You're using a free limited version of DrugPatentWatch: Upgrade for Complete Access

Last Updated: February 12, 2025

CLINICAL TRIALS PROFILE FOR MYCOPHENOLATE MOFETIL HYDROCHLORIDE


✉ Email this page to a colleague

« Back to Dashboard


505(b)(2) Clinical Trials for Mycophenolate Mofetil Hydrochloride

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials
Trial Type Trial ID Title Status Sponsor Phase Start Date Summary
New Formulation NCT00717470 ↗ A Study in Kidney Transplant Subjects to Investigate the Optimal Suppression of Immunity to Help Prevent Kidney Rejection Completed Astellas Pharma Inc Phase 4 2008-05-14 To compare how well the new formulation of Tacrolimus® used once daily, in combination with other drugs helps prevent the rejection of a new kidney after transplantation compared to the twice daily dose of Tacrolimus
New Combination NCT03249831 ↗ A Blood Stem Cell Transplant for Sickle Cell Disease Recruiting California Institute for Regenerative Medicine (CIRM) Phase 1 2019-01-04 Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism. Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor). Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications. This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because: 1. Half-matched related donors will be used, and 2. A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and 3. Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to improve transplant outcomes. It is hoped that the research transplant: 1. Will reverse sickle cell disease and improve patient quality of life, 2. Will reduce side effects and help the patient recover faster from the transplant, 3. Help the patient keep the transplant longer and 4. Reduce serious transplant-related complications.
New Combination NCT03249831 ↗ A Blood Stem Cell Transplant for Sickle Cell Disease Recruiting City of Hope Medical Center Phase 1 2019-01-04 Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism. Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor). Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications. This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because: 1. Half-matched related donors will be used, and 2. A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and 3. Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to improve transplant outcomes. It is hoped that the research transplant: 1. Will reverse sickle cell disease and improve patient quality of life, 2. Will reduce side effects and help the patient recover faster from the transplant, 3. Help the patient keep the transplant longer and 4. Reduce serious transplant-related complications.
New Combination NCT04104438 ↗ Examination of Immunosuppression Adjustment Impact on Kidney Function in Liver Transplant Not yet recruiting Fady M Kaldas, M.D., F.A.C.S. Phase 4 2019-11-01 This is a study to help understand how well new combinations of immunosuppressive medications (medications that weaken your immune system to prevent your body from rejecting the transplanted liver) work compared to standard immunosuppressive medications after your liver transplant. Also the study will assess how safe the new combination of immunosuppressive medicines are and if there are any changes in how your kidneys work after taking these medicines.
>Trial Type >Trial ID >Title >Status >Phase >Start Date >Summary

All Clinical Trials for Mycophenolate Mofetil Hydrochloride

Trial ID Title Status Sponsor Phase Start Date Summary
NCT00000936 ↗ A Study To Test An Anti-Rejection Therapy After Kidney Transplantation Terminated National Institute of Allergy and Infectious Diseases (NIAID) Phase 3 1999-11-01 Kidney transplantation is often successful. However, despite aggressive anti-rejection drug therapy, some patients will reject their new kidney. This study is designed to test two anti-rejection approaches. Two medications in this study are currently used in children, but there is no information regarding which drug is safer or more effective. Survival rates in renal transplantation are unacceptably low. Therefore, there is a need for an improved post-transplant treatment, such as the induction therapy used in this study.
NCT00001764 ↗ Mycophenolate Mofetil to Treat Wegener's Granulomatosis and Related Vascular Inflammatory Conditions Completed National Institute of Allergy and Infectious Diseases (NIAID) Phase 1 1998-04-01 This study will examine the safety and effectiveness of the drug mycophenolate mofetil (MPM) in treating Wegener's granulomatosis and related inflammatory vessel diseases. Blood vessel inflammation in these patients may involve different parts of the body, including the brain, nerves, eyes, sinuses, lungs, kidneys, intestinal tract, skin, joints, heart, and other sites. The more severe the involvement, the more likely the disease will be life-threatening. Standard treatment consists of combination drug therapy with prednisone and a cytotoxic agent-usually cyclophosphamide or methotrexate. However, some patients in whom this treatment is initially successful have a disease relapse; other patients cannot take the medications because of other health problems or because of severe side effects of the drugs. MPM is approved by the Food and Drug Administration to prevent kidney transplant rejection. It is chemically similar to another cytotoxic drug called azathioprine, which has been beneficial in maintaining remission in patients with Wegener's granulomatosis who have been treated successfully with cyclophosphamide. Because MPM is more effective than azathioprine in preventing organ rejection, it may also prove beneficial as a second-line treatment for Wegener's granulomatosis. Patients with Wegener's granulomatosis or related inflammatory vessel diseases who have had a relapse following treatment with cyclophosphamide and methotrexate or who cannot take one or both of these drugs may be eligible for this study. Only patients who have been treated at NIH in the methotrexate protocol or the cyclophosphamide switching to methotrexate protocol, or who have received the exact same treatment from their own physician may participate. Participants will have a complete medical evaluation including laboratory studies. Consultations, X-rays and biopsies of affected organs may also be done if indicated for diagnosis or treatment. Patients with active disease will be given MPM and prednisone, both in tablet form. Patients with inactive disease will receive only prednisone if they are already taking it. In both cases, the prednisone will be reduced gradually and discontinued if the disease improves significantly. MPM therapy will continue for at least 2 years. If after 2 years the disease remains in remission, the MPM dose will be gradually reduced and then stopped. If active disease recurs while on MPM therapy, the treatment plan will likely be changed. The new regimen will be determined by the severity of disease, other medical conditions, and history of side effects to previous medications. Patients will be followed at the NIH clinic every month for the first 3 months on MPM and then every 3 months for another 18 months. Those whose disease has remained in remission and have stopped all medications will then be followed every 6 months for 4 visits. The follow-up visits will include a physical examination, blood draws, and, if needed, X-rays. Visits may be scheduled more frequently if medically indicated.
NCT00001964 ↗ Combination Therapy of Severe Aplastic Anemia Completed National Heart, Lung, and Blood Institute (NHLBI) Phase 2 2000-03-17 This study will test the safety and effectiveness of a combination of three drugs in treating severe aplastic anemia and preventing its recurrence. Two drugs used in this trial ATG and cyclosporine are standard combination therapy for aplastic anemia. This study will try to improve this therapy in three ways: 1) by altering the drug regimen to allow the drugs to work better; 2) by reducing the risk of kidney damage; and 3) by adding a third drug mycophenolate mofetil to try to prevent disease relapse. Patients with severe aplastic anemia who do not have a suitable bone marrow donor or who decline bone marrow transplantation may participate in this study. Patients will have a skin test for ATG allergy, chest X-ray, blood test, and bone marrow aspiration before treatment begins. ATG will then be started, infused through a vein continuously for 4 days. Ten days after ATG is stopped, cyclosporine treatment will begin, taken twice a day by mouth in either liquid or capsule form and will continue for 6 months. Also, in the first 2 weeks of treatment, patients will be given a full dose of corticosteroid (prednisone) to prevent serum sickness that could develop as a side effect of ATG therapy. The dosage will be decreased after that. Mycophenolate will be started at the same time as ATG, in two daily doses by mouth, and will continue for 18 months. Patients will be hospitalized at the beginning of the study. During this time, blood will be drawn at 3-week intervals and a bone marrow examination will be repeated 3 months after treatment has begun. Additional tests, including X-rays may be required. After hospital discharge, patients will be followed on an outpatient basis at 3-month intervals. The patients own physician will perform blood tests weekly and kidney and liver function tests every 2 weeks during cyclosporine therapy. Transfusions may be required initially.
NCT00003145 ↗ Fludarabine Phosphate, Low-Dose Total-Body Irradiation, and Peripheral Blood Stem Cell Transplant Followed by Donor Lymphocyte Infusion in Treating Older Patients With Chronic Myeloid Leukemia Completed National Cancer Institute (NCI) Phase 2 1997-08-01 This clinical trial studies fludarabine phosphate, low-dose total-body irradiation, and peripheral blood stem cell transplant followed by donor lymphocyte infusion in treating older patients with chronic myeloid leukemia. Giving chemotherapy and total-body irradiation before a donor bone marrow transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after the transplant may stop this from happening. Once the donated stem cells begin working, the patient's immune system may see the remaining cancer cells as not belonging in the patient's body and destroy them (called graft-versus-tumor effect). Giving an infusion of the donor's white blood cells (donor lymphocyte infusion) may boost this effect.
NCT00003145 ↗ Fludarabine Phosphate, Low-Dose Total-Body Irradiation, and Peripheral Blood Stem Cell Transplant Followed by Donor Lymphocyte Infusion in Treating Older Patients With Chronic Myeloid Leukemia Completed Fred Hutchinson Cancer Research Center Phase 2 1997-08-01 This clinical trial studies fludarabine phosphate, low-dose total-body irradiation, and peripheral blood stem cell transplant followed by donor lymphocyte infusion in treating older patients with chronic myeloid leukemia. Giving chemotherapy and total-body irradiation before a donor bone marrow transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after the transplant may stop this from happening. Once the donated stem cells begin working, the patient's immune system may see the remaining cancer cells as not belonging in the patient's body and destroy them (called graft-versus-tumor effect). Giving an infusion of the donor's white blood cells (donor lymphocyte infusion) may boost this effect.
>Trial ID >Title >Status >Phase >Start Date >Summary

Clinical Trial Conditions for Mycophenolate Mofetil Hydrochloride

Condition Name

Condition Name for Mycophenolate Mofetil Hydrochloride
Intervention Trials
Kidney Transplantation 80
Leukemia 79
Lymphoma 65
Myelodysplastic Syndromes 64
[disabled in preview] 0
This preview shows a limited data set
Subscribe for full access, or try a Trial

Condition MeSH

Condition MeSH for Mycophenolate Mofetil Hydrochloride
Intervention Trials
Leukemia 185
Myelodysplastic Syndromes 157
Preleukemia 149
Syndrome 130
[disabled in preview] 0
This preview shows a limited data set
Subscribe for full access, or try a Trial

Clinical Trial Locations for Mycophenolate Mofetil Hydrochloride

Trials by Country

Trials by Country for Mycophenolate Mofetil Hydrochloride
Location Trials
Canada 150
China 108
Italy 84
Spain 82
Germany 76
This preview shows a limited data set
Subscribe for full access, or try a Trial

Trials by US State

Trials by US State for Mycophenolate Mofetil Hydrochloride
Location Trials
California 152
New York 124
Washington 116
Texas 113
Ohio 97
This preview shows a limited data set
Subscribe for full access, or try a Trial

Clinical Trial Progress for Mycophenolate Mofetil Hydrochloride

Clinical Trial Phase

Clinical Trial Phase for Mycophenolate Mofetil Hydrochloride
Clinical Trial Phase Trials
Phase 4 204
Phase 3 139
Phase 2/Phase 3 32
[disabled in preview] 395
This preview shows a limited data set
Subscribe for full access, or try a Trial

Clinical Trial Status

Clinical Trial Status for Mycophenolate Mofetil Hydrochloride
Clinical Trial Phase Trials
Completed 485
Recruiting 133
Terminated 115
[disabled in preview] 140
This preview shows a limited data set
Subscribe for full access, or try a Trial

Clinical Trial Sponsors for Mycophenolate Mofetil Hydrochloride

Sponsor Name

Sponsor Name for Mycophenolate Mofetil Hydrochloride
Sponsor Trials
National Cancer Institute (NCI) 171
Fred Hutchinson Cancer Research Center 81
Hoffmann-La Roche 62
[disabled in preview] 80
This preview shows a limited data set
Subscribe for full access, or try a Trial

Sponsor Type

Sponsor Type for Mycophenolate Mofetil Hydrochloride
Sponsor Trials
Other 1090
Industry 404
NIH 281
[disabled in preview] 4
This preview shows a limited data set
Subscribe for full access, or try a Trial

Mycophenolate Mofetil Hydrochloride: Clinical Trials, Market Analysis, and Projections

Introduction

Mycophenolate mofetil hydrochloride, commonly known as mycophenolate mofetil (MMF) or CellCept, is a potent immunosuppressant used primarily to prevent organ rejection in transplant patients and to treat various autoimmune diseases. This article delves into the recent clinical trials, market analysis, and future projections for this drug.

Clinical Trials and Efficacy

Transplantation Trials

Mycophenolate mofetil was initially approved for preventing acute rejection in renal transplant patients based on three large, multicenter, prospective, double-blind trials. These trials demonstrated a significant reduction in acute rejection rates compared to placebo or azathioprine[3].

Autoimmune Diseases

In addition to its use in transplantation, MMF has been studied for treating autoimmune diseases. A notable clinical trial compared methotrexate and mycophenolate mofetil for treating noninfectious uveitis. The study found that while both drugs were effective, methotrexate achieved corticosteroid-sparing control of inflammation in 66.7% of patients, compared to 57.1% for mycophenolate mofetil, although the difference was not statistically significant[1].

Mechanism of Action

MMF acts as a reversible inhibitor of inosine monophosphate dehydrogenase (IMPDH), preventing the proliferation of immune cells and the formation of antibodies that cause transplant rejection or exacerbate autoimmune diseases[4].

Market Analysis

Current Market Size

The global mycophenolate mofetil market was valued at USD 5.2 billion in 2023. This market is segmented based on application (e.g., pemphigus, systemic lupus erythematosus, psoriasis) and product form (tablets, capsules, topical suspension, injection)[2].

Growth Projections

The market is expected to grow at a compound annual growth rate (CAGR) of 4.9% from 2024 to 2031, reaching USD 9.84 billion by 2031. Another report projects the market to reach USD 2735.54 million by 2030, growing at a CAGR of 6.28% from 2023 to 2030[2][5].

Drivers of Growth

Several factors are driving the growth of the MMF market:

  • Increasing Organ Transplants: The rising number of organ transplants globally increases the demand for effective immunosuppressive treatments like MMF[2].
  • Autoimmune Diseases: The growing prevalence of autoimmune diseases such as lupus and rheumatoid arthritis, for which MMF is an effective treatment, contributes to market expansion[2].
  • Advances in Pharmaceutical Formulations: Improvements in drug delivery technologies and formulations enhance the accessibility and effectiveness of MMF, further boosting its market share[2].
  • Healthcare Infrastructure and Reimbursement: Advances in healthcare infrastructure and favorable reimbursement regulations also contribute to the increased adoption of MMF[2].

Market Dynamics

Porter’s Five Forces Analysis

The market analysis includes a Porter’s Five Forces analysis, which examines the bargaining power of buyers, suppliers, the threat of new entrants, the threat of substitutes, and the degree of competition. This analysis helps in understanding the competitive landscape and the various factors influencing market growth[2].

Competitive Landscape

The MMF market is characterized by a competitive landscape involving major pharmaceutical companies. The report highlights the strategies and market positions of these key players, including their product offerings, market share, and research and development activities[2].

Applications and Uses

Transplantation

MMF is primarily used in combination with other immunosuppressants to prevent the rejection of kidney, heart, or liver transplants in adult and pediatric patients[4].

Autoimmune Diseases

Off-label uses of MMF include the treatment of autoimmune hepatitis, lupus-associated nephritis, and dermatitis in children. It is also used for conditions like pemphigus, bullous pemphigoid, and psoriasis[2][4].

Future Projections and Trends

Research and Development

Continuous research and development efforts are expected to maximize the therapeutic potential of MMF. This includes exploring new drug delivery methods and formulations that improve its efficacy and safety profile[2].

Expanding Indications

The scope of MMF is likely to expand as it is studied for use in special patient populations, such as those at high immunological risk or with deteriorating kidney function[3].

Global Market Expansion

The MMF market is expected to grow globally, driven by increasing demand in regions such as North America, Europe, Asia-Pacific, South America, and the Middle East and Africa[2].

Key Takeaways

  • Mycophenolate mofetil is a crucial immunosuppressant with proven efficacy in preventing organ rejection and treating autoimmune diseases.
  • Recent clinical trials have compared its efficacy with other immunosuppressants, such as methotrexate, in treating noninfectious uveitis.
  • The global MMF market is projected to grow significantly, driven by increasing organ transplants, the prevalence of autoimmune diseases, and advances in pharmaceutical formulations.
  • The market is characterized by a competitive landscape with major pharmaceutical companies playing key roles.

FAQs

What is the primary use of mycophenolate mofetil?

Mycophenolate mofetil is primarily used to prevent the rejection of kidney, heart, or liver transplants in combination with other immunosuppressants.

How does mycophenolate mofetil work?

MMF acts as a reversible inhibitor of inosine monophosphate dehydrogenase (IMPDH), preventing the proliferation of immune cells and the formation of antibodies that cause transplant rejection or exacerbate autoimmune diseases.

What are the projected growth rates for the mycophenolate mofetil market?

The global MMF market is expected to grow at a CAGR of 4.9% from 2024 to 2031, reaching USD 9.84 billion by 2031, or at a CAGR of 6.28% from 2023 to 2030, reaching USD 2735.54 million.

What factors are driving the growth of the mycophenolate mofetil market?

The growth is driven by increasing organ transplants, the growing prevalence of autoimmune diseases, advances in pharmaceutical formulations, and favorable healthcare infrastructure and reimbursement regulations.

What are some off-label uses of mycophenolate mofetil?

Off-label uses include the treatment of autoimmune hepatitis, lupus-associated nephritis, and dermatitis in children, as well as conditions like pemphigus, bullous pemphigoid, and psoriasis.

Sources

  1. Effect of Corticosteroid-Sparing Treatment With Mycophenolate Mofetil vs Methotrexate in Patients With Noninfectious Uveitis: A Randomized Clinical Trial. JAMA, 2019.
  2. Mycophenolate Mofetil (MMF) Market Size, Trends and Projections. Market Research Intellect, 2024.
  3. Review of major clinical trials with mycophenolate mofetil in renal transplantation. PubMed, 2005.
  4. Mycophenolate Mofetil: Uses, Interactions, Mechanism of Action. DrugBank.
  5. Mycophenolate Mofetil Market Size, Share, Scope, Trends And Forecast. Verified Market Reports.

More… ↓

⤷  Try for Free

Make Better Decisions: Try a trial or see plans & pricing

Drugs may be covered by multiple patents or regulatory protections. All trademarks and applicant names are the property of their respective owners or licensors. Although great care is taken in the proper and correct provision of this service, thinkBiotech LLC does not accept any responsibility for possible consequences of errors or omissions in the provided data. The data presented herein is for information purposes only. There is no warranty that the data contained herein is error free. thinkBiotech performs no independent verification of facts as provided by public sources nor are attempts made to provide legal or investing advice. Any reliance on data provided herein is done solely at the discretion of the user. Users of this service are advised to seek professional advice and independent confirmation before considering acting on any of the provided information. thinkBiotech LLC reserves the right to amend, extend or withdraw any part or all of the offered service without notice.