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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)
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to three years post-transplantation
Awards & highlights

Study 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.Check my eligibility
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.See study design
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.
Select...
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.
Select...
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.
Select...
I have had acute chest syndrome.
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I have Beta Thalassemia Major.

Timeline

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

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Incidence of acute graft vs. host disease (GVHD)
Incidence of chronic graft vs. host disease (GVHD)
Rate of graft failure
+1 more
Secondary outcome measures
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%
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%
Infection with normal ANC or Grade 1 or 2 neutrophils
5%
Diarrhea
5%
Bilirubin (hyperbilirubinemia)
5%
Allbumin, serum-low (hypoalbuminemia)
5%
Tumor lysis syndrome
5%
Rash/desquamation
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%
Pain, head/headache
3%
Sodium, serum-high (hypernatremia)
3%
Ataxia (incoordination)
3%
Pain, Abdomen NOS
3%
Acidosis (metabolic or respiratory)
3%
Mucositis/stomatitis (functional/symptomatic), oral cavity
2%
GGT (gamma-Glutamyl transpeptidase)
2%
Neuropathy: motor
2%
Esophagitis
2%
Gastritis (including bile reflux gastritis)
2%
Magnesium, serum-low (hypomagnesemia)
2%
Alkalosis (metabolic or respiratory)
2%
Syncope (fainting)
2%
Infection with unknown ANC, blood
2%
Uric acid, serum-high (hyperuricemia)
2%
Ileus, GI (functional obstruction of bowel, i.e., neuroconstipation)
2%
Enteritis (inflammation of the small bowel)
2%
Potassium, serum-ghigh (hyperkalemia)
2%
Hemorrhage, pulmonary/upper respiratory, nose
2%
Prolonged QTc interval
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%
Seizure
1%
Magnesium, serum-high (hypermagnesemia)
1%
Calcium, serum-high (hypercalcemia)
1%
Distension/bloating, abdominal
1%
Gastrointestinal - Other
1%
Lipase
1%
Fibrinogen
1%
Supraventricular and nodal arrhythmia, sinus tachycardia
1%
Hemorrhage, GI, Lower GI NOS
1%
Somnolence/depressed level of consciousness
1%
Neuropathy: cranial, CN IX Motor-pharynx; sensory-ear, pharynx, tongue
1%
Neurology - other
1%
Infection with unknown ANC, catheter-related
1%
Infection with unknown ANC, urinary tract NOS
1%
DIC (disseminated intravascular coagulation)
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

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
708 Previous Clinical Trials
8,581,283 Total Patients Enrolled
Timothy OlsonLead Sponsor
Timothy Olson, MD, PhDPrincipal InvestigatorChildren's Hospital of Philadelphia
2 Previous Clinical Trials
75 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

Frequently Asked Questions

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

Is enrollment still open for this clinical experiment?

"Affirmative. Clinicaltrials.gov indicates that this clinical trial is actively recruiting, having been established on May 14th 2020 and last modified on December 22nd 2021. This particular study needs to recruit 20 individuals from a single research site."

Answered by AI

Is eligibility for this research project limited to those 18 years and older?

"This research seeks to enrol individuals aged between two and 25 years."

Answered by AI

What is the participant capacity of this medical experiment?

"Affirmative. The clinicaltrial.gov website shows that this research study, which was initially launched on May 14th 2020, is still recruiting participants. Approximately 20 individuals need to be recruited from one medical facility."

Answered by AI

To whom does this trial offer enrollment opportunities?

"This medical trial is enrolling 20 individuals, between 2 and 25 years of age, that suffer from thalassemia. Furthermore, they must have experienced a stroke or other neurologic deficit lasting longer than 24 hours in the past; two VOE episodes annually for the previous two years despite hydroxyurea therapy being administered for at least 6 months; one manifestation such as Hemoglobin SS/SC/SD/SOArab/SBeta Thalassemia type genotypes, priapism, acute chest syndrome or recurrent pain episodes requiring intravenous treatment and hospitalization."

Answered by AI
Recent research and studies
clinicaltrials.govStudy
Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies
Timothy Olson 2020. "Pilot Study PBSCT With TCRab Depletion For Hemoglobinopathies". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04523376.
All > Thalassemia > Sickle Cell Anemia
~3 spots leftby Dec 2024
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