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Topical KB105 Gene Therapy for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)

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: NCT04047732
Recruitment Status : Active, not recruiting
First Posted : August 7, 2019
Last Update Posted : September 13, 2021
Information provided by (Responsible Party):
Krystal Biotech, Inc.

Brief Summary:
This 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.

Condition or disease Intervention/treatment Phase
TGM-1 Related Autosomal Recessive Congenital Ichthyosis Biological: KB105 Phase 1 Phase 2

Detailed Description:
Up to six adult subjects are planned for the Phase I portion of this study. Subjects are enrolled upon obtaining consent and meeting entry criteria. This study is an intra-patient comparison of KB105 and placebo-administered Target Areas. Patients will be evaluated for safety, and Target Areas will be assessed individually with the IGA and VIIS scales. Target Areas will be imaged and evaluated for safety and efficacy. Subjects will be on-trial for approximately 3.5 months.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I/II Clinical Trial of Topical KB105, a Replication-incompetent, Non-integrating HSV-1 Vector Expressing Human Transglutaminase 1 (TGM1) for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
Actual Study Start Date : August 27, 2019
Estimated Primary Completion Date : October 2022
Estimated Study Completion Date : March 2025

Arm Intervention/treatment
Experimental: Topical KB105
HSV1-TGM1 vector (KB105)
Biological: KB105
KB105, a replication-incompetent, non-integrating HSV-1 vector expressing human transglutaminase 1 (TGM1) formulated as a topical gel
Other Name: HSV1-TGM1

Primary Outcome Measures :
  1. Safety and tolerability [ Time Frame: Up to 12 weeks ]
    Incidence of treatment-emergent adverse events

  2. Investigator's Global Assessment (IGA) of disease severity [ Time Frame: Up to 12 weeks ]
    Improvement of disease severity in the treatment area assessment through Investigator's Global Assessment (IGA)

Secondary Outcome Measures :
  1. Visual Index for Ichthyosis Severity (VIIS) scale, lamellar (L) standard [ Time Frame: Up to 12 weeks ]
    Improvement of disease severity in the treatment area through use of the Visual Index for Ichthyosis Severity scale, lamellar (VIIS-L) standard assessment. The VIIS-L is a 4-point visual scale with 1 representing normal skin and 5 representing severe ichthyosis.

  2. Immunofluorescence microscopy [ Time Frame: Up to 12 weeks ]
    Level of transglutaminase 1 in KB105-administered skin as measured by immunofluorescence microscopy

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Genetic diagnosis of TGM1-deficient ARCI with a null TGM-1 mutation;
  • Clinical diagnosis of lamellar ichthyosis;
  • Age: 18 years old or older;
  • Individual site IGA score of 3 to 4 at the target areas
  • Subject is, in the opinion of the Investigator, able to understand thestudy, cooperate with the study procedures, and is willing to return to the clinic for all required follow-up visits;
  • Except for their moderate-to-severe ARCI, subject is in good general health; and
  • Willing and able to give consent/assent.

Exclusion Criteria:

  • Medical instability limiting ability to travel to the investigative center;
  • Medical illness expected to complicate participation, such as an active infection with: HIV, hepatitis B (as determined by hepatitis B surface antigen screening), hepatitis C (as determined by detection of hepatitis C antibodies or a positive result of hepatitis C);
  • Patient has a physical condition or other dermatological disorder (e.g., atopic, seborrheic, or contact dermatitis, psoriasis, tinea infections, etc.) which, in the Investigator's opinion, might impair evaluation of the Target Areas or which exposes the patient to unacceptable risk by study participation;
  • Pregnant or breast-feeding women, or women planning to become pregnant or to breast-feed. Women of childbearing potential must have a negative urine pregnancy test at the Screening visit and Day 1 visit and must commit to using an acceptable form of contraception during the entire study period, up to three months after last KB105 administration. Women using oral contraception must also have done so for 3 months prior to Baseline or will be willing to use a combination of barrier methods. To be considered not of childbearing potential, women must be post-menopausal for at least 1 year or surgically sterile
  • Known allergy to any of the constituents of the product
  • Hypersensitivity to local anesthesia (e.g., lidocaine/prilocainecream)
  • Current enrollment in a clinical trial
  • Treatment with an investigational drug or investigational device within 30 days prior to Day 1
  • Male who is not surgically sterile nor willing to use effective forms of contraception from Day 1 until 3 months following the last dose of study drug.
  • Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator

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): NCT04047732

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United States, Illinois
Northwestern University
Chicago, Illinois, United States, 60611
United States, Pennsylvania
Paddington Testing Company, Inc.
Philadelphia, Pennsylvania, United States, 19103
Sponsors and Collaborators
Krystal Biotech, Inc.
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Responsible Party: Krystal Biotech, Inc.
ClinicalTrials.gov Identifier: NCT04047732    
Other Study ID Numbers: KB105-001
First Posted: August 7, 2019    Key Record Dates
Last Update Posted: September 13, 2021
Last Verified: September 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Ichthyosis, Lamellar
Ichthyosiform Erythroderma, Congenital
Skin Abnormalities
Congenital Abnormalities
Infant, Newborn, Diseases
Skin Diseases
Skin Diseases, Genetic
Genetic Diseases, Inborn