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Stem Cell Transplant with TCRab Depletion for Sickle Cell Disease and Beta Thalassemia

N/A
Waitlist Available
Led By Timothy Olson, MD, PhD
Research Sponsored by Children's Hospital of Philadelphia
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Clinically symptomatic neurologic event (stroke) or any neurologic deficit lasting greater than 24 hours at any time prior to enrollment
Splenic sequestration (defined as a 2 g/dL drop in hemoglobin in the setting of an acutely enlarging spleen. This will be determined as part of clinical care and prior to the research)
Must not have
Previous Hematopoietic stem cell transplant (HSCT)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to three years post-transplantation
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing a new way to do stem cell transplants for sickle cell disease and beta thalassemia.

Who is the study for?
This trial is for patients with severe forms of sickle cell disease or beta thalassemia major who have had strokes, frequent pain episodes, or require regular blood transfusions. It's not open to those who've had a previous stem cell transplant, are pregnant, or can't receive blood transfusions due to severe allergies.
What is being tested?
The study tests a peripheral stem cell transplantation technique using the CliniMACS device to remove certain T cells and B cells from donor grafts in patients with sickle cell disease and beta thalassemia major.
What are the potential side effects?
Potential side effects may include reactions related to the immune system (like fever or chills), complications from receiving donor cells (such as graft-versus-host disease), and typical risks associated with stem cell transplants like infections.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have had a stroke or lasting neurological issues for more than a day.
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My spleen is enlarged and my hemoglobin levels have dropped.
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My genetic test confirms I have Beta Thalassemia.
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I've had 8 or more blood transfusions in the last year for my sickle cell disease.
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I have had pain so severe it needed IV pain management or a hospital stay.
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I need blood transfusions regularly for my thalassemia.
Select...
I have severe Sickle Cell Disease.
Select...
I have a specific type of sickle cell disease.
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I have had acute chest syndrome.
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I have Beta Thalassemia Major.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have had a stem cell transplant before.

Timeline

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

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Incidence of acute graft vs. host disease (GVHD)
Incidence of chronic graft vs. host disease (GVHD)
Rate of graft failure
+1 more
Secondary study objectives
Incidence of viral reactivation and symptomatic viral infection
Number of deaths due to treatment
Probability of event-free survival (EFS)
+1 more

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

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: Sickle Cell DiseaseExperimental Treatment1 Intervention
Patients with Sickle Cell Disease (SCD) will be given previously established, disease-specific chemotherapy based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from closely matched unrelated donors.
Group II: Beta Thalassemias MajorExperimental Treatment1 Intervention
Patients with Beta Thalassemias Major (BTM) will be given previously established, disease-specific chemotherapy based conditioning regimens prior to hematopoietic stem cell transplantation using TCRalpha/beta and B cell depleted peripheral blood stem cells from closely matched unrelated donors.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
CliniMACS
2005
Completed Phase 3
~770

Find a Location

Who is running the clinical trial?

Children's Hospital of PhiladelphiaLead Sponsor
729 Previous Clinical Trials
8,470,252 Total Patients Enrolled
Timothy OlsonLead Sponsor
Timothy Olson, MD, PhDPrincipal InvestigatorChildren's Hospital of Philadelphia
6 Previous Clinical Trials
425 Total Patients Enrolled

Media Library

CliniMACS Clinical Trial Eligibility Overview. Trial Name: NCT04523376 — N/A
Sickle Cell Disease Research Study Groups: Beta Thalassemias Major, Sickle Cell Disease
Sickle Cell Disease Clinical Trial 2023: CliniMACS Highlights & Side Effects. Trial Name: NCT04523376 — N/A
CliniMACS 2023 Treatment Timeline for Medical Study. Trial Name: NCT04523376 — N/A
~0 spots leftby Dec 2024
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