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.
Krystal Biotech, Inc.
- Biological: KB105
Phase 1/Phase 2
- 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
- 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