Safety and tolerability of PHP-303 in Adults with AATD
Research type
Research Study
Full title
A Multi-Center, 12-Week, Randomized, Double-Blind, Placebo Controlled Study to Evaluate Safety and Tolerability of the Neutrophil Elastase Inhibitor PHP 303 in Adults with Alpha-1 Antitrypsin Deficiency (AATD)
IRAS ID
273088
Contact name
Alice Turner
Contact email
Sponsor organisation
pH Pharma Inc.
Eudract number
2019-002501-22
Clinicaltrials.gov Identifier
137165, IND
Duration of Study in the UK
2 years, 0 months, 1 days
Research summary
The study is randomized, double-blind, placebo-controlled Study to Evaluate Safety and Tolerability of the Neutrophil Elastase Inhibitor PHP-303 in Adults with Alpha-1 Antitrypsin Deficiency (AATD).
Alpha-1 antitrypsin (AAT) is a protease inhibitor secreted into the blood primarily by the Liver. AATD is an inherited disorder caused by mutations in the SERPINA1 gene on chromosome 14, which encodes for AAT. The current interventions available for AATD provide short-term benefits but do not treat the underlying causes of the condition. In later stages, patients require oxygen therapy. End-stage disease is treated by lung transplantation and/or lung volume reduction surgery.
This Phase 2 study is being conducted to support future investigation of PHP-303 for the treatment of AATD. This study is designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of PHP-303 20 mg QD versus PHP-303 10 mg QD, in order to optimize and enable the future clinical development of PHP-303 for treatment of AATD.
The total duration of a patient’s participation in the study will be around 20 weeks. This involves a 4-week screening period during which the study doctor will check whether they can participate, 12 weeks of treatment and a 4-week follow-up after the treatment. The patient will have approximately 8 study visits to the study site and 7 telephone contacts over a period of 20 weeks.
The study will take place at NHS sites in the UK.
REC name
West Midlands - Edgbaston Research Ethics Committee
REC reference
20/WM/0001
Date of REC Opinion
4 May 2020
REC opinion
Further Information Favourable Opinion