Topical Gene Therapy for Ichthyosis

Recruiting at 1 trial location

Overseen By Hirak B Routh, MDDS

Age: 18+

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Krystal Biotech, Inc.

Disqualifiers: HIV, Hepatitis B, Hepatitis C, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

6 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Eligibility Criteria

Adults with a genetic diagnosis of TGM1-deficient ARCI, specifically lamellar ichthyosis, and moderate-to-severe symptoms. Participants must be in good general health aside from their skin condition, able to follow study procedures, and not pregnant or breastfeeding. They should have no other major medical issues or skin disorders that could interfere with the trial.

Inclusion Criteria

The specific area being looked at has an IGA score of 3 to 4.

I have been diagnosed with lamellar ichthyosis.

I am 18 years old or older.

Exclusion Criteria

Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator

I do not have HIV, hepatitis B, or 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

Treatment Details

Interventions

• KB105

Trial Overview The trial is testing KB105 gene therapy applied topically to affected areas compared to a placebo. The main goals are to check for safety and improvement in the severity of the skin condition as measured by an Investigator Global Assessment scale.

Participant Groups

Group I: Topical KB105 Experimental Treatment 1 Intervention

HSV1-TGM1 vector (KB105)

Who Is Running the Clinical Trial?

Krystal Biotech, Inc.

Lead Sponsor

Trials

15

Recruited

770+