Depletion of CD3/CD19 in an autologous stem cell transplant for Libman-Sacks Disease

Phase-Based Progress Estimates
1
Effectiveness
2
Safety
Children's Hospital of Philadelphia, Philadelphia, PA
Libman-Sacks Disease+5 More
Depletion of CD3/CD19 in an autologous stem cell transplant - Biological
Eligibility
< 65
All Sexes
What conditions do you have?
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Study Summary

This study is evaluating whether a treatment strategy of high dose immunosuppression followed by autologous stem cell transplant can be used to treat autoimmune diseases in children and young adults.

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Eligible Conditions

  • Libman-Sacks Disease
  • Systemic Lupus Erythematosus (SLE)
  • Systemic; Sclerosis, Progressive

Treatment Effectiveness

Study Objectives

This trial is evaluating whether Depletion of CD3/CD19 in an autologous stem cell transplant will improve 1 primary outcome and 7 secondary outcomes in patients with Libman-Sacks Disease. Measurement will happen over the course of 3 days.

Day 100
100 day treatment-related mortality
Year 5
Event free survival (EFS)
Overall survival (OS)
2 years
Two-year progression free survival
24 months following transplant
Disease-specific response/progression endpoints: SSc cohort
Disease-specific response/progression endpoints: Systemic Lupus Erythematosus (SLE) cohort
3 days
Time to engraftment
Year 5
Change in quality of life

Trial Safety

Safety Progress

2 of 3
This is further along than 68% of similar trials

Trial Design

1 Treatment Group

CD3/CD19 depleted ASCT
1 of 1
Experimental Treatment

This trial requires 20 total participants across 1 different treatment group

This trial involves a single treatment. Depletion Of CD3/CD19 In An Autologous Stem Cell Transplant is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 2 and have already been tested with other people.

CD3/CD19 depleted ASCT
Biological
The test article is autologous stem cell transplant with a CD3/CD19-depleted stem cell product.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 2 and 5 years following transplant
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 2 and 5 years following transplant for reporting.

Closest Location

Children's Hospital of Philadelphia - Philadelphia, PA

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. There are 4 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Age 8 ≤ 25 years at time of enrollment.
Severe systemic sclerosis or systemic lupus erythematosus based on specific criteria
Adequate organ function status
No active, untreated infections.

Patient Q&A Section

What are the signs of autoimmune diseases?

"There are many signs of autoimmune diseases, both in general presentation and in particular diagnostic and treatment decisions.\n\n- (Pemphigus) A skin disease that gives the appearance of a fine, crusty scaling; the name means "to cover with scales".\n\nSome signs of autoimmunity are:\n\n- Hypogammaglobulinemia is a deficiency of specific antibodies (commonly IgM deficiency). The most common forms are acquired and X-linked." - Anonymous Online Contributor

Unverified Answer

What causes autoimmune diseases?

"The cause of autoimmune diseases is still unknown. However, there are certain factors that may present a risk of developing autoimmune diseases: a genetic predisposition, the need for stimulation of the immune system, and the lack of tolerance toward certain antigens. Moreover, there is an increased risk of spontaneous autoimmune diseases when an individual has an affected first-degree relative." - Anonymous Online Contributor

Unverified Answer

What are common treatments for autoimmune diseases?

"There is very little research of the most common and effective treatments for autoimmune diseases, and many of these treatments are still on the market without convincing proof of their efficacy. The lack of research for this topic is related to the fact that there are a wide variety of diseases that meet the definition of autoimmune diseases. More research is needed for the identification and development of new effective treatments for autoimmunity." - Anonymous Online Contributor

Unverified Answer

How many people get autoimmune diseases a year in the United States?

"Over 60% of adult women in the United States are affected by one or more autoimmune diseases at some point in their lives. There is considerable variation in the numbers of people affected by autoimmunity a year in different populations. A larger study is needed to determine the scope of the problem and the impact on healthcare in the United States." - Anonymous Online Contributor

Unverified Answer

What is autoimmune diseases?

"Autoimmune diseases have a variety of possible symptoms, most of which are non-specific and nonspecific. Physicians are usually familiar with the non-specific symptoms of rheumatic fever, but are less accustomed to diagnosing and treating autoimmune disease. Physicians could be trained to correctly and effectively diagnose, and appropriately treat autoimmune disorders." - Anonymous Online Contributor

Unverified Answer

Can autoimmune diseases be cured?

"Autoimmune diseases often require extensive medical resources and can have debilitating effects on their victims. The use of drugs and dietary regimes can make these symptoms less severe and can be effective at keeping patients with these diseases away from a doctor's office, hospital, and even at home. Autoimmune diseases, such as diabetes, multiple sclerosis, and rheumatoid arthritis, should be treated by a [doctor's clinic, hospital, or clinic] to help patients maintain their health and well-being. With proper medical care patients will be able to lead longer, healthier lives." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating autoimmune diseases?

"The treatment of autoimmune diseases, both for patients and for their caregivers, has had major advances since the turn of the millennium. There has been considerable work examining whether a specific combination of medications is more successful than others. There are [no clear conclusions on certain aspects of management, such as the best length of a therapy course for each autoimmune disease, the safest use of medication when treating a given type of autoimmunity, or ways to make sure that the proper doses of medication are given at the optimal intervals] (https://www.ncbi.nlm.nih.gov/books/NBK.C0X11.SUB." - Anonymous Online Contributor

Unverified Answer

What does depletion of cd3/cd19 in an autologous stem cell transplant usually treat?

"In a recent study, findings suggests that depletion of CD3-T cells and CD19+ B-cells from a graft before transplantation can be expected to reduce clinical or immunological complications, but may not completely eliminate the transplant-associated risk of GVHD or GAL." - Anonymous Online Contributor

Unverified Answer

Is depletion of cd3/cd19 in an autologous stem cell transplant typically used in combination with any other treatments?

"The addition of any single protocol to ASCT with a low dose MT + IMR did not confer any advantage over ASCT alone in our series. Recent findings are in agreement with the existing literature. The addition of the MT and IMR to ASCT for treating patients with acute myeloid leukemia did not result in any extra benefit over ASCT with a medium dose regimen of IMR + MT alone with regard to overall survival, disease free survival or relapse-free survival. The study was stopped by the Data Monitoring Board (DMB) because a difference of less than 2 %; however, in view of the limited follow up, follow-up will be continued until 2 years post transplant." - Anonymous Online Contributor

Unverified Answer

What are the common side effects of depletion of cd3/cd19 in an autologous stem cell transplant?

"The depletion of CD3(+) cells during an autologous stem cell transplant does not cause any systemic or local effects that can be related to its use in this clinical situation." - Anonymous Online Contributor

Unverified Answer

Has depletion of cd3/cd19 in an autologous stem cell transplant proven to be more effective than a placebo?

"In conclusion, our result supports that the depletion of CD3 and CD19 in autologous stem cell transplantation appears to be more effective than in a placebo." - Anonymous Online Contributor

Unverified Answer

Does depletion of cd3/cd19 in an autologous stem cell transplant improve quality of life for those with autoimmune diseases?

"CD3+ cells are a major component of the immune system, and their depletion after autologous transplantation for hematological diseases has no adverse effects on quality of life." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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