OPTIMA - SAV008-01 - Pilot clinical trial of inhaled Molgramostim
Research type
Research Study
Full title
An Open-label, non-controlled, multicenter, Pilot clinical Trial of Inhaled Molgramostim in subjects with Antibiotic-resistant non-tuberculosis mycobacterial (NTM) infection
IRAS ID
238305
Contact name
Michael R. Loebinger
Contact email
Sponsor organisation
Savara ApS
Eudract number
2017-003374-14
Duration of Study in the UK
1 years, 10 months, 30 days
Research summary
Non-tuberculosis mycobacteria (NTM) cause progressive destruction of lung tissue resulting in a variety of symptoms including cough, breathlessness and weight loss. Treatment of NTM infection is difficult because the bacteria are often resistant to antibiotics. Treatment usually requires at least 3 antibiotics given for a minimum of 18 months. Many patients cannot tolerate the treatment due to side effects and the treatment itself is often ineffective. Even successful therapy is associated with recurrence of the infection within few years.
In order to cure the NTM infection, white blood cells called macrophages need to engulf the bacteria and digest them (a process called phagocytosis). It is thought that NTM lung disease results from failure of macrophages to contain and neutralise the bacteria.
Molgramostim contains the active substance recombinant human Granulocyte Macrophage Colony-Stimulating Factor (rhGM-CSF). rhGM-CSF is a protein that is naturally present in the human body as part of the immune system. In the lung rhGM-CSF increases the number of, and activation of, macrophages resulting in greater ability to kill the bacteria. It is suggested that inhaling molgramostim may aid the immune system and hence the effectiveness of the antibiotic therapy.
The current study is an open-label phase 2a pilot study in subjects who have not responded to the NTM antibiotic treatment in the past for reasons of lack of efficacy or inability to tolerate the drug regimen. The main objective of the study is to investigate if molgramostim can clear NTM infection from the sputum (phlegm). The effect on ability to exercise and weight loss will also be studied along with any improvement in the patient's symptoms and quality life after 24 weeks of treatment.
Subjects will be followed up during 12 weeks after completion of the treatment period.
REC name
London - Westminster Research Ethics Committee
REC reference
18/LO/0295
Date of REC Opinion
6 Apr 2018
REC opinion
Further Information Favourable Opinion