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

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ClinicalTrials.gov Identifier: NCT04712435
Recruitment Status : Not yet recruiting
First Posted : January 15, 2021
Last Update Posted : March 9, 2021
Sponsor:
Information provided by (Responsible Party):
Jamilla Neves Cavalcante, Instituto Brasileiro de Controle do Cancer

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.

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Study Type : Interventional  (Clinical Trial)
Estimated 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-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
Estimated Study Start Date : April 2021
Estimated Primary Completion Date : March 31, 2023
Estimated Study Completion Date : March 31, 2023

Resource links provided by the National Library of Medicine


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




Primary Outcome Measures :
  1. 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)


Secondary Outcome Measures :
  1. Severity of SOS [ Time Frame: within 30 days of bone marrow transplant ]
    Death as noted in medical record

  2. 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



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04712435


Contacts
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Contact: Jamilla N Cavalcante, MD +55113474-4428 millacavalcante@hotmail.com
Contact: Alayne D Yamada, PhD +55113474-4242 alayne.pesquisa@ibcc-mooca.org.br

Locations
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Brazil
Jamilla Neves Cavalcante
Sao Paulo, SP, Brazil, 03102-002
Contact: Jamilla N Cavalcante, MD    +55113474-4428    millacavalcante@hotmail.com   
Contact: Alayne D Yamada, PhD    +55113474-4242    alayne.pesquisa@ibcc-mooca.org.br   
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         
Sponsors and Collaborators
Instituto Brasileiro de Controle do Cancer
Investigators
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Principal Investigator: Jamilla N Cavalcante, MD IBCC Oncologia
Publications:

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Responsible Party: Jamilla Neves Cavalcante, Principal Investigator, Instituto Brasileiro de Controle do Cancer
ClinicalTrials.gov Identifier: NCT04712435    
Other Study ID Numbers: 33788420.4.0000.0072
First Posted: January 15, 2021    Key Record Dates
Last Update Posted: March 9, 2021
Last Verified: January 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Jamilla Neves Cavalcante, Instituto Brasileiro de Controle do Cancer:
Stem cell transplant
Hematopoietic stem cell transplant (HSCT)
Sinusoidal obstruction syndrome (SOS)
Veno-occlusive disease (VOD)
N-acetylcysteine
Additional relevant MeSH terms:
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Hepatic Veno-Occlusive Disease
Syndrome
Disease
Pathologic Processes
Liver Diseases
Digestive System Diseases
Vascular Diseases
Cardiovascular Diseases
Acetylcysteine
N-monoacetylcystine
Antiviral Agents
Anti-Infective Agents
Expectorants
Respiratory System Agents
Free Radical Scavengers
Antioxidants
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs
Antidotes