Clinical trial NCT03422640: Trial Evaluating the Efficacy of Apremilast for the Treatment of Frontal Fibrosing Alopecia
Official title: Open Label Trial Evaluating the Efficacy of Apremilast for the Treatment of Frontal Fibrosing Alopecia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE4
Conditions: Frontal Fibrosing Alopecia
Keywords: Frontal Fibrosing Alopecia
Apremilast
Lead sponsor: Bellevue Dermatology
Sponsor class: OTHER
Collaborators: name: Celgene
First posted: 2018-02-06
Enrollment: 20
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: This is open label single side study involvement 20 patient treated with Apremilast. Each enrolled patient may be evaluated at by a dermatologist using the Lichen Planopilaris Activity Index and Frontal Fibrosing Alopecia Index. Other measures include physician global assessment, dermatology quality of life and patients analogue score for pruritus. Pt will have visits at Week 0,2,4,8,12,16,20,24
Primary purpose: TREATMENT

Brief summary: This is open label single side study involvement 20 patient treated with Apremilast. Each enrolled patient may be evaluated at by a dermatologist using the Lichen Planopilaris Activity Index and Frontal Fibrosing Alopecia Index. Other measures include physician global assessment, dermatology quality of life and patients analogue score for pruritus. Pt will have visits at Week 0,2,4,8,12,16,20,24

Detailed description: Frontal fibrosing alopecia (FFA) is a chronic immune mediated inflammatory disease characterized by inflammation of the hair follicle and scaring hair loss. Clinically, FFA presents as a progressive recession of the hairline in a frontal temporal distribution. Evidence suggests that timely and effective management can prevent the permanent loss of hair. Unfortunately, most current treatment have been disappointing with poor efficacy or high risk profile. The available of a safe effective treatment for this disease remains an unmet need Apremilast is a novel phosphodiesterase 4 inhibitor currently FDA approved to treat psoriasis and is under investigation for other auto immune conditions. The medication has a good safety profile with no required laboratory monitoring. This study primarily aimsto determine whether Apremilast offers any benefit for this difficult to treatment population

Eligibility criteria: Inclusion Criteria:

1. Must be in general good health (except for disease under study) as judged by the Investigator, based on medical history, physical examination, clinical laboratories, and urinalysis. (NOTE: The definition of good health means a subject does not have uncontrolled significant co-morbid conditions).
2. Male or Female and is at least 18 years of age, at the time of enrollment.
3. Females of childbearing potential (FCBP)† must have a negative pregnancy test at Screening and Baseline. While on investigational product and for at least 28 days after taking the last dose of investigational product, FCBP who engage in activity in which conception is possible must use one of the approved contraceptive§ options described below: Option 1: Any one of the following highly effective methods: hormonal contraception (oral, injection, implant, transdermal patch, vaginal ring); intrauterine device (IUD); tubal ligation; or partner's vasectomy; 3. OR Option 2: Male or female condom (latex condom or nonlatex condom NOT made out of natural \[animal\] membrane \[for example, polyurethane\]; PLUS one additional barrier method: (a) diaphragm with spermicide; (b) cervical cap with spermicide; or (c) contraceptive sponge with spermicide. Male subjects (including those who have had a vasectomy) who engage in activity in which conception is possible must use barrier contraception (male latex condom or nonlatex condom NOT made out of natural \[animal\] membrane \[for example, polyurethane\]) while on investigational product and for at least 28 days after the last dose of investigational product.

   * † A female of childbearing potential is a sexually mature female who 1) has not undergone a hysterectomy (the surgical removal of the uterus) or bilateral oophorectomy (the surgical removal of both ovaries) consecutive months (that is, has had menses at any time during the preceding 24 consecutive months).
   * § The female subject's chosen form of contraception must be effective by the time the female subject is randomized into the study (for example, hormonal contraception should be initiated at least 28 days before randomization).
4. Patients with an established diagnosis of FFA based on the enrolling investigator's clinical judgment
5. Patients who have been treated and failed one standard therapy including

   * Topical steroids
   * Short course systemic steroids
   * Systemic antibiotics
6. Patients who are on stable dose of topical steroids or systemic antibiotics
7. Patient and/or legal guardian has voluntarily signed and dated an informed consent/patient authorization form approved by an Institutional Review Board (IRB)/Ethics Committee (EC) if applicable according to local law, after the nature of the study has been explained and the patient has had the opportunity to ask questions.

Exclusion Criteria:

1. Other than disease under study, any clinically significant (as determined by the Investigator) cardiac, endocrinologic, pulmonary, neurologic, psychiatric, hepatic, renal, hematologic, immunologic disease, or other major disease that is currently uncontrolled.
2. Any condition, including the presence of laboratory abnormalities, which would place the subject at unacceptable risk if he/she were to participate in the study.
3. Prior history of suicide attempt at any time in the subject's life time prior to screening or randomization, or major psychiatric illness requiring hospitalization within the last 3 years.
4. Pregnant or breast feeding.
5. Active substance abuse or a history of substance abuse within 6 months prior to Screening.
6. Malignancy or history of malignancy, except for: a. treated \[ie, cured\] basal cell or squamous cell in situ skin carcinomas; b. treated \[ie, cured\] cervical intraepithelial neoplasia (CIN) or carcinoma in situ of cervix with no evidence of recurrence within the previous 5 years.
7. Use of any investigational drug within 4 weeks prior to randomization, or 5 pharmacokinetic/pharmacodynamic half lives, if known (whichever is longer).
8. Prior treatment with apremilast.
9. Patient who have underlying chronic infections including HIV, Hep B and C.
10. History of uncontrolled depression.

Primary outcomes:
measure: Lichen Planopilaris index
timeFrame: Week 0 to 24, patient visit week0,2,4,8,12,16,20,24
Secondary outcomes:
measure: Physicians global assessment
timeFrame: Weeks 0 to 24, patient visit week 0,2,4,8,12,16,20,24
measure: Visual Analogue Scale Pruritus
timeFrame: Weeks 0 to 24, patient visit week 0,2,4,8,12,16,20,24
measure: Frontal Fibrosing Alopecia Index
timeFrame: Weeks 0 to 24, patient visit weeks 0,2,4,8,12,16,20,24
measure: Dermatology Quality of Life (DLQI) (Appendix E) Dermatology Quality of Life (DLQI) (Appendix E) Dermatology Quality of Life (DLQI)
timeFrame: Weeks 0 to 24, patient visit weeks 0,2,4,8,12,16,20,24
Other outcomes:
Locations:
city: Bellevue
country: United States
facility: Bellevue Dermatology
state: Washington
Study officials:
affiliation: Bellevue Dermatology
name: Clive Liu, MD
role: STUDY_DIRECTOR
References: