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

Thrombocytopenia Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT03391856
• Other study ID #: 2017PHB220
• Status: Terminated
• Phase: Phase 2/Phase 3
• Start date: March 1, 2018
• Est. completion date: December 30, 2018

Study information

• Verified date: September 2020
• Source: Peking University People's Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

1. 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.)

2. 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.

3. 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 15
• Est. completion date: December 30, 2018
• Est. primary completion date: December 30, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 14 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Stem Cell Transplant Complications
• Thrombocytopenia

Intervention

Drug:
• N-acetyl-L-cysteine (NAC)
NAC treatment for PT: 400mg p.o tid from day 60 to 90 post transplant

Other:
• supportive therapy
prophylactic platelet transfusion was given when platelet count <20000/ul

Locations

Country	Name	City	State
China	Peking University People's Hospital	Beijing	Beijing

Sponsors (3)

Lead Sponsor	Collaborator
Peking University People's Hospital	Nanfang Hospital of Southern Medical University, The First Affiliated Hospital of Soochow University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	response rate	rate of response after 30 days of treatment	30 days after the start of enrollement
Secondary	overall survival	survival proportion at the last followup after transplantation	the day of last follow-up
Secondary	non-relapse mortality	non-relapse mortality at the last followup after transplantation	the day of last follow-up
Secondary	grade 2-4 acute graft versus host disease	grade 2-4 aGVHD at day 100 after transplant	day 100 after transplant
Secondary	chronic graft versus host disease	chronic graft versus host disease at the day of last follow-up	the day of last follow-up