Study on Early Diffuse Cutaneous Systemic Sclerosis: Comparing Stem Cell Transplantation with Cyclophosphamide, Filgrastim, and Mycophenolate Mofetil

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What is this study about?

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.

1 joining the study

Upon joining the study, you will be randomly assigned to one of two groups. One group will receive a stem cell transplantation, and the other will receive immunosuppressive medication. This process is called randomization and ensures that the study results are unbiased.

2 initial assessment

An initial assessment will be conducted to evaluate your current health status. This may include physical examinations, blood tests, and other necessary evaluations to establish a baseline for your health condition.

3 treatment phase

If you are in the stem cell transplantation group, you will receive a series of medications to prepare your body for the procedure. These medications include anhydrous cyclophosphamide administered by injection, filgrastim given through subcutaneous use, mycophenolate mofetil taken orally, and anti-t lymphocyte immunoglobulin administered via intravenous infusion.

If you are in the immunosuppressive medication group, you will receive medications designed to suppress your immune system and manage your condition. The specific medications, their dosages, and frequency will be determined by the study protocol and your healthcare provider.

4 monitoring and follow-up

Throughout the study, regular monitoring will be conducted to assess your response to the treatment. This includes scheduled visits for physical examinations, blood tests, and other necessary evaluations.

You will be monitored for any side effects or adverse reactions to the treatment. It is important to report any new symptoms or concerns to the study team promptly.

5 end of study participation

At the end of your participation in the study, a final assessment will be conducted to evaluate your health status and the effects of the treatment. This may include physical examinations, blood tests, and other necessary evaluations.

You will receive information about the study results and any further steps or recommendations for your health care.

Who Can Join the Study?

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 Study?

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.

Where you can join this trial?

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Other Sites

Site Name	City	Country
Ospedale San Raffaele S.r.l.	Milan	Italy
Region Skane Skanes Universitetssjukhus	Lund	Sweden
Universitair Medisch Centrum Utrecht	Utrecht	The Netherlands
Academisch Ziekenhuis Leiden	Leiden	The Netherlands
Universita’ Campus Bio-medico Di Roma	Rome	Italy
Karolinska Institutet	Stockholm	Sweden
Athqoyobf Uxl	Amsterdam	The Netherlands
Siencbpiy Rscvxtj Uzlaxtcyfh Mlbefgp Cauggy	Nijmegen	The Netherlands

Trial status

Country	Status	Recruitment Start
Italy	Recruiting	30.10.2020
Sweden	Recruiting	30.10.2020
The Netherlands	Recruiting	30.10.2020

Trial locations

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.

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 ID:

2023-505877-34-00

Protocol code:

NL72607.041.20

NCT ID:

NCT04464434

Trial Phase:

Therapeutic confirmatory (Phase III)

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Study on Early Diffuse Cutaneous Systemic Sclerosis: Comparing Stem Cell Transplantation with Cyclophosphamide, Filgrastim, and Mycophenolate Mofetil

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?