Efficacy and Safety Study of PRN1008 in Patients with Pemphigus
Research type
Research Study
Full title
A Randomized, Double-Blind, Placebo-Controlled, Multicenter Trial to Evaluate the Efficacy and Safety of Oral BTK Inhibitor PRN1008 in Moderate to Severe Pemphigus
IRAS ID
255486
Contact name
Ana Sousa
Contact email
Sponsor organisation
Principia Biopharma, Inc.
Eudract number
2018-002261-19
Clinicaltrials.gov Identifier
134595, IND
Duration of Study in the UK
2 years, 10 months, 10 days
Research summary
Pemphigus is a rare, chronic, autoimmune (person’s immune system attacks parts of his/her own body ) skin disorder characterised by blistering of skin and mucous membranes. There are two common types of pemphigus:
- Pemphigus vulgaris (PV) - involves painful sores and blisters on skin and in the mouth.
- Pemphigus foliaceus (PF) – involves itchy blisters on the chest, back and shoulders.
Pemphigus can occur at any age, but it's most often seen in people who are middle-aged or older.
There's currently no cure for pemphigus but existing treatment options e.g. corticosteroids can help keep the symptoms under control.
The purpose of this study is to compare the effectiveness and safety of investigational medicine called PRN1008 when taken with a corticosteroid (CS) to the effectiveness and safety of a corticosteroid (prednisone or prednisolone) taken alone. The study medicine is expected to have more targeted action then corticosteroids.
Approximately 120 patients suffering from PV and PF, age 18 to 80 years old, are invited to take part in this study at approximately 100 sites in 20 countries.
Patients’ participation is expected to last about 68 weeks (17 months) and will involve Screening, Part 1, Part 2 and Follow up periods. Patients who during the screening are confirmed as eligible will enter the 1st part of treatment period and will be randomly assigned to one of 2 treatment groups:
• Group A – patients will receive study medicine (PRN1008) and corticosteroids (standard of care for the treatment of pemphigus)
• Group B – patients will receive placebo (a tablet that looks like PRN1008, but does not contain the active ingredient) and corticosteroids (standard of care for the treatment of pemphigus).
Patient will have one in two changes (50%) to receive placebo.
This part of the study will last 36 weeks (9 months) and will be ‘blinded’ which means that neither patient nor the study doctor will know what treatment group patient is assigned to, until the study is over.
Patients who complete the first part of the study, will be eligible to enter Part 2 of the study, which will last 24 weeks (approx. 6 months). In this part every participant will receive PRN1008 and a corticosteroid. The treatment will be ‘open label’, meaning that both patient and Study Doctor will know that patient is receiving PRN1008.
The study treatment will be given in the form of tablets and patients will be required to take assigned doses twice a day by mouth.
After the treatment period patients will enter 4-week follow-up period during which patient will continue to take corticosteroid and patient’s safety will be monitored.
During the study patients will undergo physical examinations, have blood and urine tests, an ECG (electrocardiogram) and may also have skin biopsy if previous results are not available. Patient will be required to complete questionnaires and dosing diaries throughout the study.
At the end of the study patients will return to their standard of care treatment.REC name
HSC REC B
REC reference
19/NI/0024
Date of REC Opinion
2 Apr 2019
REC opinion
Further Information Favourable Opinion