Sarcoidosis
Conditions
Keywords
sarcoidosis, Acthar, CXCL9, chemokine ligand 9
Brief summary
The objective is this study is to test whether use of Acthar gel in the context of sarcoidosis will lead to improved symptoms and lung function and correlate with decreased levels of predictive blood biomarkers, like chemokine ligand 9 (CXCL9).
Detailed description
The investigators will test whether Acthar gel's anti-inflammatory properties will modulate immune cells and lead to decreases in blood biomarkers and improvements in clinical parameters. Specific Aim 1 will examine the levels of the predictive biomarker, chemokine ligand 9 (CXCL9), and related transcripts, and determine whether they decrease in participants over time while taking Acthar. Specific Aim 2 will test whether the biologic changes measured in blood correlate to clinical markers, including lung function and symptom scores. Since the investigators have found that CXCL9 predicts clinical course, they hypothesize that CXCL9 transcript levels in the blood will decrease over time in pulmonary sarcoidosis participants whose clinical outcome measures improve with Acthar.
Interventions
DRUGActhar gel
Acthar gel given IM or SQ as per package insert
Sponsors
University of California, San Francisco
Mallinckrodt
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to 65 Years
Healthy volunteers
No
Inclusion criteria
* Biopsy-proven diagnosis of sarcoidosis with demonstrated pulmonary involvement * Refractoriness to or intolerance of immunosuppressive agents like prednisone or methotrexate
Exclusion criteria
* Smoking * Cancer * Chronic infections (e.g. tuberculosis, viral, fungal, bacterial) * Inflammatory conditions * Coexisting lung disease * Congestive heart failure * Uncontrolled hypertension * Recent surgery * Active peptic ulcers * Osteoporosis
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants That Show a Decrease in Blood CXCL9 Levels by 50% | within 6 months | Quantitative polymerase chain reaction assay was used to measure the gene expression for the gene CXCL9 using whole blood samples. The change in cycle threshold from before starting medication to the end of treatment was calculated. From this calculation, the number of participants that met the criteria of a 50% or greater drop in CXCL9 levels is reported. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Participants With Improvement in Forced Vital Capacity (FVC) Liters by 5 Percent or More | within six months | — |
| Participants With Improvement in Dyspnea Score | within 6 months | The University of California San Diego Shortness of Breath Questionnaire (UCSD SOBQ) is a 24-item patient-reported outcome measure that assesses the severity of dyspnea during activities of daily living. Participants rate shortness of breath for each activity on a 6-point scale from 0 ("not at all") to 5 ("maximally or unable to do because of breathlessness"). Item scores are summed to generate a total score ranging from 0 to 120. Higher total scores represent worse dyspnea severity, while lower scores indicate less dyspnea. The SOBQ does not contain separate subscales; all 24 items are equally weighted and combined by simple summation to calculate the total score. If a participant does not routinely perform an activity, they are instructed to estimate anticipated dyspnea, minimizing missing data. |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORLaura Koth, MD
University of California, San Francisco
Baseline characteristics
| Characteristic | — |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 0 Participants |
| Age, Categorical Between 18 and 65 years | 2 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants |
| Race (NIH/OMB) Black or African American | 1 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 | 1 Participants |
| Region of Enrollment United States | 2 Participants |
| Sex: Female, Male Female | 1 Participants |
| Sex: Female, Male Male | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 0 / 2 |
| other Total, other adverse events | 0 / 2 |
| serious Total, serious adverse events | 0 / 2 |
Outcome results
None listed