NCT01379209

Intravenous Administration of RGI-2001 in Patient Undergoing Allogenic Hematopoietic Stem Cell Transplantation (AHSCT)

A Phase 1/2a, Open-Label, Multicenter, Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravenous Administration of RGI-2001 in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation (AHSCT)

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 68 (actual)
• Sponsor: Regimmune Corporation · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The clinical trial is a Phase 1/2a, open-label, multi-center, dose-escalation study to evaluate the safety, tolerability and pharmacokinetic profile of RGI-2001 in patients undergoing AHSCT, with radiation or non-radiation myeloablative preparative treatment. The study will be separated into two parts; a dose escalation phase to assess safety, followed by a large expansion phase to further evaluate the pharmacologic effects of either a Maximum Tolerated Dose, Maximum Feasible Dose or optimal pharmacologically active dose of RGI-2001. The initial dose escalation safety portion of the study (Part 1) will include higher risk patients and limit the unrelated donor transplants. After safety is established in part 1 of the study, the second portion of the study will expand the enrollment criteria and allow transplantation by either related or unrelated donors. This study will endeavor to identify the dose range at which RGI-2001 has an acceptable safety profile, at which biologic activity is observed, and to guide possible dose levels to utilize in later phase studies based on biological activity.

Detailed description

The clinical trial is a Phase 1/2a, open-label, multi-center, dose-escalation study to evaluate the safety, tolerability and pharmacokinetic profile of RGI-2001 in patients undergoing AHSCT, with radiation or non-radiation myeloablative preparative treatment. In Part 1 (Phase 1: Dose Escalation Phase), patients will receive a single intravenous administration of RGI-2001 approximately 30 minutes after completion of the transplant (either allogeneic PBSCs or allogenic bone marrow transplantation (unmodified)) with the dosage based upon the assigned treatment cohort. Eligible patients will be enrolled in five to seven centers in the United States. Patients who are undergoing AHSCT will be enrolled in a sequential group dose-escalating fashion to determine the safety, tolerability, pharmacokinetic profile, and the MTD or MFD of RGI-2001. It is anticipated that up to six dose levels will be evaluated in Part 1, with an option for an additional cohort (Cohort 7) if the MTD is not reached and pharmacodynamic markers suggest higher doses are warranted. In Part 2 (Expansion Phase), one or more doses below the MTD or MFD will be selected based on a potential correlation between GvHD and biological activity to further assess safety and biologic activity. Approximately 30 patients who are undergoing either allogeneic PBSCs or allogenic bone marrow transplantation (unmodified) will be enrolled in Part 2 of the study. Patients will be monitored for safety for 29 days after the transplant procedure. All patients will be followed for 100 days following transplant procedure for the incidence of acute GvHD, according to the Modified Keystone Criteria for grading acute GvHD (Przepiorka D, et al)

Conditions

• Graft Versus Host Disease

Interventions

Type	Name	Description
DRUG	RGI-2001	A single administration of RGI-2001 on Day 0 post AHSCT.
DRUG	Calcineurin Inhibitors	GVHD prophylaxis according to institutional guidelines. Subjects could have received any number/combinations of treatments.
PROCEDURE	Allogeneic Hematopoietic Stem Cell Transplantation	According to institutional guidelines.
DRUG	Conditioning Regimen	Myeloablative preparative treatment according to institutional guidelines. Subjects could have received any number/combinations of treatments.
PROCEDURE	Allogeneic Bone Marrow Transplantation	According to institutional guidelines
DRUG	Methotrexate
DRUG	Mofetil Mycophenolate
DRUG	sirolimus	Administered for GVHD prophylaxis as per institutional guidelines

Timeline

• Start date: 2011-09-01
• Primary completion: 2015-06-01
• Completion: 2017-04-01
• First posted: 2011-06-23
• Last updated: 2019-10-23

Locations

6 sites across 1 country: United States