A Study to Test Whether Spesolimab Helps People With a Skin Disease Called Netherton Syndrome
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT05856526 |
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Recruitment Status :
Recruiting
First Posted : May 12, 2023
Last Update Posted : May 14, 2024
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This study is open to people with a skin disease called Netherton syndrome (NS). People can join the study if they are 12 years and older. The purpose of this study is to find out whether a medicine called spesolimab helps people with NS.
Participants are divided into a spesolimab and a placebo group. Placebo injections look like spesolimab injections but do not contain any medicine. Every participant has a 2 in 3 chance of being in the spesolimab group. In the beginning, participants get the study medicine as an injection into a vein. Afterwards, they get it as an injection under the skin every month. After 4 months, participants in the placebo group switch to spesolimab treatment.
Participants are in the study for about 1 year. During this time, they visit the study site 16 times. Where possible, 4 of 16 visits can be done at the participant's home instead of the study site. The doctors regularly check participants' NS symptoms. The results are compared between the groups to see whether spesolimab works. The doctors also regularly check participants' general health and take note of any unwanted effects.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Netherton Syndrome | Drug: Spesolimab - solution for infusion Drug: Placebo matching to spesolimab - solution for infusion Drug: Spesolimab - solution for injection Drug: Placebo matching to spesolimab - solution for injection | Phase 2 Phase 3 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 39 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | EvasayilTM : A Placebo-controlled Trial to Evaluate the Efficacy and Safety of Spesolimab in the Treatment of Patients With Netherton Syndrome |
| Actual Study Start Date : | May 15, 2023 |
| Estimated Primary Completion Date : | December 30, 2024 |
| Estimated Study Completion Date : | January 3, 2028 |
| Arm | Intervention/treatment |
|---|---|
| Placebo Comparator: Placebo |
Drug: Spesolimab - solution for infusion
Solution for infusion
Other Name: BI 655130 Drug: Placebo matching to spesolimab - solution for infusion Solution for infusion Drug: Spesolimab - solution for injection Solution for injection
Other Name: BI 655130 Drug: Placebo matching to spesolimab - solution for injection Solution for injection |
| Experimental: Spesolimab |
Drug: Spesolimab - solution for infusion
Solution for infusion
Other Name: BI 655130 Drug: Placebo matching to spesolimab - solution for infusion Solution for infusion Drug: Spesolimab - solution for injection Solution for injection
Other Name: BI 655130 |
- IASI response [ Time Frame: At baseline and at Week 16. ]Ichthyosis Area Severity Index (IASI) response is defined as a decrease of at least 50 % absolute change in IASI score from baseline at Week 16.
- Key secondary endpoint: IGA response [ Time Frame: at Week 16. ]Investigator Global Assessment (IGA)
- IGA response [ Time Frame: up to Week 12. ]Investigator Global Assessment (IGA)
- IASI response [ Time Frame: At baseline and up to Week 12. ]Ichthyosis Area Severity Index (IASI)
- IASI-E subscore response [ Time Frame: At baseline and up to Week 16. ]Ichthyosis Area Severity Index - Erythema (IASI-E)
- IASI-S subscore response [ Time Frame: At baseline and up to Week 16 ]Ichthyosis Area Severity Index - Scaling (IASI-S)
- Percent change from baseline in IASI score [ Time Frame: At baseline and up to Week 16. ]Ichthyosis Area Severity Index (IASI)
- Absolute change from baseline in NRS pain [ Time Frame: At baseline and up to Week 16. ]The Numeric Pain Rating Scale (NRS)
- Absolute change from baseline in NRS itch [ Time Frame: At baseline and up to Week 16. ]The Numeric Rating Scale (NRS)
- Absolute change from baseline in DLQI/CDLQI score [ Time Frame: At baseline and up to Week 16. ]Dermatology Life Quality Index (DLQI)/Children Dermatology Life Quality Index (CDLQI)
- The occurrence of treatment emergent adverse events including serious and/or opportunistic infections [ Time Frame: up to 172 weeks. ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 12 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Male or female patients, aged 12 years and older (weight minimum is 35kg).
- Confirmed diagnosis of Netherton syndrome (NS) (causative SPINK5 mutations) at baseline (Visit 2).
- At least moderate severity of erythema at baseline (visit 2) (Ichthyosis Area Severity Index (IASI) score ≥ 16 and IASI-Erythema (E) score ≥8) and ≥ 3 on Investigator Global Assessment (IGA) score.
- Signed and dated written informed consent and assent in accordance with International Council on Harmonisation-Good Clinical Practice (ICH-GCP) and local legislation prior to admission in the trial
- Women of childbearing potential (WOCBP) must be ready and able to use highly effective methods of birth control per ICH M3 (R2) that result in a low failure rate of less than 1% per year when used consistently and correctly. A list of contraception methods meeting these criteria is provided in the clinical trial protocol (CTP) as well as in the patient, parent(s) (or patient's legal guardian) information.
Exclusion Criteria:
- Patients who have used topical corticosteroids (medium to high, US class I-V), topical retinoids, topical calcineurin inhibitors or keratolytics within 1 week prior to randomisation
- Patients who have used emollient on the area to be biopsied in the previous 24 hours
- Patients who have used systemic retinoids, other systemic immunosuppressants, systemic corticosteroids or phototherapy within 4 weeks prior to randomisation
- Patients who have used systemic antibiotics within 2 weeks prior to randomisation
- Patients who have received live vaccines within 4 weeks prior to randomisation
- Patients who have received investigational products, biologics or immunoglobulins within 4 weeks or 5 half-lives (whichever is longer) prior to randomisation
- Severe, progressive, or uncontrolled hepatic disease, defined as >3-fold Upper Limit of Normal (ULN) elevation in Aspartate Aminotransferase (AST) or Alanine Aminotransferase (ALT) or alkaline phosphatase, or >2-fold ULN elevation in total bilirubin
- Patients who have any prior exposure to BI 655130 or another interleukin 36 receptor (IL-36R) inhibitor biologics
- Further exclusion criteria apply
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05856526
| Contact: Boehringer Ingelheim | 1-800-243-0127 | clintriage.rdg@boehringer-ingelheim.com |
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| Responsible Party: | Boehringer Ingelheim |
| ClinicalTrials.gov Identifier: | NCT05856526 |
| Other Study ID Numbers: |
1368-0104 2022-501104-10-00 ( Registry Identifier: CTIS (EU) ) U1111-1289-6825 ( Registry Identifier: WHO International Clinical Trials Registry Platform (ICTRP) ) |
| First Posted: | May 12, 2023 Key Record Dates |
| Last Update Posted: | May 14, 2024 |
| Last Verified: | May 2024 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | Once the criteria in section "Time Frame" are fulfilled, researchers can use the following link https://www.mystudywindow.com/msw/datasharing to request access to the clinical study documents regarding this study, and upon a signed "Document Sharing Agreement". Furthermore, researchers can request access to the clinical study data, for this and other listed studies, after the submission of a research proposal and according to the terms outlined in the website. |
| Supporting Materials: |
Study Protocol Statistical Analysis Plan (SAP) Clinical Study Report (CSR) |
| Time Frame: | After structured results have been posted, all regulatory activities are completed in the US and EU for the product and indication, and after the primary manuscript has been accepted for publication. |
| Access Criteria: | For study documents - upon signing of a 'Document Sharing Agreement'. For study data - 1. after the submission and approval of the research proposal (checks will be performed by the sponsor and/or the independent review panel, including checking that the planned analysis does not compete with sponsor's publication plan); 2. and upon signing of a legal agreement. |
| URL: | https://www.mystudywindow.com/msw/datasharing |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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Netherton Syndrome Syndrome Disease Pathologic Processes Abnormalities, Multiple Congenital Abnormalities Ichthyosiform Erythroderma, Congenital Ichthyosis |
Skin Abnormalities Skin Diseases, Genetic Genetic Diseases, Inborn Infant, Newborn, Diseases Keratosis Skin Diseases Pharmaceutical Solutions |