![Timber Pharmceuticals LLC](https://trials-media.s3.amazonaws.com/orgs/timber-pharmceuticals-llc.jpeg)
The ASCEND Study: Evaluating TMB-001 in the Treatment of RXLI or ARCI Ichthyosis
IchthyosisThis is a randomized, double-blind and vehicle-controlled Phase III study to evaluate the safety and efficacy of topical TMB-001 0.05% ointment for the treatment of CI in subjects with either the RXLI or ARCI subtypes. In addition, a subset of preselected centers will recruit subjects in parallel with either the RXLI or ARCI subtypes for enrollment into an Optional Maximal Use arm for evaluation of the systemic exposure and safety of topical TMB-001 0.05% ointment for the treatment of CI. The Phase III Study is designed in three periods: • Period 1 - Induction (3 weeks): At the beginning of the 3-week Induction Period, eligible subjects will be randomized (2:1 ratio) to either TMB-001 0.05% once-a-day (QD) or Vehicle QD treatment, with use of mandatory standardized bland emollient (Cetaphil™) provided by the Sponsor. • Period 2 - Treatment (9 weeks): The dosing frequency in the 9-week treatment period will be increased in each treatment group to TMB-001 0.05% BID or Vehicle BID. Mandatory bland emollient will be discontinued. • Period 3 - Maintenance (12 weeks): At Week 12, eligible subjects in the TMB-001 treatment group will be randomized (1:1 ratio) to an open-label treatment with TMB-001 0.05% BID or TMB-001 0.05% QD. To be eligible, subjects must have achieved a ≥1-point reduction in IGA score from Baseline. Subjects with less than a 1-point reduction in IGA score from Baseline will be discontinued from the study. Vehicle-treated subjects who achieved <1-point reduction in IGA score from Baseline are eligible to cross over to the TMB-001 0.05% BID treatment group. Subjects with a ≥1-point reduction in IGA score from Baseline will be discontinued from the study. Subjects at the end of the study or subjects discontinued from the study at any time will be followed-up for additional 2 weeks for AEs.
![Northwestern University](https://trials-media.s3.amazonaws.com/orgs/northwestern-university.jpeg)
An Open-Label and Long-Term Extension Study to Evaluate the Efficacy and Safety of Ustekinumab in...
IchthyosisThe ichthyoses are a group of lifelong genetic disorders that 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 more common "orphan" forms of ichthyosis are autosomal recessive congenital ichthyosis (ARCI; includes lamellar ichthyosis/LI and congenital ichthyosiform erythroderma/CIE), Netherton syndrome (NS) and epidermolytic ichthyosis (EI). However, there are dozens of other syndromic and non-syndromic ichthyotic disorders as well. Therapy is time-consuming for patients or parents and is supportive, focusing on clearance of the scaling. There are no therapies based on our growing understanding of what causes the disease. We have recently found marked elevations in Th17/IL-23 pathway cytokines and chemokines in the skin of individuals with ichthyosis, most similar to the inflammatory pattern of psoriasis. While the significance of the high expression of Th17/IL-23 pathway genes across all forms of ichthyosis studied to date is unknown, the high expression of genes of the Th17/IL-23 pathway in psoriasis is thought to be causative for the disease manifestations. We propose that IL-12/IL-23 -targeting therapeutics will safely suppress the inflammation and possibly the other features of ichthyosis, improving quality of life. As a proof-of-concept study, we propose to treat children (6 years of age and higher) and adults with ichthyotic disorders with ustekinumab in an open-label trial to serially assess clinical response to and safety of ustekinumab for this group of disorders.
![Krystal Biotech, Inc.](https://trials-media.s3.amazonaws.com/orgs/krystal-biotech-inc.jpeg)
Topical KB105 Gene Therapy for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis...
TGM-1 Related Autosomal Recessive Congenital IchthyosisThis study is an intra-patient comparison of KB105 and placebo-administered Target Areas. The primary objectives of this study are to evaluate safety and Investigator Global Assessment (IGA) scale improvement of topically administered KB105.
![University Hospital, Toulouse](https://trials-media.s3.amazonaws.com/orgs/university-hospital-toulouse.jpeg)
Evaluate the Evolution of Body and Scalp Skin Discomfort in Patients With Hereditary Ichthyosis...
Ichthyosis InheritedIchthyosis is a group of rare and chronic genetic diseases beginning at birth, in which the patient's skin is covered with scales of variable appearance and severity. This disease is disabling, in particular due to sensations of pruritus, skin pain and the possible presence of associated extra-cutaneous abnormalities. The treatment is symptomatic and is based on the daily application of moisturizing creams with a high lipid content for better effectiveness, keratolytic creams, and for severe forms, oral retinoids may be necessary. Nevertheless, the effectiveness of these treatments is limited and are considered very painful by the patients. Wraps are local treatments carried out by specialized nurses and which consist in, following a therapeutic bath, applying a large quantity of cream to the whole body, followed by an occlusion. This type of care is simple to perform, but requires nursing expertise, to date, it is not carried out in standard practice in France, which is why we wish to evaluate the effect of wraps on different parameters reflecting the skin condition in the short and medium term.
![Krystal Biotech, Inc.](https://trials-media.s3.amazonaws.com/orgs/krystal-biotech-inc.jpeg)
Topical KB105 for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)...
Autosomal Recessive IchthyosisKB105-02 is an intrasubject randomized, placebo-controlled, double-blind study to evaluate the safety and efficacy of KB105 in children and adults with autosomal recessive congenital ichthyosis (ARCI).
![Maastricht University Medical Center](https://trials-media.s3.amazonaws.com/orgs/maastricht-university-medical-center.jpeg)
Quality of Life in Middle-aged and Older Patients With Congenital Ichthyosis
Congenital IchthyosisThe goal of this observational study is to learn about the quality of life in patients with congenital ichthyosis. The main question it aims to answer are: • What is the impact of ichthyosis on the biological-psychological-social quality of life in adult and elderly patients with inherited ichthyosis ? Participants will take part in individual interviews in which the investigators will explore if and how ichthyosis impacts their quality of life. Examples of such questions are whether participants experience pain, experience struggles in their personal or professional lives, with sports activities or if participants experience a financial burden.
![Northwestern University](https://trials-media.s3.amazonaws.com/orgs/northwestern-university.jpeg)
Defining the Skin and Blood Biomarkers of Ichthyosis
IchthyosisNetherton SyndromeIchthyosis is a group of genetic skin disorders that present with dry, thickened, scaly, or flaky skin. As of today, there is no cure or treatment. Doctors can only treat the dry skin with different types of emollients to soften the scale. A deeper understanding of this disease is required to develop better treatments. There are different types of cells and cell-produced signals (biomarkers) that are being studied in order to help find these new treatments. Looking at biomarkers has been successful in helping us to understand other skin disorders better. The purpose of this study is to determine which blood and skin biomarkers characterize ichthyosis. Hypothesis: We predict that the biomarkers correlating with disease activity in Netherton syndrome will be different than the biomarkers found to correlate with the lamellar and other ichthyosis phenotype.
![Assistance Publique - Hôpitaux de Paris](https://trials-media.s3.amazonaws.com/orgs/assistance-publique-hopitaux-de-paris.jpeg)
Study of in Vivo and in Vitro Transcriptomic and Proteomic Signatures in Unhereditary Ichtyosis...
Autosomal Recessive Congenital IchthyosisEpidermolytic IchthyosisThe goal of this study is to identify important biological pathways involved in a variety of ichtyosis, using transcriptomic and proteomic techniques, with the aim of guiding the development of new therapeutis.
![AnaptysBio, Inc.](https://trials-media.s3.amazonaws.com/orgs/anaptysbio-inc.jpeg)
A Study to Evaluate the Efficacy and Safety of Imsidolimab (ANB019) in the Treatment of Participants...
IchthyosisEfficacy and Safety of imsidolimab in Participants with Ichthyosis
![Mayne Pharma International Pty Ltd](https://trials-media.s3.amazonaws.com/orgs/mayne-pharma-international-pty-ltd.jpeg)
A Safety, Efficacy and Systemic Exposure Study of CD5789 Cream in Adults and Adolescents With Lamellar...
Lamellar IchthyosisThis is a phase 2 randomized, multi-center, double-blind, vehicle controlled, 90 day, safety, efficacy, and systemic exposure study followed by a 90 day open-label extension of trifarotene cream in adults and adolescents with autosomal recessive ichthyosis with lamellar scale.