Thymalin
Thymulin, Thymic Factor, Serum Thymic Factor, Facteur Thymique Serique
Recruiting
PHASE2
INTERVENTIONAL
NCT03579875
Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders
View on ClinicalTrials.gov
Updated Dec 15, 2025
Brief Summary
This is a phase II trial of T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation in patients with inherited bone marrow failure (BMF) disorders to eliminate the need for routine graft-versus-host disease (GVHD) immune suppression leading to earlier immune recovery and potentially a reduction in the risk of severe infections after transplantation.
Interventions
Name:
Total Body Irradiation (TBI) (Plan 1)
Type:
DRUG
Description:
300 cGy with thymic shielding on day -6
Name:
Cyclophosphamide (CY) (Plan 1)
Type:
DRUG
Description:
10 mg/kg IV daily on days -5, -4, -3, and -2
Name:
Fludarabine (FLU)
Type:
DRUG
Description:
35 mg/m2 IV daily on days -5, -4, -3, and -2
Name:
Methylprednisolone (MP)
Type:
DRUG
Description:
1 mg/kg IV q12h on days -5, -4, -3, -2, and -1
Name:
Donor mobilized PBSC infusion
Type:
DEVICE
Description:
T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation on day 0
Name:
G-CSF
Type:
DRUG
Description:
Initiate G-CSF 5mcg/kg per day IV on day +1 (continue until ANC \>2.5 x 10\^9/L for 3 consecutive days or single day ANC \>3000 Arm 1 and Arm 3)
Name:
Cyclophosphamide (CY) (Plan 2)
Type:
DRUG
Description:
5 mg/kg IV daily on days -5, -4, -3, and -2
Name:
Rituximab
Type:
DRUG
Description:
200 mg/m2 IV once on day -1
Name:
Busulfan
Type:
DRUG
Description:
Busulfan 0.6 mg/kg if \> 4 years old and/or \>12 kg (0.8 mg/kg IV if ≤ 4 years old and/or ≤ 12 kg) is given IV over 2 hours every 12 hours for 2 days.
Name:
Alemtuzumab
Type:
DRUG
Description:
Alemtuzumab 0.2 mg/kg is given IV over 2 hours daily for 5 days (total dose 1 mg/kg)
Name:
Melphalan
Type:
DRUG
Description:
If available, MEL dosing will be model-based using Bayesian methodology. If Bayesian methodology is unavailable, MEL dosing will be weight-based: MEL 70 mg/m2 for patients ≥10 kg (2.35 mg/kg for patients \<10 kg\^) IV for one dose over 30 minutes.
Name:
Rituximab
Type:
DRUG
Description:
Rituximab will be given once on treatment plans 1-3 on day -1.
Primary Outcomes
Measure:
Grade II-IV acute graft versus host disease (GVHD)
TimeFrame:
Day 100
Description:
incidence of grade II-IV acute graft versus host disease (GVHD)
Trial Information
NCT ID
NCT03579875
Status
Recruiting
Study Type
INTERVENTIONAL
Phases
PHASE2
Sponsor
Masonic Cancer Center, University of Minnesota
Last Updated
December 15, 2025