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

NCT03391856 | Terminated Phase 2 DMC

• 1 other identifier: 2017PHB220
• Study Type: interventional
• Target: 15
• Locations: 1 country
• Sites: 1

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.

Conditions

Thrombocytopenia; Stem Cell Transplant Complications

Outcome Measures

Primary Outcomes (1)

• response rate

  rate of response after 30 days of treatment

  30 days after the start of enrollement

Secondary Outcomes (4)

• overall survival

  the day of last follow-up

• non-relapse mortality

  the day of last follow-up

• grade 2-4 acute graft versus host disease

  day 100 after transplant

• chronic graft versus host disease

  the day of last follow-up

Study Arms (2)

intervention arm

EXPERIMENTAL

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

Drug: N-acetyl-L-cysteine (NAC)

controlled arm

OTHER

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

Other: supportive therapy

Interventions

N-acetyl-L-cysteine (NAC) DRUG

NAC treatment for PT: 400mg p.o tid from day 60 to 90 post transplant

intervention arm

supportive therapy OTHER

prophylactic platelet transfusion was given when platelet count \<20000/ul

controlled arm

Eligibility Criteria

• Age: 14 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

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

Sponsors & Collaborators

• Peking University People's Hospital: lead
• The First Affiliated Hospital of Soochow University: collaborator
• Nanfang Hospital, Southern Medical University: collaborator

Study Sites (1)

Peking University People's Hospital

Beijing, Beijing Municipality, 100044, China

Location

MeSH Terms

Conditions

Thrombocytopenia

Interventions

Acetylcysteine; Palliative Care

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia

Intervention Hierarchy (Ancestors)

Cysteine; Amino Acids, Sulfur; Sulfur Compounds; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins; Patient Care; Therapeutics; Health Services; Health Care Facilities Workforce and Services

Study Officials

• Xiao-Jun Huang, MD

  Peiking University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Head of Peking University Institute of Hematology

Study Record Dates

• First Submitted: January 2, 2018
• First Posted: January 5, 2018
• Study Start: March 1, 2018
• Primary Completion: December 30, 2018
• Study Completion: December 30, 2018
• Last Updated: September 25, 2020

Record last verified: 2020-09

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)