CLINICAL TRIALS PROFILE FOR CAMPATH

All Clinical Trials for CAMPATH

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00023192 ↗	Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	2001-08-01	This study will compare the health and well being of children treated with a modified stem cell transplantation procedure for chronic granulomatous disease (CGD) with that of children receiving standard of care treatment. CGD is an inherited disorder of neutrophils-a type of infection-fighting white blood cell-that leaves patients vulnerable to life-threatening infections. Standard treatment with antibiotics, and sometimes surgery, is not always successful, and patients with persisting infections have a poor long-term prognosis. Transplantation of donated stem cells (cells produced by the bone marrow that mature into white and red blood cells and platelets) can improve immune function in patients with CGD and possibly cure the disease. However, this procedure carries a significant risk of death, because it requires complete suppression of the immune system with high-dose chemotherapy. In addition, lymphocytes-another type of infection-fighting white blood cell-from the donor may cause what is called graft versus host disease (GvHD), in which the donor cells 'see' patient's cells as foreign and mount an immune response to reject them. To try to reduce these risks, patients in this study will be given low-dose chemotherapy that is easier for the body to tolerate and involves a shorter period of complete immune suppression. Also, the donor's lymphocytes will be removed from the rest of the stem cells to be transplanted, reducing the risk of GvHD. Patients with CGD between 2 and 17 years of age who 1) are currently free of active infection, and 2) have a history of at least one life-threatening infection or a family member with CGD and a history of at least one life-threatening infection, and 3) a family member that is a suitable donor may be eligible for this study. Candidates will have a medical history, physical examination and blood tests, lung and heart function tests, x-rays or CT scans of the body, and dental and eye examinations. They will fill out questionnaires that measure emotional well being, quality of life, and intelligence (ability to learn and understand). Stem cells will be collected from both the patient and donor. To do this, the hormone G-CSF will be injected under the skin for several days to move stem cells from the bone marrow to the bloodstream. Then, the stem cells will be collected by apheresis. In this procedure the blood is drawn through a needle placed in one arm and pumped into a machine where the required cells are separated out and removed. Then, the rest of the blood is returned through a needle in the other arm. Several days before the transplant procedure, patients will start a 'conditioning regimen' of chemotherapy with cyclophosphamide, fludarabine and Campath 1H. When the conditioning therapy is completed, the donor's stem cells will be infused. To help prevent rejection of donor cells, cyclosporine will be given by mouth or by vein starting 1 month after the transplant procedure. The average hospital stay for stem cell transplantation is 21 days. After discharge, patients will return to the NIH clinic for follow-up clinic visits weekly or twice weekly for 2 to 3 months. These visits will include a symptom check, physical examination and blood tests. Subsequent clinic visits will be scheduled 1 to 3 times a year for at least 5 years.
NCT00006390 ↗	Alemtuzumab Plus Peripheral Stem Cell Transplantation in Treating Patients With Chronic Lymphocytic Leukemia	Completed	National Cancer Institute (NCI)	Phase 2	2001-02-01	RATIONALE: Monoclonal antibodies such as alemtuzumab can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells. Peripheral stem cell transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy. Combining monoclonal antibody therapy, chemotherapy, radiation therapy, and peripheral stem cell transplantation may kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of alemtuzumab plus peripheral stem cell transplantation in treating patients who have chronic lymphocytic leukemia.
NCT00006390 ↗	Alemtuzumab Plus Peripheral Stem Cell Transplantation in Treating Patients With Chronic Lymphocytic Leukemia	Completed	Eastern Cooperative Oncology Group	Phase 2	2001-02-01	RATIONALE: Monoclonal antibodies such as alemtuzumab can locate tumor cells and either kill them or deliver tumor-killing substances to them without harming normal cells. Peripheral stem cell transplantation may be able to replace immune cells that were destroyed by chemotherapy or radiation therapy. Combining monoclonal antibody therapy, chemotherapy, radiation therapy, and peripheral stem cell transplantation may kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of alemtuzumab plus peripheral stem cell transplantation in treating patients who have chronic lymphocytic leukemia.
NCT00004857 ↗	Fludarabine Followed by Alemtuzumab in Treating Patients With Chronic Lymphocytic Leukemia	Completed	National Cancer Institute (NCI)	Phase 2	2000-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Monoclonal antibodies such as alemtuzumab can locate cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells. PURPOSE: Phase II trial to study the effectiveness of fludarabine followed by alemtuzumab in treating patients who have chronic lymphocytic leukemia.
NCT00004857 ↗	Fludarabine Followed by Alemtuzumab in Treating Patients With Chronic Lymphocytic Leukemia	Completed	Alliance for Clinical Trials in Oncology	Phase 2	2000-01-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Monoclonal antibodies such as alemtuzumab can locate cancer cells and either kill them or deliver cancer-killing substances to them without harming normal cells. PURPOSE: Phase II trial to study the effectiveness of fludarabine followed by alemtuzumab in treating patients who have chronic lymphocytic leukemia.
NCT00001984 ↗	Effectiveness of the Investigational Drug Campath-1H in Preventing Rejection of Transplanted Kidneys	Completed	National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)	Phase 2	1999-11-01	This protocol will test a humanized monoclonal antibody known as Campath-1H for its ability to induce a state of permanent allograft acceptance, or tolerance, when administered in combination with a brief course of the immunosuppressive drug deoxyspergualin (DSG) at the time of human renal allotransplantation. Campath-1H is specific for the common lymphocyte and monocyte antigen CD52. Its administration temporarily depletes mature lymphocytes and some monocytes without altering neutrophils or hematopoietic stem cells. Deoxyspergualin inhibits the NFkB pathway thus preventing monocyte and macrophage activation. Recipients of living or cadaveric donor kidneys will be treated with one dose of Campath-1H prior to transplantation to insure that peripheral depletion is achieved at the time of graft reperfusion. Three subsequent doses of Campath-1H will be administered on the first, third and fifth days after the transplant to deplete passenger donor leukocytes and residual recipient cells that mobilize in response to the allograft. In addition, patients will be treated with DSG for 14 days beginning on the day prior to surgery. This trial expands on pilot studies at the NIH of 15 patients in which Campath was given alone at the time of transplantation. In those studies, excellent peripheral depletion occurred after just one dose of Campath though central depletion required additional dosing. This allowed for greatly reduced immunosuppression to be used to prevent rejection, but to date, all patients have required some immunosuppressive medication. It is hoped that the addition of DSG will eliminate the need for long-term immunosuppression. Patients will be followed closely in the post transplant period. If patients experience rejection, they will be treated with methylprednisolone and have immunosuppression added using sirolimus as the predominant immunosuppressive agent. In the previous phase of this study without DSG, this maneuver has in all cases been successful in returning the allograft to normal function. In addition to evaluating graft function following transplantation, this protocol will also characterize and evaluate the function of the immune system and the composition of the T cell repertoire following the administration of Campath-1H and DSG, and during immune system recovery after transplantation.
NCT00004143 ↗	Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes	Completed	David Rizzieri, MD	Phase 2	1999-09-01	RATIONALE: Although used primarily to treat malignant disorders of the blood, allogeneic stem cell transplantation can also cure a variety of non-cancerous, inherited or acquired disorders of the blood. Unfortunately, the conventional approach to allogeneic stem cell transplantation is a risky procedure. For some non-cancerous conditions, the risks of this procedure outweigh the potential benefits. This protocol is designed to test a new approach to allogeneic stem cell transplantation. It is hoped that this approach will be better suited for patients with non-cancerous blood and bone marrow disorders.

Clinical Trial Conditions for CAMPATH

Condition Name

Condition Name for CAMPATH
Intervention	Trials
Leukemia	35
Lymphoma	20
Chronic Lymphocytic Leukemia	12
Sickle Cell Disease	12

Condition MeSH

Condition MeSH for CAMPATH
Intervention	Trials
Leukemia	53
Leukemia, Lymphoid	39
Lymphoma	34
Leukemia, Lymphocytic, Chronic, B-Cell	30

Clinical Trial Locations for CAMPATH

Trials by Country

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Trials by Country for CAMPATH
Location	Trials
United States	343
United Kingdom	8
Canada	7
Italy	5
Austria	4

Trials by US State

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−

Trials by US State for CAMPATH
Location	Trials
Texas	45
Maryland	23
Illinois	22
New York	20
Ohio	18

Clinical Trial Progress for CAMPATH

Clinical Trial Phase

Clinical Trial Phase for CAMPATH
Clinical Trial Phase	Trials
Phase 4	14
Phase 3	9
Phase 2/Phase 3	3

Clinical Trial Status

Clinical Trial Status for CAMPATH
Clinical Trial Phase	Trials
Completed	109
Terminated	53
Recruiting	14

Clinical Trial Sponsors for CAMPATH

Sponsor Name

Sponsor Name for CAMPATH
Sponsor	Trials
National Cancer Institute (NCI)	24
Baylor College of Medicine	21
Bayer	19

Sponsor Type

Sponsor Type for CAMPATH
Sponsor	Trials
Other	292
Industry	58
NIH	43

CAMPATH (Alemtuzumab): Clinical Trials, Market Analysis, and Projections

Introduction

CAMPATH, also known as alemtuzumab, is a monoclonal antibody used in the treatment of chronic lymphocytic leukemia (CLL) and multiple sclerosis (MS). Here, we will delve into the recent clinical trials, market analysis, and future projections for this drug.

Clinical Trials Update

Recent Trials and Outcomes

In recent years, CAMPATH has been involved in several significant clinical trials. One notable example is the Phase I BALLI-01 trial conducted by Cellectis, where CAMPATH (under the designation CLLS52) was used as part of a lymphodepletion regimen along with fludarabine and cyclophosphamide to support the administration of UCART22, a CAR-T therapy for relapsed/refractory B cell acute lymphoblastic leukemia (ALL)[1].

The trial showed positive preliminary efficacy and safety results, with the addition of alemtuzumab to the fludarabine and cyclophosphamide regimen associated with sustained lymphodepletion and significantly higher UCART22 cell expansion, leading to greater clinical activity[1].

Historical Trials

Historically, CAMPATH has demonstrated its efficacy in treating CLL. The CAM307 Phase III trial compared CAMPATH with chlorambucil in previously untreated patients with B-cell CLL. This study showed that CAMPATH significantly reduced the risk of disease progression or death by 42% and exhibited higher overall and complete response rates compared to chlorambucil[3].

Market Analysis

Current Market Landscape

The chronic lymphocytic leukemia therapeutics market has evolved significantly since CAMPATH's initial approval in 2001. In 2010, CAMPATH was the dominant drug in this market, accounting for 42.32% of total sales[2].

However, by 2020, the market was expected to be equally dominated by other drugs such as GA101/RG7159 and Arzerra. The market for CLL therapeutics is segmented into chemotherapy regimens (e.g., FC and FCR regimens) and single drugs like Fludara (fludarabine), Campath, Treanda, and Arzerra[2].

Geographical Distribution

North America, particularly the U.S. and Canada, has been a major contributor to the CLL therapeutics market, accounting for 61.21% of total sales in 2010. Europe, including countries like the U.K., Germany, Italy, France, and Spain, also plays a significant role in this market[2].

Market Dynamics

Drivers

• Innovative Therapies: The development of innovative therapies, including CAR-T cell therapies and targeted treatments, is driving the market for leukemia treatments. The integration of CAMPATH into these new therapeutic regimens, such as the UCART22 trial, highlights its ongoing relevance[1][2].
• High Unmet Needs: Oncology, particularly in the realm of CLL, has high unmet medical needs, which creates a significant market opportunity for effective treatments like CAMPATH[2].
• Off-Label Prescribing: Although CAMPATH is not currently indicated as a first-line treatment for CLL, off-label prescribing has contributed to its market growth[2].

Restraints

• Production Capabilities: Low production capabilities for efficient drugs can be a restraint, especially for complex biologics like alemtuzumab[2].

Opportunities

• Growing Older Population: The incidence of CLL is high in individuals over 50 years old, and the growing older male population presents an opportunity for market growth[2].
• Limited Players: The limited number of players in the CLL therapeutics market offers opportunities for companies like Cellectis and Sanofi to expand their market share[2].

Economic and Regulatory Aspects

FDA Designations and Approvals

CAMPATH has received several significant designations and approvals. Recently, Cellectis’s CLLS52 (alemtuzumab) was granted an orphan drug designation by the FDA for use in a type of leukemia as part of a lymphodepletion regimen associated with UCART22[1].

Historically, CAMPATH received accelerated approval in 2001 for the treatment of B-CLL in patients who had failed fludarabine therapy. The CAM307 trial supported full approval, although it is not currently indicated as a first-line treatment[3].

Withdrawal and Relaunch

In 2012, Campath was withdrawn from the markets to prevent off-label use for MS and to prepare for its relaunch under the brand name Lemtrada, which is used for MS treatment. This move was part of a strategy to manage the drug's use and pricing[5].

Future Projections

Integration with CAR-T Therapies

The future of CAMPATH looks promising, especially with its integration into CAR-T cell therapies. The positive results from the BALLI-01 trial suggest that alemtuzumab can enhance the efficacy of these new treatments by ensuring sustained lymphodepletion and higher cell expansion[1].

Market Growth

The CLL therapeutics market is expected to grow at a CAGR of 13.43% from 2015 to 2020, driven by innovative therapies and the growing older population. While CAMPATH may not dominate the market as it once did, its role in combination therapies and its potential in new indications ensure its continued relevance[2].

Strategic Partnerships

Cellectis’s partnership with AstraZeneca, which includes an equity investment and research collaboration, positions the company to develop up to ten new cell and gene therapy products. This partnership could further enhance the market position of CAMPATH and related therapies[1].

Key Takeaways

• Clinical Trials: CAMPATH has shown positive results in recent clinical trials, particularly in combination with CAR-T therapies.
• Market Analysis: The CLL therapeutics market is evolving, with CAMPATH playing a significant role despite the emergence of new drugs.
• Market Dynamics: Innovative therapies, high unmet needs, and off-label prescribing drive the market, while production capabilities and regulatory strategies are key factors.
• Future Projections: Integration with CAR-T therapies and strategic partnerships position CAMPATH for continued relevance and growth.

FAQs

What is CAMPATH used for?

CAMPATH (alemtuzumab) is used to treat chronic lymphocytic leukemia (CLL) and multiple sclerosis (MS).

What was the outcome of the CAM307 trial?

The CAM307 trial showed that CAMPATH significantly reduced the risk of disease progression or death by 42% and exhibited higher overall and complete response rates compared to chlorambucil in CLL patients[3].

Why was Campath withdrawn from the market in 2012?

Campath was withdrawn to prevent off-label use for MS and to prepare for its relaunch under the brand name Lemtrada for MS treatment[5].

What is the current market landscape for CLL therapeutics?

The market is dominated by various drugs and regimens, including GA101/RG7159, Arzerra, and traditional chemotherapy regimens like FC and FCR[2].

How does CAMPATH work?

CAMPATH works by targeting the CD52 antigen on B and T cells, activating the immune system to destroy these cells[3].

Sources

1. Pharmaceutical Technology: "Cellectis starts alemtuzumab revival with FDA orphan drug win in leukaemia"
2. MarketsandMarkets: "Chronic Lymphocytic Leukemia Therapeutics Market in G8 Countries (2010 - 2020)"
3. Biospace: "Genzyme Corporation Release: Data From Phase III Comparative Study Show Campath(R) Superior to Chlorambucil as a First-Line Therapy in B-CLL"
4. NIDDK: "Clinical Trials - Molecular & Clinical Hematology Branch - NIDDK"
5. Wikipedia: "Alemtuzumab"