RhuDex vs placebo in PBC
Research type
Research Study
Full title
Double-blind, randomised, placebo-controlled, phase II dose-finding study comparing different doses of RhuDex granules with placebo in the treatment of primary biliary cholangitis
IRAS ID
289044
Contact name
Michael Stieß
Contact email
Sponsor organisation
Dr. Falk Pharma GmbH
Eudract number
2020-001961-34
Duration of Study in the UK
2 years, 3 months, 2 days
Research summary
Researchers want to find out if the drug Rhudex can improve the disease Primary Biliary Cholangitis (PBC).
Rhudex is an investigational (experimental) drug that is being tested and is currently not approved by any
regulatory agency for sale. RhuDex is a novel, orally bioavailable, low molecular weight modulator of T-lymphocyte co-stimulation.This double-blind, randomised, multi-centre, placebo-controlled, comparative phase II trial will compare oral treatment with 50, 100 or 200 mg/day RhuDex granules vs placebo granules for the treatment of PBC.
As all patients will continue standard of care treatment with UDCA, a placebo arm will be included as control due to regulatory recommendations to evaluate dose-related benefits and adverse effects in randomised, double-blind, placebo controlled studies.
The study treatment duration will last 12 weeks ad each patient will have 7 study visits.
The study has three main purposes:
• To evaluate the efficacy of 3 doses of RhuDex vs placebo for the treatment of PBC in patients with an inadequate response to UDCA.
• To identify efficacious RhuDex dose(s) for the treatment of PBC for further evaluation in phase III,
• To study safety and tolerability of RhuDex.The study plans to enrol approximately 136 patients with PBC who are between the age of 18 and 74 years at up to
60 centres in Europe.Summary of Results
17 Jan 2024 (Final Version 1.0) Study code: RDG-1/PBC Page 1 of 3 Clinical study results - lay summary A study to compare different doses of RhuDex granules with placebo for the treatment of primary biliary cholangitis Research sponsor: Dr. Falk Pharma GmbH Drug studied: RhuDex See the end of the summary for the full title of the study. This is a summary of results from one clinical study.
We thank all study participants for their time and effort to conduct this study. They helped researchers learn more about how RhuDex works in patients with primary biliary cholangitis.
General information about the clinical study The study started in March 2021 and ended in May 2023. It was conducted in 10 countries (Belgium, Czech Republic, Germany, Hungary, Italy, the Netherlands, Poland, Slovakia, Spain, and UK). The aim of the study was to find out whether the new drug RhuDex is effective in the treatment of primary biliary cholangitis (PBC).
The researchers stopped the study early because they discovered that RhuDex was not effective after about half of the planned patients had completed the treatment.
Who took part in this study?
This study was open to adults with PBC aged between 18 and 74 years. The patients needed to have taken ursodeoxycholic acid for at least 6 months and have shown a poor response to the treatment. Patients with other liver diseases could not participate. 79 patients (74 women and 5 men) with an average age of 56 years were treated in the study. The youngest participant was 37 years old and the oldest was 70 years old.
Which drugs were studied?
The study participants were randomly divided into 4 groups of equal size with the help of a computer program. Participants in 3 groups received different doses of RhuDex: low
(50 mg/day), medium (100 mg/day), or high (200 mg/day). Participants in 1 group received a placebo. A placebo looks like the active drug but does not contain any active ingredient.
The study participants did not know which treatment they received, nor did the study doctors or study staff (a practice known as “double-blind”). The participants took the study medication twice a day, in the morning and evening, for 12 weeks. The medication was in granule form and was taken orally.
17 Jan 2024 (Final Version 1.0) Study code: RDG-1/PBC Page 2 of 3 What were the results of the trial?
The study participants’ blood was tested for alkaline phosphatase (ALP) levels at the beginning and end of the 12-week study treatment. Elevated ALP levels in the blood typically indicate liver damage in patients with PBC.
After 12 weeks of treatment, patients who took RhuDex had no improvement in their ALP levels compared to those who took the placebo. This is why the study was stopped early.
What were the side effects?
Side effects are unwanted health problems that the doctors think were caused by RhuDex or placebo. The number of patients who experienced side effects after taking the placebo or one of the 3 doses of RhuDex is shown below.
The table below shows the most common side effects (occurring in 2 or more patients) and the number and percentage of patients who experienced a side effect in each treatment group.
Side effect Placebo
RhuDex
low dose
RhuDex
medium dose
RhuDex
high dose
Out of 19
patients
Out of 20
patients
Out of 21
patients
Out of 19
patients
Stomachache (abdominal
pain)
1 patient (5%) - 2 patients (10%) 1 patient (5%) Diarrhea - - 1 patient (5%) 2 patients (11%) Nausea 2 patients (11%) 1 patient (5%) 1 patient (5%) 2 patients (11%) Vomiting 1 patient (5%) - 1 patient (5%) 1 patient (5%) Fatigue - 1 patient (5%) - 1 patient (5%) Liver value (alanine
aminotransferase) increased
- 1 patient (5%) 1 patient (5%) -
Liver value (aspartate
aminotransferase) increased
- 1 patient (5%) 1 patient (5%) -
Liver (hepatic) enzyme
increased
- - - 2 patients (11%)
Headache 1 patient (5%) 1 patient (5%) 2 patients (10%) 3 patients (16%) Itchy skin (pruritus) - 1 patient (5%) 1 patient (5%) 1 patient (5%) None of the side effects required hospital care or a prolonged hospital stay. The side effects were not life-threatening and did not cause lasting problems for the participants.
17 Jan 2024 (Final Version 1.0) Study code: RDG-1/PBC Page 3 of 3 How has the study helped patients and researchers?
This study helped researchers learn that RhuDex may not be effective in the treatment of PBC.
Please remember that this summary shows only the main results from one study. Results from other studies may be different. Generally, many studies are done to find out how well a medicine works and if it is safe.REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
20/NE/0243
Date of REC Opinion
7 Dec 2020
REC opinion
Further Information Favourable Opinion