Academisch Ziekenhuis Leiden
Leiden, The Netherlands
RecruitingRare disease
Study on Early Diffuse Cutaneous Systemic Sclerosis: Comparing Stem Cell Transplantation with Cyclophosphamide, Filgrastim, and Mycophenolate Mofetil
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What is this trial about?
A plain-language summary of the goals, design and what participants do
This clinical trial is focused on studying the treatment of Diffuse Cutaneous Systemic Sclerosis, a condition that causes hardening and tightening of the skin and connective tissues. The study aims to compare two treatment strategies: one involving autologous hematopoietic stem cell transplantation and the other using immunosuppressive medications. The immunosuppressive medications being studied include Cyclophosphamide, Filgrastim, Mycophenolate Mofetil, and Anti-T Lymphocyte Immunoglobulin derived from rabbits. These treatments are being evaluated to determine their effectiveness in improving survival and preventing major organ failure in patients with early stages of this disease.
Participants in the study will be randomly assigned to receive either the stem cell transplantation or the immunosuppressive medications. The study will monitor the participants over a period to assess the outcomes of each treatment approach. The goal is to find out which treatment strategy is more beneficial for patients with early diffuse cutaneous systemic sclerosis. The study will also involve the use of a placebo for comparison purposes.
The trial will track various health indicators to evaluate the success of the treatments, including survival rates and the prevention of organ failure. The study is designed to provide valuable insights into the best treatment options for managing this challenging condition, ultimately aiming to improve the quality of life for those affected by it.
The research process
The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.
Who can join the trial?
7 criteria
- Age between 18 and 65 years.
- Diagnosis of Diffuse cutaneous systemic sclerosis according to the 2013 ACR-EULAR classification criteria. This is a specific type of a rare disease that affects the skin and sometimes other organs.
- Disease duration of 3 years or less from the onset of the first symptoms that are not related to Raynaud's phenomenon. Raynaud's phenomenon is a condition where fingers and toes change color and may feel numb or painful in response to cold or stress.
- A skin score of 15 or more, indicating a diffuse skin pattern, or significant organ involvement, which can include:
- Respiratory (lung) involvement
- Renal (kidney) involvement
- Cardiac (heart) involvement
- Alternatively, disease duration of 1 year or less with diffuse skin disease, a skin score greater than 10, and a high risk of organ-based disease or an acute phase response. An acute phase response is a rapid inflammatory response to disease.
Who cannot join the trial?
7 criteria
- Patients who do not have diffuse cutaneous systemic sclerosis according to specific medical criteria cannot participate. This is a condition that affects the skin and other organs.
- Patients who are not within the specified age range for the study cannot participate.
- Patients who are not willing or able to follow the study procedures cannot participate.
- Patients who have other serious health conditions that might interfere with the study cannot participate.
- Patients who are pregnant or breastfeeding cannot participate.
- Patients who have participated in another clinical trial recently may not be eligible.
- Patients who have a history of certain medical treatments that could affect the study results cannot participate.
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Responsive Responsive Stichting Radboud University Medical Center
Nijmegen, The NetherlandsResponsive Universitair Medisch Centrum Utrecht
Utrecht, The Netherlands
Trial locations
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Investigated drugs
Autologous Hematopoietic Stem Cell Transplantation (HSCT)
is a procedure where a patient's own stem cells are collected, stored, and then given back to them after they have received high-dose chemotherapy. This process helps to rebuild the immune system and is being tested to see if it can be an effective early treatment for a condition called diffuse cutaneous systemic sclerosis, which affects the skin and internal organs.
Immunosuppressive Medication
refers to drugs that help reduce the activity of the immune system. In this trial, these medications are being used to see if they can effectively manage early diffuse cutaneous systemic sclerosis by preventing the immune system from attacking the body's own tissues, which can lead to organ damage.
What is already known about the treatment
Autologous Hematopoietic Stem Cell Transplantation (HSCT) – This treatment involves collecting and using a patient's own stem cells to help regenerate healthy blood and immune cells. It is currently being studied as a potential treatment for early diffuse cutaneous systemic sclerosis, a rare autoimmune disease. The main goal is to improve survival and prevent major organ failure. At the molecular level, HSCT works by resetting the immune system, which may help reduce the disease's progression. It is classified as a form of regenerative medicine.
Investigated diseases
Diffuse cutaneous systemic sclerosis – This is a chronic autoimmune disease characterized by the hardening and tightening of the skin and connective tissues. It primarily affects the skin but can also involve internal organs such as the heart, lungs, and kidneys. The disease progresses with the thickening of the skin, starting from the fingers and hands, and can spread to the arms, face, and trunk. As it advances, it may lead to reduced mobility and function of the affected areas. Internal organ involvement can occur, leading to complications in the affected organs. The progression of the disease varies among individuals, with some experiencing rapid changes and others having a more gradual course.
Trial detailsLast updated 2 Oct 2026
Age18+ yearsPhasePhase IIITrial ID2023-505877-34-00Protocol codeNL72607.041.20Estimated enrolment88 patientsSponsorUniversitair Medisch Centrum Utrecht
sourced from the EU Clinical Trials Register and site verification
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