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Cell Therapy

Stem Cell Transplant for Leukemia (HAPLOTAB Trial)

N/A
Recruiting
Led By Erin Morales, MD
Research Sponsored by Baylor College of Medicine
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Lack of suitable conventional donor (10/10 HLA matched related or unrelated donor) or presence of rapidly progressive disease not permitting time to identify an HLA-matched unrelated donor (excluding cord blood unit availability)
Lansky/Karnofsky score > 50
Must not have
Symptomatic cardiac disease or left ventricular shortening fraction less than 25% or ejection fraction < 40%
Serious concurrent uncontrolled medical disorder or mental illness
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to one year post-hct
Awards & highlights

Summary

This trial will test a new, safer way to do a stem cell transplant to treat underlying medical conditions and eliminate the risk of graft versus host disease.

Who is the study for?
This trial is for patients without a fully matched donor or with rapidly progressing diseases who need a stem cell transplant. It's open to those with certain blood cancers, hemoglobin disorders like thalassemia or sickle cell disease, severe viral infections, immune deficiencies, and bone marrow failures. Participants must be in good enough health to undergo the procedure.Check my eligibility
What is being tested?
The study tests a new way of doing transplants using half-matched family donors' blood cells treated to reduce GvHD risk. The treatment uses CliniMACS® TCRα/β-Biotin System to remove specific immune cells that could cause complications post-transplant.See study design
What are the potential side effects?
Potential side effects may include reactions related to the transplantation process such as infection risks due to weakened immunity, graft failure, mild-to-severe GvHD despite preventive measures taken during the procedure, and possible infusion-related reactions.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I don't have a perfect match donor or my disease is progressing too quickly to wait for one.
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I am mostly active and can do things for myself.
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I have a serious blood disorder or bone marrow failure.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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My heart is weak, with an ejection fraction below 40%.
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I do not have any serious uncontrolled health or mental conditions.
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I am currently on immunosuppressive drugs for GVHD from a past transplant.
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I am currently living with HIV.
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My kidney function is severely reduced.
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My liver tests are significantly higher than normal.
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I do not have an active infection that would prevent me from receiving strong chemotherapy or a transplant.
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I have severe lung disease with very low lung function.
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I am currently pregnant or breastfeeding.
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I have severe graft versus host disease from a past transplant.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to one year post-hct
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to one year post-hct for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Rate of acute graft versus host disease (GvHD) by grades
Rate of neutrophil engraftment
Rate of platelet engraftment
Secondary outcome measures
Overall survival (OS)
Rate of transplant-related mortality (TRM)

Side effects data

From 2020 Phase 3 trial • 324 Patients • NCT00703820
68%
Febrile neutropenia (fever of unknown origin: not clinically or microbiologically documented)
53%
Infection (documented clinically or microbioogically) with Grade 3 or 4 neutrophils (ANC<1.0x10e9/L)
34%
Potassium, serum-low (hypokalemia)
25%
ALT, SGPT (serum glutamic pyruvic transaminase)
16%
Colitis, infectious (e.g., Clostridium difficile)
15%
Infection (documented clinically or microbiologically documented, ANC<1.0x10e9/L, fever>38.5C),blood
15%
AST, SGOT (serum glutamic oxaloacetic transaminase)
13%
Phosphate, serum-low (hypophosphatemia)
12%
Glucose, serum-high (hyperglycemia)
12%
Calcium, serum-low (hypocalcemia)
8%
Anorexia
8%
Allergic reaction/hypersensitivity (including drug fever)
8%
Hypoxia
8%
Hypertension
8%
Fever (in the absence of neutropenia, where neutropenia is defined as ANC <1.0x10e9/L)
7%
Nausea
6%
Sodium, serum-low (hyponatremia)
6%
Hypotension
5%
Bilirubin (hyperbilirubinemia)
5%
Diarrhea
5%
Infection with normal ANC or Grade 1 or 2 neutrophils
5%
Rash/desquamation
5%
Allbumin, serum-low (hypoalbuminemia)
5%
Tumor lysis syndrome
5%
Typhlitis (cecal inflammation)
5%
Vomiting
5%
Colitis
4%
Mucositis/stomatitis (clinical exam), oral cavity
4%
Pain, rectum
4%
Speech impairment (e.g., dysphasia or aphasia)
3%
Sodium, serum-high (hypernatremia)
3%
Pain, Abdomen NOS
3%
Mucositis/stomatitis (functional/symptomatic), oral cavity
3%
Pain, head/headache
3%
Ataxia (incoordination)
3%
Acidosis (metabolic or respiratory)
2%
Infection with unknown ANC, blood
2%
GGT (gamma-Glutamyl transpeptidase)
2%
Alkalosis (metabolic or respiratory)
2%
Magnesium, serum-low (hypomagnesemia)
2%
Esophagitis
2%
Uric acid, serum-high (hyperuricemia)
2%
Ileus, GI (functional obstruction of bowel, i.e., neuroconstipation)
2%
Gastritis (including bile reflux gastritis)
2%
Enteritis (inflammation of the small bowel)
2%
Syncope (fainting)
2%
Neuropathy: motor
2%
Prolonged QTc interval
2%
Hemorrhage, pulmonary/upper respiratory, nose
2%
Potassium, serum-ghigh (hyperkalemia)
2%
Weight loss
2%
Injection site reaction/extravasation changes
2%
Left ventricular systolic dysfunction
2%
Pericardial effusion (non-malignant)
2%
Thrombosis/thrombus/embolism
2%
Hematoma
2%
PTT (Partial Thromboplastin Time)
2%
Renal failure
2%
Confusion
2%
Cholecystitis
2%
Dysphagia (difficulty swallowing)
2%
Encephalopathy
2%
Infection - other
2%
Adult Respiratory Distress Syndrome (ARDS)
2%
Pneumonitis/pulmonary infiltrates
2%
Metabolic/Laboratory
2%
CNS cerebrovascular ischemia
2%
Pulmonary/upper respiratory - other
1%
Somnolence/depressed level of consciousness
1%
Magnesium, serum-high (hypermagnesemia)
1%
Gastrointestinal - Other
1%
Lipase
1%
Distension/bloating, abdominal
1%
Fibrinogen
1%
Calcium, serum-high (hypercalcemia)
1%
Hemorrhage, GI, Lower GI NOS
1%
Seizure
1%
Neurology - other
1%
Neuropathy: cranial, CN IX Motor-pharynx; sensory-ear, pharynx, tongue
1%
Supraventricular and nodal arrhythmia, sinus tachycardia
1%
Infection with unknown ANC, catheter-related
1%
DIC (disseminated intravascular coagulation)
1%
Infection with unknown ANC, urinary tract NOS
1%
Neutrophils/granulocytes (ANC/AGC)
1%
Ocular surface disease
1%
Keratitis (corneal inflammation/corneal ulceration)
1%
Vision-photophobia
1%
Death not associated with CTCAE term, multi-organ failure
1%
Constitutional symptoms - other
1%
Pain - other
1%
Death not associated with CTCAE term, sudden death
1%
Dyspnea (shortness of breath)
1%
Pleural effusion (non-malignant)
1%
Apnea
1%
Valvular heart disease
1%
Supraventricular and nodal arrhythmia, sinus bradycardia
1%
Left ventricular diastolic dysfunction
1%
Rash: acne/acneiform
1%
Pruritus/itching
1%
Acute vascular leak syndrome
1%
Pain, back
1%
Urinary frequency/urgency
1%
Mood alteration, euphoria
1%
Tinnitus
1%
Pain, vagina
1%
Hemorrhage, pulmonary/upper respiratory, bronchopulmonary NOS
1%
Right ventricular dysfunction (cor pulmonale)
1%
Pain, extremity-limb
1%
INR (International Normalized Ratio of prothrombin time)
1%
Perforation, GI, appendix
1%
Pain, dental/teeth/peridontal
1%
Pain, stomach
1%
Mucotitis/stomatitis (functional/symptomatic), esophagus
1%
Pain, lip
1%
Neuropathy: sensory
1%
Hemorrhage, CNS
1%
Hemorrhage, GU, iuterus
1%
Dehydration
1%
Amylase
1%
Creatinine
1%
CNS necrosis/cystic progression
1%
Hemorrhage, GI, Oral cavity
100%
80%
60%
40%
20%
0%
Study treatment Arm
Cytarabine+Daunorubicin+Etoposide
Clofarabine+Cytarabine
Stem Cell Donors

Trial Design

1Treatment groups
Experimental Treatment
Group I: Alpha beta+ T cell depleted CD34+ stem cellsExperimental Treatment1 Intervention
The patient will be receiving a donor stem cell transplant with a preceding conditioning regimen (chemotherapy with, or without, radiation). The investigators will be specially treating the donor's blood cells used for the stem cell transplant.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
CliniMACS
2005
Completed Phase 3
~770

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.
Mechanism Of Action
Side Effect Profile
Prior Approvals
Other Research
Common treatments for leukemia include allogeneic hematopoietic cell transplantation (HCT), CAR T-cell therapy, and targeted agents such as BTK inhibitors and BCL2 inhibitors. Allogeneic HCT involves transplanting healthy donor stem cells to replace the patient's diseased bone marrow, leveraging the graft-versus-leukemia (GVL) effect to eradicate cancer cells. CAR T-cell therapy modifies a patient's T-cells to target and destroy leukemia cells, offering a personalized approach. BTK inhibitors (e.g., ibrutinib) and BCL2 inhibitors (e.g., venetoclax) disrupt key survival pathways in leukemia cells, leading to their death. The trial involving ex-vivo T cell receptor Alpha/Beta+ and CD19 depletion aims to reduce GvHD by removing specific T cells and B cells, thus minimizing immune complications and improving patient outcomes. These mechanisms are crucial as they offer targeted, effective treatments while reducing the risk of severe side effects, enhancing the quality of life and survival rates for leukemia patients.
New Approaches to the Management of Adult Acute Lymphoblastic Leukemia.CD19 chimeric antigen receptor (CD19 CAR)-redirected adoptive T-cell immunotherapy for the treatment of relapsed or refractory B-cell Non-Hodgkin's Lymphomas.Allogeneic Transplantation for Chronic Lymphocytic Leukemia in the Age of Novel Treatment Strategies.

Find a Location

Who is running the clinical trial?

Baylor College of MedicineLead Sponsor
1,006 Previous Clinical Trials
6,003,261 Total Patients Enrolled
30 Trials studying Leukemia
1,337 Patients Enrolled for Leukemia
Center for Cell and Gene Therapy, Baylor College of MedicineOTHER
111 Previous Clinical Trials
2,776 Total Patients Enrolled
22 Trials studying Leukemia
478 Patients Enrolled for Leukemia
The Methodist Hospital Research InstituteOTHER
275 Previous Clinical Trials
80,490 Total Patients Enrolled
21 Trials studying Leukemia
454 Patients Enrolled for Leukemia

Media Library

Haploidentical Hematopoietic Cell Transplantation (Cell Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT05236764 — N/A
Leukemia Research Study Groups: Alpha beta+ T cell depleted CD34+ stem cells
Leukemia Clinical Trial 2023: Haploidentical Hematopoietic Cell Transplantation Highlights & Side Effects. Trial Name: NCT05236764 — N/A
Haploidentical Hematopoietic Cell Transplantation (Cell Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05236764 — N/A
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