Frontal Fibrosing Alopecia
Conditions
Brief summary
This purpose of this trial was to investigate the molecular signature of frontal fibrosing alopecia (FFA) and the effect of delgocitinib cream 2% on reversing the FFA disease signature in active lesions. The trial also investigated the clinical effect of delgocitinib cream on FFA compared to a placebo cream.
Interventions
Cream for topical application
The cream vehicle is similar to the delgocitinib cream except that it does not contain any active ingredient.
Sponsors
LEO Pharma
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
Yes
Inclusion criteria
For Group 1 only (subjects with FFA): 1. Male or female subject aged 18 years of age or older at the time of consent. 2. Subject has clinically confirmed diagnosis of FFA. 3. Subject has a target area with a perifollicular erythema score ≥ 2 and a perifollicular scale score ≥ 2 at Screening and Day 1. For Group 2 only (healthy subjects): 1. Female subject aged 45 years of age or older at the time of consent. 2. Female is postmenopausal. 3. Subject is in good general health.
Exclusion criteria
For all subjects: 1. Subject is a female who is breastfeeding, pregnant, or who is planning to become pregnant during the trial. 2. Presence of hepatitis B or C infection or HIV infection at screening. For Group 1 only (subjects with FFA): 1. History of other scalp/hair disease including discoid lupus erythematosus and central centrifugal cicatricial alopecia. 2. Subject who has undergone scalp reduction surgery or hair transplantation. 3. Subject is known to have immune deficiency or is immunocompromised. 4. Subject has used intralesional scalp corticosteroids or platelet rich plasma injection in the last 4 weeks prior to randomization. 5. Subject has used systemic treatment with immunosuppressive/modulating medication or medication within 4 weeks prior to randomization. 6. Subject has used any topical medicated treatment that could affect FFA within 2 weeks prior to randomization. 7. Subject has received any phototherapy within 4 weeks prior to randomization. For Group 2 only (healthy subjects): 1. Subject has a history of skin disease or presence of skin condition that, in the opinion of the investigator, would interfere with the trial assessments. 2. Subject has used a topical medicated treatment on the targeted skin sites within 2 weeks prior to trial assessments.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Expression of Chemokine (C-X-C Motif) Ligand 9 (CXCL9), Chemokine (C-X-C Motif) Ligand 10 (CXCL10), and Interferon (IFN)-γ From Baseline to Week 12. | Baseline and Week 12 | CXCL9, CXCL10 and IFN-γ are small proteins that act as chemical messengers, especially in the immune system. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of Treatment-emergent Adverse Events (TEAEs) From Baseline to Week 12. | Between baseline and Week 12 | Treatment emergent adverse events (TEAEs) were defined as any AEs with onset date on or after the first study treatment dosing. |
Countries
United States
Participant flow
Recruitment details
The trial was conducted in the United States and consisted of 2 cohorts: Cohort 1 included 30 subjects with FFA and Cohort 2 included 5 healthy postmenopausal female subjects. Both cohorts were conducted in parallel.
Pre-assignment details
Eligible subjects were randomized into the trial after a screening period. They were assigned to receive either delgocitinib cream or a placebo twice daily for 12 weeks. After completing this period, they continued using delgocitinib cream for another 12 weeks.
Participants by arm
| Arm | Count |
|---|---|
| Delgocitinib - Delgocitinib Participants were blinded and randomised to delgocitinib cream treatment for the first 12 weeks, followed by an open label treatment with delgocitinib cream treatment for another 12 weeks.
Delgocitinib cream: Cream for topical application | 15 |
| Placebo - Delgocitinib Participants were blinded and randomised to placebo cream treatment for the first 12 weeks, followed by an open label treatment with delgocitinib cream treatment for another 12 weeks.
Delgocitinib cream: Cream for topical application
Delgocitinib cream vehicle: The cream vehicle is similar to the delgocitinib cream except that it does not contain any active ingredient. | 15 |
| No Treatment Participants did not receive any treatment. They will only provide a molecular signature of healthy skin to act as a control. | 5 |
| Total | 35 |
Baseline characteristics
| Characteristic | Delgocitinib - Delgocitinib | Total | No Treatment | Placebo - Delgocitinib |
|---|---|---|---|---|
| Age, Continuous | 66.2 years STANDARD_DEVIATION 6.82 | 64.8 years STANDARD_DEVIATION 10 | 67.4 years STANDARD_DEVIATION 9.81 | 62.6 years STANDARD_DEVIATION 12.64 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants | 2 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 13 Participants | 33 Participants | 5 Participants | 15 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Height (cm) | 163.34 units on a scale STANDARD_DEVIATION 7.557 | 163.24 units on a scale STANDARD_DEVIATION 7.257 | 165.20 units on a scale STANDARD_DEVIATION 8.415 | 162.49 units on a scale STANDARD_DEVIATION 6.97 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 1 Participants | 1 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 14 Participants | 34 Participants | 5 Participants | 15 Participants |
| Region of Enrollment United States | 15 participants | 35 participants | 5 participants | 15 participants |
| Sex: Female, Male Female | 15 Participants | 35 Participants | 5 Participants | 15 Participants |
| Sex: Female, Male Male | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Weight (kg) | 68.41 units on a scale STANDARD_DEVIATION 15.245 | 71.98 units on a scale STANDARD_DEVIATION 15.95 | 72.40 units on a scale STANDARD_DEVIATION 17.418 | 75.41 units on a scale STANDARD_DEVIATION 16.503 |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 15 | 0 / 15 | 0 / 0 |
| other Total, other adverse events | 6 / 15 | 10 / 15 | 0 / 0 |
| serious Total, serious adverse events | 0 / 15 | 0 / 15 | 0 / 0 |
Outcome results
Primary
Change in Expression of Chemokine (C-X-C Motif) Ligand 9 (CXCL9), Chemokine (C-X-C Motif) Ligand 10 (CXCL10), and Interferon (IFN)-γ From Baseline to Week 12.
CXCL9, CXCL10 and IFN-γ are small proteins that act as chemical messengers, especially in the immune system.
Time frame: Baseline and Week 12
Population: No data was collected for No Treatment Arm/Groups.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Delgocitinib - Delgocitinib | Change in Expression of Chemokine (C-X-C Motif) Ligand 9 (CXCL9), Chemokine (C-X-C Motif) Ligand 10 (CXCL10), and Interferon (IFN)-γ From Baseline to Week 12. | (C-X-C motif) ligand 9 (CXCL9) | -3.10 Fold-changes |
| Delgocitinib - Delgocitinib | Change in Expression of Chemokine (C-X-C Motif) Ligand 9 (CXCL9), Chemokine (C-X-C Motif) Ligand 10 (CXCL10), and Interferon (IFN)-γ From Baseline to Week 12. | Chemokine (C-X-C motif) ligand 10 (CXCL10) | -2.60 Fold-changes |
| Delgocitinib - Delgocitinib | Change in Expression of Chemokine (C-X-C Motif) Ligand 9 (CXCL9), Chemokine (C-X-C Motif) Ligand 10 (CXCL10), and Interferon (IFN)-γ From Baseline to Week 12. | Interferon (IFN)-γ | -1.49 Fold-changes |
| Placebo - Delgocitinib | Change in Expression of Chemokine (C-X-C Motif) Ligand 9 (CXCL9), Chemokine (C-X-C Motif) Ligand 10 (CXCL10), and Interferon (IFN)-γ From Baseline to Week 12. | (C-X-C motif) ligand 9 (CXCL9) | -1.12 Fold-changes |
| Placebo - Delgocitinib | Change in Expression of Chemokine (C-X-C Motif) Ligand 9 (CXCL9), Chemokine (C-X-C Motif) Ligand 10 (CXCL10), and Interferon (IFN)-γ From Baseline to Week 12. | Chemokine (C-X-C motif) ligand 10 (CXCL10) | -1.10 Fold-changes |
| Placebo - Delgocitinib | Change in Expression of Chemokine (C-X-C Motif) Ligand 9 (CXCL9), Chemokine (C-X-C Motif) Ligand 10 (CXCL10), and Interferon (IFN)-γ From Baseline to Week 12. | Interferon (IFN)-γ | -1.097 Fold-changes |
Secondary
Number of Treatment-emergent Adverse Events (TEAEs) From Baseline to Week 12.
Treatment emergent adverse events (TEAEs) were defined as any AEs with onset date on or after the first study treatment dosing.
Time frame: Between baseline and Week 12
Population: No investigational product was administered in Cohort 2 No Treatment Arm/Groups and thereby were no treatment-emergent AESIs reported in this trial for No Treatment Arm/Groups.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Delgocitinib - Delgocitinib | Number of Treatment-emergent Adverse Events (TEAEs) From Baseline to Week 12. | 4 events |
| Placebo - Delgocitinib | Number of Treatment-emergent Adverse Events (TEAEs) From Baseline to Week 12. | 9 events |