Trial Outcomes & Findings for ACTHar in the Treatment of Lupus Nephritis (NCT NCT02226341)
NCT ID: NCT02226341
Last Updated: 2025-03-07
Results Overview
Complete response (CR) is defined as all of the following criteria having been achieved: 1. Stabilization of estimated glomerular filtration rate (eGFR) (i.e., a 6-month eGFR level ± 10% of baseline) or improvement if the screening value is changed from patient's baseline 2. Inactive urinary sediment (red blood cells per high-power field \[RBCs/HPF\] \< 5-10, not due to gyn bleeding) 3. Urine protein/creatinine ratio \< 0.5
Recruitment status
TERMINATED
Study phase
PHASE4
Target enrollment
8 participants
Primary outcome timeframe
6 Months
Results posted on
2025-03-07
Participant Flow
Participant milestones
| Measure |
CellCept Daily & ACTHar Gel BIW (Two Times Per Week)
Patients will be treated with CellCept 3 grams daily and ACTHar gel 80 U BIW for 3 months per Aim 1. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U biw for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
CellCept Daily & ACTHar Gel TIW (Three Times Per Week)
Patients will be treated with CellCept 3 grams daily for 3 months, and ACTHar gel 80 U TIW for the first month and ACTHar gel 80 U BIW for the following 2 months per Aim 2. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U BIW for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
|---|---|---|
|
Overall Study
STARTED
|
4
|
4
|
|
Overall Study
COMPLETED
|
4
|
4
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
ACTHar in the Treatment of Lupus Nephritis
Baseline characteristics by cohort
| Measure |
CellCept Daily & ACTHar Gel BIW (Two Times Per Week)
n=4 Participants
Patients will be treated with CellCept 3 grams daily and ACTHar gel 80 U BIW for 3 months per Aim 1. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U biw for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
CellCept Daily & ACTHar Gel TIW (Three Times Per Week)
n=4 Participants
Patients will be treated with CellCept 3 grams daily for 3 months, and ACTHar gel 80 U TIW for the first month and ACTHar gel 80 U BIW for the following 2 months per Aim 2. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U BIW for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
Total
n=8 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
26.5 years
n=5 Participants
|
28.5 years
n=7 Participants
|
26.5 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
4 Participants
n=7 Participants
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
1 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
3 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
1 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
2 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
3 Participants
n=5 Participants
|
2 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
4 participants
n=5 Participants
|
4 participants
n=7 Participants
|
8 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 6 MonthsComplete response (CR) is defined as all of the following criteria having been achieved: 1. Stabilization of estimated glomerular filtration rate (eGFR) (i.e., a 6-month eGFR level ± 10% of baseline) or improvement if the screening value is changed from patient's baseline 2. Inactive urinary sediment (red blood cells per high-power field \[RBCs/HPF\] \< 5-10, not due to gyn bleeding) 3. Urine protein/creatinine ratio \< 0.5
Outcome measures
| Measure |
CellCept Daily & ACTHar Gel BIW (Two Times Per Week)
n=4 Participants
Patients will be treated with CellCept 3 grams daily and ACTHar gel 80 U BIW for 3 months per Aim 1. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U biw for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
CellCept Daily & ACTHar Gel TIW (Three Times Per Week)
n=4 Participants
Patients will be treated with CellCept 3 grams daily for 3 months, and ACTHar gel 80 U TIW for the first month and ACTHar gel 80 U BIW for the following 2 months per Aim 2. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U BIW for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
|---|---|---|
|
Number of Patients With a Complete Response (CR)
|
2 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 6 MonthsResponders = Complete Responders (CR) + Partial Responders (PR). PR = improvement from baseline of at least ≥ 50% in all abnormal renal parameters (proteinuria and serum creatinine) without deterioration of any measurements at 6 months
Outcome measures
| Measure |
CellCept Daily & ACTHar Gel BIW (Two Times Per Week)
n=4 Participants
Patients will be treated with CellCept 3 grams daily and ACTHar gel 80 U BIW for 3 months per Aim 1. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U biw for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
CellCept Daily & ACTHar Gel TIW (Three Times Per Week)
n=4 Participants
Patients will be treated with CellCept 3 grams daily for 3 months, and ACTHar gel 80 U TIW for the first month and ACTHar gel 80 U BIW for the following 2 months per Aim 2. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U BIW for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
|---|---|---|
|
Number of Responders
|
2 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 6 MonthsFrequency of extra-renal flares as defined by the SELENA-SLEDAI Flare Index. Extra-renal disease activity measured by SELENA-SLEDAI and BILAG
Outcome measures
| Measure |
CellCept Daily & ACTHar Gel BIW (Two Times Per Week)
n=4 Participants
Patients will be treated with CellCept 3 grams daily and ACTHar gel 80 U BIW for 3 months per Aim 1. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U biw for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
CellCept Daily & ACTHar Gel TIW (Three Times Per Week)
n=4 Participants
Patients will be treated with CellCept 3 grams daily for 3 months, and ACTHar gel 80 U TIW for the first month and ACTHar gel 80 U BIW for the following 2 months per Aim 2. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U BIW for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
|---|---|---|
|
Number of Patients With Extra-renal Flares
|
0 Participants
|
1 Participants
|
SECONDARY outcome
Timeframe: 6 MonthsSteroid -like side effects: increase in blood pressure (BP) by 20 mmHg for both systolic blood pressure (SBP) and diastolic blood pressure (DBP), increased blood sugar with a fasting plasma glucose level ≥ 126 mg/dl, weight gain ≥ 10% of the initial weight, infections
Outcome measures
| Measure |
CellCept Daily & ACTHar Gel BIW (Two Times Per Week)
n=4 Participants
Patients will be treated with CellCept 3 grams daily and ACTHar gel 80 U BIW for 3 months per Aim 1. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U biw for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
CellCept Daily & ACTHar Gel TIW (Three Times Per Week)
n=4 Participants
Patients will be treated with CellCept 3 grams daily for 3 months, and ACTHar gel 80 U TIW for the first month and ACTHar gel 80 U BIW for the following 2 months per Aim 2. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U BIW for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
|---|---|---|
|
Number of Patients With Steroid -Like Side Effects
|
0 Participants
|
0 Participants
|
SECONDARY outcome
Timeframe: 6 MonthsNumber of participants who experienced side effects from taking ACTHar
Outcome measures
| Measure |
CellCept Daily & ACTHar Gel BIW (Two Times Per Week)
n=4 Participants
Patients will be treated with CellCept 3 grams daily and ACTHar gel 80 U BIW for 3 months per Aim 1. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U biw for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
CellCept Daily & ACTHar Gel TIW (Three Times Per Week)
n=4 Participants
Patients will be treated with CellCept 3 grams daily for 3 months, and ACTHar gel 80 U TIW for the first month and ACTHar gel 80 U BIW for the following 2 months per Aim 2. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U BIW for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
|---|---|---|
|
Number of Patients With Side Effects
|
0 Participants
|
0 Participants
|
OTHER_PRE_SPECIFIED outcome
Timeframe: Upon study completion, up to 30 monthsPopulation: Data was not collected due to lack of funding leading to early termination of the study.
Urinary markers of active inflammatory nephritis
Outcome measures
Outcome data not reported
OTHER_PRE_SPECIFIED outcome
Timeframe: Upon study completion, up to 30 monthsPopulation: Data was not collected due to lack of funding leading to early termination of the study.
Cortisol levels 8 hours after ACTHar dose in 2-3 patients per dosing arm
Outcome measures
Outcome data not reported
Adverse Events
CellCept Daily & ACTHar Gel BIW (Two Times Per Week)
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
CellCept Daily & ACTHar Gel TIW (Three Times Per Week)
Serious events: 1 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
CellCept Daily & ACTHar Gel BIW (Two Times Per Week)
n=4 participants at risk
Patients will be treated with CellCept 3 grams daily and ACTHar gel 80 U BIW for 3 months per Aim 1. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U biw for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
CellCept Daily & ACTHar Gel TIW (Three Times Per Week)
n=4 participants at risk
Patients will be treated with CellCept 3 grams daily for 3 months, and ACTHar gel 80 U TIW for the first month and ACTHar gel 80 U BIW for the following 2 months per Aim 2. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U BIW for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
|---|---|---|
|
General disorders
Extra-renal flare
|
0.00%
0/4 • 6 months
|
25.0%
1/4 • 6 months
|
Other adverse events
| Measure |
CellCept Daily & ACTHar Gel BIW (Two Times Per Week)
n=4 participants at risk
Patients will be treated with CellCept 3 grams daily and ACTHar gel 80 U BIW for 3 months per Aim 1. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U biw for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
CellCept Daily & ACTHar Gel TIW (Three Times Per Week)
n=4 participants at risk
Patients will be treated with CellCept 3 grams daily for 3 months, and ACTHar gel 80 U TIW for the first month and ACTHar gel 80 U BIW for the following 2 months per Aim 2. After 3 months, patients with complete response will stop ACTHar gel but continue CellCept 3 grams daily for another 3 months whereas patients with partial response will continue CellCept 3 grams daily and be offered the option to continue ACTHar 80 U BIW for another 3 months. After 6 months, all patients will continue CellCept at a dose of 2 grams daily for another 18 months.
|
|---|---|---|
|
Renal and urinary disorders
Renal flare
|
25.0%
1/4 • 6 months
|
0.00%
0/4 • 6 months
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place