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Alkylating agents

Immunosuppression vs. Stem Cell Transplant for Severe Aplastic Anemia

Q: How many locations are covered by this research endeavor?

For this trial, there are currently 13 locations available for enrolment. This includes the Cleveland Clinic in Cleveland, Fred Hutchinson <a href="https://www.withpower.com/clinical-trials/cancer">Cancer</a> Research Center in Seattle and Children's Hospital Los Angeles among other options.

Q: To what ailments is Matched Unrelated Stem Cell Transplant typically applied?

The most common treatment for nephrotic syndrome is Matched Unrelated Stem Cell <a href="https://www.withpower.com/clinical-trials/transplant">Transplant</a>. This medical therapy has also been seen to be effective at treating <a href="https://www.withpower.com/clinical-trials/leukemia">leukemia</a>, ocular <a href="https://www.withpower.com/clinical-trials/rosacea">rosacea</a>, and <a href="https://www.withpower.com/clinical-trials/lung-cancer">lung cancer</a>.

Q: Are researchers currently enrolling for this clinical investigation?

The information held on clinicaltrials.gov currently indicates that this trial has ceased its recruitment efforts, with the study being originally posted in August 2016 and last updated February 2022. Despite this, there are a further 1261 medical studies open for patient participation at present.

Q: What is the expected enrollment of this experiment?

As of this moment, no further candidates are being accepted for this trial. This was first posted to clinicaltrials.gov on August 1st 2016 and most recent updates were made on February 10th 2022. However, individuals who still wish to partake in a medical study may be interested in the 249 trials actively searching for participants with aplastic anemia or one of the 1012 research projects recruiting patients for Matched Unrelated Stem Cell Transplantations.

Q: Is Matched Unrelated Stem Cell Transplant a common practice in medical studies?

Presently, there are 1012 clinical studies in progress regarding Matched Unrelated Stem Cell <a href="https://www.withpower.com/clinical-trials/transplant">Transplant</a>. Of these trials, 196 have reached the 3rd phase of testing and 30980 medical centres worldwide are conducting research on this topic with a particular concentration of locations centred around Philadelphia, <a href="https://www.withpower.com/clinical-trials/pennsylvania">Pennsylvania</a>.

N/A

Waitlist Available

Research Sponsored by Michael Pulsipher, MD

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Confirmed diagnosis of idiopathic SAA with bone marrow cellularity <25%, or <30% hematopoietic cells and two out of three of the following (in peripheral blood): neutrophils <0.5 x109/L, platelets <20 x109/L, reticulocyte count <60 x109/L with hemoglobin <8g/dL.

No suitable fully matched related donor available (minimum 6/6 match for Human Leukocyte antigen (HLA) -A and B at intermediate or high resolution and DRB1 at high resolution using DNA based typing).

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 4 years

Awards & highlights

Study Summary

This trial will compare outcomes of immunosuppressive therapy vs. stem cell transplant to treat pediatric severe aplastic anemia.

Who is the study for?

This trial is for children and young adults up to 25 years old with severe aplastic anemia, who don't have a closely matched family donor for stem cell transplant. They should have specific low blood counts, no inherited bone marrow failure syndromes or certain other conditions, and at least two potential unrelated donors.Check my eligibility

What is being tested?

The study compares two treatments: immunosuppressive therapy (IST) versus a stem cell transplant from an unrelated donor (MUD HSCT). It includes drugs like fludarabine and cyclophosphamide, anti-thymocyte globulin from rabbits or horses, methotrexate, cyclosporine, and low-dose total body irradiation.See study design

What are the potential side effects?

Possible side effects include allergic reactions to medications especially horse ATG if known allergy exists; damage to organs such as the liver or kidneys; increased risk of infections due to immune suppression; mouth sores; nausea; diarrhea.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

I have severe aplastic anemia with very low blood cell counts.

Select...

I don't have a perfectly matched family donor for a transplant.

Select...

I have at least two unrelated bone marrow donors who match me closely.

Select...

I am 25 years old or younger.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 4 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~4 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and 4 years for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Percentage of patients randomized to HSCT that actually complete HSCT

Secondary outcome measures

Paroxysmal nocturnal hemoglobinuria

Incidence of significant infection in both treatment arms

Number of patients that fail to receive their primary assigned therapy (HSCT or IST).

+15 more

Trial Design

2Treatment groups

Active Control

Group I: Matched Unrelated Stem Cell TransplantActive Control7 Interventions

Patient will under go matched unrelated donor transplant of hematopoietic stem cells as their therapy using fludarabine, cyclophosphamide, rabbit anti-thymocyte globulin (ATG), and low-dose total body irradiation (TBI) as preparative regimen and cyclosporine and methotrexate for graft versus host disease (GVHD) prevention.

Group II: Immunosuppressive TherapyActive Control3 Interventions

Patient will receive standard immunosuppressive therapy combination of drugs: horse anti-thymocyte globulin (ATG) and cyclosporine.

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

Michael Pulsipher, MDLead Sponsor

4 Previous Clinical Trials

204 Total Patients Enrolled

David A Williams, MDStudy ChairBoston's Childrens Hospital

2 Previous Clinical Trials

126 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

How many locations are covered by this research endeavor?

"For this trial, there are currently 13 locations available for enrolment. This includes the Cleveland Clinic in Cleveland, Fred Hutchinson Cancer Research Center in Seattle and Children's Hospital Los Angeles among other options."

Answered by AI

To what ailments is Matched Unrelated Stem Cell Transplant typically applied?

"The most common treatment for nephrotic syndrome is Matched Unrelated Stem Cell Transplant. This medical therapy has also been seen to be effective at treating leukemia, ocular rosacea, and lung cancer."

Answered by AI

Are researchers currently enrolling for this clinical investigation?

"The information held on clinicaltrials.gov currently indicates that this trial has ceased its recruitment efforts, with the study being originally posted in August 2016 and last updated February 2022. Despite this, there are a further 1261 medical studies open for patient participation at present."

Answered by AI

What is the expected enrollment of this experiment?

"As of this moment, no further candidates are being accepted for this trial. This was first posted to clinicaltrials.gov on August 1st 2016 and most recent updates were made on February 10th 2022. However, individuals who still wish to partake in a medical study may be interested in the 249 trials actively searching for participants with aplastic anemia or one of the 1012 research projects recruiting patients for Matched Unrelated Stem Cell Transplantations."

Answered by AI

Is Matched Unrelated Stem Cell Transplant a common practice in medical studies?

"Presently, there are 1012 clinical studies in progress regarding Matched Unrelated Stem Cell Transplant. Of these trials, 196 have reached the 3rd phase of testing and 30980 medical centres worldwide are conducting research on this topic with a particular concentration of locations centred around Philadelphia, Pennsylvania."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

Michael Pulsipher, MD 2016. "Unrelated Donor Transplant Versus Immune Therapy in Pediatric Severe Aplastic Anemia". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02845596.

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