NCT05249452Completed
Adding Azathioprine/Hydroxyurea Preconditioning to Alemtuzumab/TBI to Reduce Risk of Graft Failure in MSD HSCT in Adult SCD Patients
Adding Azathioprine/Hydroxyurea Preconditioning to Alemtuzumab/TBI to Reduce Risk of Graft Failure in Matched Sibling Donor Allogeneic HSCT in Adult Sickle Cell Patients
Source: ClinicalTrials.gov · Last updated 2 Oct 2025
Brief summary (registry, in English)
In this study the investigators will prospectively investigate whether the addition of a 3-months long preconditioning with azathioprine to the alemtuzumab/TBI non-myeloablative conditioning results in improved disease-free survival and donor chimerism after allo-SCT in SCD patients. Furthermore, the investigators will evaluate whether azathioprine/hydroxyurea preconditioning leads to more patients being able to taper and discontinue sirolimus at 12 months post-transplantation.
- Status (registry)
- Completed
- Study type
- Observational
- Enrollment
- 20 (actual)
- First submitted
- 6 Jan 2022
- Start date
- 8 Mar 2018
- Primary completion date
- 1 Dec 2023
- Lead sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
- Diseases
- Sickle cell disease
- NCT number
- NCT05249452
What you commit to
- Study type: Observational
- Participants enrolled: 20
- From 8 Mar 2018 to the primary completion date, 1 Dec 2023
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Patients who have any debilitating medical or psychiatric illness that would preclude their giving informed consent or their receiving optimal treatment and follow-up. However, patients with history of stroke and significant cognitive deficit, that would preclude giving informed consent or assent will not be excluded, if they have a family member or significant other with Power of Attorney to also consent of their behalf.
Other illness
- HIV-positive
Lab values
- Poor cardiac function: left ventricular ejection fraction\<35%.
- Poor liver function: direct bilirubin \>3.1 mg/dl
Other
- Poor performance status (ECOG\>1).
- Poor pulmonary function: FEV1 and FVC\<40% predicted.
- Women of childbearing potential who currently are pregnant (Beta-HCG+) or who are not practicing adequate contraception.
Who the study is looking for
- SCD patients with an HLA-identical matched sibling donor eligible for allogeneic stem cell 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 and donating hematopoietic stem cells respectively. The sibling donor should be willing to donate.
- Patients must be geographically accessible and willing to participate in all stages of treatment.
- Eligible diagnoses: Patients with sickle cell disease such as sickle cell anemia (Hb SS), Hb/Sβ0-thalassemia, Hb/Sβ+-thalassemia, HbSC disease, HbSE disease, HbSD disease and Hemoglobin SO- Arab disease.
Age in the registry: 16 years to 60 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Save your city in your profile to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Amsterdam Medical Centre Coordinator of this site? Confirm its status | Amsterdam | Netherlands | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.