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New treatment option for Sickle Cell Disease
Official title A Study of Immune Suppression Treatment for People With Sickle Cell Disease or β-Thalassemia Who Are Going to Receive an Allogeneic Hematopoietic Cell Transplantation (HCT)
ClinicalTrials.gov ID: NCT05736419
What this study is testing
What is Fludarabine?
Fludarabine is an investigational medicine, being studied as a potential treatment for sickle cell disease.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- Hematopoietic Cell Transplantation/HCT involves receiving healthy blood-forming cells (stem cells) from a donor to replace the diseased or damaged cells in participants' bone marrow. The researchers think giving participants treatment with fludarabine and dexamethasone, drugs that lower the activity of the body's immune system (immune suppression), before standard conditioning therapy and HCT may help prevent serious side effects, including graft failure and GvHD.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 2 to 50
You may be able to join if
- Age ≥ 2 and ≤ 50 years
- Suitable haploidentical donor.
- Performance score ≥ 70% by Karnofsky Performance Scale or 0 to 1 by ECOG (age \> 16 years), or Lansky Play-Performance Scale ≥ 70% (age ≤ 16 years).
- Adequate major organ system function as demonstrated by:
- For patients ≥ 18 years of age:
You likely can't join if
- Prior myeloablative allogeneic HCT.
- Overt stroke or CNS instrumentation (e.g. for Moyamoya disease) within 6 months of enrollment.
- Liver cirrhosis. Mild fibrosis will be permitted, i.e. fine reticulin or grade 1 of 4, with bridging fibrosis.
- Hepatic iron content ≥ 3 mg Fe/g liver dry weight, if applicable
- Active hepatitis B or C.
- Other uncontrolled infections.
See the full eligibility criteria
- Age ≥ 2 and ≤ 50 years
- Suitable haploidentical donor.
- Performance score ≥ 70% by Karnofsky Performance Scale or 0 to 1 by ECOG (age \> 16 years), or Lansky Play-Performance Scale ≥ 70% (age ≤ 16 years).
- Adequate major organ system function as demonstrated by:
- For patients ≥ 18 years of age:
- eGFR ≥ 50 mL/min by Cockcroft-Gault formula Formula: ((140 - Age) x Weight (kg)) / (72 x Serum Creatinine (mg/dL) Female Adjustment: Multiply result by 0.85
- For patients \< 18 years of age:
- Serum creatinine clearance: glomerular filtration rate [GFR]) must be \>50 mL/min/1.73 m2 as calculated by the Schwartz formula
- Conjugated (direct) bilirubin less than 3x upper limit of normal.
- ALT or AST ≤ 3 times institutional upper limit of normal.
- Left ventricular ejection fraction ≥ 50%.
- Diffusing capacity for carbon monoxide (DLCO) ≥ 50% predicted, corrected for hemoglobin. For children \ 92% on room air by pulse oximetry.
- For SCD patients: HbSS, HbSC, HbS/β° with one or more of the following complications:
- Acute chest syndrome: 2 or more episodes in the 2 years preceding enrollment
- Vaso-occlusive episodes: 3 or more episodes in the 2 years preceding enrollment
- Recurrent priapism: 2 or more episodes in the 2 years preceding enrollment
- History of osteomyelitis or osteonecrosis
- Cerebrovascular disease:
- Imaging evidence of prior overt or silent stroke
- History of a neurologic event resulting in focal neurologic deficits lasting \> 24 hours
- Abnormal transcranial Doppler: Timed average maximum mean velocity ≥ 200 cm/sec in terminal portion of the carotid or proximal portion of the middle cerebral artery or \> 185 cm/sec plus evidence of intracranial...
- Pulmonary hypertension: Confirmed by right heart catheterization with mean pulmonary arterial pressure ≥ 25 mmHg or mean pulmonary vascular resistance \> 2 Wood units
- Red blood cell alloimmunization (\> 3 alloantibodies)
- For thalassemia patients: Any genotype, with all of the following:
- Onset of red blood cell transfusion dependence during the first 3 years of life
- RBC transfusion history \> 225 mL/kg/year or \> 15 lifetime RBC transfusions
- Pre-transfusion hemoglobin ≤ 7 g/dL
- Hepatosplenomegaly
- Patient or the patient's legal representative, parent(s) or guardian should be able to provide written informed consent. Assent of a minor if participant's age is at least seven and less than eighteen years.
- For sexually active men and women of childbearing potential, must agree to use a form of contraception considered effective and medically acceptable by the Investigator.
- Prior myeloablative allogeneic HCT.
- Overt stroke or CNS instrumentation (e.g. for Moyamoya disease) within 6 months of enrollment.
- Liver cirrhosis. Mild fibrosis will be permitted, i.e. fine reticulin or grade 1 of 4, with bridging fibrosis.
- Hepatic iron content ≥ 3 mg Fe/g liver dry weight, if applicable
- Active hepatitis B or C.
- Other uncontrolled infections.
- Other malignancy/cancer diagnosis unless in remission after definitive therapy for a minimum of 2 years. Exceptions: Ductal carcinoma in situ, basal cell carcinoma, cervical intraepithelial neoplasia.
- Positive pregnancy test in a woman with child-bearing potential, defined as not post-menopausal for 12 months or no previous surgical sterilization.
- Inability to comply with medical therapy or follow-up.
- Known history of allergic reactions to any constituents of the stem cell product, including a known history of allergic reactions to DMSO.
The study team makes the final eligibility decision.
Where it's taking place
- Basking Ridge, New Jersey, United States
- Middletown, New Jersey, United States
- Montvale, New Jersey, United States
- Commack, New York, United States
- Harrison, New York, United States
- Rockville Centre, New York, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 2 years to 50 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Basking Ridge, New Jersey, United States; Middletown, New Jersey, United States; Montvale, New Jersey, United States; Commack, New York, United States; Harrison, New York, United States; Rockville Centre, New York, United States. Enter your location above to see the nearest site and check your eligibility.
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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.