Trial Outcomes & Findings for Apremilast in the Treatment of Central Centrifugal Cicatricial Alopecia (CCCA) (NCT NCT03521687)

NCT ID: NCT03521687

Last Updated: 2022-05-09

Results Overview

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).

Recruitment status

COMPLETED

Study phase

PHASE4

Target enrollment

20 participants

Primary outcome timeframe

Week 0 and Week 24

Results posted on

2022-05-09

Participant Flow

Enrollment period from November 2018 to July 2020

Participant milestones

Participant milestones
Measure
Apremilast
Patients with CCCA Apremilast: 30 mg BID
Overall Study
STARTED
20
Overall Study
COMPLETED
15
Overall Study
NOT COMPLETED
5

Reasons for withdrawal

Reasons for withdrawal
Measure
Apremilast
Patients with CCCA Apremilast: 30 mg BID
Overall Study
Adverse Event
4
Overall Study
Physician Decision
1

Baseline Characteristics

Apremilast in the Treatment of Central Centrifugal Cicatricial Alopecia (CCCA)

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Apremilast
n=20 Participants
Patients with CCCA Apremilast: 30 mg BID
Age, Continuous
49.8 years
STANDARD_DEVIATION 11.11 • n=39 Participants
Sex: Female, Male
Female
20 Participants
n=39 Participants
Sex: Female, Male
Male
0 Participants
n=39 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
1 Participants
n=39 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
19 Participants
n=39 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
0 Participants
n=39 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=39 Participants
Race (NIH/OMB)
Asian
0 Participants
n=39 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=39 Participants
Race (NIH/OMB)
Black or African American
20 Participants
n=39 Participants
Race (NIH/OMB)
White
0 Participants
n=39 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=39 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=39 Participants

PRIMARY outcome

Timeframe: Week 0 and Week 24

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).

Outcome measures

Outcome measures
Measure
Apremilast
n=15 Participants
Patients with CCCA Apremilast: 30 mg BID
Mean Change in Physician Global Assessment of Improvement (PGA-I)
0.07 score on a scale
Standard Deviation 0.51

SECONDARY outcome

Timeframe: Week 0 and Week 24

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).

Outcome measures

Outcome measures
Measure
Apremilast
n=15 Participants
Patients with CCCA Apremilast: 30 mg BID
Mean Change in CCCA Investigator Global Severity Score (IGSS)
-0.31 score on a scale
Standard Deviation 0.65

SECONDARY outcome

Timeframe: Week 0 and week 24

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).

Outcome measures

Outcome measures
Measure
Apremilast
n=15 Participants
Patients with CCCA Apremilast: 30 mg BID
Mean Change in Central Hair Loss Grade (CHLG)
-0.44 score on a scale
Standard Deviation 0.86

SECONDARY outcome

Timeframe: Week 0 and Week 24

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).

Outcome measures

Outcome measures
Measure
Apremilast
n=15 Participants
Patients with CCCA Apremilast: 30 mg BID
Mean Change in Subject Visual Analog Scale (VAS) of Hair Loss Severity
-1.94 score on a scale
Standard Deviation 1.86

SECONDARY outcome

Timeframe: Week 0 and Week 24

Mean change in PaGA-I at Week 24 as compared to Baseline. PaGA-I will range from -3 (significant worsening) to 3 (significant improvement).

Outcome measures

Outcome measures
Measure
Apremilast
n=15 Participants
Patients with CCCA Apremilast: 30 mg BID
Mean Change in Subject Global Assessment of Improvement
1 score on a scale
Standard Deviation 1.45

SECONDARY outcome

Timeframe: Week 0 and Week 24

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.

Outcome measures

Outcome measures
Measure
Apremilast
n=15 Participants
Patients with CCCA Apremilast: 30 mg BID
Change in Subject Rating of Symptom Severity Questionnaire (NRS)
0.94 score on a scale
Standard Deviation 3.99

SECONDARY outcome

Timeframe: Week 0 and Week 24

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).

Outcome measures

Outcome measures
Measure
Apremilast
n=15 Participants
Patients with CCCA Apremilast: 30 mg BID
Mean Change From Baseline in Dermatology Life Quality Index (DLQI) Total Score
-2.18 score on a scale
Standard Deviation 3.79

Adverse Events

Apremilast

Serious events: 0 serious events
Other events: 19 other events
Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure
Apremilast
n=20 participants at risk
Patients with CCCA Apremilast: 30 mg BID
Gastrointestinal disorders
Nausea
25.0%
5/20 • 24 weeks
Gastrointestinal disorders
Flatulence
15.0%
3/20 • 24 weeks
Gastrointestinal disorders
Reflux
10.0%
2/20 • 24 weeks
Nervous system disorders
Headache
25.0%
5/20 • 24 weeks
Gastrointestinal disorders
Loose stools
25.0%
5/20 • 24 weeks
Gastrointestinal disorders
Diarrhea
15.0%
3/20 • 24 weeks
Psychiatric disorders
Depression
15.0%
3/20 • 24 weeks
General disorders
Decreased appetite
25.0%
5/20 • 24 weeks
Gastrointestinal disorders
Abdominal pain
20.0%
4/20 • 24 weeks
General disorders
Weight Loss
15.0%
3/20 • 24 weeks
Infections and infestations
Common cold
10.0%
2/20 • 24 weeks
Infections and infestations
Upper respiratory infection
15.0%
3/20 • 24 weeks
General disorders
Tiredness
10.0%
2/20 • 24 weeks
Respiratory, thoracic and mediastinal disorders
Cough
10.0%
2/20 • 24 weeks
Gastrointestinal disorders
Increased bowel movements
25.0%
5/20 • 24 weeks
Infections and infestations
Covid like illness
15.0%
3/20 • 24 weeks
Skin and subcutaneous tissue disorders
Increased hair loss
10.0%
2/20 • 24 weeks
General disorders
Nasal congestion
5.0%
1/20 • 24 weeks
General disorders
Cracked tooth
5.0%
1/20 • 24 weeks
Psychiatric disorders
Anxiety
5.0%
1/20 • 24 weeks
Musculoskeletal and connective tissue disorders
Lower back muscle strain
5.0%
1/20 • 24 weeks
Musculoskeletal and connective tissue disorders
left knee pain
5.0%
1/20 • 24 weeks
Skin and subcutaneous tissue disorders
Itchy scalp
5.0%
1/20 • 24 weeks
General disorders
Seasonal allergies
5.0%
1/20 • 24 weeks
General disorders
Sinus headache
5.0%
1/20 • 24 weeks
Psychiatric disorders
Hair breakage
5.0%
1/20 • 24 weeks
General disorders
Sinusitis
5.0%
1/20 • 24 weeks
Skin and subcutaneous tissue disorders
Scalp pain
5.0%
1/20 • 24 weeks
Skin and subcutaneous tissue disorders
Worsening urticaria
5.0%
1/20 • 24 weeks
Nervous system disorders
Intention tremor
5.0%
1/20 • 24 weeks
Psychiatric disorders
Insomnia
5.0%
1/20 • 24 weeks
Skin and subcutaneous tissue disorders
Subjective PIH
5.0%
1/20 • 24 weeks
Psychiatric disorders
Hyperactivity
5.0%
1/20 • 24 weeks
Reproductive system and breast disorders
Abnormal mammogram
5.0%
1/20 • 24 weeks
General disorders
Post nasal drip
5.0%
1/20 • 24 weeks
Reproductive system and breast disorders
Irregular periods
5.0%
1/20 • 24 weeks
Skin and subcutaneous tissue disorders
Perioral dermatitis
5.0%
1/20 • 24 weeks
Eye disorders
Eye Infection
5.0%
1/20 • 24 weeks

Additional Information

Ingrid Sanabria-Gonzalez

Icahn School of Medicine at Mount Sinai

Phone: 212-523-3812

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place