NCT03579875

Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders

MT2017-17:T Cell Receptor Alpha/Beta T Cell Depleted Hematopoietic Cell Transplantation in Patients With Inherited Bone Marrow Failure (BMF) Disorders

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 48 (estimated)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 65 Years
• Healthy volunteers: Not accepted

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.

Conditions

• Fanconi Anemia
• Severe Aplastic Anemia
• Myelodysplastic Syndromes
• T Cell Receptor Alpha/Beta Depletion
• Telomere Biology Disorder
• Bone Marrow Failure
• Dyskeratosis Congenita
• Telomere Biology Disorders

Interventions

Type	Name	Description
DRUG	Total Body Irradiation (TBI) (Plan 1)	300 cGy with thymic shielding on day -6
DRUG	Cyclophosphamide (CY) (Plan 1)	10 mg/kg IV daily on days -5, -4, -3, and -2
DRUG	Fludarabine (FLU)	35 mg/m2 IV daily on days -5, -4, -3, and -2
DRUG	Methylprednisolone (MP)	1 mg/kg IV q12h on days -5, -4, -3, -2, and -1
DEVICE	Donor mobilized PBSC infusion	T cell receptor alpha/beta depletion (α/β TCD) peripheral blood stem cell (PBSC) transplantation on day 0
DRUG	G-CSF	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)
DRUG	Cyclophosphamide (CY) (Plan 2)	5 mg/kg IV daily on days -5, -4, -3, and -2
DRUG	Rituximab	200 mg/m2 IV once on day -1
DRUG	Busulfan	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.
DRUG	Alemtuzumab	Alemtuzumab 0.2 mg/kg is given IV over 2 hours daily for 5 days (total dose 1 mg/kg)
DRUG	Melphalan	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.
DRUG	Rituximab	Rituximab will be given once on treatment plans 1-3 on day -1.

Timeline

• Start date: 2018-11-13
• Primary completion: 2027-01-01
• Completion: 2029-01-05
• First posted: 2018-07-09
• Last updated: 2026-01-30

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated device study