Rapamune

corticosteroid therapy, Kidney Transplant, Lymphangioleiomyomatosis + 5 more
Treatment
3 FDA approvals
20 Active Studies for Rapamune

What is Rapamune

SirolimusThe Generic name of this drug
Treatment SummarySirolimus, also known as rapamycin, is a medication derived from bacteria found in the soil of Easter Island. It is used to suppress the immune system and treat tumors. It works by targeting and blocking the mammalian target of rapamycin (mTOR), a protein that regulates cell growth, proliferation and survival. This drug was approved by the FDA in 1999 for kidney transplants and in 2015 for lymphangioleiomyomatosis. In 2021, it was approved for the treatment of malignant perivascular epithelioid cell tumours. Sirolimus has also been investigated for use in treating
Rapamuneis the brand name
image of different drug pills on a surface
Rapamune Overview & Background
Brand Name
Generic Name
First FDA Approval
How many FDA approvals?
Rapamune
Sirolimus
1999
43

Approved as Treatment by the FDA

Sirolimus, also known as Rapamune, is approved by the FDA for 3 uses such as corticosteroid therapy and Organ Transplantation .
corticosteroid therapy
Used to treat corticosteroid therapy in combination with Cyclosporine
Organ Transplantation
Used to treat Transplanted Organ Rejection in combination with Cyclosporine
Kidney Transplant
Used to treat Kidney Transplantation in combination with Cyclosporine

Effectiveness

How Rapamune Affects PatientsSirolimus is a drug that helps prevent organ rejection in transplant patients, as well as having anti-fungal and anti-tumor properties. In clinical studies, sirolimus was found to reduce the risk of organ rejection in low- to moderate-immunologic risk renal transplant patients when taken orally twice daily. Even after discontinuing the drug, its effects can last up to six months. It works by blocking the body’s immune response, and has been found to be effective in treating autoimmune diseases such as lupus, arthritis, diabetes, myocarditis, and uveoretinitis
How Rapamune works in the bodySirolimus prevents cells from growing and reproducing. It does this by blocking a protein kinase called mTOR from working, which stops signals from activating the growth of cells. In addition, sirolimus can prevent the body from producing antibodies and other growth factors. It is used to treat lymphangioleiomyomatosis, a disorder that affects the lungs, in people with tuberous sclerosis complex, which is an inherited condition caused by mutations in certain genes. Sirolimus works by blocking the mTOR pathway and preventing the abnormal growth of cells that occurs with this condition.

When to interrupt dosage

The measure of Rapamune is contingent upon the recognized disorder, including Kidney Transplantation, Chronic Graft Versus Host Disease and Rejection; Transplant, Liver. The quantity of dosage fluctuates as per the approach of delivery (e.g. Tablet or Tablet, coated) featured in the table beneath.
Condition
Dosage
Administration
Tuberous Sclerosis
, 2.0 mg, 1.0 mg, 0.5 mg, 5.0 mg, 1.0 mg/mL, 5.0 mg/mL, 0.002 mg/mg
Tablet - Oral, , Oral, Tablet, Tablet, sugar coated - Oral, Tablet, sugar coated, Tablet, film coated - Oral, Tablet, film coated, Solution - Oral, Solution, Tablet, coated, Tablet, coated - Oral, Injection, powder, lyophilized, for suspension - Intravenous, Injection, powder, lyophilized, for suspension, Intravenous, Gel - Topical, Topical, Gel
Renal Angiomyolipomas
, 2.0 mg, 1.0 mg, 0.5 mg, 5.0 mg, 1.0 mg/mL, 5.0 mg/mL, 0.002 mg/mg
Tablet - Oral, , Oral, Tablet, Tablet, sugar coated - Oral, Tablet, sugar coated, Tablet, film coated - Oral, Tablet, film coated, Solution - Oral, Solution, Tablet, coated, Tablet, coated - Oral, Injection, powder, lyophilized, for suspension - Intravenous, Injection, powder, lyophilized, for suspension, Intravenous, Gel - Topical, Topical, Gel
Chronic Graft Versus Host Disease
, 2.0 mg, 1.0 mg, 0.5 mg, 5.0 mg, 1.0 mg/mL, 5.0 mg/mL, 0.002 mg/mg
Tablet - Oral, , Oral, Tablet, Tablet, sugar coated - Oral, Tablet, sugar coated, Tablet, film coated - Oral, Tablet, film coated, Solution - Oral, Solution, Tablet, coated, Tablet, coated - Oral, Injection, powder, lyophilized, for suspension - Intravenous, Injection, powder, lyophilized, for suspension, Intravenous, Gel - Topical, Topical, Gel
Organ Transplantation
, 2.0 mg, 1.0 mg, 0.5 mg, 5.0 mg, 1.0 mg/mL, 5.0 mg/mL, 0.002 mg/mg
Tablet - Oral, , Oral, Tablet, Tablet, sugar coated - Oral, Tablet, sugar coated, Tablet, film coated - Oral, Tablet, film coated, Solution - Oral, Solution, Tablet, coated, Tablet, coated - Oral, Injection, powder, lyophilized, for suspension - Intravenous, Injection, powder, lyophilized, for suspension, Intravenous, Gel - Topical, Topical, Gel
corticosteroid therapy
, 2.0 mg, 1.0 mg, 0.5 mg, 5.0 mg, 1.0 mg/mL, 5.0 mg/mL, 0.002 mg/mg
Tablet - Oral, , Oral, Tablet, Tablet, sugar coated - Oral, Tablet, sugar coated, Tablet, film coated - Oral, Tablet, film coated, Solution - Oral, Solution, Tablet, coated, Tablet, coated - Oral, Injection, powder, lyophilized, for suspension - Intravenous, Injection, powder, lyophilized, for suspension, Intravenous, Gel - Topical, Topical, Gel
Kidney Transplant
, 2.0 mg, 1.0 mg, 0.5 mg, 5.0 mg, 1.0 mg/mL, 5.0 mg/mL, 0.002 mg/mg
Tablet - Oral, , Oral, Tablet, Tablet, sugar coated - Oral, Tablet, sugar coated, Tablet, film coated - Oral, Tablet, film coated, Solution - Oral, Solution, Tablet, coated, Tablet, coated - Oral, Injection, powder, lyophilized, for suspension - Intravenous, Injection, powder, lyophilized, for suspension, Intravenous, Gel - Topical, Topical, Gel
Lymphangioleiomyomatosis
, 2.0 mg, 1.0 mg, 0.5 mg, 5.0 mg, 1.0 mg/mL, 5.0 mg/mL, 0.002 mg/mg
Tablet - Oral, , Oral, Tablet, Tablet, sugar coated - Oral, Tablet, sugar coated, Tablet, film coated - Oral, Tablet, film coated, Solution - Oral, Solution, Tablet, coated, Tablet, coated - Oral, Injection, powder, lyophilized, for suspension - Intravenous, Injection, powder, lyophilized, for suspension, Intravenous, Gel - Topical, Topical, Gel
Chordoma
, 2.0 mg, 1.0 mg, 0.5 mg, 5.0 mg, 1.0 mg/mL, 5.0 mg/mL, 0.002 mg/mg
Tablet - Oral, , Oral, Tablet, Tablet, sugar coated - Oral, Tablet, sugar coated, Tablet, film coated - Oral, Tablet, film coated, Solution - Oral, Solution, Tablet, coated, Tablet, coated - Oral, Injection, powder, lyophilized, for suspension - Intravenous, Injection, powder, lyophilized, for suspension, Intravenous, Gel - Topical, Topical, Gel

Warnings

Rapamune Contraindications
Condition
Risk Level
Notes
Severe Hypersensitivity Reactions
Do Not Combine
Sirolimus may interact with Pulse Frequency
Severe Hypersensitivity Reactions
Do Not Combine
Sirolimus may interact with Pulse Frequency
Severe Hypersensitivity Reactions
Do Not Combine
Sirolimus may interact with Pulse Frequency
There are 20 known major drug interactions with Rapamune.
Common Rapamune Drug Interactions
Drug Name
Risk Level
Description
2-Methoxyethanol
Major
The risk or severity of adverse effects can be increased when Sirolimus is combined with 2-Methoxyethanol.
9-(N-methyl-L-isoleucine)-cyclosporin A
Major
The risk or severity of adverse effects can be increased when Sirolimus is combined with 9-(N-methyl-L-isoleucine)-cyclosporin A.
Abetimus
Major
The risk or severity of adverse effects can be increased when Sirolimus is combined with Abetimus.
Acteoside
Major
The risk or severity of adverse effects can be increased when Sirolimus is combined with Acteoside.
Antilymphocyte immunoglobulin (horse)
Major
The risk or severity of adverse effects can be increased when Sirolimus is combined with Antilymphocyte immunoglobulin (horse).
Rapamune Toxicity & Overdose RiskThe lowest toxic dose of sirolimus in rats has been found to be 800 mg/kg and 2500 mg/kg in mice. Sirolimus is a drug with a small safe range, so overdoses can occur. Symptoms of an overdose may be similar to the usual side effects of sirolimus. Treatment usually involves general supportive care, since sirolimus is not likely to be removed from the blood by dialysis.
image of a doctor in a lab doing drug, clinical research

Rapamune Novel Uses: Which Conditions Have a Clinical Trial Featuring Rapamune?

80 active studies are examining the use of Rapamune in providing relief from Renal Angiomyolipomas, Lymphangioleiomyomatosis and Kidney Transplantation.
Condition
Clinical Trials
Trial Phases
Kidney Transplant
37 Actively Recruiting
Phase 2, Phase 1, Not Applicable, Phase 4, Phase 3
Organ Transplantation
0 Actively Recruiting
corticosteroid therapy
0 Actively Recruiting
Lymphangioleiomyomatosis
0 Actively Recruiting
Chordoma
0 Actively Recruiting
Chronic Graft Versus Host Disease
24 Actively Recruiting
Phase 4, Phase 1, Phase 2, Not Applicable, Phase 3
Renal Angiomyolipomas
0 Actively Recruiting
Tuberous Sclerosis
4 Actively Recruiting
Phase 3, Phase 4, Phase 2, Phase 1

Rapamune Reviews: What are patients saying about Rapamune?

5Patient Review
5/7/2010
Rapamune for Prevent Kidney Transplant Rejection
I had a liver transplant in September 2008 and was placed on Prograft and Cellcept. Unfortunately, I had a stroke in November of that year as well; luckily, my liver MDs said this was a very rare side effect. They placed me on Rapamune and I've been doing much better since then. My only issues now are high cholesterol and blood pressure, but those are being treated with medication.
5Patient Review
10/6/2015
Rapamune for Lymphangioleiomyomatosis
This has been a really great medication for me. I've had it for four years now and it's kept my lung function stable the whole time. The only downside is that I sometimes get mouth ulcers from it.
5Patient Review
3/21/2011
Rapamune for Prevent Kidney Transplant Rejection
Six months ago, I had a kidney transplant. The Rapamune and Prograf have been working well for me with no side effects at the current dosage (1 mg/day and 4mg/day respectively).
5Patient Review
9/11/2011
Rapamune for Prevent Kidney Transplant Rejection
I had a kidney transplant in April of 2009 and my son was the donor. I was on cellcept, prograf, and prednisone, but on July of 2010 I was diagnosed with central nervous system lymphoma. I had intense chemo for the last 14 months, but they switched me to 2mg.daily use of rapamune with no side effects. My cancer is in remission and the kidney is doing perfect.
5Patient Review
2/11/2012
Rapamune for Prevention of Cardiac Transplant Rejection
The only downside is that it increases your cholesterol and triglicerides, so you need to take medication for them (statins). Otherwise, the drug does great things for your kidney health.
5Patient Review
8/12/2011
Rapamune for Prevention of Cardiac Transplant Rejection
I've been on this medication for 3 years following my heart transplant. So far, I haven't had any issues with rejection.
4.7Patient Review
2/13/2011
Rapamune for Prevent Kidney Transplant Rejection
I was very happy to start on this medication. It has a lot fewer side effects for me than the Cyclosporin I was taking before I started on the trial for it. I take 4mg per day and I have never had a rejection problem in the 7 years I have been on it.
4.3Patient Review
2/22/2012
Rapamune for Prevent Kidney Transplant Rejection
I had a kidney transplant 19 years ago and have been on Rapamune for 10 years. It's really effective, with only minor side effects. The one downside is that now my cholesterol is high and I need to take Zetia every day.
3.7Patient Review
8/9/2011
Rapamune for Prevent Kidney Transplant Rejection
I've had my transplant for ten years now, and Rapamune has been a part of my daily medication list. Recently, I developed severe edema and swelling in my right leg. For the past three and a half years, I've been wearing compression stockings to help manage the problem.
3Patient Review
2/20/2013
Rapamune for Prevent Kidney Transplant Rejection
I had my transplant in 2007 and was switched from myfortic to rapamune in 2009. I developed lymphodemia in 2010 and am hoping to be taken off the medication soon.
1Patient Review
8/22/2014
Rapamune for Prevent Kidney Transplant Rejection
I wasn't a fan of having to mix this medication with juice, and my cholesterol shot up to an unhealthy level as a result.
image of drug pills surrounding a glass of water symbolizing drug consumption

Patient Q&A Section about rapamune

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What drug class is Rapamune?

"This medication belongs to a class of drugs that suppress the immune system. It does this by weakening the body's defenses to help the body accept a new organ as if it were the person's own."

Answered by AI

What is the drug Rapamune used for?

"After this, the corticosteroids can be tapered off by your doctor.

Rapamune is a medicine that helps to prevent the body from rejecting a newly transplanted kidney. It is most commonly used in adults who have a low to moderate risk of rejection. It is often recommended that Rapamune is used in combination with other rejection prevention medicines such as ciclosporin and corticosteroids for a period of two to three months. After this, the corticosteroids can be tapered off as decided by your doctor."

Answered by AI

What are the side effects of Rapamune?

"You may have a fever, cold symptoms such as a stuffy nose, sneezing, or a sore throat; mouth sores; nausea, stomach pain, or diarrhea; a headache, muscle aches; chest pain; dizziness; or acne."

Answered by AI

Is Rapamune the same as rapamycin?

"The medication rapamycin was originally discovered by scientists from a soil sample on Easter Island. It was later given the generic name sirolimus by the U.S. pharmaceutical world."

Answered by AI

Clinical Trials for Rapamune

Image of New York University Langone Health in New York, United States.

GGTA1 KO Thymokidney for Kidney Failure

40 - 70
All Sexes
New York, NY
The purpose of this study is to evaluate the safety and efficacy of the GGTA1 KO Thymokidney in patients with end-stage renal disease (ESRD) who are either not eligible for conventional allogeneic kidney transplantation (Group 1) or are on an Organ Procurement and Transplantation Network (OPTN) kidney transplant waitlist, but are more likely to die or go untransplanted within 5 years than receive a kidney transplant (Group 2). The study consists of xenotransplantation followed by a 24-week Post-transplant Follow-up Period (Part A) to evaluate the efficacy and safety objectives followed by a Long-term Follow-up Period (Part B) to evaluate participant survival, GGTA1 KO Thymokidney survival, and screening for zoonotic infections. Part B will continue for the lifetime of the participant or for 52 weeks following nephrectomy, if required.
Phase 1 & 2
Recruiting
New York University Langone HealthUnited Therapeutics
Image of University of California, San Francisco in San Francisco, United States.

Trimethoprim-Sulfamethoxazole for Urinary Tract Infections

13 - 29
All Sexes
San Francisco, CA
The goal of this clinical trial is to learn if a common antibiotic called trimethoprim-sulfamethoxazole (TMP-SMX) can help prevent urinary tract infections (UTIs) in children and young adults who recently had a kidney transplant. Most people take TMP-SMX for about 6 months after getting a kidney transplant. In this study, researchers want to see what happens if people keep taking it for 6 more months. The main questions this study is asking are: * Does TMP-SMX lower the number of UTIs in the first year after transplant? * What side effects or problems do participants have while taking TMP-SMX? Researchers will compare TMP-SMX to a placebo (a look-alike pill that does not contain any medication) to see if TMP-SMX works to prevent UTIs. Participants will: * Take either TMP-SMX or a placebo pill by mouth every day for 6 months * Have three visits to touch base with the study team about any issues * Complete short monthly online surveys about any symptoms or side effects * Share blood and urine test results from their regular transplant clinic visits
Phase 4
Waitlist Available
University of California, San FranciscoAlexandra Bicki, MD
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Image of City of Hope Medical Center in Duarte, United States.

Emapalumab for Graft-versus-Host Disease

18 - 75
All Sexes
Duarte, CA
This phase I trial tests the safety, side effects and effectiveness of emapalumab with post-transplant cyclophosphamide, tacrolimus, and mycophenolate mofetil in preventing graft-versus-host disease (GVHD) in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS) after reduced-intensity donor (allogeneic) hematopoietic cell transplant (HCT). Giving chemotherapy, such as fludarabine, melphalan, or busulfan, before a donor \[peripheral blood stem cell\] transplant helps kill cancer cells in the body and helps make room in the patient's bone marrow for new blood-forming cells (stem cells) to grow. When healthy stem cells for a donor are infused into a patient (allogeneic HCT), they may help the patient's bone marrow make more healthy cells and platelets. Allogeneic HCT is an established treatment, however, GVHD continues to be a major problem of allogeneic HCT that can complicate therapy. GVHD is a disease caused when cells from a donated stem cell graft attack the normal tissue of the transplant patient. Emapalumab binds to an immune system protein called interferon gamma. This may help lower the body's immune response and reduce inflammation. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid and may kill cancer cells. It may also lower the body's immune response. Tacrolimus is a drug used to help reduce the risk of rejection by the body of organ and bone marrow transplants. Mycophenolate mofetil is a drug used to prevent GVHD after organ transplants. It is also being studied in the prevention of GVHD after stem cell transplants for cancer, and in the treatment of some autoimmune disorders. Mycophenolate mofetil is a type of immunosuppressive agent. Giving emapalumab with post-transplant cyclophosphamide, tacrolimus and mycophenolate mofetil may be safe, tolerable and/or effective in preventing GVHD in patients with AML or MDS after a reduced-intensity allogeneic HCT.
Phase 1
Waitlist Available
City of Hope Medical CenterAmandeep Salhotra
Image of Cedars Sinai Medical Center in Los Angeles, United States.

Berinert for Kidney Transplant Outcomes

18 - 75
All Sexes
Los Angeles, CA
The purpose of this study is to find out if Berinert can improve kidney function in the first year after transplant and to find out what effects, good or bad, Berinert will have in the kidney recipient. This research study will compare Berinert to placebo. The placebo looks exactly like Berinert but does not contain any active drug. Placebos are used in research studies to see if the results are due to the study drug or due to other reasons. Neither you or the study doctor can choose or know which group is assigned. The primary objective is to test whether intrarenal artery C1 esterase inhibitor (C1INH) injection into the donor kidney prior to transplantation improves kidney function in recipients of high risk, deceased donor kidney transplants as measured by 12-month Estimated Glomerular Filtration Rate (eGFR) Chronic Kidney Disease Epidemiology Collaboration (CDK-EPI)
Phase 2
Recruiting
Cedars Sinai Medical Center (+5 Sites)Peter S Heeger, MD
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Vedolizumab + Cyclophosphamide + Tacrolimus for Graft-versus-Host Disease

18 - 80
All Sexes
Duarte, CA
This phase II trial studies how well vedolizumab plus post-transplant cyclophosphamide (PTCy) and short course tacrolimus work for the prevention of graft versus host disease (GVHD) in patients undergoing allogeneic hematopoietic cell transplantation (HCT) after reduced intensity conditioning. Allogeneic HCT is a procedure in which a person receives blood-forming stem cells (cells from which all blood cells develop) from a donor. Giving reduced conditioning chemotherapy before an allogeneic HCT helps kill cancer cells in the body and helps make room in the patient's bone marrow for new stem cells to grow using less than standard doses of chemotherapy. Sometimes, the transplanted cells from a donor can attack the body's normal cells (called graft-versus-host disease). Vedolizumab is a monoclonal antibody, which is a type of protein that can bind to certain targets in the body, such as molecules that cause the body to make an immune response (antigens). It may reduce inflammation. Cyclophosphamide is in a class of medications called alkylating agents. It works by damaging the cell's deoxyribonucleic acid and may kill cancer cells. It may also lower the body's immune response. Tacrolimus suppresses the immune system by preventing the activation of certain types of immune cells. Giving vedolizumab plus PTCy and short course tacrolimus may be effective at preventing GVHD after allogeneic HCT.
Phase 2
Recruiting
City of Hope Medical CenterMonzr M. Al Malki
Have you considered Rapamune clinical trials? We made a collection of clinical trials featuring Rapamune, we think they might fit your search criteria.Go to Trials
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