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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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04047732
Recruitment Status : Recruiting
First Posted : August 7, 2019
Last Update Posted : August 29, 2019
Sponsor:
Information provided by (Responsible Party):
Krystal Biotech, Inc.

Tracking Information
First Submitted Date  ICMJE July 29, 2019
First Posted Date  ICMJE August 7, 2019
Last Update Posted Date August 29, 2019
Actual Study Start Date  ICMJE August 27, 2019
Estimated Primary Completion Date March 2020   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 6, 2019)
  • Safety and tolerability [ Time Frame: Up to 12 weeks ]
    Incidence of treatment-emergent adverse events
  • 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)
Original Primary Outcome Measures  ICMJE
 (submitted: August 5, 2019)
  • Safety [ Time Frame: Up to 12 weeks ]
    Evaluate safety through the incidence of adverse events (AEs) associated with KB105 post-administration
  • 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)
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 6, 2019)
  • 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.
  • Immunofluorescence microscopy [ Time Frame: Up to 12 weeks ]
    Level of transglutaminase 1 in KB105-administered skin as measured by immunofluorescence microscopy
Original Secondary Outcome Measures  ICMJE
 (submitted: August 5, 2019)
  • Visual Index for Ichthyosis Severity (VIIS) scale, lamellar (L) standard [ Time Frame: Up to 12 weeks ]
    Improvement of disease severity in the treatment area assessment through Visual Index for Ichthyosis Severity (VIIS) scale, lamellar (L) standard assessment
  • Immunofluorescence microscopy [ Time Frame: Up to 12 weeks ]
    Level of transglutaminase 1 in KB105-administered skin as measured by immunofluorescence microscopy
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Topical KB105 Gene Therapy for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
Official Title  ICMJE 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)
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.
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.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 1
Phase 2
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE TGM-1 Related Autosomal Recessive Congenital Ichthyosis
Intervention  ICMJE 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
Study Arms  ICMJE Experimental: Topical KB105
HSV1-TGM1 vector (KB105)
Intervention: Biological: KB105
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: August 5, 2019)
6
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE March 2025
Estimated Primary Completion Date March 2020   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Lawrence C Parish, M.D. (215) 563-7330 larryderm@yahoo.com
Contact: Hirak B Routh, MDDS (215) 563-7330 hirakbrouth@gmail.com
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04047732
Other Study ID Numbers  ICMJE KB105-001
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: No
Responsible Party Krystal Biotech, Inc.
Study Sponsor  ICMJE Krystal Biotech, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Krystal Biotech, Inc.
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP