Necrobiosis Lipoidica
Conditions
Brief summary
This research study is evaluating the safety and efficacy of a topical drug treatment Ruxolitinib in treating Necrobiosis Lipoidica (NL).
Interventions
DRUGRuxolitinib
Ruxolitinib cream 1.5%, topical application will be used twice daily on lesions of Necrobiosis Lipoidica
Sponsors
Aaron R. Mangold
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Subjects must be able to understand and comply with the requirements of the study and communicate with the investigator. Subjects must give written, signed, and dated informed consent before any study related activity is performed. When appropriate, a legal representative will sign the informed consent according to local laws and regulation * Both men and women must be at least 18 years of age at the time of screening * Subjects must have clinical and histological features of NL * Subjects must have at least one NL lesion measuring at least 1.7 cm * NL must not affect greater than 10% BSA
Exclusion criteria
* On excluded therapies, not on a stable dose of a therapy, or incompletely washed out for a therapy (Table-1). * Known hypersensitivity to Ruxolitinib formulation. * Pregnant or nursing (lactating) women (pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive human chorionic gonadotropin (hCG) laboratory test). * Women of childbearing potential \[Post-menopausal or not of child-bearing potential is defined by: 1 year of natural (spontaneous) amenorrhea or Surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy or tubal ligation at least 6 weeks ago. Oophorectomy alone must be confirmed by follow up hormone level assessment to be considered not of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using medically acceptable methods of contraception which includes: * Total abstinence (periodic abstinence and withdrawal are not acceptable methods of contraception). * Female sterilization (bilateral oophorectomy with or without hysterectomy), total hysterectomy, or tubal ligation at least 6 weeks before taking study treatment. Oophorectomy alone requires follow up hormone level assessment for fertility. * Male sterilization (at least 6 months prior to screening). The vasectomized male partner should be the sole partner for that subject. * Barrier methods of contraception: condom or occlusive cap. * Use of oral, injected or implanted hormonal methods of contraception or other forms or hormonal contraception that have complete efficacy (failure \<1%). (The dose of the contraceptive should be stable for 3 months). * Active ongoing inflammatory diseases of the skin other than NL that might confound the evaluation of the benefit of ruxolitinib cream. * Underlying condition (including, but not limited to metabolic, hematologic, renal, hepatic, pulmonary, neurologic, endocrine, cardiac, infectious or gastrointestinal conditions) which, in the opinion of the investigator, significantly immunocompromised the subject and/or places the subject at unacceptable risk for receiving an immunomodulatory therapy. * Active systemic infections during the 2 weeks prior to randomization (common cold viruses not included) or any infection that reoccurs on a regular basis. * Current severe progressive or uncontrolled disease which the investigator renders the subject unsuitable for the trial or puts the subject at increased risk.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Mean Necrobiosis Lipoidica (NL) Lesion Score | Baseline, week 12 | The NL skin scoring system examines the index treatment lesion on a scale from 0 (none) to 3 (severe) for each of the following areas: erythema, infiltration, ulceration, and pain. A total possible score of 0-12, with lower scores indicating none and higher scores indicating more severe. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Necrobiosis Lipoidica Adults with cutaneous Necrobiosis Lipoidica (NL) up to 10% of the body surface area (BSA) were treated with Ruxolitinib cream
Ruxolitinib: Ruxolitinib cream 1.5%, topical application was used twice daily on lesions of Necrobiosis Lipoidica | 12 |
| Total | 12 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Withdrawal by Subject | 1 |
Baseline characteristics
| Characteristic | Necrobiosis Lipoidica |
|---|---|
| Age, Continuous | 43.6 years STANDARD_DEVIATION 12.9 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 10 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants |
| Race (NIH/OMB) White | 12 Participants |
| Region of Enrollment United States | 12 participants |
| Sex: Female, Male Female | 12 Participants |
| Sex: Female, Male Male | 0 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 12 |
| other Total, other adverse events | 10 / 12 |
| serious Total, serious adverse events | 0 / 12 |
Outcome results
Primary
Change in Mean Necrobiosis Lipoidica (NL) Lesion Score
The NL skin scoring system examines the index treatment lesion on a scale from 0 (none) to 3 (severe) for each of the following areas: erythema, infiltration, ulceration, and pain. A total possible score of 0-12, with lower scores indicating none and higher scores indicating more severe.
Time frame: Baseline, week 12
Population: 1 Subject withdrew. Week 12 data was not collected nor analyzed
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Necrobiosis Lipoidica | Change in Mean Necrobiosis Lipoidica (NL) Lesion Score | -2.7 score on a scale | Standard Deviation 1.6 |
p-value: 0.005Wilcoxon (Mann-Whitney)