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Prolonged Isolated Thrombocytopenia After Allo-SCT : N-acetyl-L-cysteine (NAC) Versus Supportive Therapy

Primary Purpose

Thrombocytopenia, Stem Cell Transplant Complications

Status
Terminated
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
N-acetyl-L-cysteine (NAC)
supportive therapy
Sponsored by
Peking University People's Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Thrombocytopenia

Eligibility Criteria

14 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • age>= 14 years
  • diagnosed as PT (defined as platelet count in peripheral blood never achieve 20000/miro-liter for 7 consecutive days without transfusion at day 60 post transplantation)
  • serum creatine level < ULN (upper limit of normal), serum ALT/AST /TBil<=2 ULN
  • without active CMV/EBV/ADV reactivation, active GVHD, without relapse or minimal residual disease at enrollment

Exclusion Criteria:

  • history of asthma
  • allergy to NAC
  • refuse to participate

Sites / Locations

  • Peking University People's Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Other

Arm Label

intervention arm

controlled arm

Arm Description

NAC 400mg p.o tid from day 60 to day 90 post transplant

Supportive therapy including platelet infusion:prophylactic platelet transfusion was given when platelet count <20000/ul

Outcomes

Primary Outcome Measures

response rate
rate of response after 30 days of treatment

Secondary Outcome Measures

overall survival
survival proportion at the last followup after transplantation
non-relapse mortality
non-relapse mortality at the last followup after transplantation
grade 2-4 acute graft versus host disease
grade 2-4 aGVHD at day 100 after transplant
chronic graft versus host disease
chronic graft versus host disease at the day of last follow-up

Full Information

First Posted
January 2, 2018
Last Updated
September 23, 2020
Sponsor
Peking University People's Hospital
Collaborators
The First Affiliated Hospital of Soochow University, Nanfang Hospital, Southern Medical University
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1. Study Identification

Unique Protocol Identification Number
NCT03391856
Brief Title
Prolonged Isolated Thrombocytopenia After Allo-SCT : N-acetyl-L-cysteine (NAC) Versus Supportive Therapy
Official Title
A Prospective Randomized Controlled Study for Intervention of Prolonged Isolated Thrombocytopenia After Allogeneic Hematopoietic Stem Cell Transplantation: N-acetyl-L-cysteine (NAC) Versus Supportive Therapy
Study Type
Interventional

2. Study Status

Record Verification Date
September 2020
Overall Recruitment Status
Terminated
Why Stopped
protocol changed
Study Start Date
March 1, 2018 (Actual)
Primary Completion Date
December 30, 2018 (Actual)
Study Completion Date
December 30, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Peking University People's Hospital
Collaborators
The First Affiliated Hospital of Soochow University, Nanfang Hospital, Southern Medical University

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
Isolated prolonged thrombocytopenia (PT) is a common complication after allogeneic stem cell transplantation with significant poor prognosis. No standard treatment is available. The current study assigned PT randomly to 2 arms: intervention arm with N-acetyl-L-cysteine (NAC) and control arm with supportive therapy.This is a prospective randomized controlled study.
Detailed Description
patients diagnosed with PT at day 60 post transplant will be randomized assigned to intervention arm (NAC) or controlled arm (supportive therapy: prophylactic platelet transfusion was given when platelet count <20000/ul.) Response will be evaluated at day 90. Response was defined as platelet recovery to >= 20000/ul for 7 consecutive days without transfusion support during the enrollment period. All the other patients not achieved above criteria was defined as no response. For those without response in both arms, patients will received NAC plus recombinant human thrombopoietin (rhTPO) for an additional 30 days. rhTPO was given at 300IU/kg/d for 28 consecutive days or platelet > 50000/ul independent of platelet transfusion.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Thrombocytopenia, Stem Cell Transplant Complications

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
15 (Actual)

8. Arms, Groups, and Interventions

Arm Title
intervention arm
Arm Type
Experimental
Arm Description
NAC 400mg p.o tid from day 60 to day 90 post transplant
Arm Title
controlled arm
Arm Type
Other
Arm Description
Supportive therapy including platelet infusion:prophylactic platelet transfusion was given when platelet count <20000/ul
Intervention Type
Drug
Intervention Name(s)
N-acetyl-L-cysteine (NAC)
Intervention Description
NAC treatment for PT: 400mg p.o tid from day 60 to 90 post transplant
Intervention Type
Other
Intervention Name(s)
supportive therapy
Intervention Description
prophylactic platelet transfusion was given when platelet count <20000/ul
Primary Outcome Measure Information:
Title
response rate
Description
rate of response after 30 days of treatment
Time Frame
30 days after the start of enrollement
Secondary Outcome Measure Information:
Title
overall survival
Description
survival proportion at the last followup after transplantation
Time Frame
the day of last follow-up
Title
non-relapse mortality
Description
non-relapse mortality at the last followup after transplantation
Time Frame
the day of last follow-up
Title
grade 2-4 acute graft versus host disease
Description
grade 2-4 aGVHD at day 100 after transplant
Time Frame
day 100 after transplant
Title
chronic graft versus host disease
Description
chronic graft versus host disease at the day of last follow-up
Time Frame
the day of last follow-up

10. Eligibility

Sex
All
Minimum Age & Unit of Time
14 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: age>= 14 years diagnosed as PT (defined as platelet count in peripheral blood never achieve 20000/miro-liter for 7 consecutive days without transfusion at day 60 post transplantation) serum creatine level < ULN (upper limit of normal), serum ALT/AST /TBil<=2 ULN without active CMV/EBV/ADV reactivation, active GVHD, without relapse or minimal residual disease at enrollment Exclusion Criteria: history of asthma allergy to NAC refuse to participate
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Xiao-Jun Huang, MD
Organizational Affiliation
Peiking University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Peking University People's Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100044
Country
China

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

Prolonged Isolated Thrombocytopenia After Allo-SCT : N-acetyl-L-cysteine (NAC) Versus Supportive Therapy

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