74 Participants Needed

Stem Cell Therapy for Epidermolysis Bullosa

CD
Overseen ByCristina Daniele
Age: Any Age
Sex: Any
Trial Phase: Phase 3
Sponsor: RHEACELL GmbH & Co. KG
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial is testing a special cell therapy given through an IV to people with a severe genetic skin condition called RDEB. The therapy aims to help heal and grow new, healthy skin. Researchers are evaluating its safety and effectiveness. Previous tests have shown increased collagen expression and faster wound healing in patients with RDEB.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. However, if you are currently or have recently been in another clinical trial, you may not be eligible to participate.

Is stem cell therapy for epidermolysis bullosa safe?

Research on ABCB5+ mesenchymal stem cells (MSCs) for treating epidermolysis bullosa shows that the treatment is generally well-tolerated. In a clinical trial, some patients experienced mild side effects like lymphadenopathy (swollen lymph nodes) and hypersensitivity reactions, but these were manageable and resolved without lasting issues.12345

How is the treatment allo-APZ2-OTS different from other treatments for epidermolysis bullosa?

The treatment allo-APZ2-OTS uses ABCB5-positive mesenchymal stem cells (a type of cell that can help reduce inflammation and promote healing) derived from skin, which is unique compared to other treatments that may use stem cells from different sources like umbilical cord blood or adipose tissue. This therapy is administered intravenously (through the veins) and has shown potential in reducing disease activity, itch, and pain in patients with recessive dystrophic epidermolysis bullosa, a condition with no effective standard treatment.13567

What data supports the effectiveness of the treatment allo-APZ2-OTS for Epidermolysis Bullosa?

Research shows that ABCB5+ mesenchymal stem cells, a component of allo-APZ2-OTS, have been effective in reducing disease activity, itch, and pain in patients with recessive dystrophic epidermolysis bullosa, a severe skin disorder. These stem cells have shown potential in promoting wound healing and improving quality of life in clinical trials.12356

Are You a Good Fit for This Trial?

This trial is for males and females from 6 months old with confirmed recessive dystrophic epidermolysis bullosa (RDEB). Participants must have a specific wound type, be in good general health, and understand the study. Women who can have children need a negative pregnancy test and agree to use birth control.

Inclusion Criteria

Subject is eligible to participate in this clinical trial based on general health condition
I have RDEB confirmed by genetic testing or skin biopsy.
My wound is between 5-50 cm2, older than 21 days but less than 9 months, and not infected.
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Exclusion Criteria

Clinically significant or unstable concurrent disease or other clinical contraindications like an uncontrolled or poorly controlled mental health condition of the subject and/or his/her legal representative that could impact on patient's safety or interfere with study compliance such as inability to attend scheduled study visits
Employees of the sponsor, or employees or relatives of the investigator
Previous participation in this clinical trial (except for screening failures)
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Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive intravenous administration of allo-APZ2-OTS or placebo

12 months

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

  • allo-APZ2-OTS
  • Placebo
Trial Overview The trial tests allo-APZ2-OTS, an intravenous treatment for RDEB against a placebo. The goal is to see if it's safe and works better than not receiving the active treatment.
How Is the Trial Designed?
2Treatment groups
Experimental Treatment
Placebo Group
Group I: VerumExperimental Treatment1 Intervention
Group II: PlaceboPlacebo Group1 Intervention

Find a Clinic Near You

Who Is Running the Clinical Trial?

RHEACELL GmbH & Co. KG

Lead Sponsor

Trials
11
Recruited
680+

Published Research Related to This Trial

In a phase I/IIa clinical trial involving 16 patients with recessive dystrophic epidermolysis bullosa (RDEB), treatment with ABCB5+ mesenchymal stem cells (MSCs) showed a statistically significant reduction in disease activity scores, indicating potential efficacy as a disease-modifying therapy.
The treatment was well-tolerated, with only three adverse events related to the cell product, all of which were manageable and resolved without lasting effects, suggesting a favorable safety profile for ABCB5+ MSCs.
Clinical trial of ABCB5+ mesenchymal stem cells for recessive dystrophic epidermolysis bullosa.Kiritsi, D., Dieter, K., Niebergall-Roth, E., et al.[2022]
In a clinical trial involving 16 patients with recessive dystrophic epidermolysis bullosa (RDEB), treatment with ABCB5+ mesenchymal stromal cells led to a significant closure of 64.9% of wounds by week 12, with many wounds closing as early as day 17 or 35.
The treatment not only facilitated wound closure but also significantly reduced the rate of new wound formation by 79.3%, suggesting a promising therapeutic efficacy for ABCB5+ MSCs in managing RDEB.
ABCB5+ mesenchymal stromal cells facilitate complete and durable wound closure in recessive dystrophic epidermolysis bullosa.Dieter, K., Niebergall-Roth, E., Daniele, C., et al.[2023]
The phase I/IIa trial involving 6 patients with recessive dystrophic epidermolysis bullosa (RDEB) demonstrated that intravenous infusions of allogeneic human umbilical cord blood-derived mesenchymal stem cells (hUCB-MSCs) were safe and well tolerated, with no serious adverse events reported.
Patients showed significant improvements in disease severity, blister counts, pain, and quality of life, with the most notable effects observed 56-112 days post-treatment, indicating potential clinical efficacy of hUCB-MSCs in managing RDEB.
Intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell therapy in recessive dystrophic epidermolysis bullosa patients.Lee, SE., Lee, SJ., Kim, SE., et al.[2021]

Citations

Clinical trial of ABCB5+ mesenchymal stem cells for recessive dystrophic epidermolysis bullosa. [2022]
ABCB5+ mesenchymal stromal cells facilitate complete and durable wound closure in recessive dystrophic epidermolysis bullosa. [2023]
Intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell therapy in recessive dystrophic epidermolysis bullosa patients. [2021]
Beneficial Effect of Systemic Allogeneic Adipose Derived Mesenchymal Cells on the Clinical, Inflammatory and Immunologic Status of a Patient With Recessive Dystrophic Epidermolysis Bullosa: A Case Report. [2020]
Skin-Derived ABCB5+ Mesenchymal Stem Cells for High-Medical-Need Inflammatory Diseases: From Discovery to Entering Clinical Routine. [2023]
ABCB5+ dermal mesenchymal stromal cells with favorable skin homing and local immunomodulation for recessive dystrophic epidermolysis bullosa treatment. [2022]
[Stem cell therapy for intractable skin diseases]. [2012]
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