment due to its ability to inhibit the mTOR pathway. The primary mechanism of action of rapamycin involves the formation of a complex with the intracellular protein FKBP12, which then inhibits the mechanistic target of rapamycin (mTOR) pathway. This inhibition blocks cell-cycle progression at the G1 to S phase transition, affecting cell growth, proliferation, and survival. Pharmacokinetic properties of rapamycin include a variable oral bioavailability due to first-pass metabolism, with a half-life of approximately 62 hours when administered orally. It is metabolized primarily by the liver. Clinically, rapamycin is FDA-approved for the prevention of organ transplant rejection and for use in Tuberous Sclerosis Complex-related seizure therapy. It is also used off-label for potential longevity benefits, although this use is not widely recognized by the clinical community. Regulatory authorities have approved its use based on its unique mechanism and efficacy in immunosuppression.

Chemical profile

Chemische Struktur

Chemical structure of Rapamycin
FormelC51H79NO13
Molekulargewicht914.2g/mol
CAS-Nummer53123-88-9
PubChem CID5284616
Mechanism

Wirkmechanismus

Rapamycin acts primarily on the mechanistic target of rapamycin (mTOR) pathway by forming a complex with the intracellular protein FKBP12. This complex inhibits mTOR, a key regulator of cell growth and proliferation, thereby blocking cell-cycle progression from G1 to S phase.

Mechanism

Signalweg

Rapamycin (sirolimus) exerts its effects by forming a complex with the intracellular protein FKBP12, which subsequently inhibits the mechanistic target of rapamycin (mTOR) signaling pathway. This inhibition disrupts the activation of downstream effectors, such as p70S6 kinase and the cdk2/cyclin E complex, leading to cell-cycle arrest at the G1/S transition and suppression of T cell proliferation and cytokine production. While the overall mechanism is well-characterized, some aspects, such as the precise role of the sirolimus effector protein (SEP) in cell-cycle progression, remain to be fully elucidated.

Half-Life & Pharmacokinetics

POOral

Approximately 62 hours

Oral bioavailability is variable due to first-pass metabolism.

Storage

Temperature

Store at room temperature (15-30C)

Light

Protect from light

Form

Stable in tablet form

Notes

Ensure container is tightly closed to protect from moisture.

Solubility

Löslichkeit

Rapamycin is poorly soluble in water but soluble in ethanol and other organic solvents.

Legal Status

🇩🇪DE

Prescription only (verschreibungspflichtig), not listed under BtMG.

🇺🇸US

FDA approved for specific uses; prescription required.

🇦🇺AU

TGA Schedule 4 (prescription only medicine).

🇬🇧UK

Prescription only medicine (POM) under MHRA.

Legal status information is provided for general reference only and may not reflect the most current regulatory changes. Always verify with official government sources before making any decisions.

Open Questions

Offene Forschungsfragen

Current evidence is limited regarding the long-term safety and efficacy of off-label rapamycin use for promoting healthspan in healthy adults, necessitating larger randomized controlled trials (RCTs) to establish definitive outcomes. Additionally, the specific mechanisms underlying the immunosuppressive and anti-aging effects of rapamycin, particularly the role of the sirolimus effector protein (SEP) in cell-cycle progression, remain poorly understood and warrant further investigation. Further research is needed to explore the effects of rapamycin across diverse populations, including older adults and those with pre-existing health conditions, to better assess its therapeutic potential and side effect profile in these groups.

73 Research Publications

4,249

Total Citations

19

Human/RCT

3.8

Avg. Influence

2025

Latest

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#01

Sirolimus: its discovery, biological properties, and mechanism of action.

Sehgal S N · Transplantation proceedings · 2003

ReviewInfluence21.0
745
Researchers observed that sirolimus, derived from Streptomyces hygroscopicus, exhibits potent immunosuppressive and antitumor activities through mTOR pathway inhibition.

Key findings

  1. 01Sirolimus was originally discovered as an antifungal agent.
  2. 02It shows significant anticancer effects in various tumor models.
  3. 03Sirolimus is effective in preventing organ rejection due to its unique mechanism of action.
#02

Rapamune (RAPA, rapamycin, sirolimus): mechanism of action immunosuppressive effect results from blockade of signal transduction and inhibition of cell cycle progression.

Sehgal S N · Clinical biochemistry · 1998

ReviewInfluence17.0
697
The study demonstrated that rapamycin's immunosuppressive effect is mediated through the blockade of signal transduction and inhibition of cell cycle progression.

Key findings

  1. 01Rapamune is currently in Phase III trials for kidney transplantation.
  2. 02It has a unique mechanism that interferes with cellular signaling pathways.
  3. 03The drug's effects provide insights into immune system regulation.
#03

Rapamune (Sirolimus, rapamycin): an overview and mechanism of action.

Sehgal S N · Therapeutic drug monitoring · 1995

AnimalInfluence6.0
273
The study demonstrated that rapamycin effectively inhibits allograft rejection in animal models, acting at a later stage in T-cell cycle progression.

Key findings

  1. 01Rapamune inhibits organ rejection in animal transplant models.
  2. 02It binds to a protein that alters T-cell activity.
  3. 03The drug affects key proteins involved in cell cycle progression.
#04

mTOR inhibitors: an overview.

ReviewInfluence5.0
191
Researchers observed that mTOR inhibitors like sirolimus offer distinct immunosuppressive mechanisms compared to other macrolides, with potential benefits in transplantation.
#05

Everolimus and sirolimus in transplantation-related but different.

ReviewInfluence6.0
157
Researchers observed distinct pharmacokinetic and pharmacodynamic properties between sirolimus and everolimus, impacting their use in transplantation and cancer therapy.
#06

Sirolimus: a comprehensive review.

HumanInfluence5.0
148
Researchers observed that sirolimus significantly reduces acute rejection episodes in renal transplantation when combined with cyclosporine, improving long-term graft survival.
#07

Treatment of Lymphatic Malformations with the mTOR Inhibitor Sirolimus: A Systematic Review.

ReviewInfluence7.0
143
The systematic review indicated that sirolimus may be effective for treating extensive lymphatic malformations, with partial remission observed in the majority of patients, although further studies are needed.
#08

Sirolimus in renal transplantation.

ReviewInfluence5.0
122
The study demonstrated that sirolimus provides a promising alternative for maintenance immunosuppression in renal transplantation due to its lower nephrotoxicity compared to calcineurin inhibitors.
#09

Benefit-risk assessment of sirolimus in renal transplantation.

ReviewInfluence4.0
107
Researchers observed that sirolimus can prolong graft survival in renal transplantation, but its combination with calcineurin inhibitors may lead to nephrotoxicity.
#10

Functional consequences of mTOR inhibition.

ReviewInfluence3.0
95
The review described the pivotal role of mTOR in various diseases and the functional consequences of its inhibition by rapamycin, with implications for therapeutic strategies.

Clinical Trials (63)

Preclinical
Phase I
Phase II
Phase III
Approved

63

Total Trials

8,613

Total Enrolled

Efficacy and Safety of Sirolimus to Vascular Anomalies

NCT03583307COMPLETED
Sponsor

West China Hospital

Enrollment

126

Started

2018

Primary outcome

Volumetric changes in complicated vascular anomalies to sirolimus

Vascular Anomaly

Pilot Trial of Sirolimus/MEC in High Risk Acute Myelogenous Leukemia (AML)

NCT01184898COMPLETED
Sponsor

Sidney Kimmel Comprehensive Cancer Center at Thomas Jefferson University

Enrollment

36

Started

2010

Primary outcome

Association Between the Magnitude of mTOR Target Inhibition Post-treatment in Leukemic Blasts and Clinical Response in Patients With High Risk AML Treated With Sirolimus MEC

AML

Impacts of Mechanistic Target of Rapamycin (mTOR) Inhibition on Aged Human Muscle (Rapamune)

NCT05414292RECRUITING
Sponsor

University of Nottingham

Enrollment

16

Started

2021

Primary outcome

Change in muscle mass from baseline

Muscle AtrophyAge-Related Sarcopenia

Neo-adjuvant Temsirolimus in Patients With Advanced Renal Cell Carcinoma

NCT01404104COMPLETED
Sponsor

St. Joseph's Healthcare Hamilton

Enrollment

11

Started

2008

Primary outcome

The rate of change in response of primary tumor and metastases (if applicable)in participants.

Renal Cell Carcinoma

Conversion to Sirolimus: Effects in Cytomegalovirus Infection Recurrence

NCT02671318Phase 4UNKNOWN
Sponsor

Hospital do Rim e Hipertensão

Enrollment

250

Started

2015

Primary outcome

Cytomegalovirus infection/disease recurrence

Cytomegalovirus Infections

Open Label Comparative Study Of De Novo Renal Allograft Recipients Receiving CSA + MMF + Corticosteroids Versus CSA + Rapamune + Corticosteroids

NCT01601821Phase 4COMPLETED
Sponsor

Pfizer

Enrollment

245

Started

2006

Primary outcome

Incidence of Efficacy Failure

Inflammation

Cosibelimab for CSCC in Patients With Kidney Transplant or Hematologic Malignancy

NCT07426484Phase 4NOT_YET_RECRUITING
Sponsor

Dana-Farber Cancer Institute

Enrollment

80

Started

2026

Primary outcome

Best Overall Response Rate (BORR)

Cutaneous Squamous Cell CarcinomaSkin Cancer

Prevention of Skin Cancer in High Risk Patients After Conversion to a Sirolimus-based Immunosuppressive Protocol

NCT01797315Phase 4TERMINATED
Sponsor

University Hospital of Berlin

Enrollment

40

Started

2007

Primary outcome

Progression of actinic keratosis or invasive squamous cell carcinoma (SCC) or incidence/reoccurrence of neoplastic skin tumors

Renal Transplant Patients at High-risk for Skin Cancer

Rapamycin In Angiomyolipomas In Patients With Tuberous Sclerosis

NCT01217125Phase 4COMPLETED
Sponsor

Fundacio Puigvert

Enrollment

18

Started

2008

Primary outcome

Angiomyolipoma volume

Angiomyolipoma

Study to Evaluate Safety and Efficacy of Early Calcineurin Inhibitor Withdrawal in Primary Renal Allografts

NCT00374647Phase 4COMPLETED
Sponsor

University of Cincinnati

Enrollment

17

Started

2005

Primary outcome

Composite endpoint of either acute rejection, biopsy proven calcineurin inhibitor toxicity, or failure of the estimated BSA indexed GFR by MDRD method to improve by 20% from time randomization to 12 months.

Primary Renal Allograft

Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome

NCT03781050Phase 4UNKNOWN
Sponsor

Peking Union Medical College Hospital

Enrollment

10

Started

2018

Primary outcome

Total load of PJS-related intestinal polyps Total load of PJS-related intestinal polyps Total load of PJS-related intestinal polyps

Peutz-Jeghers Syndrome

Study to Evaluate the Safety and Efficacy of Sirolimus, in Subject With Refractory Solid Tumors

NCT02688881Phase 4COMPLETED
Sponsor

Samsung Medical Center

Enrollment

6

Started

2017

Primary outcome

Progression Free survival

Refractory Solid Tumors

Study Comparing Bevacizumab + Temsirolimus vs. Bevacizumab + Interferon-Alfa In Advanced Renal Cell Carcinoma Subjects

NCT00631371Phase 3COMPLETED
Sponsor

Pfizer

Enrollment

791

Started

2008

Primary outcome

Progression-Free Survival (PFS): Independent-Assessment

Renal Cell Carcinoma

Study Evaluating Sirolimus in Kidney Transplant Recipients.

NCT00167947Phase 3COMPLETED
Sponsor

Wyeth is now a wholly owned subsidiary of Pfizer

Enrollment

150

Started

2005

Primary outcome

To evaluate the safety and efficacy of an immunosuppressive regimen using synergic action of SRL and CsA, but which will limit (ab initio), the nephrotic effect of such combination. To compare Rapamune/low dose Cyclosporine vs. Rapamune/Steroids.

Kidney FailureGraft vs Host Disease

Efficacy and Safety of Sirolimus in Active Systemic Lupus Erythematosus

NCT04582136Phase 3ACTIVE_NOT_RECRUITING
Sponsor

Chinese SLE Treatment And Research Group

Enrollment

146

Started

2021

Primary outcome

The Proportion of Patients Who Achieve an SLE Responder Index-4 (SRI-4) Composite Response at Week 24

Systemic Lupus Erythematosus

Topical Sirolimus in Chemoprevention of Facial Squamous Cell Carcinomas in Solid Organ Transplant Recipients (SiroSkin)

NCT05860881Phase 3ACTIVE_NOT_RECRUITING
Sponsor

Melanoma and Skin Cancer Trials Limited

Enrollment

146

Started

2024

Primary outcome

KC development

Solid Organ Transplant RecipientsSkin Cancer

Sirolimus-based Immunosuppression Treatment Regimen for Liver Transplantation

NCT03500848Phase 2/3UNKNOWN
Sponsor

Southern Medical University, China

Enrollment

130

Started

2018

Primary outcome

HCC recurrence free survival

Liver Transplantation

Trial of Sirolimus for Cognitive Impairment in Sturge-Weber Syndrome

NCT03047980Phase 2/3COMPLETED
Sponsor

Anne Comi, MD

Enrollment

10

Started

2017

Primary outcome

Change in Cognitive Function as Assessed by the National Institute of Health (NIH) Toolbox Cognitive Battery

Sturge-Weber Syndrome

TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer

NCT02693535Phase 2RECRUITING
Sponsor

American Society of Clinical Oncology

Enrollment

4,200

Started

2016

Primary outcome

Objective Response Rate defined as % of participants in a cohort with complete or partial response or with stable disease according to standard response criteria

Lymphoma, Non-HodgkinMultiple MyelomaAdvanced Solid Tumors

Temsirolimus and Bevacizumab in Treating Patients With Advanced Endometrial, Ovarian, Liver, Carcinoid, or Islet Cell Cancer

NCT01010126Phase 2COMPLETED
Sponsor

National Cancer Institute (NCI)

Enrollment

252

Started

2009

Primary outcome

Progression Free Survival Rate

Adult Hepatocellular CarcinomaAdvanced Adult Hepatocellular CarcinomaEndometrial Serous AdenocarcinomaLocalized Non-Resectable Adult Liver CarcinomaLung Carcinoid TumorMalignant Pancreatic GastrinomaMalignant Pancreatic GlucagonomaMalignant Pancreatic InsulinomaMalignant Pancreatic SomatostatinomaMetastatic Digestive System Neuroendocrine Tumor G1Ovarian CarcinosarcomaOvarian Endometrioid AdenocarcinomaOvarian Seromucinous CarcinomaOvarian Serous Surface Papillary AdenocarcinomaPancreatic Alpha Cell AdenomaPancreatic Beta Cell AdenomaPancreatic Delta Cell AdenomaPancreatic G-Cell AdenomaPancreatic Polypeptide TumorRecurrent Adult Liver CarcinomaRecurrent Digestive System Neuroendocrine Tumor G1Recurrent Fallopian Tube CarcinomaRecurrent Ovarian CarcinomaRecurrent Pancreatic Neuroendocrine CarcinomaRecurrent Primary Peritoneal CarcinomaRecurrent Uterine Corpus CarcinomaRegional Digestive System Neuroendocrine Tumor G1Stage IIIA Fallopian Tube CancerStage IIIA Ovarian CancerStage IIIA Primary Peritoneal CancerStage IIIA Uterine Corpus CancerStage IIIB Fallopian Tube CancerStage IIIB Ovarian CancerStage IIIB Primary Peritoneal CancerStage IIIB Uterine Corpus CancerStage IIIC Fallopian Tube CancerStage IIIC Ovarian CancerStage IIIC Primary Peritoneal CancerStage IIIC Uterine Corpus CancerStage IV Fallopian Tube CancerStage IV Ovarian CancerStage IV Primary Peritoneal CancerStage IVA Uterine Corpus CancerStage IVB Uterine Corpus CancerUterine Carcinosarcoma

Advancing Transplantation Outcomes in Children

NCT06055608Phase 2RECRUITING
Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Enrollment

200

Started

2024

Primary outcome

Incidence of de novo Donor Specific Antibody (dnDSA) (central lab) OR decline in estimated glomerular filtration rate (eGFR) >7.5 mL/min/1.73m^2 (central lab)

Kidney Transplant

Rapamycin Versus Mycophenolate Mofetil in Kidney-Pancreas Recipients

NCT00533442Phase 2COMPLETED
Sponsor

University of Miami

Enrollment

170

Started

2000

Primary outcome

Event-Specific Survival Comparisons

Type 1 Diabetes

Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy

NCT02979873Phase 2ACTIVE_NOT_RECRUITING
Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Enrollment

84

Started

2016

Primary outcome

To determine if the rate of relapse at 24 months after CSA discontinuation can be improved by conversion to sirolimus in severe aplastic anemia patients who have responded to IST.

Severe Aplastic Anemia

Study Evaluating The Safety, Efficacy & Pharmacokinetics Of Temsirolimus(CCI-779) In Subjects With Advanced Renal Cell Carcinoma

NCT00494091Phase 2COMPLETED
Sponsor

Pfizer

Enrollment

82

Started

2007

Primary outcome

Percentage of Participants With Clinical Benefit

Advanced Renal Cell Carcinoma

HLA-Mismatched Unrelated Donor Bone Marrow Transplantation With Post-Transplantation Cyclophosphamide

NCT02793544Phase 2COMPLETED
Sponsor

Center for International Blood and Marrow Transplant Research

Enrollment

80

Started

2016

Primary outcome

Overall Survival

Myelodysplastic Syndrome (MDS)Chronic Lymphocytic Leukemia (CLL)Chemotherapy-sensitive LymphomaAcute Lymphoblastic Leukemia (ALL)/T Lymphoblastic LymphomaAcute Myelogenous Leukemia (AML)Acute Biphenotypic Leukemia (ABL)Acute Undifferentiated Leukemia (AUL)

Hemorrhagic Brainstem Cavernous Malformations Treatment With Sirolimus: aSingle Centre, Randomized, Placebo-controlled Pilot Trial

NCT06091332Phase 2RECRUITING
Sponsor

Huashan Hospital

Enrollment

75

Started

2024

Primary outcome

The primary outcome is to explore the safety of sirolimus in the management of BSCMs.

Cavernous MalformationsIntracerebral HemorrhageBrainstem Stroke

Itacitinib, Tacrolimus, and Sirolimus for the Prevention of GVHD in Patients With Acute Leukemia, Myelodysplastic Syndrome, or Myelofibrosis Undergoing Reduced Intensity Conditioning Donor Stem Cell Transplantation

NCT04339101Phase 2COMPLETED
Sponsor

City of Hope Medical Center

Enrollment

59

Started

2020

Primary outcome

Graft-versus-host Disease Free Relapse Free (GRFS) at 1 Year

Acute LeukemiaHematologic and Lymphocytic DisorderMyelodysplastic SyndromePrimary MyelofibrosisSecondary Myelofibrosis

Intravitreal Injections of Sirolimus in the Treatment of Geographic Atrophy

NCT01675947Phase 2TERMINATED
Sponsor

National Eye Institute (NEI)

Enrollment

52

Started

2012

Primary outcome

Rate of change in area of geographic atrophy

Geographic Atrophy

Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations

NCT02042326Phase 2UNKNOWN
Sponsor

Centre Hospitalier Universitaire, Amiens

Enrollment

50

Started

2014

Primary outcome

Treatment efficacy at M12

Arteriovenous Malformations

Efficacy Study of Temsirolimus to Treat Head and Neck Cancer

NCT01172769Phase 2COMPLETED
Sponsor

Hannover Medical School

Enrollment

42

Started

2010

Primary outcome

Progression free rate

Head and Neck Squamous Cell Carcinoma

Avastin and Temsirolimus Following Tyrosine Kinase Inhibitor Failure in Patients With Advanced Renal Cell Carcinoma

NCT00782275Phase 2COMPLETED
Sponsor

Beth Israel Deaconess Medical Center

Enrollment

41

Started

2009

Primary outcome

4-month Progression-Free Survival Rate

Renal Cell CarcinomaKidney Cancer

Rapamycin - Effects on Alzheimer's and Cognitive Health

NCT04629495Phase 2ACTIVE_NOT_RECRUITING
Sponsor

The University of Texas Health Science Center at San Antonio

Enrollment

40

Started

2021

Primary outcome

Number of adverse events

Mild Cognitive ImpairmentAlzheimer Disease

Discovery of Sirolimus Sensitive Biomarkers in Blood

NCT03304678Phase 2COMPLETED
Sponsor

National Heart, Lung, and Blood Institute (NHLBI)

Enrollment

33

Started

2017

Primary outcome

Number of Sirolimus-Sensitive Plasma Protein Biomarkers in Lymphangioleiomyomatosis (LAM) Participants

Lymphangioleiomyomatosis

CCI-779 in Treating Patients With Recurrent Glioblastoma Multiforme

NCT00016328Phase 2COMPLETED
Sponsor

National Cancer Institute (NCI)

Enrollment

33

Started

2001

Primary outcome

Percentage of patients being progression free

Adult Giant Cell GlioblastomaAdult GlioblastomaAdult GliosarcomaRecurrent Adult Brain Tumor

Sirolimus Treatment in Hospitalized Patients With COVID-19 Pneumonia

NCT04341675Phase 2COMPLETED
Sponsor

University of Cincinnati

Enrollment

32

Started

2020

Primary outcome

Percentage of Patients Who Are Alive and Free From Advanced Respiratory Support Measures at Day 28.

COVID-19

CCI-779 in Treating Patients With Mantle Cell Non-Hodgkin's Lymphoma

NCT00033267Phase 2COMPLETED
Sponsor

National Cancer Institute (NCI)

Enrollment

27

Started

2002

Primary outcome

Proportion of patients who achieve a confirmed CR or PR during the first 24 weeks of treatment defined by the International Workshop criteria

Recurrent Mantle Cell Lymphoma

Temsirolimus as Second-line Therapy in HCC

NCT01567930Phase 2UNKNOWN
Sponsor

University of Tennessee Cancer Institute

Enrollment

25

Started

2010

Primary outcome

Disease Progression

Unresectable or Metastatic Hepatocellular Carcinoma

Safety Study for the Use of Rapamycin in Children With Familial Adenomatous Polyposis

NCT06308445Phase 2NOT_YET_RECRUITING
Sponsor

University Hospital, Toulouse

Enrollment

25

Started

2026

Primary outcome

Evaluation of the safety profile of two doses of rapamycin in adolescents with Familial Adenomatous Polyposis.

Familial Adenomatous Polyposis

Enhancing Self Regulation Among Smokers

NCT04161144Phase 2COMPLETED
Sponsor

Medical University of South Carolina

Enrollment

22

Started

2018

Primary outcome

Craving Levels in Response to Cue

Smoking CessationSmoking, CigaretteNicotine Dependence

Sirolimus for Leigh Syndrome

NCT06843811Phase 2ENROLLING_BY_INVITATION
Sponsor

Matthew Demczko

Enrollment

15

Started

2025

Primary outcome

Rate of Adverse Events

Leigh Syndrome

Prospective Pilot Trial to Assess a Multimodal Molecular Targeted Therapy in Children, Adolescent and Young Adults With Relapsed or Refractory High-grade Pineoblastoma

NCT02596828Phase 2COMPLETED
Sponsor

University of Regensburg

Enrollment

4

Started

2016

Primary outcome

The primary endpoint is progression-free survival (PFS)

Pineoblastoma

Sirolimus & Mycophenolate Mofetil as GvHD Prophylaxis in Myeloablative, Matched Related Donor HCT

NCT01220297Phase 2TERMINATED
Sponsor

Stanford University

Enrollment

3

Started

2006

Primary outcome

Acute Graft-vs-Host Disease (GvHD) (Grade 2 to 4)

Hematologic DiseasesAcute-graft-versus-host DiseaseLeukemiaNon-Hodgkin Lymphoma (NHL)Hodgkin Lymphoma

High Dose Busulfan and Bortezomib in Treating Patients With High Risk Multiple Myeloma Undergoing Stem Cell Transplant

NCT01534143Phase 2TERMINATED
Sponsor

Barbara Ann Karmanos Cancer Institute

Enrollment

1

Started

2012

Primary outcome

Incidence and Severity of Acute GVHD Using Fludarabine Phosphate / Busulfan / Bortezomib Preparative Regimen and Triple Immune Suppression With Tacrolimus, Sirolimus and Anti-thymocyte Globulin

Refractory Multiple MyelomaStage I Multiple MyelomaStage II Multiple MyelomaStage III Multiple Myeloma

CCI-779 in Treating Patients With Malignant Glioma

NCT00022724Phase 1/2COMPLETED
Sponsor

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Enrollment

49

Started

2001

Brain and Central Nervous System Tumors

Cixutumumab and Temsirolimus in Treating Patients With Locally Recurrent or Metastatic Breast Cancer

NCT00699491Phase 1/2COMPLETED
Sponsor

National Cancer Institute (NCI)

Enrollment

48

Started

2008

Primary outcome

Recommended Dose Level for Phase II Testing (RPTD) (Phase I)

Male Breast CarcinomaRecurrent Breast CarcinomaStage IV Breast Cancer AJCC v6 and v7

Low-dose Interleukin-2 and Rapamycin on sjögren's Syndrome

NCT05605665Phase 1/2COMPLETED
Sponsor

Peking University People's Hospital

Enrollment

30

Started

2022

Primary outcome

Proportion of Treg cells in peripheral blood

Sjögren's Syndrome

SARC023: Ganetespib and Sirolimus in Patients With MPNST (Malignant Peripheral Nerve Sheath Tumors)

NCT02008877Phase 1/2COMPLETED
Sponsor

Sarcoma Alliance for Research through Collaboration

Enrollment

20

Started

2013

Primary outcome

Number of Dose Limiting Toxicities of Ganetespib When Administered in Combination With Sirolimus.

Malignant Peripheral Nerve Sheath Tumors (MPNST)Sarcoma

CESAR Study in Prostate Cancer With Temsirolimus Added to Standard Docetaxel Therapy (CEPTAS)

NCT01206036Phase 1/2COMPLETED
Sponsor

Central European Society for Anticancer Drug Research

Enrollment

19

Started

2010

Primary outcome

recommended dose

Prostatic Neoplasms

Evaluating Rapamycin Treatment in Alzheimer's Disease Using Positron Emission Tomography

NCT06022068Phase 1/2COMPLETED
Sponsor

Karolinska Institutet

Enrollment

14

Started

2023

Primary outcome

Change in cerebral glucose metabolism

Alzheimer Disease

Safety of Simvastatin in LAM and TSC

NCT02061397Phase 1/2COMPLETED
Sponsor

University of Pennsylvania

Enrollment

10

Started

2014

Primary outcome

Safety of Simvastatin in the Treatment of LAM-S and LAM-TS Patients

LymphangioleiomyomatosisTuberous Sclerosis Complex

Lenalidomide in Combination With Bevacizumab, Sorafenib, Temsirolimus, or 5-Fluorouracil, Leucovorin, Oxaliplatin (FOLFOX)

NCT01183663Phase 1COMPLETED
Sponsor

M.D. Anderson Cancer Center

Enrollment

180

Started

2010

Primary outcome

Maximum Tolerated Dose (MTD) of Lenalidomide in Combination With Bevacizumab, Sorafenib, Temsirolimus, or 5-Fluorouracil, Leucovorin, Oxaliplatin (FOLFOX)

Advanced Cancers

Bevacizumab and Temsirolimus Alone or in Combination With Valproic Acid or Cetuximab in Treating Patients With Advanced or Metastatic Malignancy or Other Benign Disease

NCT01552434Phase 1TERMINATED
Sponsor

M.D. Anderson Cancer Center

Enrollment

154

Started

2012

Primary outcome

Maximum tolerated dose (MTD) of bevacizumab, defined as the dose level below the dose at which 2 of 6 patients experience drug-related dose limiting toxicity (DLT)

Advanced Malignant NeoplasmCastleman DiseaseDigestive System CarcinomaErdheim-Chester DiseaseLip and Oral Cavity CarcinomaLymphangioleiomyomatosisMalignant Endocrine NeoplasmMalignant Female Reproductive System NeoplasmMalignant Male Reproductive System NeoplasmMalignant NeoplasmMalignant Respiratory Tract NeoplasmMalignant Thoracic NeoplasmMalignant Urinary System NeoplasmMesothelial NeoplasmMetastatic Malignant NeoplasmMetastatic Urothelial CarcinomaNeurofibromatosis Type 2Recurrent Adult Soft Tissue SarcomaRecurrent Breast CarcinomaRecurrent Childhood Soft Tissue SarcomaRecurrent Digestive System CarcinomaRecurrent Female Reproductive System CarcinomaRecurrent Male Reproductive System CarcinomaRecurrent Malignant NeoplasmRecurrent Pharyngeal CarcinomaRecurrent Thyroid Gland CarcinomaRefractory Malignant NeoplasmSoft Tissue NeoplasmStage III Breast Cancer AJCC v7Stage III Pharyngeal CancerStage IIIA Breast Cancer AJCC v7Stage IIIB Breast Cancer AJCC v7Stage IIIC Breast Cancer AJCC v7Stage IV Breast Cancer AJCC v6 and v7Stage IV Pharyngeal CancerStage IVA Pharyngeal CancerStage IVB Pharyngeal CancerStage IVC Pharyngeal CancerThyroid Gland Neoplasm

Rapamycin With Grapefruit Juice for Advanced Malignancies

NCT00375245Phase 1COMPLETED
Sponsor

University of Chicago

Enrollment

41

Started

2006

Primary outcome

Pharmacokinetic interaction

TumorsNeoplasm Metastasis

Rapamycin Plus Bevacizumab in Advanced Cancers

NCT00667485Phase 1COMPLETED
Sponsor

University of Chicago

Enrollment

28

Started

2008

Primary outcome

Toxicity

Advanced CancerMetastatic Cancer

Study of Inotuzumab Ozogamicin + Temsirolimus in Patients With Relapsed or Refractory CD22+ B-cell NHLymphoma

NCT01535989Phase 1COMPLETED
Sponsor

Cristiana Sessa

Enrollment

25

Started

2011

Primary outcome

number of participants with adverse events based on the CTCAE v.4

B-cell Lymphoma Refractory

131I-apamistamab-based Conditioning for Hematopoietic Stem Cell Transplant (HSCT) in Advanced Sickle Cell Disease (SCD)

NCT07015684Phase 1RECRUITING
Sponsor

Columbia University

Enrollment

24

Started

2025

Primary outcome

Graft failure rate

Sickling Disorder Due to Hemoglobin S

Temsirolimus and Bryostatin 1 in Treating Patients With Unresectable or Metastatic Solid Tumors

NCT00112476Phase 1COMPLETED
Sponsor

National Cancer Institute (NCI)

Enrollment

24

Started

2005

Primary outcome

MTD of CCI-779 and Bryostatin-1 administered in combination, graded according to NCI Common Toxicity Criteria, Version 3.0

Recurrent MelanomaRecurrent Renal Cell CancerStage IV MelanomaStage IV Renal Cell CancerUnspecified Adult Solid Tumor, Protocol Specific

A Study of Metformin With or Without Rapamycin as Maintenance Therapy After Induction Chemotherapy in Subjects With Pancreatic Cancer

NCT02048384Phase 1COMPLETED
Sponsor

Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Enrollment

22

Started

2014

Primary outcome

safety and feasibility

Metastatic Pancreatic Adenocarcinoma

Temsirolimus and Dexamethasone in Treating Patients With Recurrent or Refractory Multiple Myeloma

NCT00693433Phase 1COMPLETED
Sponsor

National Cancer Institute (NCI)

Enrollment

15

Started

2008

Primary outcome

Maximum tolerated dose of temsirolimus

Refractory Multiple MyelomaStage I Multiple MyelomaStage II Multiple MyelomaStage III Multiple Myeloma

Safety Study of Sirolimus and Hydroxychloroquine in Women With Lymphangioleiomyomatosis

NCT01687179Phase 1COMPLETED
Sponsor

Brigham and Women's Hospital

Enrollment

14

Started

2012

Primary outcome

Safety of Combination Therapy With Sirolimus and Hydroxychloroquine in LAM Patients

Lymphangioleiomyomatosis

NPC-12G Gel 0.2% Sirolimus PK Bridging Study

NCT03972462Phase 1COMPLETED
Sponsor

Nobelpharma

Enrollment

12

Started

2019

Primary outcome

AUC0-48

Bioavailability Study

Temsirolimus in Combination With Gemcitabine in Previously Untreated Metastatic Pancreatic Cancer

NCT00593008Phase 1TERMINATED
Sponsor

Massachusetts General Hospital

Enrollment

5

Started

2007

Primary outcome

To determine the dose-limiting toxicities and maximal tolerated doses of gemcitabine combined with temsirolimus

Pancreatic Adenocarcinoma

Anti-Third Party T Lymphocytes With Nonmyeloablative Stem Cell Transplantation for Indolent Lymphoid Malignancies

NCT00473551Phase 1TERMINATED
Sponsor

M.D. Anderson Cancer Center

Enrollment

4

Started

2007

Primary outcome

Number of Participants achieving engraftment without severe Graft-versus-host disease (GVHD)

LeukemiaLymphomaMyeloma

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