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A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital Ichthyosis

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
Sponsor:
Information provided by (Responsible Party):
Timber Pharmceuticals LLC

Brief Summary:

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

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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)
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




Primary Outcome Measures :
  1. 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


Secondary Outcome Measures :
  1. Reduction in overall Ichthyosis severity [ Time Frame: 12 weeks ]
    Investigator Global Assessment decrease in disease severity by at least 2 grades

  2. Patient Reported Outcomes [ Time Frame: 12 weeks ]
    Change in Itch-Numeric Rating Scale

  3. Patient Reported Outcomes [ Time Frame: 12 weeks ]
    Change in Dermatology Life Quality Index



Information from the National Library of Medicine

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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
Criteria

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

Information from the National Library of Medicine

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


Locations
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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
Sponsors and Collaborators
Timber Pharmceuticals LLC
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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

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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
Additional relevant MeSH terms:
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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