Safety and Efficacy of N-Acetylcysteine Mouthwash in Prevention of Mucositis in HSCT Patients

NCT07325383 | Active Not Recruiting Phase 3

• 2 other identifiers: IR.TUMS.DENTISTRY.REC.1403.048IRCT20140818018842N42
• Study Type: interventional
• Target: 106
• Locations: 1 country
• Sites: 1

Brief Summary

Oral mucositis (OM) is one of the most frequent complications in patients who undergo HSCT. It not only causes severe discomfort but also affects treatment adherence and increases hospital stays. In this study, we aim to assess whether using N-acetylcysteine mouthwash can safely and effectively prevent OM. The trial is randomized and conducted in the hematology wards of TUMS-affiliated RIOHCT, including both autologous and allogeneic HSCT patients.

Conditions

Oral Mucositis; Hematopoietic Stem Cell Transplantation (HSCT)

Keywords

N-Acetylcysteine; Oral Mucositis; Hematopoietic Stem Cell Transplantation; Chemotherapy-Induced Mucositis; Mouthwash

Outcome Measures

Primary Outcomes (3)

• Incidence of oral mucositis

  Oral mucositis is a painful and debilitating complication of hematopoietic stem cell transplantation, characterized by erythema, edema, and ulcerations of the oral mucosa. The incidence of mucositis is assessed daily from the beginning of the conditioning regimen until day +14 post-transplantation. The World Health Organization (WHO) Oral Toxicity Scale is used to grade the severity of mucositis. The presence of grade ≥1 mucositis is recorded as incidence.

  From day 1 of conditioning to day +14 post-transplantation.

• Incidence of N-acetylcysteine-related Adverse Events

  Participants are monitored daily for any adverse events potentially related to the use of N-acetylcysteine mouthwash. Adverse events include non-immune anaphylaxis reactions, cardiovascular reactions, dermatologic reactions (such as rash or pruritus), and gastrointestinal reactions (such as nausea or vomiting).

  Clinical examination is performed daily from the beginning of the conditioning regimen until day +14 post-transplantation.

• Incidence of Severe Oral Mucositis

  Severe oral mucositis is defined as grade 3 or 4 according to the World Health Organization (WHO) Oral Toxicity Scale. Participants are assessed daily to determine the presence and severity of mucositis. The incidence of severe mucositis (grade ≥3) is recorded during the study period.

  From day 1 of conditioning to day +14 post-transplantation

Secondary Outcomes (1)

• Length of Hospitalization

  From hospital admission through hospital discharge, up to Day +60 post-transplantation

Study Arms (2)

N-Acetylcysteine group

EXPERIMENTAL

Participants in this group receive 2400 mg of N-acetylcysteine daily as an oral mouthwash, divided into four doses per day. The intervention starts at the beginning of the conditioning regimen and continues until day 14 after hematopoietic stem cell transplantation.

Drug: N-Acetylcysteine 600mg/tab

Placebo group

PLACEBO COMPARATOR

Participants in this group receive a placebo mouthwash with identical appearance, taste, and administration schedule as the NAC mouthwash. The placebo contains no active pharmaceutical ingredients and is administered four times daily from the start of conditioning until day 14 post-transplantation.

Drug: Placebo

Interventions

N-Acetylcysteine 600mg/tab DRUG

Each dose consists of one 600 mg effervescent tablet of N-acetylcysteine (NAC) dissolved in 20 mL of sodium carbonate solution. Participants receive this mouthwash four times daily, totaling 2400 mg of NAC per day. The solution is used as an oral rinse (swish and spit) and is not intended to be swallowed. Administration begins with the conditioning regimen and continues until day 14 after HSCT.

Also known as: NAC

N-Acetylcysteine group

Placebo DRUG

Each dose consists of one placebo effervescent tablet, identical in appearance and packaging to the N-acetylcysteine tablets, dissolved in 20 mL of sodium carbonate solution. Participants use this placebo mouthwash four times daily as an oral rinse (swish and spit), starting from the conditioning regimen and continuing until day 14 post-transplantation. The placebo contains no active pharmaceutical ingredients.

Placebo group

Eligibility Criteria

• Age: 18 Years - 66 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients undergoing hematopoietic stem cell transplantation (HSCT), including both allogeneic and autologous transplants
• Age ≥18 years
• Ability to provide informed consent

You may not qualify if:

• Known hypersensitivity to N-acetylcysteine or any components of the effervescent tablets
• Inability to adhere to the proper use of mouthwash
• Systemic use of N-acetylcysteine during the study period
• Pregnancy or lactation

Sponsors & Collaborators

• Research Institute for Oncology- Hematology and Cell Therapy (RIOHCT), Tehran University of Medical Sciences, Iran: collaborator
• Tehran University of Medical Sciences: lead

Study Sites (1)

Research Institute of Oncology, Hematology, and Cell Therapy, Shariati Hospital

Tehran, Iran

Location

Related Publications (1)

• Moslehi A, Taghizadeh-Ghehi M, Gholami K, Hadjibabaie M, Jahangard-Rafsanjani Z, Sarayani A, Javadi M, Esfandbod M, Ghavamzadeh A. N-acetyl cysteine for prevention of oral mucositis in hematopoietic SCT: a double-blind, randomized, placebo-controlled trial. Bone Marrow Transplant. 2014 Jun;49(6):818-23. doi: 10.1038/bmt.2014.34. Epub 2014 Mar 10.

  PMID: 24614837 BACKGROUND

MeSH Terms

Conditions

Stomatitis

Interventions

Acetylcysteine

Condition Hierarchy (Ancestors)

Mouth Diseases; Stomatognathic Diseases

Intervention Hierarchy (Ancestors)

Cysteine; Amino Acids, Sulfur; Sulfur Compounds; Organic Chemicals; Amino Acids; Amino Acids, Peptides, and Proteins

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: PREVENTION
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: PharmD, iBCPS, FCCP Assistant; Professor at Tehran University of Medical Sciences (TUMS)

Study Record Dates

• First Submitted: November 16, 2025
• First Posted: January 8, 2026
• Study Start: August 18, 2024
• Primary Completion: March 8, 2025
• Study Completion: February 1, 2026
• Last Updated: January 8, 2026

Record last verified: 2026-01

Locations

IR (1)