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This is a single-center, open-label clinical study to study the efficacy of apremilast in the treatment of mild to moderate central centrifugal cicatricial alopecia. The investigators hypothesize that the anti-inflammatory properties of apremilast may play a role in the decreasing scalp inflammation in patients with CCCA and may prevent further hair loss and potentially induce hair regrowth in patients with mild to moderate disease.
Central centrifugal cicatricial alopecia (CCCA) is a type of scarring alopecia commonly seen in women of African American descent. The etiology is not completely understood, but CCCA likely results from a combination of hair-grooming practices, a pro-inflammatory state within the hair follicles, and genetic factors. The management of CCCA remains a challenge as there are no published treatment guidelines. Current therapies aim to decrease inflammation in order to prevent further hair loss.
Apremilast, an oral phosphodiesterase-4 inhibitor, has been shown to be effective in the treatment of moderate to severe plaque psoriasis and psoriatic arthropathy. In vitro studies have demonstrated anti-inflammatory properties via inhibition of inflammatory mediators. Therefore, apremilast offers a possible therapeutic option for CCCA. This will be a single-center, open-label clinical study to determine the efficacy of apremilast in the treatment of mild to moderate central centrifugal cicatricial alopecia.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Apremilast | Experimental | Patients with CCCA |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Apremilast | Drug | 30 mg BID |
|
| Measure | Description | Time Frame |
|---|---|---|
| Mean Change in Physician Global Assessment of Improvement (PGA-I) | Mean change in PGA-I at Week 24 compared to Baseline. Trained study personnel will take standardized photographs of the scalp. These photographs will be provided to a panel of three dermatologists with expertise in CCCA, each of whom will review the photographs at these time points. Investigators will assess the improvement in hair loss severity using PGA-I. PGA-I will range from -3 (significant worsening) to 3 (significant improvement). | Week 0 and Week 24 |
| Measure | Description | Time Frame |
|---|---|---|
| Mean Change in CCCA Investigator Global Severity Score (IGSS) | Mean change in IGSS at Week 24 compared to Baseline. Treatment response will be considered no change or improvement in IGSS. CCCA Investigator Global Severity Score (IGSS) assess subjects on a scale of 0 (no hair loss) to 6 (severe CCCA, e.g. >75% involvement of vertex). | Week 0 and Week 24 |
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Inclusion Criteria:
Exclusion Criteria:
Only female subjects will be enrolled.
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| Name | Affiliation | Role |
|---|---|---|
| Saakshi Khattri, MD | Icahn School of Medicine at Mount Sinai | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Mount Sinai West Dermatology | New York | New York | 10023 | United States |
IPD will be shared with Celgene, who is providing a grant for this study.
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Enrollment period from November 2018 to July 2020
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| ID | Title | Description |
|---|---|---|
| FG000 | Apremilast | Patients with CCCA Apremilast: 30 mg BID |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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| ID | Title | Description |
|---|---|---|
| BG000 | Apremilast | Patients with CCCA Apremilast: 30 mg BID |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Mean Change in Physician Global Assessment of Improvement (PGA-I) | Mean change in PGA-I at Week 24 compared to Baseline. Trained study personnel will take standardized photographs of the scalp. These photographs will be provided to a panel of three dermatologists with expertise in CCCA, each of whom will review the photographs at these time points. Investigators will assess the improvement in hair loss severity using PGA-I. PGA-I will range from -3 (significant worsening) to 3 (significant improvement). | Posted | Mean | Standard Deviation | score on a scale | Week 0 and Week 24 |
|
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24 weeks
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Apremilast | Patients with CCCA Apremilast: 30 mg BID | 0 |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Ingrid Sanabria-Gonzalez | Icahn School of Medicine at Mount Sinai | 212-523-3812 | ingrid.sanabria@mountsinai.org |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Feb 1, 2021 | Apr 1, 2022 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 5, 2022 | Apr 7, 2022 | SAP_001.pdf |
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| ID | Term |
|---|---|
| C505730 | apremilast |
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This is an open-label study that will enroll 20 subjects with central centrifugal cicatricial alopecia. All subjects will receive apremilast.
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| Mean Change in Central Hair Loss Grade (CHLG) | Mean change in CHLG at Week 24 compared to Baseline. Degree of severity of hair loss is graded on a 6-point visual scale (pattern 0: no hair loss, pattern 1-2: mild hair loss, pattern 3-5: more severe hair loss). | Week 0 and week 24 |
| Mean Change in Subject Visual Analog Scale (VAS) of Hair Loss Severity | Mean change in VAS at Week 24 compared to Baseline. The VAS is a numerical scale used to assess patients' perception of hair loss severity. The evaluation is a 10cm long line on which the subjects indicate the severity of their condition from "0" (complete loss of hair in affected area - ie no visible hairs on central scalp) to "10" (full growth/regrowth in affected area-ie no visible hair loss on central scalp). | Week 0 and Week 24 |
| Mean Change in Subject Global Assessment of Improvement | Mean change in PaGA-I at Week 24 as compared to Baseline. PaGA-I will range from -3 (significant worsening) to 3 (significant improvement). | Week 0 and Week 24 |
| Change in Subject Rating of Symptom Severity Questionnaire (NRS) | Change in NRS at Week 24 as compared to Baseline. Subjects will complete a symptom severity questionnaire consisting of 3 numeric rating scales (NRS) measuring severity of pruritus, burning, and pain. The NRS will range from 0 (no symptoms) to 10 (severe symptoms). Patients indicate the intensity of each symptom (pruritus, burning, or pain) by choosing a number from 0 to 10 that corresponds to the severity of that symptom. | Week 0 and Week 24 |
| Mean Change From Baseline in Dermatology Life Quality Index (DLQI) Total Score | Mean change in DLQI total score at Week 24 as compared to Baseline. DLQi is a 10-item questionnaire, each question is scored from 0 to 3, giving a possible score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life). | Week 0 and Week 24 |
| years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Participants |
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| Secondary | Mean Change in CCCA Investigator Global Severity Score (IGSS) | Mean change in IGSS at Week 24 compared to Baseline. Treatment response will be considered no change or improvement in IGSS. CCCA Investigator Global Severity Score (IGSS) assess subjects on a scale of 0 (no hair loss) to 6 (severe CCCA, e.g. >75% involvement of vertex). | Posted | Mean | Standard Deviation | score on a scale | Week 0 and Week 24 |
|
|
|
| Secondary | Mean Change in Central Hair Loss Grade (CHLG) | Mean change in CHLG at Week 24 compared to Baseline. Degree of severity of hair loss is graded on a 6-point visual scale (pattern 0: no hair loss, pattern 1-2: mild hair loss, pattern 3-5: more severe hair loss). | Posted | Mean | Standard Deviation | score on a scale | Week 0 and week 24 |
|
|
|
| Secondary | Mean Change in Subject Visual Analog Scale (VAS) of Hair Loss Severity | Mean change in VAS at Week 24 compared to Baseline. The VAS is a numerical scale used to assess patients' perception of hair loss severity. The evaluation is a 10cm long line on which the subjects indicate the severity of their condition from "0" (complete loss of hair in affected area - ie no visible hairs on central scalp) to "10" (full growth/regrowth in affected area-ie no visible hair loss on central scalp). | Posted | Mean | Standard Deviation | score on a scale | Week 0 and Week 24 |
|
|
|
| Secondary | Mean Change in Subject Global Assessment of Improvement | Mean change in PaGA-I at Week 24 as compared to Baseline. PaGA-I will range from -3 (significant worsening) to 3 (significant improvement). | Posted | Mean | Standard Deviation | score on a scale | Week 0 and Week 24 |
|
|
|
| Secondary | Change in Subject Rating of Symptom Severity Questionnaire (NRS) | Change in NRS at Week 24 as compared to Baseline. Subjects will complete a symptom severity questionnaire consisting of 3 numeric rating scales (NRS) measuring severity of pruritus, burning, and pain. The NRS will range from 0 (no symptoms) to 10 (severe symptoms). Patients indicate the intensity of each symptom (pruritus, burning, or pain) by choosing a number from 0 to 10 that corresponds to the severity of that symptom. | Posted | Mean | Standard Deviation | score on a scale | Week 0 and Week 24 |
|
|
|
| Secondary | Mean Change From Baseline in Dermatology Life Quality Index (DLQI) Total Score | Mean change in DLQI total score at Week 24 as compared to Baseline. DLQi is a 10-item questionnaire, each question is scored from 0 to 3, giving a possible score range from 0 (meaning no impact of skin disease on quality of life) to 30 (meaning maximum impact on quality of life). | Posted | Mean | Standard Deviation | score on a scale | Week 0 and Week 24 |
|
|
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| 20 |
| 0 |
| 20 |
| 19 |
| 20 |
| Flatulence | Gastrointestinal disorders | Systematic Assessment |
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| Reflux | Gastrointestinal disorders | Systematic Assessment |
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| Headache | Nervous system disorders | Systematic Assessment |
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| Loose stools | Gastrointestinal disorders | Systematic Assessment |
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| Diarrhea | Gastrointestinal disorders | Systematic Assessment |
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| Depression | Psychiatric disorders | Systematic Assessment |
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| Decreased appetite | General disorders | Systematic Assessment |
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| Abdominal pain | Gastrointestinal disorders | Systematic Assessment |
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| Weight Loss | General disorders | Systematic Assessment |
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| Common cold | Infections and infestations | Systematic Assessment |
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| Upper respiratory infection | Infections and infestations | Systematic Assessment |
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| Tiredness | General disorders | Systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | Systematic Assessment |
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| Increased bowel movements | Gastrointestinal disorders | Systematic Assessment |
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| Covid like illness | Infections and infestations | Systematic Assessment |
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| Increased hair loss | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Nasal congestion | General disorders | Systematic Assessment |
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| Cracked tooth | General disorders | Systematic Assessment |
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| Anxiety | Psychiatric disorders | Systematic Assessment |
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| Lower back muscle strain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| left knee pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
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| Itchy scalp | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Seasonal allergies | General disorders | Systematic Assessment |
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| Sinus headache | General disorders | Systematic Assessment |
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| Hair breakage | Psychiatric disorders | Systematic Assessment |
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| Sinusitis | General disorders | Systematic Assessment |
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| Scalp pain | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Worsening urticaria | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Intention tremor | Nervous system disorders | Systematic Assessment |
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| Insomnia | Psychiatric disorders | Systematic Assessment |
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| Subjective PIH | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Hyperactivity | Psychiatric disorders | Systematic Assessment |
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| Abnormal mammogram | Reproductive system and breast disorders | Systematic Assessment |
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| Post nasal drip | General disorders | Systematic Assessment |
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| Irregular periods | Reproductive system and breast disorders | Systematic Assessment |
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| Perioral dermatitis | Skin and subcutaneous tissue disorders | Systematic Assessment |
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| Eye Infection | Eye disorders | Systematic Assessment |
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