Efficacy of N-acetylcysteine Versus Placebo as Prophylaxis of Sinusoidal Obstruction Syndrome in Patients Undergoing Hematopoietic Stem Cell Transplantation (ALISON)

March 8, 2021 updated by: Jamilla Neves Cavalcante, Instituto Brasileiro de Controle do Cancer

Prospective, Randomized Study Using N-Acetylcysteine as Prophylaxis of Sinusoidal Obstruction Syndrome in Patients Undergoing Hematopoietic Stem Cell Transplantation

Sinusoidal obstruction syndrome (SOS) or hepatic veno-occlusive disease (VOD) is a serious complication that occurs, most often occurring in patients undergoing hematopoietic stem cell transplantation (HSCT), especially in its first thirty days. The morbidity and mortality in this syndrome are considerable, since severe SOS is associated with a mortality of more than 90% in the first hundred days of HSCT.

Some risk factors are modifiable, especially those related to transplantation, but when non-alterable factors are present, preventive measures are needed that can reduce the incidence and / or severity of SOS.

Study Overview

Status

Not yet recruiting

Conditions

Intervention / Treatment

Detailed Description

This is a single-center, randomized, placebo-controlled study to evaluate the efficacy and safety of N-acetylcysteine as prophylaxis of sinusoidal obstruction syndrome in patients undergoing hematopoietic stem cell transplantation Up to date, no approved medications are available for prophylactic treatment of sinusoidal obstruction syndrome in patients undergoing hematopoietic stem cell transplantation. N-acetylcysteine is a drug that has been long used as a mucolytic. And placebo responses contribute to help assess the appropriateness of randomized clinical trials in determining the size of drug effect.

The study will enroll patients undergoing hematopoietic stem cell transplantation to receive sequentially one of two treatment: N-acetylcysteine or Placebo.

Study Type

Interventional

Enrollment (Anticipated)

80

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • Brazil
    • SP
      • Sao Paulo, SP, Brazil, 03102-002
        • Jamilla Neves Cavalcante
        • Contact:
        • Contact:
        • Principal Investigator:
          • Jamilla N Cavalcante, MD
        • Sub-Investigator:
          • Maria Cristina M Macedo, PhD
        • Sub-Investigator:
          • Larissa Y Okada, MD
        • Sub-Investigator:
          • Lauro Augusto C Leite, MD

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

Inclusion Criteria:

  • Men or women
  • Age ≥ 18 years
  • A proven diagnosis of one of the hematological malignancies: Acute Myeloid Leukemia, Acute Lymphoid Leukemia, Chronic Myeloid Leukemia, Chronic Lymphoid Leukemia, Multiple Myeloma, Non-Hodgkin's Lymphoma, Hodgkin's Lymphoma
  • Patients must be scheduled to undergo of Allogeneic hematopoietic transplantation or HLA (Human Leukocyte Antigen) haploidentical related or Unrelated or HLA-matched related or autologous stem cell transplant
  • Patients must be able to understand and sign a written informed consent

Exclusion Criteria:

  • Patient has a serious active disease or co-morbid medical condition, as judged by the investigator, which would interfere with the conduct of this study
  • Patient, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study
  • Known hypersensitivity to N-acetylcysteine
  • Contraindications to perform any procedure provided for in this study
  • Patients who have already undergone a previous transplant

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Prevention
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Experimental: N-acetylcysteine
Sachets containing N-acetylcysteine 200 mg Powder for Oral Solution administered every 8 hours (Daily dose 600 mg/day). is administered on the first day of conditioning and will continue until Day +30 post HSCT or hospital discharge, whichever is sooner
Drug: N-acetylcysteine
Eligible patients will be randomised to receive N-acetylcysteine versus matching placebo
Placebo Comparator: Placebo Comparator: Placebo
200mg granulated solution of matching placebo administered every 8 hours (Daily dose 600 mg/day), is administered on the first day of conditioning and will continue until Day +30 post HSCT or hospital discharge, whichever is sooner
Drug: Placebo
Eligible patients will be randomised to receive N-acetylcysteine versus matching placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Presence or absence of SOS
Time Frame: Day + 30 post HSCT
To evaluate if prophylactic of N-acetylcysteine has an impact on the incidence of Sinusoidal obstruction syndrome (SOS)
Day + 30 post HSCT

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Severity of SOS
Time Frame: within 30 days of bone marrow transplant
Death as noted in medical record
within 30 days of bone marrow transplant
Sinusoidal obstruction syndrome (SOS) -Free Survival at Day 30 Post-Hematopoietic Stem Cell Transplant (HSCT)
Time Frame: 30 Days Post-Transplant
Comparison of efficacy of N-acetylcysteine vs Placebo for the prevention of SOS
30 Days Post-Transplant

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Instituto Brasileiro de Controle do Cancer

Investigators

  • Principal Investigator: Jamilla N Cavalcante, MD, IBCC Oncologia

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Anticipated)

April 1, 2021

Primary Completion (Anticipated)

March 31, 2023

Study Completion (Anticipated)

March 31, 2023

Study Registration Dates

First Submitted

January 13, 2021

First Submitted That Met QC Criteria

January 13, 2021

First Posted (Actual)

January 15, 2021

Study Record Updates

Last Update Posted (Actual)

March 9, 2021

Last Update Submitted That Met QC Criteria

March 8, 2021

Last Verified

January 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 33788420.4.0000.0072

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

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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