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

Recruitment & Eligibility

Status: ACTIVE_NOT_RECRUITING

Sex: All

Target Recruitment: 6

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 entire study period, up to three months after last KB105 administration. Women using oral contraception must also have done so for 3 months prior to Baseline or will be willing to use a combination of barrier methods. To be considered not of childbearing potential, women must be post-menopausal for at least 1 year or surgically sterile
Known allergy to any of the constituents of the product
Hypersensitivity to local anesthesia (e.g., lidocaine/prilocainecream)
Current enrollment in a clinical trial
Treatment with an investigational drug or investigational device within 30 days prior to Day 1
Male who is not surgically sterile nor willing to use effective forms of contraception from Day 1 until 3 months following the last dose of study drug.
Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Topical KB105	KB105	HSV1-TGM1 vector (KB105)

Primary Outcome Measures

Name	Time	Method
Safety and tolerability	Up to 12 weeks	Incidence of treatment-emergent adverse events
Investigator's Global Assessment (IGA) of disease severity	Up to 12 weeks	Improvement of disease severity in the treatment area assessment through Investigator's Global Assessment (IGA)

Secondary Outcome Measures

Name	Time	Method
Visual Index for Ichthyosis Severity (VIIS) scale, lamellar (L) standard	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	Up to 12 weeks	Level of transglutaminase 1 in KB105-administered skin as measured by immunofluorescence microscopy

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

Recruitment & Eligibility

Study & Design

Trial Locations

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