The primary objective of the trial is to demonstrate a non-flat curve and evaluate the doseresponse relationship for 3 subcutaneous dosing regimens of BI 655130 (spesolimab) (with each regimen consisting of a single loading dose and a separate…
ID
Source
Brief title
Condition
- Epidermal and dermal conditions
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Time to first GPP flare (defined by increase in GPPGA score by * 2 from
baseline and the pustular component of GPPGA * 2) up to week 48.
Secondary outcome
Key Secondary Endpoint
- The occurrence of at least one GPP flare (defined by increase in GPPGA score
by * 2 from baseline and the pustular component of GPPGA * 2) up to week 48.
Secondary Endpoints:
- Time to first worsening of Psoriasis Symptom Scale (PSS) up to week 48
defined as a 4-point increase in total score from baseline.
- Time to first worsening of Dermatology Quality of Life Index (DLQI) up to
week 48 defined as a 4-point increase in total score from baseline.
- Sustained remission, defined as a patient with a GPPGA score of 0 or 1 (clear
or almost clear) at all visits up to week 48, without intake of rescue
medication, or investigator-prescribed Standard of Care.
- The occurrence of treatment emergent adverse events (TEAEs)
Background summary
GPP is a rare and serious disease which it difficult to treat. A flare can be
very dangerous and immediate treatment is required. At this moment, little is
known about this rare disease and in Europe, no medical treatment is approved
for GPP. The need for better treatment is high.
Study objective
The primary objective of the trial is to demonstrate a non-flat curve and
evaluate the doseresponse relationship for 3 subcutaneous dosing regimens of BI
655130 (spesolimab) (with each regimen consisting of a single loading dose and
a separate maintenance subcutaneous dosing regimen) versus placebo, on the
primary endpoint, the time to the first GPP flare onset up to week 48.
Study design
This is a multicenter, randomized, double-blind, placebo-controlled Phase IIb
study comprising of 3 active doses compared to placebo in patients (adult and
adolescent) with history of GPP and currently presenting (at screening and at
randomization) with a GPPGA score of 0 or 1 (clear or almost clear).
Intervention
About 120 patiënt including at least 8 adolescent patients aged 12 to 17 years
will be included in this trial.
30 patients on BI 655130 300 mg s.c. q4 weeks (Arm 1)
30 patients on BI 655130 300 mg s.c. q12weeks (Arm 2)
30 patients on BI 655130 150 mg s.c. q12 weeks (Arm 3)
30 patients on placebo (Arm 4)
Study burden and risks
Burden:
Regual visits to the research location.
Physical examination: 15 times
ECG: 15 times
Vital functions 15 times
Vena punction: 15 times
Urine collection: 15 times
Pregnancy test: 15 times
Photo's from skin lesions: 14 times
Questionnaires about patients health: 13 times
Injection (subcutan) study medication: 12 times
Skin biopsy (OPTIONAL SUBSTUDY): 2 times
Extra bloodsampling for biobanking (OPTIONAL SUBSTUDY): 1 time
Obligated use of birth control and/of seksual abstination
Risks:
Risks on side effects or allergic reation to study medication.
Risks on side effects from study procedures, e.g. bruising after vena punction.
Comeniusstraat 6
Alkmaar 1817MS
NL
Comeniusstraat 6
Alkmaar 1817MS
NL
Listed location countries
Age
Inclusion criteria
1. Patients with a known and documented history of GPP per ERASPEN criteria
(see protocol section 3.3.1) regardless of IL36RN mutation status.
2. Patients with a GPPGA score of 0 or 1 at screening and randomization.
3. Male or female patients, aged 12 to 75 years at screening. For all patients,
a minimum weight of 40 kg is required.
4. Signed and dated written informed consent.
5. Women of childbearing potential must be ready and able to use highly
effective methods of birth control
Additional inclusion criteria will apply, see protocol section 3.3.2.
Exclusion criteria
1. Patients with SAPHO (Synovitis*acne*pustulosis*hyperostosis*osteitis)
syndrome.
2. Patients with primary erythrodermic psoriasis vulgaris.
3. Severe, progressive, or uncontrolled hepatic disease
4. Treatment with:
a. Any restricted medication as specified in the CTP, or any drug considered
likely to interfere with the safe conduct of the study, as assessed by the
investigator.
b. Any prior exposure to BI 655130 or another IL36R inhibitor biologic.
5. Increased risk of infectious complications as assessed by the investigator.
6. Relevant chronic or acute infections at the time of randomization. A patient
can be re-screened if the patient was treated and is cured from the acute
infection.
7. Active or Latent TB
Additional exclusion criteria will apply, see protocol sectie 3.3.3.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2018-003081-14-NL |
| ClinicalTrials.gov | NCT04399837 |
| CCMO | NL72068.056.19 |