CI

At a glance

ClinicalIndex Comparison Record
Phase 2Active· 71 enrolled
Drug / intervention
e-ATG +16 moredrug
Likely dose
GVHD Prophylaxis 50 mg/kgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT03663933
NCT03663933Phase 2Active

Phase II Trial of Allogeneic Hematopoietic Cell Transplantation for Disorders of T-cell Proliferation and/or Dysregulation

National Cancer Institute (NCI)·interventional·Posted Sep 10, 2018·Updated Mar 17, 2026

In Brief

A Phase 2 clinical trial evaluating e-ATG, Immunosuppression Only Conditioning, and 15 other interventions for Lymphoproliferative Disorders and 4 related conditions. Active but no longer recruiting, targeting 71 participants across 2 sites.

Detailed Summary

Background: Blood stem cells in the bone marrow make all the cells to normally defend a body against disease. Allogeneic blood or marrow transplant is when these stem cells are transferred from one person to another. Researchers think this treatment can provide a new, healthy immune system to correct T-cell problems in some people. Objective: To see if allogeneic blood or bone marrow transplant is safe and effective in treating people with T-cell problems. Eligibility: Donors: Healthy people ages 4 and older Recipients: People the same age with abnormal T-cell function causing health problems Design: All participants will be screened with: * Medical history * Physical exam * Blood, heart, and urine tests Donors will also have an electrocardiogram and chest x-ray. They may have veins tested or a pre-anesthesia test. Recipients will also have lung tests. Some participants will have scans and/or bone marrow collected by needle in the hip bones. Donors will learn about medicines and activities to avoid and repeat some screening tests. Some donors will stay in the hospital overnight and have bone marrow collected with anesthesia. Other donors will get shots for several days to stimulate cells. They will have blood removed by plastic tube (IV) in an arm vein. A machine will remove stem cells and return the rest of the blood to the other arm. Recipients will have: * More bone marrow and a small fragment of bone removed * Dental, diet, and social worker consultations * Scans * Chemotherapy and antibody therapy for 2 weeks * Catheter inserted in a chest or neck vein to receive donor stem cells * A hospital stay for several weeks with more medicines and procedures * Multiple follow-up visits

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
CountriesUnited States
Collaborators--

Timeline

Phase 2Active
201920202021202220232024202520262027202820292030
First PostedSep 10, 2018
Enrollment StartSep 4, 2018
Primary CompletionApr 3, 2025
Study CompletionApr 3, 2030
TodayJul 2, 2026
Enrollment to primary: 6.6 yearsPosted 7.8 years ago

Interventions

e-ATGdrug

During Immunosuppression Only Conditioning (IOC) and Reduced Intensity Conditioning (RIC).

Immunosuppression Only Conditioningprocedure

Equine anti-thymocyte globulin (e-ATG) 40 mg/kg intravenous (IV) once daily for days -14 and -13. Prednisone: Tapering doses, given orally daily, and given prior to each daily dose of e-ATG on days -14 and -13, Pentostatin:4 mg/m\^2/day IV on days -9 and -5, cyclophosphamide:5 mg/kg orally daily on days -9 through -2.

Reduced Intensity Conditioningprocedure

Equine anti-thymocyte globulin (e-ATG) 40 mg/kg intravenous (IV) once daily for days -14 and -13. Prednisone: Tapering doses, given orally daily, and given prior to each daily dose of e-ATG on days -14 and -13, Pentostatin:4 mg/m\^2/day IV on days -11 and -7, cyclophosphamide: 5 mg/kg orally daily on days -11 through -4, Busulfan IV, pharmacokinetically dosed, on days -3 and -2.

GVHD Prophylaxisdrug

High-dose, post-transplantation cyclophosphamide (PTCy) 25-50 mg/kg on days +3 and +4, Mesna: 25-50 mg/kg weight-based dosing, Tacrolimus 0.02 mg/kg on days +5 through +90, and mycophenolate mofetil (MMF) 15 mg/kg on days +5 through +25.

Allogeneic HSCprocedure

Stem cell transplant

Bisulfandrug

During Reduced Intensity Conditioning (RIC).

Prednisonedrug

During Immunosuppression Only Conditioning (IOC) and Reduced Intensity Conditioning (RIC).

Cyclophosphamidedrug

During Immunosuppression Only Conditioning (IOC), Reduced Intensity Conditioning (RIC) and Graft-versus-host disease prophylaxis (GVHD).

MMFdrug

During Graft-versus-host disease prophylaxis (GVHD).

Mesnadrug

During Graft-versus-host disease prophylaxis (GVHD).

Tacrolimusdrug

During Graft-versus-host disease prophylaxis (GVHD).

Pentostatindrug

During Immunosuppression Only Conditioning (IOC) and Reduced Intensity Conditioning (RIC).

PFTsother

Screening ≤4 weeks pretreatment (rx), Day +180 (≤ 14 days), Day +36 (± 21 days), Day +548 (18 months) (± 28 days), and at 2 years and yearly thereafter through +5 years (± 56 days).

DEXAother

Baseline, Day +365 (± 21 days), at 2 years and yearly thereafter through +5 years (± 56 days), and as clinically indicated after hematopoietic cell transplant (HCT).

Bone Marrow Aspirate & Biopsyprocedure

Baseline, Day +60 (± 3 days) and Day +365 (±21 days).

EKGother

Baseline

2D ECHOother

Screening ≤4 weeks pretreatment (rx), Day +180 (≤ 14 days), Day +36 (± 21 days), Day +548 (18 months) (± 28 days), and at 2 years and yearly thereafter through +5 years (± 56 days).