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Cell Therapy
Stem Cell Transplant for Blood Diseases
N/A
Recruiting
Led By Alice Bertaina, MD, PhD
Research Sponsored by Alice Bertaina
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Lansky/Karnofsky score > 50
The donor and recipient must be identical, as determined by high resolution typing, in at least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, and HLA-DRB1
Must not have
Current active infectious disease (including positive HIV serology or viral RNA)
Greater than Grade II acute GvHD or severe, unmanaged chronic extensive GvHD due to a previous allograft at the time of inclusion
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year after hsct
Awards & highlights
No Placebo-Only Group
Summary
This trial will help researchers learn whether a particular cell-depleting device is effective in making haploidentical (partially matched) transplants safer and more successful.
Who is the study for?
This trial is for children and young adults under 60 with life-threatening blood diseases, who need a stem cell transplant but don't have a fully matched donor. They must be able to use birth control and have a partial genetic match with the donor. People can't join if they're pregnant, breastfeeding, have severe organ dysfunction or uncontrolled diseases.
What is being tested?
The study tests a new method using CliniMACS TCR α/β Reagent Kit and CD19 to remove certain cells from donor stem cells before transplanting them into patients without fully matched donors. It aims to make transplants safer and more effective for treating various malignant and non-malignant disorders.
What are the potential side effects?
Potential side effects may include reactions related to the immune system's response such as graft-versus-host disease (GvHD), where the donated cells attack the patient's body, infection risks due to weakened immunity, or complications from organ inflammation.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I can do most activities but need help with some.
Select...
My donor and I match in at least one gene at four specific genetic locations.
Select...
I have a serious blood disorder that may improve with a stem cell transplant.
Select...
I am considered eligible for a stem cell transplant from a donor.
Select...
I am between 1 month and 60 years old.
Select...
My genetic match for the treatment is at least half identical.
Exclusion Criteria
You may be eligible for the trial if you check “No” for criteria below:Select...
I do not have any active infections, including HIV.
Select...
I have severe graft-versus-host disease from a past transplant.
Select...
My liver or kidney function is not severely impaired.
Select...
I do not have severe heart problems like bad heart rhythms, heart failure, or very weak heart pumping.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ 1 year after hsct
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 year after hsct
Treatment Details
Study Objectives
Study objectives can provide a clearer picture of what you can expect from a treatment.Secondary study objectives
Leukemia-free survival at 1 year after HSCT
Leukemia-free survival at 2 years after HSCT
Side effects data
From 2019 Phase 2 trial • 124 Patients • NCT009489229%
Fever (in the absence of neutropenia)
9%
Death not associated with CTCAE term
6%
Dyspnea
6%
Renal failure
3%
Pain - Back
3%
Infection with normal ANC or Grade 1 or 2 neutrophils - Brain
3%
Febrile neutropenia
3%
Infection with normal ANC or Grade 1 or 2 neutrophils - Paranasal
3%
Pulmonary/Upper Respiratory - Other, Pneumonia
3%
Pulmonary/Upper Respiratory - Other, Pulmonary edema
3%
Infection with normal ANC or Grade 1 or 2 neutrophils - Salivary gland
3%
Blood/Bone Marrow - Other
3%
Cardiac General - Other
3%
Hypotension
3%
Dysphagia
3%
Gastrointestinal - Other
3%
Mucositis/stomatitis
3%
Nausea
3%
Hypoxia
3%
Secondary malignancy - possibly related to cancer treatment
3%
Thrombosis/embolism (vascular access-related)
3%
Thrombosis/thrombus/embolism
3%
Esophagitis
3%
Infection with unknown ANC - Salivary gland
3%
Opportunistic infection associated with >= Grade 2 Lymphopenia
3%
ALT, SGPT - Increase
3%
AST, SGOT - Increase
3%
Alkaline phosphatase
3%
Encephalopathy
100%
80%
60%
40%
20%
0%
Study treatment Arm
A: Allogeneic Stem Cell Transplant
B: Autologous Stem Cell Transplant
BE: Group B Expansion
Awards & Highlights
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.
Trial Design
2Treatment groups
Experimental Treatment
Group I: Stem Cell Transplant -MalignantExperimental Treatment2 Interventions
The participant with a malignancy will undergo a stem cell transplant using donor cells that have been manipulated through an investigational device. Participants will be followed for outcomes for two years.
Group II: Stem Cell Transplant - Non-MalignantExperimental Treatment2 Interventions
The participant with a non-malignant disease will undergo a stem cell transplant using donor cells that have been manipulated through an investigational device. Participants will be followed for outcomes for two years.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Allogeneic Stem Cell Transplant
2009
Completed Phase 2
~130
Find a Location
Who is running the clinical trial?
Alice BertainaLead Sponsor
1 Previous Clinical Trials
12 Total Patients Enrolled
Alice Bertaina, MD, PhDPrincipal Investigator - Associate Professor of Pediatrics, Stem Cell Transplantation
Stanford University
1 Previous Clinical Trials
22 Total Patients Enrolled
Media Library
Research Study Groups:
This trial has the following groups:- Group 1: Stem Cell Transplant -Malignant
- Group 2: Stem Cell Transplant - Non-Malignant
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.