NCT04278040

Inhalations of Ultra-low Doses of Melphalan for the Treatment of Non-cystic Fibrosis Bronchiectasis

Safety, Tolerability and Efficacy of Ultra-low Doses of Alkylating Drug Melphalan Inhalations for the Treatment of Non-cystic Fibrosis Bronchiectasis

• Status: Unknown
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 7 (estimated)
• Sponsor: Federal State Budgetary Institution, Pulmonology Scientific Research Institute · Other Government
• Sex: All
• Age: 18 Years – 70 Years
• Healthy volunteers: Not accepted

Summary

This non-randomised open-label prospective pilot study evaluates the safety and efficacy of inhalations of ultra-low doses of alkylating drug melphalan for the treatment of non-cystic fibrosis bronchiectasis. All patients will receive 0,1 mg of melphalan in 5 daily inhalations 1 time per day.

Detailed description

It was previously shown that in ultra-low (more than 100 times lower than conventional therapeutic) doses inhalations of alkylating drug (melphalan) are effective in severe steroid-resistant bronchial asthma, a form of the disease often characterized by neutrophilic type of inflammation. The exacerbation frequency reduced after the treatment, steroid-sparing effect was shown, morphological signs of bronchial epithelial regeneration were revealed and quality of life of asthmatic patients, treated with ultra-low doses of melphalan, improved. In preclinical studies and studies with volunteers, it was found that inhalations of ultra-low doses of melphalan do not have cytotoxic properties, but have local and systemic anti-inflammatory effects and decrease the activation of lymphocytes due to blockade of heavy β-chain of the interleukin (IL)-2 surface receptor. In addition, in ultra-low concentrations, alkylating agents are able to disrupt the cell signalling through the receptor for tumor necrosis factor, thereby exerting a protective effect from the cytotoxic activity of tumor necrosis factor (TNF)-α, which leads to the anti-inflammatory response. It can be assumed that the inhalation use of ultra-low doses of melphalan can be effective in patients with bronchiectasis, as in a disease characterized by a neutrophilic type of inflammation, leading to the improvement of quality of life, increasing the time to the first exacerbation and decreasing of exacerbation frequency.

Conditions

• Non-cystic Fibrosis Bronchiectasis

Interventions

Type	Name	Description
DRUG	Melphalan	Inhalations with low doses of Melphalan for 5 consequent days

Timeline

• Start date: 2018-06-20
• Primary completion: 2021-12-01
• Completion: 2021-12-30
• First posted: 2020-02-20
• Last updated: 2020-02-20

Locations

1 site across 1 country: Russia