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

Instituto Brasileiro de Controle do Cancer

Status and phase: Unknown; Phase 2

Conditions: Hematopoietic Stem Cell Transplant (HSCT); Sinusoidal Obstruction Syndrome (SOS)

Treatments: Drug: Placebo; Drug: N-acetylcysteine

Study type: Interventional

Funder types: Other

Identifiers

NCT04712435

33788420.4.0000.0072

Details and patient eligibility

About

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.

Full 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.

Enrollment: 80 estimated patients

Sex: All

Ages: 18+ years old

Volunteers: No 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

Trial design

Primary purpose: Prevention

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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80 participants in 2 patient groups, including a placebo group

Experimental: N-acetylcysteine

Experimental group

Description:

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

Treatment:

Drug: N-acetylcysteine

Placebo Comparator: Placebo

Placebo Comparator group

Description:

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

Treatment:

Drug: Placebo