Darier’s Disease or Hailey-Hailey Disease MedDRA version: 20.0 Level: LLT Classification code 10011860 Term: Darier's disease System Organ Class: 100000004850 MedDRA version: 20.1 Level: LLT Classification code 10019029 Term: Hailey-Hailey disease System Organ Class: 100000004850
Conditions
Interventions
Product Name: LX3305 250 mg capsule
Product Code: LX3305
Pharmaceutical Form: Capsule
INN or Proposed INN: None
CAS Number: 1078151-47-9
Current Sponsor code: LX3305 Dihydrate
Other descriptive name:
Sponsors
Dermecular Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1.Male or female =18 years of age. 2.Able to read, understand, and sign the informed consent. 3.Stable health in the opinion of the investigator and as confirmed by medical history with no acute hematologic, renal, neurologic, or liver disease within the past 12 months. 4.Histological diagnosis of Darier’s Disease or Hailey Hailey Disease. 5.Grade 3 (moderate) or Grade 4 (severe) lesions as defined in the IGSA. 6.Willing to abstain from any other medical treatments for HHD or DD while in the study, except as specified in the protocol. 7.Willing and able to participate in the study and to comply with all study requirements, including concomitant medication and other treatment restrictions. 8.Female subjects must have a negative serum pregnancy test at Screening. A female of childbearing potential is a sexually mature woman who: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 consecutive months (ie, has had menses at any time in the preceding 24 consecutive months, must agree to use two effective methods of contraception for the duration of the study and at least 1 month after the last dose of study drug. The two forms of contraception authorized are defined as the use of a barrier method of contraception (condom with spermicide) in association with one of the following methods of contraception: •Combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation: oral, intravaginal, or transdermal •Progestogen-only hormonal contraception associated with inhibition of ovulation: oral, injectable or implantable •Intrauterine device (IUD) •Intrauterine hormone -releasing system (IUS) •Bilateral tubal; occlusion •Vasectomized partner (provided that partner is the sole sexual partner and that vasectomized partner has received medical assessment of the surgical success) •Sexual abstinence (refraining from heterosexual intercourse during the entire study period; the reliability of sexual abstinence needs to be evaluated in relation to the duration of the clinical study and the preferred and usual lifestyle of the patient) 9.If the subject is a male with a female sexual partner who is of childbearing potential, the couple is willing to use two highly effective methods of birth control during the duration of the trial and for one month after the last dose of study drug. The two forms of birth control authorized are defined as the use of a barrier method of contraception (condom with spermicide) in association with one of the methods of birth control listed in Exclusion Criterion 8. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 40 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: A participant who meets any of the following criteria will be excluded from this study: 1.Female participant who is pregnant, lactating, breastfeeding, or planning to become pregnant during the study period. 2.Prior treatment as follows: a.Acitretin =4 weeks prior to study Baseline b.Alitretinoin =4 weeks prior to study Baseline c.Any other systemic retinoid =26 weeks prior to study Baseline d.Systemic corticosteroids =4 weeks prior to study Baseline e.Systemic antibiotics =2 weeks prior to study Baseline f.Local treatments such as dermabrasion, laser ablation, or botulinum toxin to the specific skin sites to be monitored during this study =26 weeks prior to study Baseline 3.Participants who are currently participating in another clinical study or have completed another clinical study with an investigational drug or device on the study area within 30 days prior to study treatment initiation. 4.Participants with a past or current medical condition or illness that, in the opinion of the investigator, would compromise their safety during the study or confound study results. 5.Participants with a history of alcohol or drug abuse. 6.Participants with a history of hypersensitivity to any of the excipients of the study drug. 7.Participants who are deprived of liberty by a judicial or administrative decision. 8.Participants who are subject to psychiatric care. 9.Participants admitted to a health or social institution for purposes other than research. 10.Adult participants subject to a legal protection measure (eg, under guardianship or judicial protection).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Primary: To evaluate the safety and tolerability of LX3305 compared to placebo in participants with Darier’s Disease (DD) or Hailey-Hailey Disease (HHD);Secondary Objective: Secondary: To evaluate the efficacy of LX3305 compared to placebo as determined by Investigator’s Global Signs Assessment (IGSA) score in participants with DD or HHD To characterize the pharmacokinetics (PK) of LX3305 in participants with DD or HHD Exploratory: To evaluate improvement in Quality of Life (QOL) and pain after receiving LX3305 compared to placebo To evaluate changes in genetic signatures of DD and HDD after receiving LX3305 compared to placebo ;Primary end point(s): Safety: Primary Endpoint: Safety and tolerability of LX3305 compared to placebo as assessed by adverse events (AEs), serious AEs (SAEs), deaths, and discontinuations due to AEs Additional safety assessments will include review and evaluation of laboratory testing including hematology, coagulation, chemistry, urinalysis; vital signs; 12-lead ECGs; and physical examination findings. ;Timepoint(s) of evaluation of this end point: Safety laboratory measures will be conducted during Screening, week 2 and every 4 weeks. A physical examination will be conducted during Screening, at Baseline, and every 4 weeks. A standard 12-lead electrocardiogram (ECG) will be performed at Baseline and at the End of Study visit. Adverse events will be monitored continuously. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Efficacy: •Proportion of participants with at least a 1-grade improvement in IGSA score from Baseline at any time during the trial and by scheduled visit (i.e. at Week 4, at Week 8, and at Week 12) •Proportion of participants achieving at least a 2-grade improvement in IGSA score from Baseline at any time during the trial and by scheduled visit •Time-to-response for 1-grade improvement, defined as duration from randomization until first improvement from baseline of at least 1-grade, or censored at last efficacy assessment •Time-to-response for 2-grade improvement, defined as duration from randomization until first improvement from baseline of at least 2-grades, or censored at last efficacy assessment •Duration of response for 1-grade improvement, defined as duration from first improvement from baseline of at least 1-grade until return to baseline grade or higher, or censored at last efficacy assessment •Duration of response for 2-grade improvement, defined as duration from first improvement from baseline of at least 2-grades until first efficacy assessment that is less than a 2-grade improvement, or censored at last efficacy assessment •Change from Baseline in affected body surface area (BSA) by scheduled visit Efficacy endpoints derived from the IGSA score will be based on the Investigator assessment. ;Timepoint(s) of evaluation of this end point: Efficacy measures (IGSA scoring, lesion photography, QOL, and pain assessment by visual analog scale [VAS]) will be conducted at Baseline and every 4 weeks.Biomarkers of DD and HHD will be assessed through skin biopsies obtained before and after study drug administration. Blood samples for trough PK analysis will be obtained at Baseline and every 4 weeks at each study visit before dosing. | — |
Countries
France
Contacts
Public ContactMarie-Caroline Leclere
BlueReg
Outcome results
None listed