Health TrialsbyHealthPanel

A Study to Investigate Safety and Efficacy of KB111 for the Treatment of Hailey-Hailey Disease

ConditionsHailey Hailey DiseaseSkin DiseasesLesion Skin
Trial statusRecruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age12+
SponsorKrystal Biotech, Inc.

About this trial

An open-label study to evaluate the safety and efficacy of KB111 in pediatric and adult subjects with Hailey-Hailey Disease

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

The participant must have read, understood, and signed an Institutional Review Board (IRB)- approved Informed Consent Form and must be willing and able to comply with study procedures and instructions

Aged ≥ 12 years of age at the time of informed consent/assent

Documented clinical diagnosis of HHD with confirmed ATP2C1 variant by genetic testing

A negative pregnancy test prior to initial dose for subjects of child-bearing potential

Disqualifiers

Participation in another clinical study or treatment with an investigational agent 30 days or 5 half-lives, whichever is longer, prior to initial dosing in Week 1

Participant who is pregnant or nursing

Participant who is unwilling to comply with contraception requirements per protocol (for subjects of child-bearing potential)

Participant who is known to be non-compliant or is unlikely to comply with the requirements of the study protocol, in the opinion of the Investigator

Trial design

Design model

Single group

Treatments tested in this trial

  • KB111

    Biological/Vaccine

    KB111 is a replication-defective, non-integrating herpes simplex virus (HSV-1)-based vector encoding functional, full-length human ATP2C1 protein combined with a gel for topical application.

Treatment groups

7 Participants
are divided into 1 treatment group
Group A: KB111Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

To evaluate the safety and tolerability of KB111

Number of subjects with treatment related adverse events as assessed by CTCAE v5

Time frame
12 weeks

Secondary outcomes

1

To evaluate the effect of KB111 on patient-reported lesion severity throughout the treatment period.

Change from baseline using a patient reported 5-point multi-item scale where signs and symptoms are rated from none (0) to extreme (4)

Time frame
12 weeks
2

To evaluate the effect of KB111 on patient-reported global impression of severity throughout the treatment period

Change from baseline using a patient reported 5-point multi-item scale where signs and symptoms are rated from none (0) to extreme (4)

Time frame
12 weeks
3

To evaluate the effect of KB111 on Investigator-based assessments of lesion severity

Change from baseline in the Investigator's assessment using a 5-point scale where 0 is clear and 4 is severe

Time frame
12 weeks
4

To evaluate the effect of KB111 on Investigator-based assessment of global severity

Change from baseline in the Investigator's assessment using a 5-point scale where 0 is clear and 4 is severe

Time frame
12 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Krystal Biotech, Inc.

Lead sponsor

Source ClinicalTrials.gov
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