IchthyosisPhase 2NCT03041038
The Efficacy and Safety of Secukinumab in Patients With Ichthyoses
A Phase 2 study testing Secukinumab in people with Ichthyosis and Autosomal Recessive Congenital Ichthyosis, run by Northwestern University at 2 sites in United States.
Trial summary
The ichthyoses are a group of lifelong genetic disorders which share characteristics of generalized skin thickening, scaling and underlying cutaneous inflammation. There are no therapies based on growing understanding of what causes the disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Ichthyosis, Autosomal Recessive Congenital Ichthyosis, Lamellar Ichthyosis
- What is being tested?
- Secukinumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 20 participants
- Where
- 2 sitesUnited States
- Study dates
- Dec 2016to 31 Aug 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Subject has provided informed consent
- Subjects are at least 18 years of age or older at the time of screening
- Female subjects must not be pregnant or breast-feeding
- Female subjects of child-bearing potential with a negative urine pregnancy test and using at least one form of contraception (abstinence allowed)
- Subjects must have a confirmed diagnosis of ARCI (divided phenotypically into ARCI-LI or ARCI-CIE), EI or NS (by genotype or willingness to be genotyped)
- Subjects must be clinically judged to be immunocompetent.
Show all 9Show fewer
- Subjects will have no allergy to secukinumab or components of the product.
- Subjects will have normal baseline laboratory testing (CMP, CBC, HIV negative, hepatitis B, C negative, QuantiFERON®-TB gold negative)
- Subjects must have an erythema score of at least 18 on IASI and an IASI-E score of 12 (at least moderate severity of erythema) at baseline
Who cannot join
- Subjects who are unable to give informed consent or assent.
- Subjects without a confirmed diagnosis ARCI, EI, or NS.
- Subjects who have a known allergy to secukinumab.
- Female subjects who are pregnant, considering becoming pregnant, or will breastfeed.
- Subjects who have prior biologic use targeting IL-17A/IL-17 receptor A or IL-12/IL-23 or who have prior use of TNF-alpha blockers.
- Subjects who have used a systemic retinoid within one month prior to initiation.
- Subjects who have used topical retinoids or keratolytics within one week prior to initiation.
- Subjects who have used emollient on the area to be biopsied in the previous 24 hours
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Department of Dermatology, Northwestern University Feinberg School of Medicine
Chicago, Illinois, 60611
Department of Dermatology Icahn School of Medicine at Mount Sinai
New York, New York, 10029
About this study
Official title: A Multicenter Study With a Randomized, Double-Blind, Placebo-Controlled Period, Followed by an Open-Label Maintenance Dosing Period to Evaluate the Efficacy and Safety of Secukinumab in Patients With Ichthyoses
The ichthyoses are a group of lifelong genetic disorders which share characteristics of generalized skin thickening, scaling and underlying cutaneous inflammation. There are no therapies based on growing understanding of what causes the disease. However, there have been recent discoveries of marked elevations in expression of interleukin-17A (IL-17A) and IL-17-related cytokines in the skin of individuals with ichthyosis, which may explain the inflammation. Investigators propose that IL-17-targeting therapeutics will safely suppress the inflammation and possibly the other features of ichthyosis, improving quality of life.
Read the detailed description
The ichthyoses are a group of lifelong genetic disorders which share characteristics of generalized skin thickening, scaling and underlying cutaneous inflammation. The vast majority are orphan disorders and are associated with extremely poor quality of life related to social ostracism from altered appearance, associated itchiness and discomfort, and functional limitations from the skin disease. Among the most common of these orphan disorders are autosomal recessive congenital ichthyosis (ARCI) with its phenotypic subsets of lamellar ichthyosis (ARCI-LI) and congenital ichthyosiform erythroderma (ARCI-CIE), epidermolytic ichthyosis (EI) and Netherton syndrome (NS). Therapy is time-consuming for patients or parents and is supportive, focusing on clearance of the scaling. There are no therapies based on growing understanding of what causes the disease. There have been recent discoveries of marked elevations in expression of interleukin-17A (IL-17A) and IL-17-related cytokines in the skin of individuals with ichthyosis, which may explain the inflammation. Psoriasis, another inflammatory skin disorder with redness and scaling, has now been shown to result from IL-17 pathway activation and IL-17A inhibition is the most effective therapy known to treat psoriasis. Investigators propose that IL-17-targeting therapeutics will safely suppress the inflammation and possibly the other features of ichthyosis, improving quality of life. In this long-term, open-label extension, Investigators propose to treat adults with ichthyosis and at least moderate erythema with subcutaneously administered anti-IL-17 antibody (secukinumab) and to serially assess clinical response to this therapy and its safety.
- Conditions
- Ichthyosis, Autosomal Recessive Congenital Ichthyosis, Lamellar Ichthyosis, Congenital Ichthyosiform Erythroderma, Epidermolytic Ichthyosis, Netherton Syndrome
- Being tested
- Secukinumab, Placebo
- Medical subject headings
- Ichthyosis, Ichthyosis, Lamellar, Ichthyosiform Erythroderma, Congenital, Hyperkeratosis, Epidermolytic, Netherton Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SecukinumabDrug
Also called Cosentyx
Anti IL-17A antibody
PlaceboDrug
Also called Sterile Saline
| Study group | Role | What they receive |
|---|---|---|
| Secukinumab | Experimental | Secukinumab 300mg (liquid formation) administered subcutaneously weekly for 5 weeks then monthly until end of trial Receives: Drug: Secukinumab |
| Placebo | Placebo comparator | Placebo (sterile saline) 2ml administered subcutaneously weekly for 5 weeks then monthly until end of trial Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Reduction at Week 16 in the Ichthyosis Area Severity Index (IASI)
Primary Efficacy Endpoint. The IASI score was modelled after the Eczema Area and Severity Index (EASI) and Psoriasis Area and Severity Index (PASI), commonly used in clinical trials for atopic dermatitis and psoriasis, respectively. This scale measures erythema and scaling and has a range of 0-48 (sum of a max score of 24 for erythema and 24 for scaling). A higher score means worse clinical severity. Mean difference IASI total score at Baseline was compared to IASI total score at Week 16.
Measured: 16 Weeks
Total Number of Bacterial or Fungal Mucocutaneous Infections Through Week 16
Primary Safety Endpoint
Measured: 16 weeks of secukinumab/placebo double blind followed by 32 week open label treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Feb 2017 (estimated)
- Study startDec 2016
- Primary completion31 Aug 2020
- Study completion31 Aug 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for IchthyosisStudies of the same condition, open and under way.
- More trials run by Northwestern UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Lead sponsor
- Northwestern University
- Sponsor type
- University, hospital or other
- Collaborators
- Icahn School of Medicine at Mount Sinai
- Responsible party
- Principal investigator, Amy Paller, Northwestern University
- Investigators
- Amy Paller, MD, Principal investigator, Northwestern University Department of DermatologyEmma Guttman-Yassky, MD, PhD, Principal investigator, Mt. Sinai Hospital Department of Dermatology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03041038
- Sponsor's protocol ID
- CAIN457AUS05T
- Status last verified
- Aug 2021
- First submitted
- 17 Jan 2017
- Last updated
- 25 Aug 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Lefferdink R, Rangel SM, Chima M, Ibler E, Pavel AB, Kim H, Wu B, Abu-Zayed H, Wu J, Jackson K, Singer G, Choate KA, Guttman-Yassky E, Paller AS. Secukinumab responses vary across the spectrum of congenital ichthyosis in adults. Arch Dermatol Res. 2023 Mar;315(2):305-315. doi: 10.1007/s00403-022-02325-3. Epub 2022 Feb 26.
PubMed 35218370
Study at a glance
From the registry record, last updated by the sponsor on 25 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter Study With a Randomized, Double-Blind, Placebo-Controlled Period, Followed by an Open-Label Maintenance Dosing Period to Evaluate the Efficacy and Safety of Secukinumab in Patients With Ichthyoses
- ClinicalTrials.gov ID
- NCT03041038
- Acronym / sponsor ID
- CAIN457AUS05T
- Conditions
- Ichthyosis, Autosomal Recessive Congenital Ichthyosis, Lamellar Ichthyosis, Congenital Ichthyosiform Erythroderma, Epidermolytic Ichthyosis, Netherton Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Northwestern UniversityUniversity, hospital or other
- Collaborators
- Icahn School of Medicine at Mount Sinai
- Study start
- Dec 2016
- Primary completion
- 31 Aug 2020 (actual)
- Study completion
- 31 Aug 2020 (actual)
- First posted
- 2 Feb 2017 (estimated)
- Results
- Posted 25 Aug 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The ichthyoses are a group of lifelong genetic disorders which share characteristics of generalized skin thickening, scaling and underlying cutaneous inflammation. There are no therapies based on growing understanding of what causes the disease. However, there have been recent discoveries of marked elevations in expression of interleukin-17A (IL-17A) and IL-17-related cytokines in the skin of individuals with ichthyosis, which may explain the inflammation. Investigators propose that IL-17-targeting therapeutics will safely suppress the inflammation and possibly the other features of ichthyosis, improving quality of life.
Detailed description
The ichthyoses are a group of lifelong genetic disorders which share characteristics of generalized skin thickening, scaling and underlying cutaneous inflammation. The vast majority are orphan disorders and are associated with extremely poor quality of life related to social ostracism from altered appearance, associated itchiness and discomfort, and functional limitations from the skin disease. Among the most common of these orphan disorders are autosomal recessive congenital ichthyosis (ARCI) with its phenotypic subsets of lamellar ichthyosis (ARCI-LI) and congenital ichthyosiform erythroderma (ARCI-CIE), epidermolytic ichthyosis (EI) and Netherton syndrome (NS). Therapy is time-consuming for patients or parents and is supportive, focusing on clearance of the scaling. There are no therapies based on growing understanding of what causes the disease. There have been recent discoveries of marked elevations in expression of interleukin-17A (IL-17A) and IL-17-related cytokines in the skin of individuals with ichthyosis, which may explain the inflammation. Psoriasis, another inflammatory skin disorder with redness and scaling, has now been shown to result from IL-17 pathway activation and IL-17A inhibition is the most effective therapy known to treat psoriasis. Investigators propose that IL-17-targeting therapeutics will safely suppress the inflammation and possibly the other features of ichthyosis, improving quality of life. In this long-term, open-label extension, Investigators propose to treat adults with ichthyosis and at least moderate erythema with subcutaneously administered anti-IL-17 antibody (secukinumab) and to serially assess clinical response to this therapy and its safety.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Secukinumab | Experimental | Drug: SecukinumabSecukinumab 300mg (liquid formation) administered subcutaneously weekly for 5 weeks then monthly until end of trial |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo (sterile saline) 2ml administered subcutaneously weekly for 5 weeks then monthly until end of trial |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Subject has provided informed consent
- Subjects are at least 18 years of age or older at the time of screening
- Female subjects must not be pregnant or breast-feeding
- Female subjects of child-bearing potential with a negative urine pregnancy test and using at least one form of contraception (abstinence allowed)
- Subjects must have a confirmed diagnosis of ARCI (divided phenotypically into ARCI-LI or ARCI-CIE), EI or NS (by genotype or willingness to be genotyped)
Key exclusion criteria (8 listed)
- Subjects who are unable to give informed consent or assent.
- Subjects without a confirmed diagnosis ARCI, EI, or NS.
- Subjects who have a known allergy to secukinumab.
- Female subjects who are pregnant, considering becoming pregnant, or will breastfeed.
- Subjects who have prior biologic use targeting IL-17A/IL-17 receptor A or IL-12/IL-23 or who have prior use of TNF-alpha blockers.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SecukinumabAlso: CosentyxAnti IL-17A antibody | Drug | Secukinumab |
| PlaceboAlso: Sterile Saline | Drug | Placebo |
Primary outcome
Reduction at Week 16 in the Ichthyosis Area Severity Index (IASI)
Time frame: 16 Weeks
Primary Efficacy Endpoint. The IASI score was modelled after the Eczema Area and Severity Index (EASI) and Psoriasis Area and Severity Index (PASI), commonly used in clinical trials for atopic dermatitis and psoriasis, respectively. This scale measures erythema and scaling and has a range of 0-48 (sum of a max score of 24 for erythema and 24 for scaling). A higher score means worse clinical severity. Mean difference IASI total score at Baseline was compared to IASI total score at Week 16.
Total Number of Bacterial or Fungal Mucocutaneous Infections Through Week 16
Time frame: 16 weeks of secukinumab/placebo double blind followed by 32 week open label treatment
Primary Safety Endpoint
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Aug 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 35218370Lefferdink R, Rangel SM, Chima M, Ibler E, Pavel AB, Kim H, Wu B, Abu-Zayed H, Wu J, Jackson K, Singer G, Choate KA, Guttman-Yassky E, Paller AS. Secukinumab responses vary across the spectrum of congenital ichthyosis in adults. Arch Dermatol Res. 2023 Mar;315(2):305-315. doi: 10.1007/s00403-022-02325-3. Epub 2022 Feb 26.
Related trials for Ichthyosis
Active, not recruitingPhase 1
A Study of the Safety, Tolerability, Pharmacokinetics, and Immunogenicity of BCX17725
Netherton Syndrome
Not yet recruiting
Acupuncture Combined With Transcutaneous Electrical Acupoint Stimulation for Constipation-Predominant Irritable Bowel Syndrome With Anxiety State
IBS, Constipation Predominant, Anxiety State
RecruitingObservational study
Characterization of Diseases With Salivary Gland Involvement
Healthy Volunteer, Sjogren's Syndrome
RecruitingPhase 2/3
Phase 2/3 Clinical Study of QRX003 Lotion in Subjects With Netherton Syndrome
Netherton Syndrome
Recruiting
HPTC Versus AFSM in Diabetic Foot Ulcers
Diabetic Foot Ulcer (DFU)
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Amy Paller, MD principal investigator
Northwestern University Department of Dermatology
Emma Guttman-Yassky, MD, PhD principal investigator
Mt. Sinai Hospital Department of Dermatology
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Secukinumab in people with Ichthyosis and Autosomal Recessive Congenital Ichthyosis, run by Northwestern University at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Aug 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Reduction at Week 16 in the Ichthyosis Area Severity Index (IASI) (time frame: 16 Weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03041038, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Aug 2021.