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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 year after hsct
Awards & highlights

Study 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.Check my eligibility
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.See study design
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 below
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I can do most activities but need help with some.
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My donor and I match in at least one gene at four specific genetic locations.
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I have a serious blood disorder that may improve with a stem cell transplant.
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I am considered eligible for a stem cell transplant from a donor.
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I am between 1 month and 60 years old.
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My genetic match for the treatment is at least half identical.

Timeline

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

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Number of participants with grade II-IV acute GvHD at day 100 after HSCT
Secondary outcome measures
Leukemia-free survival at 1 year after HSCT
Leukemia-free survival at 2 years after HSCT
Number of participants with chronic GVHD at 1 year after HSCT
+4 more

Side effects data

From 2019 Phase 2 trial • 124 Patients • NCT00948922
9%
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

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

Blood Diseases Research Study Groups: Stem Cell Transplant -Malignant, Stem Cell Transplant - Non-Malignant
Blood Diseases Clinical Trial 2023: Allogeneic Stem Cell Transplant Highlights & Side Effects. Trial Name: NCT04249830 — N/A

Frequently Asked Questions

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

Are participants of the age 25 or younger being included in this clinical investigation?

"The enrollment requirements dictate that individuals between the ages of 1 month and 60 years old can participate in this study. Conversely, there are 30 trials available for minors and 42 studies open to seniors above 65 years old."

Answered by AI

Am I eligible to partake in this experiment?

"This clinical trial is recruiting 50 individuals with hematological illnesses aged between one month and sixty years old. They must also meet the following conditions: be younger than 60, have a life expectancy of more than 10 weeks, qualify for allogeneic HSCT according to their medical facility's guidelines, suffer from either cancerous or non-cancerous blood diseases that can benefit from stem cell transplantation, and possess a Lansky/Karnofsky score higher than fifty."

Answered by AI

How many participants are receiving treatment through this trial?

"Correct. According to the information displayed on clinicaltrials.gov, this medical experiment is currently recruiting candidates. The trial was first posted on February 1st 2020 and most recently updated two days ago - 2/2/2022. 50 individuals are needed for the study from a single location."

Answered by AI

Are there any vacancies open in this clinical experiment?

"Affirmative, the particulars of this clinical trial are available on clinicaltrials.gov and indicate that it is now recruiting participants. Originally posted in February 2020, the study was last updated in February 2022 and requires 50 enrollees from 1 centre."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Young Adults
Alice Bertaina 2020. "Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Young Adults". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04249830.
~23 spots leftby Feb 2025
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