Last updated: June 7, 2023
Sponsor: Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Overall Status: Active - Recruiting
Phase
N/A
Condition
Sickle Cell Disease
Red Blood Cell Disorders
Treatment
Preconditioning with azathiprine and hydroxyurea (3 months)
Clinical Study ID
NCT05249452
W21_160
Ages 16-60 All Genders
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
- SCD patients with an HLA-identical matched sibling donor eligible for allogeneic stemcell transplantation.
- Age 16 - 60 years
- Good performance status (ECOG 0 or 1; Karnofsky and Lansky 70-100)
- Patients and donors (MSD) must be able to sign consent forms for receiving anddonating hematopoietic stem cells respectively. The sibling donor should be willing todonate.
- Patients must be geographically accessible and willing to participate in all stages oftreatment.
- Eligible diagnoses: Patients with sickle cell disease such as sickle cell anemia (HbSS), Hb/Sβ0-thalassemia, Hb/Sβ+-thalassemia, HbSC disease, HbSE disease, HbSD diseaseand Hemoglobin SO- Arab disease.
Exclusion
Exclusion Criteria:
- Poor performance status (ECOG>1).
- Poor cardiac function: left ventricular ejection fraction<35%.
- Poor pulmonary function: FEV1 and FVC<40% predicted.
- Poor liver function: direct bilirubin >3.1 mg/dl
- HIV-positive
- Women of childbearing potential who currently are pregnant (Beta-HCG+) or who are notpracticing adequate contraception.
- Patients who have any debilitating medical or psychiatric illness that would precludetheir giving informed consent or their receiving optimal treatment and follow-up.However, patients with history of stroke and significant cognitive deficit, that wouldpreclude giving informed consent or assent will not be excluded, if they have a familymember or significant other with Power of Attorney to also consent of their behalf.
Study Design
Total Participants: 20
Treatment Group(s): 1
Primary Treatment: Preconditioning with azathiprine and hydroxyurea (3 months)
Phase:
Study Start date:
March 08, 2018
Estimated Completion Date:
December 01, 2023
Study Description
Connect with a study center
Amsterdam Medical Centre
Amsterdam,
NetherlandsActive - Recruiting
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