Topical KB105 Gene Therapy for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ich… (NCT04047732) | Clinical Trial Compass
UnknownPhase 1/2
Topical KB105 Gene Therapy for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)
United States6 participantsStarted 2019-08-27
Plain-language 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.
Who can participate
Age range18 Years
SexALL
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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 enti…
What they're measuring
1
Safety and tolerability
Timeframe: Up to 12 weeks
2
Investigator's Global Assessment (IGA) of disease severity