Inhaled Molgramostim (rhGM-CSF) in Healthy Adult Subjects
United Kingdom42 participantsStarted 2015-05
Plain-language summary
This is a randomized, double-blind, placebo-controlled, single ascending (SAD), and multiple ascending dose (MAD) study conducted at a single clinical site within the UK. Healthy male and female subjects (on non-child bearing potential) will be enrolled to investigate single inhaled doses of molgramostim at 3 dose levels (Part 1) and multiple inhaled doses at 2 dose levels (Part 2). The 2 doses in the multiple ascending dose regimens will be administered once daily (QD) for 6 consecutive days. The clinical indication for inhaled molgramostim is the treatment of respiratory diseases such as aPAP, bronchiectasis and cystic fibrosis. The Clinical trial will involve 42 healthy participants. The trial is expected to last approximately 4 months.
Who can participate
Age range
18 Years – 55 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Healthy, adult, male or female, 18- 55 years of age, inclusive, at screening.
. Life-long non smoker who has not used nicotine containing products.
. Body mass index (BMI) ≥ 18.5 and ≤ 32.0 kg/m2 at screening.
. Medically healthy with no clinically significant medical history, physical examination, laboratory profiles, spirometry, vital signs, or ECGs, as deemed by the PI.
. Females must be of non-child bearing potential who must have undergone one of the following sterilization procedures at least 6 months prior to dosing in Part 1 or prior to the first dose in Part 2:
. A non vasectomized, male subject must agree to use a condom with spermicide or abstain from sexual intercourse during the study until 90 days beyond the last dose of study medication. (No restrictions are required for a vasectomized male provided his vasectomy has been performed 4 months or more prior to study start. A male who has been vasectomized less than 4 months prior to study start must follow the same restrictions as a non vasectomized male).
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number and severity of treatment-emergent Adverse Events (AEs) following single and multiple inhaled doses of molgramostim
. If male must agree not to donate sperm from dosing in Part 1 or from the first dose in Part 2, until 90 days following last dose.
. Understands the study procedures in the informed consent form (ICF), and be willing and able to comply with the protocol.
Exclusion criteria
. Subject is mentally or legally incapacitated or has significant emotional problems at the time of screening visit or expected during the conduct of the study.
. History or presence of clinically significant medical condition(s) (including ongoing active infections) or psychiatric condition(s) or disease(s) in the opinion of the PI.
. History of any illness that, in the opinion of the PI, might confound the results of the study or poses an additional risk to the subject by their participation in the study.
. Abnormal spirometry test results, in the opinion of the PI.
. History of severe or unexplained adverse reactions during aerosol delivery of any medicinal product.
. History or presence of alcoholism or drug abuse within the past 2 years prior to screening.
. History or presence of hypersensitivity or idiosyncratic reaction to molgramostim or to related compounds (i.e., Growgen®, Leucomax®, Leukine®, Topleucon™).
. Clinically significant history or presence of ECG abnormalities such as second- or third degree atrioventricular block, evidence, or family history of prolonged QT syndrome.