A study to test whether spesolimab helps people with skin disease called Netherton syndrome

JRCT ID: jRCT2031220722

Registered date:21/03/2023

Basic Information

• Recruitment status: Recruiting
• Health condition(s) or Problem(s) studied: Netherton Syndrome
• Date of first enrollment: 01/05/2023
• Target sample size: 39
• Countries of recruitment: Australia,Japan,Austria,Japan,Belgium,Japan,Bulgaria,Japan,China,Japan,Czech Republic,Japan,Finland,Japan,France,Japan,Germany,Japan,Israel,Japan,Italy,Japan,Malaysia,Japan,Netherlands,Japan,Portugal,Japan,Spain,Japan,Switzerland,Japan,United Kingdom,Japan,United States,Japan
• Study type: Interventional

Intervention(s)

Active arm: spesolimab i.v. and s.c. dose
Placebo arm: placebo matching speolimab i.v. and s.c. dose

Outcome(s)

• Primary Outcome: IASI response, defined as a decrease of at least 50 % absolute change in IASI score from baseline at Week 16 (Yes/No).

Secondary Outcome

[Key Secondary]
IGA response, defined as IGA score of 0 or 1 at Week 16 (Yes/No)
[Efficacy]
1) IGA response, defined as IGA score of 0 or 1 at Weeks 4, 8, 12 (Yes/No)
2) Occurrence of bacterial or fungal mucocutaneous infection through Week 16
3) IASI response, defined as a decrease of at least 50 % absolute change in IASI score from baseline at Weeks 4, 8, 12 (Yes/No)
4) IASI subscore response, defined as a decrease of at least 50 % absolute change in IASI-E subscore AND a decrease of at least 50 % absolute change in IASI-S subscore from baseline at Weeks 4, 8, 12 (Yes/No)
5) Percent change from baseline in IASI score at Weeks 4, 8, 12 and 16
6) Percent change from baseline in NASA score at Weeks 4, 8, 12 and 16
7) Absolute change from baseline in NRS pain at Weeks 4, 8, 12 and 16
8) Absolute change from baseline in NRS itch at Weeks 4, 8, 12 and 16
9) Absolute change from baseline in DLQI/CDLQI score at Weeks 8 and 16
[Safety]
1) The occurrence of treatment emergent adverse events

Key inclusion & exclusion criteria

• Age minimum: >= 12age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1) Male or female patients, aged 12 years and older (weight minimum is 35 kg)
2) Confirmed diagnosis of NS (SPINK5 causative mutations) at baseline
3) At least moderate severity of erythema at baseline (IASI score morethan equal to 16 and IASI-E score morethan equal to 8) and more than equal to 3 on IGA score
4) Signed and dated written informed consent and assent in accordance with ICH-GCP and local legislation prior to admission in the trial
5) 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 percent per year when used consistently and correctly. A list of contraception methods meeting these criteria is provided in the CTP as well as in the patient, parent(s) (or patient's legal guardian) information.

Exclude criteria

1) 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
2) Patients who have used emollient on the area to be biopsied in the previous 24 hours
3) Patients who have used systemic retinoids, other systemic immunosuppressants, systemic corticosteroids or phototherapy within 4 weeks prior to randomisation
4) Patients who have used systemic antibiotics within 2 weeks prior to randomisation
5) Patients who have received live vaccines within 4 weeks prior to randomisation
6) Patients who have received investigational products, biologics or immunoglobulins within 4 weeks or 5 half-lives (whichever is longer) prior to randomisation
Relevant chronic or acute infections including active tuberculosis, human immunodeficiency virus (HIV) infection or viral hepatitis at the time of randomisation. A patient can be re-screened if the patient was treated and is cured from the acute infection

Related Information

• Primary Sponsor: Taguchi Aya
• Secondary ID(s): NCT05856526