JAKPPPOT Trial
Research type
Research Study
Full title
JAnus Kinase inhibitors in PalmoPlantar PustulOsis: a mixed-methods feasibiliTy trial.
IRAS ID
346783
Contact name
Catherine Smith
Contact email
Sponsor organisation
King's College London
ISRCTN Number
ISRCTN61751241
Duration of Study in the UK
2 years, 0 months, 0 days
Research summary
Research Summary:
Aims:
Palmoplantar pustulosis (PPP) is a rare, debilitating condition, causing painful, red skin with pus-filled lumps on the hands and feet that are difficult to treat. A new group of drugs, called Janus kinase (JAK) inhibitors, show promise in the treatment of PPP in some reported cases. However, these drugs also carry a risk of side effects which are more common in people with long-term health conditions.
We need to know whether JAK inhibitors are an acceptable treatment for PPP. To do this, we will carry out a small-scale trial investigating whether people with PPP are happy to take JAK inhibitors and whether a larger clinical trial investigating JAK inhibitors
in PPP is possible.Methods:
This is a single-arm, open-label trial exploring the feasibility of using JAK inhibitors as a treatment for PPP. 20 adult participants (>18 years old) will receive a daily tablet of upadacitinib (30mg once-a-day) for 8 weeks. The trial will be run by King’s College London and Guy’s and St Thomas’ NHS Trust, and last for 2 years.
We will assess how willing individuals are to participate in the trial, how well they take the medication throughout the trial, and the overall acceptability of the treatment from the perspective of both people with PPP and healthcare professionals. Scoring systems, questionnaires, and in-depth interviews will be used to understand the barriers to running a full-scale clinical trial.We will compare the results in this study with a previous trial (APRICOT Trial) in which participants received a non-active (placebo) treatment to determine how many participants will be required for a future full-scale trial. Based on the findings from in-depth interviews with people with PPP and healthcare professionals, changes will be made to the recruitment process throughout the trial, identifying strategies that may improve recruitment in the future.
Summary of Results:
Palmoplantar pustulosis (PPP) is a rare, long-term inflammatory skin disease that causes painful, pus-filled blisters on the palms of the hands and soles of the feet. It can make everyday activities such as walking and using the hands difficult and has a major impact on quality of life. There are currently no licensed treatments for PPP in the United Kingdom or Europe.
Researchers from King's College London and Guy's and St Thomas' NHS Foundation Trust, UK, wanted to find out whether it would be possible to run a larger clinical trial of a medicine called upadacitinib, a Janus kinase (JAK) inhibitor, in patients with PPP. JAK inhibitors work by blocking several signals in the immune system that contribute to inflammation. Twenty adults with moderate-to-severe PPP received upadacitinib for 8 weeks. The researchers assessed whether people were willing to join the study, whether they took the medication as prescribed, and whether they found the treatment acceptable. They also compared the results with carefully matched placebo (dummy treatment) data from an earlier clinical trial to provide an early indication of how well the treatment worked.
More than 40% of people invited to take part joined the study, almost all participants took their medication as prescribed, and most considered the treatment acceptable. Participants receiving upadacitinib showed much greater improvement in disease severity than the earlier placebo group, although this comparison should be interpreted with caution because the groups were not randomly assigned and the numbers of participants in the study was small. Side effects were generally mild, and no new safety concerns were identified.
These findings show that a larger clinical trial of upadacitinib in PPP is feasible. They also provide encouraging early evidence that this treatment may improve disease severity. A future multicentre clinical trial comparing upadacitinib with an active treatment rather than placebo is now needed to determine whether it is an effective treatment for people with PPP.
REC name
North East - Newcastle & North Tyneside 2 Research Ethics Committee
REC reference
24/NE/0147
Date of REC Opinion
11 Sep 2024
REC opinion
Further Information Favourable Opinion