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Hematopoietic Stem Cell Transplantation

Stem Cell Transplant for Blood Diseases

Phase 2
Waitlist Available
Led By Christen L Ebens, MD, MPH
Research Sponsored by Masonic Cancer Center, University of Minnesota
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Sickle Cell Disease (SCD) with specific disease characteristics including stroke, CNS hemorrhage, abnormal cerebral MRI, acute chest syndrome, recurrent vaso-occlusive pain, impaired neuropsychological function, sickle lung disease, sickle nephropathy, proliferative retinopathy, osteonecrosis of multiple joints, requirement for chronic transfusions, RBC alloimmunization, transfusion dependent Alpha- or Beta-Thalassemia, and other non-malignant hematologic disorders
Be younger than 65 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year
Awards & highlights

Study Summary

This trial is for people with high-risk non-malignant diseases who don't have a suitable HLA-matched sibling donor, using a reduced intensity conditioning T-cell replete haploidentical donor.

Who is the study for?
This trial is for people aged 0-55 with high-risk non-malignant diseases like certain blood, metabolic disorders, and adrenoleukodystrophy. They must lack a suitable HLA-matched sibling donor but have adequate organ function and no major disabilities. Pregnant or breastfeeding individuals, those with uncontrolled infections, HIV positive or who have an available matched related donor cannot participate.Check my eligibility
What is being tested?
The study tests T-cell replete reduced intensity conditioning haploidentical donor allogeneic hematopoietic cell transplantation (HaploHCT) in patients without a matching sibling donor. It's designed to see if this approach is effective for treating high-risk non-malignant diseases.See study design
What are the potential side effects?
Potential side effects include complications from the transplant procedure such as infection risk due to immune suppression, graft-versus-host disease where the donated cells attack the body, organ damage due to conditioning treatment, and possible infusion-related reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
You have sickle cell disease with specific severe complications such as stroke, abnormal brain MRI, lung or kidney problems, or the need for frequent blood transfusions.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Neutrophil Recovery
Secondary outcome measures
Overall Survival (OS)
Primary Graft Failure (neutropenic and non-neutropenic)

Trial Design

1Treatment groups
Experimental Treatment
Group I: rATG, FLU/CY/TBI, & ThiotepaExperimental Treatment1 Intervention
Anti-Thymocyte Globulin - Rabbit (rATG), Fludarabine (Fludara), Cyclophosphamide (Cytoxan, Neosar), Total Body Irradiation (TBI), & Thiotepa

Find a Location

Who is running the clinical trial?

Masonic Cancer Center, University of MinnesotaLead Sponsor
271 Previous Clinical Trials
14,620 Total Patients Enrolled
Christen L Ebens, MD, MPHPrincipal InvestigatorUniversity of Minnesota - Pediatrics Blood and Marrow Transplant

Media Library

Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases (Hematopoietic Stem Cell Transplantation) Clinical Trial Eligibility Overview. Trial Name: NCT03367546 — Phase 2
Hematologic Disorders Research Study Groups: rATG, FLU/CY/TBI, & Thiotepa
Hematologic Disorders Clinical Trial 2023: Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases Highlights & Side Effects. Trial Name: NCT03367546 — Phase 2
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases (Hematopoietic Stem Cell Transplantation) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03367546 — Phase 2

Frequently Asked Questions

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

Is the age threshold for this experiment over or under twenty years old?

"This experiment has established that only those between 0 and 25 years old are eligible. Furthermore, there exist 432 studies for younger individuals and 831 trials for more senior citizens."

Answered by AI

How many participants have enrolled in this clinical trial?

"Affirmative. Clinicaltrials.gov provides evidence that this clinical trial, first posted on July 2nd 2018, is actively recruiting participants. The research requires 20 individuals to be enrolled at only one medical site."

Answered by AI

What are the eligibility criteria for involvement in this clinical exploration?

"This trial is taking on 20 individuals between 0 and 25 years old who have thalassemia. Furthermore, the following prerequisites must be met: bilateral proliferative retinopathy with major vision impairment in one eye, multiple joint necrosis with observable damage, need for perpetual transfusions, recurrent hospitalizations or exchange transfusions due to acute chest syndrome."

Answered by AI

Are there still opportunities for participants to join this research project?

"Affirmative. The clinical trial is actively recruiting, as the information available on clinicaltrials.gov confirms. Initially posted in July 2018 and most recently edited in May 2022, this medical study requires 20 participants across 1 site for completion."

Answered by AI

What risks should a patient consider before undergoing Blood and Marrow Transplantation?

"Our assessment of the safety profile of Blood and Marrow Transplant is a 2, as this Phase 2 trial has shown some evidence in favour of its security but not efficacy."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases
2018. "Haploidentical Allogeneic Hematopoietic Stem Cell Transplantation (HaploHCT) Following Reduced Intensity Conditioning (RIC) for Selected High Risk Non-Malignant Diseases". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03367546.
~1 spots leftby Apr 2025
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