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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT03738800




Registration number
NCT03738800
Ethics application status
Date submitted
24/10/2018
Date registered
13/11/2018

Titles & IDs
Public title
A Safety, Efficacy and Systemic Exposure Study of CD5789 Cream in Adults and Adolescents With Lamellar Ichthyosis
Scientific title
A Phase 2 Randomized, Multicenter, Double-blind, Vehicle Controlled, 90-Day, Safety, Efficacy & Systemic Exposure Study of Trifarotene (CD5789) Cream HE1 in Adults and Adolescents With Autosomal Recessive Ichthyosis With Lamellar Scale
Secondary ID [1] 0 0
18-ICH-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Lamellar Ichthyosis 0 0
Condition category
Condition code
Skin 0 0 0 0
Other skin conditions
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - CD5789 Cream 200 µg/g
Treatment: Drugs - CD5789 Cream 100 µg/g
Treatment: Drugs - CD5789 Cream Vehicle

Experimental: CD5789 Cream 200 µg/g - CD5789 200 µg/g, topical, 50g

Experimental: CD5789 Cream 100 µg/g - CD5789 100 µg/g, topical, 50g

Placebo comparator: CD5789 Cream Vehicle - CD5789 Cream Vehicle, topical, 50g


Treatment: Drugs: CD5789 Cream 200 µg/g
A fixed dose (determined at Visit 1) of 200 µg/g applied topically twice weekly to up to 90% BSA

Treatment: Drugs: CD5789 Cream 100 µg/g
A fixed dose (determined at Visit 1) of 100 µg/g applied topically twice weekly to up to 90% BSA

Treatment: Drugs: CD5789 Cream Vehicle
A fixed dose (determined at Visit 1) applied topically twice weekly, up to 36 g per dose up to 90% BSA
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
The Percentage of Subjects in Each Treatment Group Who Experienced Successful Resolution of LI.
Timepoint [1] 0 0
90 Days
Secondary outcome [1] 0 0
Total 16-point Visual Index for Ichthyosis Severity (VIIS)
Timepoint [1] 0 0
90 Days
Secondary outcome [2] 0 0
The Difference in Mean Scores Using Individual Score for Roughness
Timepoint [2] 0 0
90 Days
Secondary outcome [3] 0 0
The Difference in Mean Scores Using Palm Sole Assessment
Timepoint [3] 0 0
90 Days
Secondary outcome [4] 0 0
The Difference in Proportion of Subjects With Presence of Fissures on Palms Between the Active and Vehicle Groups
Timepoint [4] 0 0
90 Days
Secondary outcome [5] 0 0
Quality of Life Measurement Per Dermatology Life Quality Index (DLQI)
Timepoint [5] 0 0
90 Days
Secondary outcome [6] 0 0
The Difference in Proportion of Subjects With Presence of Fissures on Soles Between the Active and Vehicle Groups
Timepoint [6] 0 0
90 Days

Eligibility
Key inclusion criteria
1. For Cohort A: subject is =18 years old; for Cohort B: subject is =12 years old.
2. Subject has known diagnosis of LI.
3. Subject has moderate to severe (IGA 3-4) LI on the IGA of LI severity.
4. Subject has signed an ICF at Screening before any investigational procedures. Subjects <18 years of age (or Age of Majority) must sign an assent form in conjunction with an ICF signed by the parent/legal representative.
5. Subject who is participating in optional photography has signed a photography ICF.
6. Subject who is participating in the optional PK substudy has signed a PK ICF. Minors, in the event of their reaching majority during the study, should be capable of giving consent to take part in the PK substudy.
7. Subject is not of childbearing potential, who is postmenopausal (absence of menstrual bleeding for 1 year before Baseline, without any other medical reason), or has documented hysterectomy, bilateral salpingectomy, or bilateral oophorectomy. For individuals with permanent infertility due to an alternate medical cause other than the above, (e.g., Mullerian agenesis, androgen insensitivity), investigator discretion should be applied to determining study entry.

OR
* Subject is a woman of childbearing potential (WOCBP), i.e., a female =12 years of age (regardless of whether they have experienced/reported menarche), or a male subject with sexual partners capable of reproduction who agrees to use 2 effective forms of contraception during the study and for at least 1 month after the last study drug application. The 2 authorized forms of contraception are condom used with 1 of the following methods of contraception:
* bilateral tubal ligation
* combined oral contraceptives (estrogens and progesterone), vaginal ring, or implanted or injectable hormonal contraceptives with a stable dose for at least 1 month before Baseline; hormonal contraceptives must inhibit ovulation
* intrauterine device (IUD) inserted at least 1 month before Baseline OR Agrees to abstain from heterosexual intercourse during study participation and for 1 month after the last application of study drug and to use a highly effective contraceptive as backup if he or she becomes sexually active during the study. Abstinence is only acceptable if this is the subject's usual lifestyle. Periodic abstinence (calendar, symptothermal, postovulation methods), withdrawal (coitus interruptus), spermicides only, and lactational amenorrhoea method are not acceptable methods of contraception.

AND Male subjects may not donate sperm during the study and for at least 1 month after the last study drug application.

Note: Female subjects who are premenstrual at screening should nonetheless follow the pregnancy testing schedule for WOCBP even if they abstain from sexual intercourse while in the study and for at least 1 month after the last study drug application.
8. Women of childbearing potential must be nonlactating and have negative pregnancy test results at Screening (serum) and on Day 1 before study drug administration (urine).
9. Subject is reliable and capable of adhering to the protocol and visit schedule, in the investigator's judgment, and has signed informed consent/assent, as applicable.
10. Subject is taking no more than 3500 IU/day Vitamin A (e.g., as in a multivitamin).
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
Exclusion criteria:

1. Subject has any variant of ichthyosis other than LI or another disorder of keratinization, including syndromic ichthyoses.
2. Subject has current moderate or severe stinging/burning at Screening.
3. Subject has an ongoing cutaneous infection or any other significant concomitant skin disease (other than the LI) which, in the investigator's opinion, may interfere with the study assessments.
4. Subject with fasting triglycerides >200 mg/dL or >2.25 mmol/L and/or total cholesterol >250 mg/dL or >6.5 mmol/L. Subjects whose triglycerides and/or total cholesterol are within normal limits with a stable dose of lipid-lowering agents for at least 6 months may be included.
5. Subject was previously treated with trifarotene/CD5789 in an acne or ichthyosis study.
6. Subject has any other significant concomitant disease, or poorly controlled medical condition other than LI that in the investigator's opinion may put him or her at risk if he or she takes part in the study, and/or that may interfere with the study assessments.
7. Subject has a medical condition that potentially alters bone metabolism (e.g., osteoporosis, thyroid dysfunction, Cushing syndrome, Crohn's disease, or ulcerative colitis). Subjects with hypothyroidism who are on a stable dose of thyroid hormone replacement therapy and whose thyroid-stimulating hormone (TSH) is normal may be included
8. Subject is being treated for major depression disorder and/or has a history of major depression or suicide attempt requiring hospitalization, medications, and close psychiatric surveillance to prevent suicide attempts.
9. Subject with positive serology for hepatitis B surface antigen, hepatitis C, or are known to be HIV positive or to have AIDS at Screening.
10. Subject with any of the following laboratory values at Screening:

1. Aspartate aminotransferase or alanine aminotransferase >1.5 × upper limit of normal defined by the laboratory
2. Total bilirubin >1.25 × ULN at Screening. Subjects with known Gilbert's syndrome may be included with total bilirubin >1.25 × ULN
3. Hemoglobin <12.5 g/dL for men and <11.5 g/dL for women
4. Platelets <150 × 109/L or >400 × 109/L.
11. Subject has any clinically other significant abnormal laboratory value (hematology, chemistry, or urinalysis) at Screening that, in the investigator's opinion, may put the subject at risk if he or she takes part in the study, and/or that may interfere with the study assessments.
12. Subject has had recent systemic malignancy (e.g., within 5 years) with exception of nonmelanoma skin cancer or cervical intraepithelial neoplasia of Grade 1 who are >6 months post-treatment.
13. Subject has a history of long QT syndrome or has clinically significant electrocardiogram (ECG) abnormalities, including clinically significant conduction disorders or significant arrhythmias, or QTcF interval >450 ms.
14. Subject has a known allergy or sensitivity to any of the components of the investigational products.
15. Subject has been exposed to excessive UV radiations on the treated zones within 1 month before Baseline visit or is planning intensive UV exposure during the study (e.g., occupational exposure to the sun, sunbathing, phototherapy, etc.).
16. Subject is inherently sensitive to sunlight.
17. Subject is unable or unwilling to stop use of topical or systemic retinoids.
18. Subject is presumed to be abusing drug or alcohol at Screening or Baseline Visits based on medical history or current clinical symptoms.
19. Subject is participating in another interventional clinical trial.
20. Subject is institutionalized.
21. Subject is in any way related to the sponsor, investigator, or site personnel.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
1/05/2019
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
3/09/2021
Sample size
Target
Accrual to date
Final
65
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Eastern Health Monash University - Box Hill
Recruitment hospital [2] 0 0
Veracity Clinical Research - Brisbane
Recruitment hospital [3] 0 0
Royal Children's Hospital - Parkville
Recruitment hospital [4] 0 0
Premier Specialists Ptd Ltd - Sydney
Recruitment postcode(s) [1] 0 0
- Box Hill
Recruitment postcode(s) [2] 0 0
- Brisbane
Recruitment postcode(s) [3] 0 0
- Parkville
Recruitment postcode(s) [4] 0 0
- Sydney
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Connecticut
Country [4] 0 0
United States of America
State/province [4] 0 0
Illinois
Country [5] 0 0
United States of America
State/province [5] 0 0
Indiana
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Pennsylvania
Country [9] 0 0
United States of America
State/province [9] 0 0
South Carolina
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas
Country [11] 0 0
Canada
State/province [11] 0 0
Toronto
Country [12] 0 0
France
State/province [12] 0 0
Paris
Country [13] 0 0
France
State/province [13] 0 0
Rouen
Country [14] 0 0
France
State/province [14] 0 0
Toulouse
Country [15] 0 0
Germany
State/province [15] 0 0
Berlin
Country [16] 0 0
Germany
State/province [16] 0 0
Frankfurt
Country [17] 0 0
Germany
State/province [17] 0 0
Hamburg
Country [18] 0 0
Germany
State/province [18] 0 0
Munich
Country [19] 0 0
Germany
State/province [19] 0 0
Rostock
Country [20] 0 0
Israel
State/province [20] 0 0
Tel Aviv
Country [21] 0 0
Spain
State/province [21] 0 0
Barcelona
Country [22] 0 0
Spain
State/province [22] 0 0
Madrid
Country [23] 0 0
Spain
State/province [23] 0 0
Pamplona
Country [24] 0 0
Ukraine
State/province [24] 0 0
Dnipro
Country [25] 0 0
Ukraine
State/province [25] 0 0
Ternopil'
Country [26] 0 0
Ukraine
State/province [26] 0 0
Uzhhorod
Country [27] 0 0
Ukraine
State/province [27] 0 0
Zaporizhzhya
Country [28] 0 0
United Kingdom
State/province [28] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Mayne Pharma International Pty Ltd
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Keith A. Choate, MD
Address 0 0
Yale University
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?




Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results are available at https://clinicaltrials.gov/study/NCT03738800