Topical Gene Therapy for Ichthyosis

Not currently 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

Duration: 12 weeks

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new topical gene therapy called KB105 for treating lamellar ichthyosis, a skin condition that causes thick, scaly skin. Researchers aim to determine if KB105 is safe and can improve skin appearance compared to a placebo (a treatment with no active medicine). Individuals with a genetic diagnosis of TGM1-deficient ARCI and moderate to severe symptoms might be suitable for this study. As a Phase 1, Phase 2 trial, the research focuses on understanding how the treatment works in people and measuring its effectiveness in an initial, smaller group, offering participants a chance to be among the first to benefit from this innovative therapy.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the study team or your doctor.

Is there any evidence suggesting that KB105 is likely to be safe for humans?

Research has shown that KB105, a skin-applied gene therapy, is safe for use. Studies found that patients using KB105 experienced no serious side effects, and no negative reactions linked to the drug were reported. This indicates the treatment is generally safe. Additionally, repeated use did not provoke negative reactions, suggesting the body does not perceive it as a threat. Overall, the safety results are promising for those considering participation in a clinical trial for KB105. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Unlike the standard treatments for ichthyosis, which often involve topical moisturizers and systemic retinoids, KB105 offers a groundbreaking approach through gene therapy. This novel treatment uses a HSV1-TGM1 vector to deliver the TGM1 gene directly to the skin, potentially addressing the root cause of certain types of ichthyosis at the genetic level. Researchers are excited because this method could provide a more targeted and effective treatment with longer-lasting results than traditional options, which mainly focus on symptom management.

What evidence suggests that KB105 might be an effective treatment for ichthyosis?

Research has shown that KB105, a topical treatment, may help with ichthyosis, a condition causing dry, scaly skin. In earlier studies, KB105 successfully delivered a protein called TGM1, crucial for healthy skin, directly to skin cells. Patients tolerated this treatment well, experiencing no serious side effects or immune reactions. Animal studies indicated that KB105 remained on the skin and did not affect other parts of the body. These findings suggest that KB105 could improve skin conditions for people with ichthyosis. ^{1 2 3 4 5}

Are You a Good Fit for This Trial?

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 understand the study and agree to follow all procedures and attend all visits.

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

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive topical KB105 and placebo on different target areas for intra-patient comparison

12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment

2-4 weeks

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. sciencedirect.com sciencedirect.com/science/article/pii/S0022202X20320704

Preclinical Evaluation of a Modified Herpes Simplex Virus ... In this study, we investigated the ability of KB105, a gene therapy vector encoding full-length human TGM1, to deliver functional human TGM1 to keratinocytes.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT04047732

Topical KB105 Gene Therapy for the Treatment of TGM1- ... Target Areas will be imaged and evaluated for safety and efficacy. Subjects will be on-trial for approximately 3.5 months. Official Title. A Phase I/II Clinical ...

3. ir.krystalbio.com ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-announces-positive-interim-results-phase-12

Investors Safety data from all three patients showed that repeat dosing with KB105 was well-tolerated with no drug related adverse events and no immune ...

4. jidonline.org jidonline.org/article/S0022-202X(19)30650-5/fulltext

383 Preclinical safety and pharmacology of KB105, an ... In a repeat dose GLP toxicology study in the same species, KB105 was well tolerated and remained localized to the application site with no systemic exposure.

5. clinicaltrial.be clinicaltrial.be/en/details/258892?per_page=20&only_recruiting=0&only_eligible=0

Topical KB105 for the Treatment of TGM1-deficient ... KB105-02 is an intrasubject randomized, placebo-controlled, double-blind study to evaluate the safety and efficacy of KB105 in children and ...