E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
|
E.1.1.1 | Medical condition in easily understood language |
|
E.1.1.2 | Therapeutic area | Body processes [G] - Immune system processes [G12] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10050185 |
E.1.2 | Term | Palmoplantar pustulosis |
E.1.2 | System Organ Class | 10040785 - Skin and subcutaneous tissue disorders |
|
E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective is to provide dose-ranging data for 4 dose regimens of BI 655130
compared to placebo on the primary endpoint of percentage change from baseline in PPP ASI
at Week 16. The target dose(s) will be estimated from the model by incorporating
information on the minimum clinically relevant effect and accounting for safety. |
|
E.2.2 | Secondary objectives of the trial |
Supportive dose-ranging assessments will also be done on pre-specified secondary endpoints. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- 18 to 75 years of legal age (according to local legislation) at screening.
- Diagnosis of Palmoplantar Pustulosis defined as presence of primary, persistent
(>3 months duration), sterile, macroscopically visible pustules on the palms and/or soles,
without or with plaque psoriasis elsewhere on the body.
- Presence of white or yellow pustules on palms and/or soles at screening and baseline.
- Pustular severity score ≥2 in at least one region and ≥10 well-demarcated pustules (white
or yellow pustules) across all regions at screening and baseline.
- PPP PGA of at least moderate severity (≥3) at screening and baseline.
- A minimum PPP ASI score of 12 at screening and baseline.
- Male or female patients. Women of childbearing potential (WOCBP) must be ready and
able to use highly effective methods of birth control per ICH M3 (R2).
- Signed and dated written informed consent in accordance with ICH-GCP and local
legislation prior to admission to the trial. |
|
E.4 | Principal exclusion criteria |
- Reduction in PPP ASI total score ≥ 5 from screening visit (Visit 1) to baseline
(randomisation visit, Visit 2).
- Patients with plaque psoriasis with worsening of plaque psoriasis within the last 3 months
prior to screening.
- Skin conditions that affect ability to score area and severity of PPP components (such as
dyshidrotic eczema, calluses, tinea, xerotic scaling on heels, or maceration of interdigital
areas).
- Women who are pregnant, nursing, or who plan to become pregnant while in the trial.
- Severe, progressive, or uncontrolled condition such as renal, hepatic, haematological,
endocrine, pulmonary, cardiac, neurologic, cerebral, or psychiatric disease, or signs and
symptoms thereof.
- Presence or known history of anti-TNF-induced PPP-like disease.
- Patient with a transplanted organ (with exception of a corneal transplant >12 weeks prior
to screening) or who have ever received stem cell therapy (e.g., Prochymal).
- Known history of lymphoproliferative disease, including lymphoma, or signs and
symptoms suggestive of possible lymphoproliferative disease, such as lymphadenopathy
and/or splenomegaly.
Further criteria apply. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
1) Percent change in PPP ASI from baseline |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
1) Change from baseline in PPP Pain Visual Analog Scale (VAS) score
2) PPP SI change from baseline
3) PPP ASI50
4) PPP ASI75
5) PPP PGA clear/almost clear
6) PPP PGA pustules clear/almost clear
7) Percent change in PPP ASI from baseline
|
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1) Weeks 4 and 16
2) Week 16
3) Week 16
4) Week 16
5) Week 16
6) Week 16
7) Week 52 |
|
E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 5 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 34 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Australia |
Belgium |
Canada |
Czech Republic |
France |
Germany |
Hungary |
Japan |
Korea, Republic of |
Netherlands |
Poland |
Russian Federation |
Switzerland |
Taiwan |
United Kingdom |
United States |
|
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
|
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | 24 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 4 |