Trial Search Results

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

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.

Stanford is currently accepting patients for this trial.

Lead Sponsor:

Krystal Biotech, Inc.

Stanford Investigator(s):


  • Biological: KB105


Phase 1/Phase 2


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

Ages Eligible for Study

18 Years - N/A

Genders Eligible for Study


Now accepting new patients

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Pediatric Derm Study
Not Recruiting