Clinical trials: Sirolimus
Chronic Granulomatous Disease
Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide
Background: CGD causes infections and inflammation. The only cure currently is a bone marrow transplant. Most often a perfectly matched bone marrow donor is used.
- Trial name
- NCT03910452
- Testing
- Busulfan, Alemtuzumab, Cyclophosphamide
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Tuberous Sclerosis Complex, Epilepsy
Stopping TSC Onset and Progression 2B: Sirolimus TSC Epilepsy Prevention Study
This trial is a Phase II randomized, double-blind, placebo controlled multi-site study to evaluate the safety and efficacy of early sirolimus to prevent or delay seizure onset in TSC infants.
- Trial name
- TSC-STEPS (NCT05104983)
- Testing
- Sirolimus
- Where
- 8 sites in United States
- Volunteers
- 64 people
- Sponsor
- Darcy Krueger
Tumor, Tumor, Solid
Phase 2 Basket Trial of Nab-sirolimus in Patients With Malignant Solid Tumors With Pathogenic Alterations in TSC1/TSC2 Genes (PRECISION 1)
A Phase 2 multi-center open-label basket trial of nab-sirolimus for adult and adolescent patients with malignant solid tumors harboring pathogenic inactivating alterations in TSC1 or TSC2 genes
- Conditions
- Tumor, Tumor, Solid, Metastasis, Metastatic Cancer
- Trial name
- NCT05103358
- Testing
- nab-sirolimus
- Where
- 166 sites in United States, Puerto Rico and 1 more
- Volunteers
- 128 people
- Sponsor
- Aadi Bioscience, Inc.
Kidney Transplant
Advancing Transplantation Outcomes in Children
This is a pediatric kidney transplant study comparing the safety and efficacy of an immunosuppressive regimen of belatacept and sirolimus to tacrolimus and Mycophenolate Mofetil (MMF).
- Trial name
- ADVANTage (NCT06055608)
- Testing
- Sirolimus, Belatacept, Mycophenolate Mofetil
- Where
- 21 sites in United States, Canada
- Volunteers
- 200 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Gaucher Disease, Type 2
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)
J3Z-MC-OJAB is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants diagnosed with Type 2 Gaucher disease (GD2).
- Trial name
- NCT04411654
- Testing
- LY3884961, Methylprednisolone, Sirolimus
- Where
- 6 sites in United States, United Kingdom
- Volunteers
- 7 people
- Sponsor
- Prevail Therapeutics
Tuberous Sclerosis Complex
Efficacy and Safety of Rapamycin Versus Vigabatrin in the Prevention of Tuberous Sclerosis Complex Symptoms in Infants
The purpose of the study is to evaluate the efficacy, tolerability, and safety of vigabatrin versus rapamycin as a preventive treatment in infants with Tuberous Sclerosis Complex (TSC).
- Trial name
- ViRap (NCT04987463)
- Testing
- Vigabatrin, Rapamycin
- Where
- 2 sites in Poland
- Volunteers
- 60 people
- Sponsor
- Katarzyna Kotulska
Tuberous Sclerosis Complex
Placebo Controlled Study to Assess the Efficacy and Safety of Rapamycin in Drug Resistant Epilepsy Associated With Tuberous Sclerosis Complex
The purpose of the RaRE-TS study is to determine safety, tolerability and efficacy of rapamycin versus placebo in a drug resistant epilepsy associated with tuberous sclerosis complex (TSC).
- Trial name
- RaRE-TS (NCT05534672)
- Testing
- Rapamycin
- Where
- 2 sites in Poland
- Volunteers
- 200 people
- Sponsor
- Katarzyna Kotulska
Acute Leukemia, Acute Lymphoblastic Leukemia
Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS)
This phase II trial investigates two strategies and how well they work for the reduction of graft versus host disease in patients with acute leukemia or MDS in remission.
- Conditions
- Acute Leukemia, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Myelodysplastic Syndrome
- Trial name
- NCT03970096
- Testing
- Total-Body Irradiation, Thiotepa, Fludarabine
- Where
- 3 sites in United States
- Volunteers
- 120 people
- Sponsor
- Fred Hutchinson Cancer Center
Chronic Granulomatous Disease Transplant
High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease
Chronic granulomatous disease (CGD) affects white blood cell function. Currently, the only curative treatment is bone marrow transplant to replace the abnormal stem cells with new ones (donor cells) capable of making a normal…
- Trial name
- NCT02629120
- Testing
- Alemtuzumab, Busulfan, Sirolimus
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Hematologic Neoplasms
Phase I/II Study to Reduce Post-transplantation Cyclophosphamide Dosing for Older or Unfit Patients Undergoing Bone Marrow Transplantation for Hematologic Malignancies
Background: Certain blood cancers can be treated with blood or bone marrow transplants. Sometimes the donor cells attack the recipient's body, called graft-versus-host disease (GVHD).
- Trial name
- NCT04959175
- Testing
- Mycophenolate Mofetil, Allogeneic HSCT, Fludarabine
- Where
- 2 sites in United States
- Volunteers
- 320 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Hematologic Neoplasms
Donor Lymphocyte Infusion After Allogeneic Hematopoietic Cell Transplantation for High-Risk Hematologic Malignancies
Background: People with blood cancers often receive blood or bone marrow transplants. But even with these treatments, the risk of relapse is high.
- Trial name
- NCT05327023
- Testing
- donor lymphocyte infusion, Cyclophosphamide, Busulfan
- Where
- United States
- Volunteers
- 430 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Peripheral Blood Stem Cell Transplantation, Hematopoietic Stem Cell Transplantation
The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation
Background: Blood cancers (such as leukemias or lymphomas) often do not respond to standard treatments. A transplant of blood stem cells from a healthy donor can help people with these cancers.
- Trial name
- NCT05436418
- Testing
- Melphalan, Sirolimus, Total Body Irradiation (TBI)
- Where
- 2 sites in United States
- Volunteers
- 260 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Chronic Granulomatous Disease
Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists
Background: Chronic granulomatous disease (CGD) affects the immune system. People with CGD are more likely to get infections. Drugs can help control infections, but these treatments can cause side effects including kidney failure…
- Trial name
- NCT05463133
- Testing
- Sirolimus, Cyclophosphamide, Alemtuzumab
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Chronic Graft-versus-host-disease
A Study to Evaluate Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256)
This study will be conducted to compare Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy.
- Trial name
- NCT07124078
- Testing
- INCA034176, Best available Treatment (BAT)
- Where
- 41 sites in United States, Belgium and 4 more
- Volunteers
- 60 people
- Sponsor
- Incyte Corporation
Severe Aplastic Anemia
Sirolimus (Rapamune ) for Relapse Prevention in People With Severe Aplastic Anemia Responsive to Immunosuppressive Therapy
Background: People with severe aplastic anemia (SAA) do not make enough red and white blood cells, and/or platelets. Their body's immune system stops the bone marrow from making these cells.
- Trial name
- NCT02979873
- Testing
- Sirolimus
- Where
- United States
- Volunteers
- 84 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Duchenne Muscular Dystrophy
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting
The primary objective of this study is to evaluate acute liver injury (ALI) rates associated with ELEVIDYS with the addition of sirolimus as an adjunct prophylactic immunosuppression agent.
- Trial name
- ENHANCE (NCT07542314)
- Testing
- ELEVIDYS, Sirolimus, Glucocorticoids
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Sarepta Therapeutics, Inc.
Alveolar Rhabdomyosarcoma, Botryoid-Type Embryonal Rhabdomyosarcoma
Combination Chemotherapy With or Without Temsirolimus in Treating Patients With Intermediate Risk Rhabdomyosarcoma
This randomized phase III trial studies how well combination chemotherapy (vincristine sulfate, dactinomycin, cyclophosphamide alternated with vincristine sulfate and irinotecan hydrochloride or vinorelbine) works compared to…
- Conditions
- Alveolar Rhabdomyosarcoma, Botryoid-Type Embryonal Rhabdomyosarcoma, Embryonal Rhabdomyosarcoma, Rhabdomyosarcoma
- Trial name
- NCT02567435
- Testing
- Cyclophosphamide, Dactinomycin, Irinotecan Hydrochloride
- Where
- 382 sites in United States, Australia and 3 more
- Volunteers
- 325 people
- Sponsor
- National Cancer Institute (NCI)
Osteosarcomas, Bone Cancer
Phase I/II Trial of Dinutuximab Beta in Combination With Irinotecan and Rapamycin in Pediatric, Adolescents and Adult Patients With Relapsed/Refractory Bone Tumors.
Osteosarcomas, like other primitive bone tumors, remain hard-to-treat malignancies, especially after failure of first-line treatments.
- Conditions
- Osteosarcomas, Bone Cancer, Ewing Sarcomas
- Trial name
- BONE DINIRRA (NCT07853105)
- Testing
- Dinutuximab beta, Sirolimus and Irinotecan
- Where
- 12 sites in France
- Volunteers
- 89 people
- Sponsor
- University Hospital, Strasbourg, France
Advanced Malignant Solid Neoplasm, Recurrent Brain Neoplasm
Dasatinib, Temsirolimus, and Cyclophosphamide in Treating Patients With Advanced, Recurrent, or Refractory Solid Tumors
This phase I trial studies the side effects and best dose of dasatinib and temsirolimus when given together with cyclophosphamide in treating patients with solid tumors that have spread to other places in the body, have come…
- Conditions
- Advanced Malignant Solid Neoplasm, Recurrent Brain Neoplasm, Recurrent Malignant Solid Neoplasm, Refractory Brain Neoplasm
- Trial name
- NCT02389309
- Testing
- Cyclophosphamide, Dasatinib, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- M.D. Anderson Cancer Center
Kaposiform Hemangioendothelioma
Sirolimus Discontinuation Strategies in Kaposiform Hemangioendothelioma
Sirolimus has demonstrated efficacy in the treatment of kaposiform hemangioendothelioma (KHE); however, a high rate of rebound growth following discontinuation has been reported, highlighting the clinical importance of…
- Trial name
- NCT07131644
- Testing
- Sirolimus
- Where
- China
- Volunteers
- 120 people
- Sponsor
- West China Hospital