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

Sponsor

Instituto Brasileiro de Controle do Cancer (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT04712435

Collaborator

(none)

Enrollment

Location

Arms

Anticipated Duration (Months)

3.3

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

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.

Condition or Disease	Intervention/Treatment	Phase
Sinusoidal Obstruction Syndrome (SOS) Hematopoietic Stem Cell Transplant (HSCT)	Drug: N-acetylcysteine Drug: Placebo	Phase 2

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 Design

Study Type:

Interventional

Anticipated Enrollment :

80 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

This is a single-center, randomized, placebo-controlled study to evaluate the efficacy and safety of N-acetylcysteineThis is a single-center, randomized, placebo-controlled study to evaluate the efficacy and safety of N-acetylcysteine

Masking:

Double (Participant, Investigator)

Masking Description:

Matching placebo

Primary Purpose:

Prevention

Official Title:

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

Anticipated Study Start Date :

Apr 1, 2021

Anticipated Primary Completion Date :

Mar 31, 2023

Anticipated Study Completion Date :

Mar 31, 2023

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

Presence or absence of SOS [Day + 30 post HSCT]
To evaluate if prophylactic of N-acetylcysteine has an impact on the incidence of Sinusoidal obstruction syndrome (SOS)

Secondary Outcome Measures

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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