CLINICAL TRIALS PROFILE FOR UVADEX

All Clinical Trials for Uvadex

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00005092 ↗	Chemotherapy, Radiation Therapy, and Peripheral Stem Cell Transplantation in Treating Patients With Hematologic Cancer	Completed	National Cancer Institute (NCI)	Phase 1	1999-05-28	RATIONALE: Peripheral stem cell transplantation may be able to replace immune cells that were destroyed by the chemotherapy or radiation therapy used to kill tumor cells. Sometimes the transplanted cells are rejected by the body's normal tissues. Transplanting donated cells that have been treated with psoralen may prevent this from happening. PURPOSE: Phase I trial to study the effectiveness of chemotherapy, radiation therapy, and psoralen-treated donor cells in treating patients who are undergoing peripheral stem cell transplantation for hematologic cancer.
NCT00005092 ↗	Chemotherapy, Radiation Therapy, and Peripheral Stem Cell Transplantation in Treating Patients With Hematologic Cancer	Completed	M.D. Anderson Cancer Center	Phase 1	1999-05-28	RATIONALE: Peripheral stem cell transplantation may be able to replace immune cells that were destroyed by the chemotherapy or radiation therapy used to kill tumor cells. Sometimes the transplanted cells are rejected by the body's normal tissues. Transplanting donated cells that have been treated with psoralen may prevent this from happening. PURPOSE: Phase I trial to study the effectiveness of chemotherapy, radiation therapy, and psoralen-treated donor cells in treating patients who are undergoing peripheral stem cell transplantation for hematologic cancer.
NCT00054600 ↗	Safety and Efficacy Study of Photopheresis With UVADEX to Prevent Graft-versus-Host Disease	Completed	Mallinckrodt	Phase 2	2002-06-01	The purpose of this study is to determine whether Extracorporeal Photopheresis with UVADEX (ECP) prior to bone marrow or peripheral blood stem cell transplantation is effective in the prevention of Graft-versus-Host Disease (GvHD).
NCT00054613 ↗	Safety and Efficacy Study of Photopheresis Plus Standard Therapy to Treat Chronic Graft-versus-Host Disease	Completed	Mallinckrodt	Phase 2	2002-06-01	The purpose of this study is to determine whether extracorporeal photoimmune therapy with UVADEX (ECP) added to standard therapy is effective in the treatment of chronic graft-versus-host disease (GvHD).
NCT00056355 ↗	Extracorporeal Photopheresis to Maintain Symptoms Remission During Steroid Withdrawal in Patients With Steroid-Dependent Crohn's Disease	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 1	2003-03-01	This study will examine the safety and effectiveness of extracorporeal photopheresis (ECP) in controlling Crohn's disease symptoms as patients taper their corticosteroid dose. Crohn's disease is a chronic inflammatory bowel disease. Patients commonly have chronic diarrhea with abdominal pain, loss of appetite and weight loss. Acute disease flares are treated with large doses of corticosteroids, but long-term use of these drugs can have harmful side effects. ECP (described below), is approved to treat skin symptoms associated with a type of cancer called cutaneous T-cell lymphoma and has been used experimentally in conditions involving abnormal inflammation. Patients 18 years of age and older who have had Crohn's disease for at least 6 months, who are corticosteroid-dependent, and whose symptoms are controlled well enough so that their Crohn's Disease Activity Index (CDAI) is less than 220, may be eligible for this study. Candidates will be screened with a medical history and review of medical records, physical examination, electrocardiogram, blood tests, urine pregnancy test for women of childbearing potential, and a questionnaire about how Crohn's disease affects their life and activities. Patients with a CDAI score of less than 150 will begin ECP treatments as soon as possible. Those with scores from 150 to 219 will have their corticosteroid dose increased enough to bring their CDAI score to below 150 before beginning ECP. Patients who do not achieve a CDAI of less than 150 after 4 to 6 weeks of increased corticosteroids will be excluded from the study. Participants will have ECP treatments for 2 consecutive days every 2 weeks for 24 weeks, for a total of 26 treatments. For ECP, patients undergo leukapheresis, a method of collecting large numbers of white blood cells, or leukocytes-cells that may be responsible for many of the medical problems in Crohn's disease. Whole blood is collected through a needle in an arm vein, similar to donating a unit of blood. The blood flows through a machine that separates it into its components by spinning. The white cells are removed and collected in a plastic bag, and the red blood cells and plasma are returned to the patient's bloodstream through the same needle. The collected white cells are mixed with a drug called UVADEX® (Registered Trademark), exposed to ultraviolet (UVA) light, and then returned to the patients' bloodstream. (The UVADEX allows the blood cells to absorb more UVA.) The UVA changes the cells in a way that, once they are back in the body, they cause changes in other cells like them. Each ECP treatment takes 3 to 4 hours. On the first day of each 2-day treatment, patients will undergo a review of symptoms, check of vital signs, and blood draw. They will complete a CDAI diary for 7 days before the first of the two ECP treatments and a questionnaire about their life and activities at 4-week intervals. During the ECP treatment period, corticosteroids will be slowly reduced as long as disease symptoms do not worsen. Patients whose disease remains under control with cessation of all steroids may begin maintenance ECP, 2 days in a row every 4 weeks for an additional 20 weeks (another 10 treatments), with the same follow-up as described above, and a full physical examination 4 weeks after the final treatment. Patients who were able to reduce, but not stop, steroid treatment may be considered for maintenance therapy if it is thought that continuing treatment may enable further reduction of steroids. Patients whose disease symptoms worsen with ECP or who have not been able to decrease their steroid dose will not be eligible for maintenance therapy and their participation in the study will end.

Clinical Trial Conditions for Uvadex

Condition Name

Condition Name for Uvadex
Intervention	Trials
Graft Versus Host Disease	2
Graft-versus-Host Disease	2
Leukemia	1
Acute Graft-versus-Host Disease	1

Condition MeSH

Condition MeSH for Uvadex
Intervention	Trials
Graft vs Host Disease	9
Lymphoma, T-Cell	2
Crohn Disease	2
Mycosis Fungoides	2

Clinical Trial Locations for Uvadex

Trials by Country

Trials by Country for Uvadex
Location	Trials
United States	70
Germany	8
United Kingdom	7
France	7
Austria	6

Trials by US State

Trials by US State for Uvadex
Location	Trials
Texas	6
Michigan	5
Massachusetts	5
Illinois	5
New York	4

Clinical Trial Progress for Uvadex

Clinical Trial Phase

Clinical Trial Phase for Uvadex
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	2
Phase 2	9

Clinical Trial Status

Clinical Trial Status for Uvadex
Clinical Trial Phase	Trials
Completed	10
Terminated	3
Not yet recruiting	2

Clinical Trial Sponsors for Uvadex

Sponsor Name

Sponsor Name for Uvadex
Sponsor	Trials
Mallinckrodt	15
M.D. Anderson Cancer Center	3
PRA Health Sciences	1

Sponsor Type

Sponsor Type for Uvadex
Sponsor	Trials
Other	22
Industry	20
NIH	2

UVADEX (Methoxsalen): Clinical Trials Update, Market Analysis, and Projections

Introduction to UVADEX

UVADEX (methoxsalen) is a critical component in the treatment of various medical conditions, particularly those involving immune system dysregulation. It is used in conjunction with the THERAKOS CELLEX Photopheresis System, a therapy that involves the extracorporeal treatment of blood cells with UVA light.

Clinical Trials Update

Pediatric Patients with Steroid Refractory Acute Graft-versus-Host Disease (aGvHD)

A recent interim analysis of a Phase 3 clinical trial for the treatment of pediatric patients (ages 1-21 years) with steroid refractory aGvHD showed promising results. The study, which involved 25 patients, reported an overall response rate of 74% at the four-week interim analysis, exceeding the specified target. This led to the decision to discontinue further enrollment. An ad hoc 12-week analysis also showed a significant response rate of 48%[1].

Chronic Graft-versus-Host Disease (cGvHD)

Another clinical trial focused on the use of UVADEX in treating moderate to severe chronic GvHD. This randomized controlled study involved patients receiving extracorporeal photopheresis (ECP) therapy with UVADEX in addition to standard of care. The trial highlighted the efficacy and safety of UVADEX in this patient population, although it noted that the patient and investigator could not be blinded due to the nature of the treatment[4].

Regulatory Approvals

UVADEX has received significant regulatory approvals, enhancing its market presence. Recently, it was approved by the Australian Therapeutic Goods Administration (TGA) for the treatment of steroid-refractory and steroid-intolerant chronic GvHD in adults following allogeneic hematopoietic stem cell transplantation. This approval was granted for use with the THERAKOS CELLEX Photopheresis System[5].

Market Analysis

Current Market Landscape

The market for treatments involving UVADEX is closely tied to the broader landscape of immunotherapy and oncology. The global market for non-small cell lung cancer (NSCLC), for example, is expected to grow significantly, driven by targeted therapies. While UVADEX is not directly used in NSCLC treatment, the growth in immunotherapy and targeted treatments indicates a favorable environment for innovative therapies like photopheresis[2].

Growth Drivers

• Increasing Incidence of Chronic Diseases: The rising prevalence of chronic diseases, including those requiring immunotherapy, is driving the demand for clinical trials and innovative treatments. This trend is expected to continue, fueling the growth of the market for drugs like UVADEX[3].
• Regulatory Approvals: Recent approvals in regions like Australia expand the market reach of UVADEX, contributing to its growth potential.
• Clinical Trial Success: Positive outcomes from clinical trials, such as the high response rates in pediatric patients with aGvHD, enhance the credibility and adoption of UVADEX.

Market Barriers

• Safety and Adverse Events: While the clinical trials have shown promising results, there have been reports of serious adverse events. For instance, the trial in pediatric patients with aGvHD reported 11 serious adverse events, leading to two subject withdrawals and two deaths, although none were considered related to the UVADEX photopheresis therapy[1].
• Competition from Generic Drugs: The broader pharmaceutical market faces challenges from generic drugs, which can impact the sales of branded therapies. However, UVADEX's unique application in photopheresis may mitigate some of this competition.

Market Projections

Global Clinical Trials Market

The global clinical trials market, which includes trials for drugs like UVADEX, is projected to grow from $61.58 billion in 2024 to $106.78 billion by 2032, at a CAGR of 7.1%. This growth is driven by the increasing number of clinical trials globally, particularly in regions outside the U.S. and EU due to cost-effectiveness and ease of process[3].

Specific Market for UVADEX

While specific financial projections for UVADEX are not readily available, the drug's approval and positive clinical trial outcomes suggest a strong growth trajectory. The market for chronic GvHD and other conditions treated by UVADEX is expected to expand as more patients become eligible for this therapy.

Targeted Therapies and Market Impact

Targeted therapies, including those used in conjunction with UVADEX, are expected to play a significant role in the growth of the pharmaceutical market. For example, in the NSCLC market, targeted therapies like Tagrisso (osimertinib) and Avastin (bevacizumab) are projected to achieve significant sales by 2025. This trend indicates a favorable market environment for innovative and targeted treatments like UVADEX[2].

Unmet Needs and Future Directions

Despite the progress made, there are still unmet needs in the treatment of conditions like GvHD. Future clinical trials and research are expected to focus on optimizing treatment protocols, reducing adverse events, and expanding the indications for UVADEX.

Key Takeaways

• Positive Clinical Trial Outcomes: UVADEX has shown significant efficacy in treating pediatric patients with steroid refractory aGvHD and adults with chronic GvHD.
• Regulatory Approvals: Recent approvals in regions like Australia have expanded the market reach of UVADEX.
• Market Growth: The global clinical trials market and the specific market for UVADEX are expected to grow, driven by increasing incidence of chronic diseases and positive clinical trial results.
• Challenges: Safety concerns and competition from generic drugs are potential barriers to growth.

FAQs

What is UVADEX used for?

UVADEX (methoxsalen) is used in conjunction with the THERAKOS CELLEX Photopheresis System for the treatment of conditions such as steroid-refractory acute graft-versus-host disease (aGvHD) and chronic GvHD.

What were the results of the recent clinical trial in pediatric patients with aGvHD?

The recent clinical trial reported an overall response rate of 74% at the four-week interim analysis and 48% at the 12-week analysis, leading to the decision to discontinue further enrollment.

Has UVADEX received any recent regulatory approvals?

Yes, UVADEX received regulatory approval in Australia by the Australian Therapeutic Goods Administration (TGA) for the treatment of steroid-refractory and steroid-intolerant chronic GvHD in adults.

What are the major drivers of growth for the market involving UVADEX?

The major drivers include the increasing incidence of chronic diseases, positive outcomes from clinical trials, and recent regulatory approvals.

What are the potential barriers to the growth of the UVADEX market?

Potential barriers include safety concerns and adverse events, as well as competition from generic drugs in the broader pharmaceutical market.

Sources

1. Mallinckrodt Pharmaceuticals: "Mallinckrodt Announces Interim Analysis Results for Therakos® Platform Phase 3 Clinical Trial in Treatment of Pediatric Patients with Steroid Refractory Acute Graft-versus-Host Disease."
2. GlobalData: "NSCLC MARKET - Global Drug Forecast & Market Analysis to 2025."
3. Fortune Business Insights: "Clinical Trials Market Size, Share, Industry Trends, Growth, 2032."
4. EudraCT: "Clinical Trial Results: A Randomized Controlled Study of Extracorporeal Photopheresis (ECP) Therapy with UVADEX TM for the Treatment of Patients with Moderate to Severe Chronic Graft-versus-Host Disease (cGvHD)."
5. Terumo BCT: "Mallinckrodt and Terumo BCT Announce UVADEX® (Methoxsalen) Regulatory Approval in Australia."

Last updated: 2025-01-07