← Back to Search

Gene Therapy

Topical Gene Therapy for Ichthyosis

Phase 1 & 2
Waitlist Available
Research Sponsored by Krystal Biotech, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Clinical diagnosis of lamellar ichthyosis
Age: 18 years old or older
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 12 weeks
Awards & highlights

Study Summary

This trial is testing a new drug, KB105, to see if it's safe and effective for treating skin conditions. The study will compare KB105 to a placebo, and measure improvement using the Investigator Global Assessment scale.

Who is the study for?
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.Check my eligibility
What is being tested?
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.See study design
What are the potential side effects?
While specific side effects are not listed here, typical concerns may include local reactions at the application site such as redness, itching or swelling; potential allergic responses; and systemic effects depending on how much of the therapy is absorbed through the skin.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have been diagnosed with lamellar ichthyosis.
Select...
I am 18 years old or older.
Select...
I understand the study and agree to follow all procedures and attend all visits.
Select...
I have a genetic form of ichthyosis caused by a specific TGM1 mutation.
Select...
Aside from my skin condition, I am in good health.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 12 weeks
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 12 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Investigator's Global Assessment (IGA) of disease severity
Safety and tolerability
Secondary outcome measures
Immunofluorescence Microscopy
Visual Index for Ichthyosis Severity (VIIS) scale, lamellar (L) standard

Trial Design

1Treatment groups
Experimental Treatment
Group I: Topical KB105Experimental Treatment1 Intervention
HSV1-TGM1 vector (KB105)

Find a Location

Who is running the clinical trial?

Krystal Biotech, Inc.Lead Sponsor
12 Previous Clinical Trials
424 Total Patients Enrolled

Media Library

KB105 (Gene Therapy) Clinical Trial Eligibility Overview. Trial Name: NCT04047732 — Phase 1 & 2
Congenital Ichthyosis Research Study Groups: Topical KB105
Congenital Ichthyosis Clinical Trial 2023: KB105 Highlights & Side Effects. Trial Name: NCT04047732 — Phase 1 & 2
KB105 (Gene Therapy) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04047732 — Phase 1 & 2

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this experiment currently seeking out test subjects?

"Clinicaltrials.gov confirms that this medical trial, posted on August 27th 2019 and updated September 10 2021, is not currently enrolling patients. However, there are 9 other trials with open participant recruitment right now."

Answered by AI
~1 spots leftby Mar 2025