Hematopoietic Stem Cell Transplant (HSCT), Sinusoidal Obstruction Syndrome (SOS)
Conditions
Keywords
Stem cell transplant, Hematopoietic stem cell transplant (HSCT), Sinusoidal obstruction syndrome (SOS), Veno-occlusive disease (VOD), N-acetylcysteine
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.
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.
Interventions
DRUGN-acetylcysteine
Eligible patients will be randomised to receive N-acetylcysteine versus matching placebo
DRUGPlacebo
Eligible patients will be randomised to receive N-acetylcysteine versus matching placebo
Sponsors
Instituto Brasileiro de Controle do Cancer
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
DOUBLE (Subject, Investigator)
Masking description
Matching placebo
Intervention model description
This is a single-center, randomized, placebo-controlled study to evaluate the efficacy and safety of N-acetylcysteine
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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
| Measure | Time frame | Description |
|---|---|---|
| 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 |
Countries
Brazil
Contacts
Primary ContactJamilla N Cavalcante, MD
Backup ContactAlayne D Yamada, PhD
Outcome results
None listed