Graft Versus Host DiseasePhase 1/2T-REGNCT01453140

In-vivo Regulatory T Cell Enhancement With Cyclophosphamide and Sirolimus

A Phase 1/2 study testing Cyclophosphamide and Sirolimus, Low dose IL-2 with Cytoxan + Sirolimus and Low dose IL-2, low dose Vidaza, cyclophosphamide & Sirolimus in people with Graft Versus Host Disease, run by Hackensack Meridian Health at 1 site in United States.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record

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Trial summary

In this study the investigators are proposing to treat patients with steroid-refractory Graft-versus-host Disease (GVHD) stabilization using IL-2 and azacitidine Read more about this study

Who is it for?: Ages 18 to 70 years
Conditions the study is for: Graft Versus Host Disease
What is being tested?: Cyclophosphamide and Sirolimus, Low dose IL-2 with Cytoxan + Sirolimus and Low dose IL-2, low dose Vidaza, cyclophosphamide & Sirolimus
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 3 participants
Where: 1 siteUnited States
Study dates: Aug 2011to Jul 2012
Phase: Phase 1/2

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

Ages 18 to 70 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Patients must have a documented clinical diagnosis of grade II-IV acute graft-versus- host disease defined as graft versus host disease (GVHD) occurring within the first 100 days of transplantation
Patients must be steroid-refractory defines as progression after 3 days of corticosteroid therapy or no response after 5 days of corticosteroid therapy.
Progression is defined as up-grading
No response is defined as no down-grading
Progression after 3 days requires patients to have received at least 2 mg/mg/day for a total of 6 mg/kg of methylprednisolone or its equivalent.
No response after 5 days requires patient to have received at least 2 mg/kg/d for a total of 10 mg/kg of methylprednisolone or its equivalent.

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Patients with exacerbation of GVHD during steroid taper will require re-treatment with 2mg/kg/d of corticosteroids and will need to meet the criteria
Age 18-70
Patients must have received an allogeneic hematopoietic stem cell transplant within 100 days of study enrollment.
Serum creatinine < 2 mg/dL

Who cannot join

Patients cannot have active central nervous system (CNS) disease.
Patients must not have received cyclophosphamide for GVHD prophylaxis
Patients must not have pneumonia requiring oxygen supplementation
Unable or unwilling to sign informed consent.

Trial locations and contacts

1 site in 1 country.

United States1 site

John Theurer Cancer Center at Hackensack University Medical Center
Hackensack, New Jersey, 07601

Other trials in United States

About this study

Official title: Phase I- II Study of in Vivo Regulatory T Cell Enhancement With Cyclophosphamide and Sirolimus With or Without Vidaza (Azacitidine) for the Treatment of Steroid-refractory Acute Graft-versus-host Disease

In this study the investigators are proposing to treat patients with steroid-refractory Graft-versus-host Disease (GVHD) stabilization using IL-2 and azacitidine

Read the detailed description

High-dose cyclophosphamide and sirolimus have been successfully used for the prevention of Graft-versus-host Disease (GVHD) and have shown to enhance the Tregs subpopulation. The addition of low dose IL-2 and a demethylating agent such as azacitidine will also be studied in an attempt to promote and stabilize the FoxP3 expression of Tregs.

Conditions: Graft Versus Host Disease
Being tested: Cyclophosphamide and Sirolimus, Low dose IL-2 with Cytoxan + Sirolimus, Low dose IL-2, low dose Vidaza, cyclophosphamide & Sirolimus, Low dose IL-2, Vidaza, Cytoxan & Sirolimus
Medical subject headings: Graft vs Host Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Cyclophosphamide and SirolimusDrug
Also called Cytoxan
On the first day of treatment, cyclophosphamide will be administered at a dose of 4g/m2 IV x 1 dose. Patients who are >40% above ideal weight will be dosed based on adjusted weight and adjusted BSA.
One day after the administration of cyclophosphamide, patients will receive sirolimus 6 mg PO x 1 and on the following day will start sirolimus at a dose of 2 mg PO daily.
Low dose IL-2 with Cytoxan + SirolimusDrug
Also called Interleukin-2, Cytoxan
Patients in treatment arm B will be receiving low-dose IL-2 in conjunction with the cyclophosphamide and sirolimus.
IL-2 will be administered at a dose of 0.5E6 IU/m2 SQ daily x 8 weeks followed by 4 weeks off, starting 14 days after the cyclophosphamide.
Low dose IL-2, low dose Vidaza, cyclophosphamide & SirolimusDrug
Also called Interleukin-2, Azactidine, Cytoxan
Patients in treatment arm C will be receiving low-dose azacitidine (Vidaza). The Vidaza will be initiated between day 27 and 32 following the cyclophosphamide.
The dose administered will be 10 mg SQ daily for 5 days followed by 3 weeks off.
Cyclophosphamide and SirolimusDrug
Also called Other names: Cytoxan
Day 1: Cyclophosphamide Day 2: Sirolimus
Low dose IL-2 with Cytoxan + SirolimusDrug
Also called Other names:, Interleukin-2, Cytoxan
treatment arm B will be receiving low-dose IL-2 in conjunction with the cyclophosphamide and sirolimus
Low dose IL-2, Vidaza, Cytoxan & SirolimusDrug
Also called Other names:, Interleukin-2, Azactidine, Cytoxan
Vidaza will be initiated between day 27 and 32 following the cyclophosphamide.

Study groups in NCT01453140
Study group	Role	What they receive
Cyclophosphamide and Sirolimus	Experimental	cyclophosphamide and sirolimus combo Receives: Drug: Cyclophosphamide and Sirolimus
Lowdose IL-2, Cytoxan + Sirolimus	Experimental	Low dose IL-2 with Cytoxan + Sirolimus Patients in treatment arm B will be receiving low-dose IL-2 in conjunction with the cyclophosphamide and sirolimus. Receives: Drug: Low dose IL-2 with Cytoxan + Sirolimus
Lowdose IL-2, Vidaza, cyclophosphamide & Sirolimus	Experimental	Lowdose IL-2, Vidaza, cyclophosphamide (Cytoxan) \& Sirolimus Patients in treatment arm C will be receiving low-dose azacitidine (Vidaza). Receives: Drug: Low dose IL-2, low dose Vidaza, cyclophosphamide & Sirolimus; Drug: Low dose IL-2, Vidaza, Cytoxan & Sirolimus

What is being measured

The primary outcome is the main question the study is designed to answer.

Response Rate of Patients With Steroid-refractory Graft-versus-host Disease (GVHD) Using Cyclophospahmide and Sirolimus Combined With 3 Variations of Low-dose IL 2 and Low-dose Vidaza.
The primary objective of this study is to determine the response rate of patients treated steroid-refractory graft-versus-host disease (GVHD) using cyclophospahmide and sirolimus combined with 3 variations of low-dose IL 2 and low-dose Vidaza with an outcome goal of promoting CD4+CD25+FoxP3+ Tregs.
Measured: 28 days to 100 days post transplant

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

Aug 2011Jul 2012

First posted17 Oct 2011 (estimated)
Study startAug 2011
Primary completionMar 2012
Study completionJul 2012

Why it stopped: Dose Limiting Toxicities

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Graft Versus Host DiseaseStudies of the same condition, open and under way.
What the trial phases meanHow a treatment moves from first tests in people to approval.

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design

Study type: Interventional
Phase: Phase 1/2
Primary purpose: Treatment
Allocation: Non-randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 3 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01453140
Sponsor's protocol ID: Pro00002219
Acronym: T-REG
Status last verified: Mar 2026
First submitted: 13 Oct 2011
Last updated: 8 Apr 2026
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

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