New ointment shows promise for rare skin condition

NCT ID NCT05295732

Disease control Sponsor: LEO Pharma Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This Phase 3 study tested an ointment called TMB-001 in 153 people aged 6 and older with X-linked or ARCI ichthyosis, conditions that cause dry, scaly, and cracked skin. Participants applied the ointment or a placebo daily for 12 weeks, with some continuing for maintenance. The goal was to see if TMB-001 could improve skin scaling and fissuring better than placebo.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance: TMB-001 0.05% ointment
What this could lead to: If successful, this could provide a new topical treatment option to reduce severe skin scaling and fissuring in people with inherited ichthyosis.
What could go wrong: This is a completed Phase 3 trial, but results are not yet published. The improvement may be modest, and some people may not respond. Side effects from the ointment are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

153 people

The number who actually took part.

Started

Jun 2022

Finished

Sep 2024

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

6 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: 1. Subject is male or female, 6 years of age and older at Visit 2 (Baseline). 2. Subject has provided written informed consent/assent. A subject under 18 years of age must provide written informed assent and be accompanied by the parent or legal guardian at the time of consent/assent signing. The parent or legal guardian must provide informed consent for the subject. If a subject becomes 18 years of age during the study, the subject must provide written informed consent at that time to continue study participation. 3. Females must be postmenopausal (defined as amenorrhea greater than 12 consecutive months in women 50 years of age and older), surgically sterile (hysterectomy, bilateral salpingectomy, or bilateral oophorectomy), or use 2 acceptable forms of birth control. WOCBP must have a negative serum pregnancy test at screening and negative urine pregnancy test (UPT) at Visit 2 (Baseline) (UPTs must have a minimum sensitivity to detect 25 mIU beta human chorionic gonadotropin \[β hCG\]/mL). Female subjects who become sexually active or begin to have relations with a partner during the study must agree to use 2 forms of birth control for 30 days prior to having relations and to continue such forms of birth control for the duration of the study. 4. Subject has clinical diagnosis of CI and has a genetic confirmation of either ARCI (including but not exclusively transglutaminase 1-deficient, ALOX-12B) or RXLI (e.g., deletion of steroid sulfatase gene) subtypes of CI. Other genetically confirmed ARCI-LI mutations can potentially be enrolled as long as the phenotype is consistent with ARCI and the other inclusion criteria are met, as determined by the Investigator. 5. The amount of CI affected skin in the Treatment Area at Baseline will be between a minimum of 10% and maximum of 90% of the total BSA (1% BSA is approximately equal to the surface area of the subject's palm and fingers, with the fingers extended yet grouped together, creating a flat oval-like surface area). • For the Optional Maximal Use arm: The amount of CI affected skin in the Treatment Area at Baseline will be between a minimum of 75% and maximum of 90% of the total BSA. 6. Documented history of moderate to severe disease at Screening. Subject's designated VIIS Assessment Areas at Baseline (not applicable for Optional Maximal Use arm): * Include any of the 4 VIIS Assessment Areas that have some CI disease involving: (a) the upper back from the posterior axillary fold to the other encompassing the T1-T10, (b) the upper arm (excluding elbows), left or right, (c) the shin/lower leg (the portion below the proximal aspect of the kneecap), left or right, and (d) dorsal foot (left or right); AND * At least 2 of the 4 VIIS Assessment Areas MUST have a scaling score of 3 or more. 7. Subject's IGA score in the Treatment Area at Baseline must be 3 or more. 8. Subject and parent/guardian (if applicable) are willing and able to apply the study treatment(s) as directed, comply with study instructions, and commit to all follow-up visits for the duration of the study. 9. Subject, in the Investigator's opinion, is in good general health and free of any disease state or physical condition that might impair evaluation of the Treatment Areas or exposes the subject to an unacceptable risk by study participation. Exclusion Criteria: 1. Subject is pregnant, lactating, or is planning to become pregnant during the study. 2. Subject has inflammatory skin diseases that confound the interpretation of results (e.g., atopic dermatitis) unrelated to ichthyosis. 3. Subject has genetic abnormality consistent with non-lamellar type or syndromic ichthyoses (including but not exclusively KRT1, KRT10, KRT2, GJB3, GJB4, CDSN) 4. Subject, in the Treatment Areas, has used: (a) any topical prescription or over-the-counter (OTC) therapies (except emollients, keratolytics, and topical steroids - see below), that are intended for, or that in the opinion of the Investigator, may improve CI within 2 weeks of Visit 2 (Baseline), or (b) keratolytics or topical corticosteroids within 5 days prior to Visit 2 (Baseline). 5. Subject, in the Treatment Areas, has used TMB-001 in the past or oral isotretinoin in the past 12 months (not applicable for Optional Maximal Use arm). 6. Subject has used any topical products in the Treatment Areas, including bland emollients, on Visit 2 (Baseline). 7. Subject has used ultraviolet (UV) treatment within 4 weeks prior to Visit 2 (Baseline). 8. Subject has undergone systemic therapies using vitamin A supplements or St. John's Wort within 4 weeks prior to Visit 2 (Baseline). Note: Use of a multivitamin including vitamin A is not exclusionary provided it is taken as directed on the packaging. 9. Subject is immunosuppressed (e.g., human immunodeficiency virus, systemic malignancy, graft host disease) or receives systemic immunotherapy. 10. Subject is currently taking concomitant immunosuppressive drugs, including systemic corticosteroids, within 2 weeks of Visit 2 (Baseline). 11. Subject has untreated secondary infections; however, subject may become eligible after successful treatment of his/her infection(s) at the Investigator's discretion. 12. Subject is currently enrolled in an investigational drug or device study or has used an investigational drug or investigational device treatment within 30 days or five half-lives prior to Visit 2 (Baseline). 13. Subject has lesions suspicious for skin cancer (if skin cancer is not ruled out by biopsy) or untreated skin cancers within the Treatment Areas. 14. Subject has a physical condition or other dermatologic disorder that, in the Investigator's opinion, might impair evaluation of CI, or that exposes the subject to unacceptable risk by study participation. 15. Subjects with ALT or AST \>2 x Upper Limit of Normal (ULN) and/or creatinine \>1.5 x ULN. 16. Subject is unable to communicate or cooperate with the Investigator due to language problems, impaired cerebral function, or physical limitations. 17. Subject has a history of drug or alcohol abuse within the past 6 months, or if suspected to be noncompliant or is unlikely to comply with the requirements of the study protocol (e.g., due to alcoholism, drug dependency, mental incapacity) in the opinion of the Investigator. 18. Subject has a history of sensitivity to any of the ingredients in the study treatments.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Autosomal recessive congenital ichthyosis are added.

Vår säkerhetsrekommendation!

Genom att skicka in godkänner du våra Användarvillkor

Conditions

The condition(s) this trial relates to.

autosomal recessive congenital ichthyosis ichthyosis inherited ichthyosis recessive X-linked ichthyosis

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Ichthyosis

Contacts and locations

Locations

About Skin Dermatology

Centennial, Colorado, 80111, United States
Ambulatorio di Malattie Rare Dermatologiche e Immunopatologia Cutanea

Florence, 50125, Italy
Ann & Robert H. Lurie Children's Hospital of Chicago

Chicago, Illinois, 60611, United States
Associated Skincare Specialists

New Brighton, Minnesota, 55112, United States
Austin Institute for Clinical Research

Houston, Texas, 77056, United States
Austin Institute for Clinical Research, Inc.

Pflugerville, Texas, 78660, United States
CHU de Nantes Hotel Dieu

Nantes, 44093, France
Charité - Universitätsmedizin Berlin

Berlin, 10117, Germany
Children's Hospital of Philadelphia (CHOP)

Philadelphia, Pennsylvania, 19104, United States
Dawes Fretzin Clinical Research Group, LLC

Indianapolis, Indiana, 46250, United States
Department of Dermatology and Cutaneous Surgery, University of Miami

Miami, Florida, 33136, United States
Dr. Chih-ho Hong Medical Inc.

Surrey, British Columbia, V3R 6A7, Canada
Fondazione IRCCS Ospedale Maggiore Policlinico Milano, Area Materno Infantile - SC pediatria Pneumoinfettivologia

Milan, Italy
Hopital Larrey CHU Toulouse

Toulouse, 31059, France
Hopital Necker APHP

Paris, 75015, France
Hôpital Femme Mère Enfant

Bron, 69677, France
Katholisches Kinderkrankenhaus Wilhelmstift GmbH

Hamburg, 22149, Germany
Medical University of South Carolina

Charleston, South Carolina, 29425, United States
Münster University Hospital

Münster, 48149, Germany
North Sound Dermatology

Mill Creek, Washington, 98012, United States
Optima Research

Boardman, Ohio, 44512, United States
SickKids Hospital

Toronto, Ontario, M5G 1X8, Canada
Stanford University School of Medicine

Palo Alto, California, 94304, United States
Stollery Children's Hospital

Edmonton, Alberta, T6G 2B7, Canada
The Indiana Clinical Trials Center

Plainfield, Indiana, 46168, United States
U.O. di Dermatologia Dipartimento di Medicina Specialistica, Diagnostica e Sperimentale

Bologna, 40138, Italy
U.O. di Dermatologia e Venereologia Universitaria

Bari, 70124, Italy
U.O.C. di Dermatologia Dipartimento Pediatrico Universitario Ospedaliero

Roma, 00165, Italy
U.S. Dermatology Partners

Phoenix, Arizona, 85006, United States
University of Mississippi Medical Center (UMMC)

Jackson, Mississippi, 39216, United States
Universitätsklinikum Erlangen

Erlangen, 91054, Germany
Virginia Clinical Research-Pariser Dermatology Specialists

Norfolk, Virginia, 23502, United States
Wake Forest University Health Sciences

Winston-Salem, North Carolina, 27104, United States
Wiseman Dermatology Research Inc.

Winnipeg, Manitoba, R3M 3Z4, Canada
Yale Center for Clinical Investigation

New Haven, Connecticut, 06519, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.