GBT440-026 (ZEPHYR)
Research type
Research Study
Full title
A Phase II open label study to evaluate the effect of GBT440 on hypoxemia in subjects with Idiopathic Pulmonary Fibrosis (IPF) who are using supplemental oxygen at rest (ZEPHYR)
IRAS ID
228635
Contact name
Dr Toby Maher
Contact email
Sponsor organisation
Global Blood Therapeutics
Eudract number
2017-001276-27
Clinicaltrials.gov Identifier
Duration of Study in the UK
1 years, 1 months, 31 days
Research summary
Idiopathic Pulmonary Fibrosis (IPF) is a disease that scars the lungs and so reduces the efficiency of the breathing. The build-up scar tissue is called fibrosis. Fibrosis causes the lungs to become stiffer and lose their elasticity so they’re less able to inflate and take oxygen from the air breathed. The cause and origin of IPF is unknown. IPF is a progressive condition and usually gets worse over time. It is estimated that the median survival rate it about 3 to 5 years after diagnosis.
In the United States, it is estimated to affect up to 200,000 people with approximately 50,000 new cases diagnosed each year and approximately 40,000 dying each year.
Nintedanib and pirfenidone are approved therapies that target the fibrotic process. Both drugs demonstrated to provide effective improvements. However, these and other therapies, have not shown significant improvement in oxygenation, patient symptoms, functional status,or
survival.Haemoglobin is a protein that has the role of carrying oxygen in the body. In this context, GBT440 is acting as a haemoglobin modulator, meaning that it increases the affinity of the oxygen to the haemoglobin protein.
Previous studies on animal models showed GBT440 safely increased haemoglobin oxygen affinity. Additional studies are being conducted in healthy volunteers and patients with Sickle cell disease.
The current study is an open label study. This means that both researchers and participants know which treatment is being administered. The study consists of a treatment period (participants receive GBT440 for 90 days) and a safety follow up (30 days). The treatment period is further divided in two parts. Part A and B only differ in the quantity of the drug that is administered to participants (900mg vs 1500mg). Together, Parts A and B will provide safety and efficacy data across two GBT440 doses that are expected to improve oxygen saturation in the enrolled participants. This study is currently enrolling participants and is on-going in the United States.
Approximately up to 32 eligible participants will be enrolled into the study.
Please note that in the UK all participants will be enrolled to part B of the study.REC name
East Midlands - Derby Research Ethics Committee
REC reference
17/EM/0246
Date of REC Opinion
21 Aug 2017
REC opinion
Further Information Favourable Opinion