HZN-825 safety and effectiveness on Patients with Diffuse Cutaneous Systemic Sclerosis
Research type
Research Study
Full title
A Randomized, Double-blind, Placebo-controlled, Repeat-dose, Multicenter Trial to Evaluate the Efficacy, Safety, Tolerability and Pharmacokinetics of HZN-825 in Patients with Diffuse Cutaneous Systemic Sclerosis
IRAS ID
1003976
Contact name
Farah Ali
Contact email
Sponsor organisation
Horizon Therapeutics Ireland DAC
Eudract number
2020-005764-62
Clinicaltrials.gov Identifier
Research summary
This is a randomised, double-blind, placebo-controlled, study in patients with diffuse cutaneous Systemic Sclerosis (SSc). Diffuse cutaneous SSc is dominated by rapidly progressive fibrosis (scarring) of the skin, lungs and other internal organs. Progressive means that the fibrosis has grown in size. The main purpose of this study is to see if the study drug, called HZN-825 is safe, tolerable and effective in the treatment of patients with cutaneous SSc compared to placebo.
Eligible partcipants will be randomised (by chance, like flipping a coin) to receive one of three oral dose regimens in a 1: 1: 1 ratio:HZN-825 300 mg once daily (QD), HZN-825 300 mg twice daily (BID) or placebo for 52 weeks.
The study consists of a 4-week Screening Period and a 52-week Double-blind Treatment Period. All subjects who complete the Double-blind Treatment Period (Week 52) will be eligible to enter a 52-week extension study (HZNP-HZN-825-302). Subjects not entering the extension trial will return to the clinic for a Safety Follow-up Visit 4 weeks after the last dose of study drug. The study will last for around 14 months.
Study procedures include vital signs, physical examination, questionnaires, High-Resolution CT scans, ECGs, blood and urine samples.
The study is sponsored by Horizon Therapeutics Ireland DAC. Approximately 300 male and non-pregnant female patients between the ages of 18 and 75 years, inclusive, with diffuse cutaneous SSc will be enrolled worldwide with 3-10 patients from 5 hospitals in the UK.
REC name
London - Westminster Research Ethics Committee
REC reference
21/LO/0857
Date of REC Opinion
23 Jan 2022
REC opinion
Further Information Favourable Opinion