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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 belowSelect...
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.
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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 weeks3 visits
Treatment ~ Varies
Follow Up ~ up to 12 weeks
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 12 weeks
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)
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Who is running the clinical trial?
Krystal Biotech, Inc.Lead Sponsor
12 Previous Clinical Trials
424 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- The specific area being looked at has an IGA score of 3 to 4.I do not have HIV, hepatitis B, or hepatitis C.I am a man who can father children and will use contraception during and 3 months after the study.I have been diagnosed with lamellar ichthyosis.I am 18 years old or older.I understand the study and agree to follow all procedures and attend all visits.I am not pregnant, breastfeeding, or planning to become pregnant and agree to use contraception during the study.I have a genetic form of ichthyosis caused by a specific TGM1 mutation.I am too unwell to travel to the research site.Aside from my skin condition, I am in good health.You are allergic to the numbing cream used for local anesthesia.
Research Study Groups:
This trial has the following groups:- Group 1: Topical KB105
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
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."
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