A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis
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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. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT04154293 |
Recruitment Status :
Completed
First Posted : November 6, 2019
Last Update Posted : October 5, 2021
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The purpose of this study is to investigate the efficacy and safety of two concentrations of topically applied ointment formulation of isotretinoin called TMB-001 (0.05% and 0.1% isotretinoin) in subjects 9 years of age and older for the treatment of congenital ichthyosis (CI), including recessive X-linked ichthyosis (RXLI) and autosomal recessive congenital ichthyosis-lamellar ichthyosis (ARCI-LI) subtypes.
Funding Source FDA-OOPD
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Congenital Ichthyosis | Drug: Isotretinoin Other: Vehicle | Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 34 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Intervention Model Description: | A Randomized, Parallel, Double-Blind, Vehicle-Controlled Study |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Masking Description: | Double-blind |
Primary Purpose: | Treatment |
Official Title: | A Randomized, Parallel, Double-Blind, Vehicle Controlled Study to Evaluate the Safety and Efficacy of Two Concentrations of Topical TMB-001 for the Treatment of Congenital Ichthyosis |
Actual Study Start Date : | December 3, 2019 |
Actual Primary Completion Date : | August 20, 2021 |
Actual Study Completion Date : | August 30, 2021 |

Arm | Intervention/treatment |
---|---|
Placebo Comparator: Vehicle Ointment (Control)
Topical, BID (Twice daily)
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Other: Vehicle
Topical Vehicle Ointment |
Experimental: TMB-001 Ointment, 0.05%
Topical, BID ( twice daily)
|
Drug: Isotretinoin
Topical Isotretinoin ointment |
Experimental: TMB-001 Ointment, 0.1%
Topical, BID (Twice daily)
|
Drug: Isotretinoin
Topical Isotretinoin ointment |
- Reduction in targeted Ichthyosis severity [ Time Frame: 12 weeks ]Proportion of subjects with Visual Index for Ichthyosis Severity (VIIS) treatment success, defined as 50% or greater decreased in VIIS scaling score
- Reduction in overall Ichthyosis severity [ Time Frame: 12 weeks ]Investigator Global Assessment decrease in disease severity by at least 2 grades
- Patient Reported Outcomes [ Time Frame: 12 weeks ]Change in Itch-Numeric Rating Scale
- Patient Reported Outcomes [ Time Frame: 12 weeks ]Change in Dermatology Life Quality Index

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Ages Eligible for Study: | 9 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Subject is male or female, 9 years of age or older at Visit 2 (Baseline)
- Subject has provided written informed consent
- Females of child bearing potential must be surgically sterile or agree to 2 forms of birth control
- Subject has clinical diagnosis of Congenital Ichthyosis with genetic confirmation of subtype
- Subject has between 10% and 90% total BSA affected by Congenital Ichthyosis
- Subject has at least 2 VIIS assessment areas with a scaling score of 3 or greater
Exclusion Criteria:
- Subject has inflammatory skin diseases unrelated to ichthyosis
- Subject has used other prohibited topical treatments in the assessment areas within certain days from baseline
- Subject has used systemic retinoids within12 weeks of baseline
- Subject has untreated secondary infections
- Subject has lesions suspicious for skin cancer or untreated skin cancers

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): NCT04154293
United States, Arizona | |
Medical Dermatology Specialists | |
Phoenix, Arizona, United States, 85006 | |
United States, California | |
Stanford University School of Medicine | |
Palo Alto, California, United States, 94304 | |
United States, Connecticut | |
Yale Center for Clinical Investigation | |
New Haven, Connecticut, United States, 06510 | |
United States, Indiana | |
The Indiana Clinical Trials Center | |
Plainfield, Indiana, United States, 46168 | |
United States, Louisiana | |
Austin Institute for Clinical Research, Inc | |
Metairie, Louisiana, United States, 70005 | |
United States, Minnesota | |
Minnesota Clinical Study Center | |
New Brighton, Minnesota, United States, 55112 | |
United States, Ohio | |
Cincinnati Children's Hospital | |
Cincinnati, Ohio, United States, 45229 | |
United States, Pennsylvania | |
The Children's Hospital of Philadelphia | |
Philadelphia, Pennsylvania, United States, 19140 | |
Australia, Queensland | |
Queensland Children's Hospital | |
Brisbane, Queensland, Australia, 4101 | |
Australia, Sydney | |
Premier Specialists, The church | |
Kogarah, Sydney, Australia, NSW 2217 | |
Australia, Victoria | |
Murdoch Children's Research Institute, RCH | |
Melbourne, Victoria, Australia, 3052 |
Responsible Party: | Timber Pharmceuticals LLC |
ClinicalTrials.gov Identifier: | NCT04154293 |
Other Study ID Numbers: |
235-9051-202 1R01FD006079-01A1 ( U.S. FDA Grant/Contract ) |
First Posted: | November 6, 2019 Key Record Dates |
Last Update Posted: | October 5, 2021 |
Last Verified: | October 2021 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Product Manufactured in and Exported from the U.S.: | No |
Ichthyosis Ichthyosis, Lamellar Ichthyosiform Erythroderma, Congenital Skin Abnormalities Congenital Abnormalities Infant, Newborn, Diseases |
Keratosis Skin Diseases Skin Diseases, Genetic Genetic Diseases, Inborn Isotretinoin Dermatologic Agents |