Ruxolitinib as GVHD Prophylaxis in AA Patients

Ruxolitinib as Graft-versus-Host Disease Prophylaxis for Allogeneic Stem Cell Transplantation in Aplastic Anemia Patients

Our study aimed to determine the prophylactic value of ruxolitinib for aGVHD in HSCT recipients of AA.Ruxolitinib was initiated at the beginning of conditioning regimen at 5 twice daily until 3 months post-transplantation. The cumulative incidence of aGVHD within 6 months after HSCT will be the primary observation item.

Study Overview

• Status: Not yet recruiting
• Conditions: The Prophylactic Value of Ruxolitinib for aGVHD in HSCT Recipients of AA
• Intervention / Treatment: Drug: Ruxolitinib 5 MG BID Oral Tablet

• Study Type: Interventional
• Enrollment (Estimated): 46
• Phase: Phase 2

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• (1) diagnosed as aplasitic anemia, including V/SAA,CAA and HAAA. (2) Age elder than or equal to 14 years; (3) Informed consent may be signed by themselves. (4) HIV negative, HBV, HCV negative; (5) Informed consent must be signed before the start of the study procedures, and informed consent must be signed by the patient himself or his immediate family. Considering the patient 's condition, if the patient' s signature is unfavorable for disease treatment, the informed consent form should be signed by the legal guardian or the patient 's immediate family member

Exclusion Criteria:

• (1) received a previous allogeneic HSCT; (2) Uncontrolled infection at enrollment; requires mechanical ventilation or is hemodynamically unstable at the time of enrollment; (3) has severe hepatic insufficiency (defined as Child-Pugh Class C; has serum aspartate aminotransferase (AST) or alanine aminotransferase (ALT) > 5 xthe upper limit of normal (ULN) or serum total bilirubin > 2.5 x ULN.

  (4) has end-stage renal impairment with a creatinine clearance less than 10 mL/min.

  (5) has both moderate hepatic insufficiency AND moderate renal insufficiency; (6) has documented positive results for human immunodeficiency virus antibody (HIVAb), hepatitis C virus antibody (HCV-Ab) with detectable HCV RNA, or hepatitis B surface antigen (HBsAg) within 90 days prior to enrollments; (7) has active solid tumor malignancies with the exception of localized basal cell or squamous cell skin cancer or the condition under treatment (e.g., lymphomas).

  (8) Suffering from mental disorders or other conditions and unable to cooperate with the requirements of study treatment and monitoring; (9) unable or unwilling to sign the consent form; (10) patients with other special conditions assessed as unqualified by the investigator

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: treatment arm	Drug: Ruxolitinib 5 MG BID Oral Tablet; ruxolitinib 5mgBID will be administrated at the beginning of the conditioning regimen until 3 months post-transplantation.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
cumulative incidence of aGVHD	cumulative incidence of aGVHD within 6 months post-HSCT	6 months post-HSCT

Collaborators and Investigators

• Sponsor: Institute of Hematology & Blood Diseases Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): July 1, 2023
• Primary Completion (Estimated): May 1, 2025
• Study Completion (Estimated): May 1, 2025

Study Registration Dates

• First Submitted: June 13, 2023
• First Submitted That Met QC Criteria: June 13, 2023
• First Posted (Actual): June 22, 2023

Study Record Updates

• Last Update Posted (Actual): June 22, 2023
• Last Update Submitted That Met QC Criteria: June 13, 2023
• Last Verified: May 1, 2023

More Information

Terms related to this study

• Other Study ID Numbers: IIT2023011-EC-2

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No