Trial Outcomes & Findings for Use of CXCL9 as a Biomarker of Acthar Efficacy (NCT NCT02523092)

NCT ID: NCT02523092

Last Updated: 2026-04-30

Results Overview

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.

Recruitment status

TERMINATED

Study phase

PHASE4

Target enrollment

2 participants

Primary outcome timeframe

within 6 months

Results posted on

2026-04-30

Participant Flow

Participant milestones

Participant milestones
Measure
Acthar Gel
After a 4-week period of baseline monitoring, Acthar gel will be administered by intramuscular or subcutaneous injection. Initial dosing will be 40 U every 72 hours (or twice per week) for 4 weeks. Dosage will then be increased to 80 U with similar frequency for 8 weeks and up to 16 weeks.
Overall Study
STARTED
2
Overall Study
COMPLETED
2
Overall Study
NOT COMPLETED
0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Use of CXCL9 as a Biomarker of Acthar Efficacy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Acthar Gel
n=2 Participants
After a 4-week period of baseline monitoring, Acthar gel will be administered by intramuscular or subcutaneous injection. Initial dosing will be 40 U every 72 hours (or twice per week) for 4 weeks. Dosage will then be increased to 80 U with similar frequency for 8 weeks and up to 16 weeks.
Sex: Female, Male
Female
1 Participants
n=14 Participants
Sex: Female, Male
Male
1 Participants
n=14 Participants
Race (NIH/OMB)
American Indian or Alaska Native
0 Participants
n=14 Participants
Race (NIH/OMB)
Asian
0 Participants
n=14 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=14 Participants
Race (NIH/OMB)
Black or African American
1 Participants
n=14 Participants
Race (NIH/OMB)
White
1 Participants
n=14 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=14 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=14 Participants
Region of Enrollment
United States
2 Participants
n=14 Participants
Age, Categorical
<=18 years
0 Participants
n=14 Participants
Age, Categorical
Between 18 and 65 years
2 Participants
n=14 Participants
Age, Categorical
>=65 years
0 Participants
n=14 Participants

PRIMARY outcome

Timeframe: within 6 months

Population: for the two participants, blood samples were analyzed before starting Acthar gel and at the last visit time point at 6 months. Therefore, two time points were analyzed per participant.

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.

Outcome measures

Outcome measures
Measure
Observational Arm
n=2 Participants
observational arm
Number of Participants That Show a Decrease in Blood CXCL9 Levels by 50%
0 Participants

SECONDARY outcome

Timeframe: within six months

Outcome measures

Outcome measures
Measure
Observational Arm
n=2 Participants
observational arm
Participants With Improvement in Forced Vital Capacity (FVC) Liters by 5 Percent or More
Yes
1 Participants
Participants With Improvement in Forced Vital Capacity (FVC) Liters by 5 Percent or More
No
1 Participants

SECONDARY outcome

Timeframe: 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.

Outcome measures

Outcome measures
Measure
Observational Arm
n=2 Participants
observational arm
Participants With Improvement in Dyspnea Score
Yes
1 Participants
Participants With Improvement in Dyspnea Score
No
1 Participants

Adverse Events

Acthar Gel

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

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Laura Koth, MD

UCSF

Phone: 415-476-9225

Results disclosure agreements

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