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