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Adrenocorticotropic Hormone (ACTH) for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome

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ClinicalTrials.gov Identifier: NCT02132195
Recruitment Status : Completed
First Posted : May 7, 2014
Results First Posted : May 10, 2019
Last Update Posted : May 29, 2019
Sponsor:
Collaborator:
Mallinckrodt
Information provided by (Responsible Party):
Larry Greenbaum, MD, PhD, Emory University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Nephrotic Syndrome
Intervention Drug: ACTH
Enrollment 31
Recruitment Details  
Pre-assignment Details Of 31 patients enrolled: 15 were randomized to receive ACTH, and 16 were randomized to No-treatment. There was an option for patients in No-treatment arm to elect to be placed on ACTH (rescue therapy). 13 patients in No-treatment arm used this option, and received ACTH as rescue therapy.
Arm/Group Title Adrenocorticotropic Hormone (ACTH) No Treatment Rescue Therapy
Hide Arm/Group Description

Patients will receive ACTH twice weekly subcutaneously The initial dosing will be based on body surface area (BSA): 80 IU/1.73 m2

The patients will receive the initial dose for 6 months. At 6 months, the dose will be reduced by 50%. Patients who have side effects may have the dose reduced by 50% during the initial 6 months. A second dose reduction would still occur at 6 months (25% of initial dose).

ACTH: Patients will receive ACTH twice weekly for 6 months, with a 50% dose reduction allowed for side effects. The dose will be reduce by 50% at 6 months and continued for an additional 6 months.

Patients in this treatment arm will receive no treatment to prevent relapses of nephrotic syndrome. A relapse, if it occurs, will be treated with prednisone and the patient will leave the no treatment arm of the study. There is an option for the patient to elect to be placed in the active treatment arm of the trial (rescue therapy).

Patients moved from No-treatment group to receive rescue ACTH therapy twice weekly for 6 months, with a 50% dose reduction allowed for side effects. The dose will be reduce by 50% at 6 months and continued for an additional 6 months.

Other Names: Acthar Adrenocorticotropic hormone

Period Title: Initial Assignment
Started 15 16 0
Completed 1 16 0
Not Completed 14 0 0
Reason Not Completed
Relapsed             14             0             0
Period Title: Rescue Therapy
Started [1] 0 0 13
Completed 0 0 2
Not Completed 0 0 11
Reason Not Completed
Relapsed             0             0             7
Physician Decision             0             0             1
Withdrawal by Subject             0             0             1
Adverse Event             0             0             2
[1]
There was an option for No-treatment pts to be placed on ACTH (rescue therapy). 13 pts used it
Arm/Group Title Adrenocorticotropic Hormone (ACTH) No Treatment Rescue Therapy Total
Hide Arm/Group Description

Patients will receive ACTH twice weekly subcutaneously The initial dosing will be based on body surface area (BSA): 80 IU/1.73 m2

The patients will receive the initial dose for 6 months. At 6 months, the dose will be reduced by 50%. Patients who have side effects may have the dose reduced by 50% during the initial 6 months. A second dose reduction would still occur at 6 months (25% of initial dose).

ACTH: Patients will receive ACTH twice weekly for 6 months, with a 50% dose reduction allowed for side effects. The dose will be reduce by 50% at 6 months and continued for an additional 6 months.

Patients in this treatment arm will receive no treatment to prevent relapses of nephrotic syndrome. A relapse, if it occurs, will be treated with prednisone and the patient will leave the no treatment arm of the study. There is an option for the patient to elect to be placed in the active treatment arm of the trial (rescue therapy). There is an option for the patient in no-treatment arm to elect to be placed in the active treatment arm of the trial (rescue therapy). Total of all reporting groups
Overall Number of Baseline Participants 15 16 13 44
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous   [1] 
Median (Inter-Quartile Range)
Unit of measure:  Years
Initial assignment Number Analyzed 15 participants 16 participants 0 participants 31 participants
7.0
(4.8 to 11.2)
9.5
(5.2 to 12.0)
8.6
(4.3 to 12.9)
Rescue therapy Number Analyzed 0 participants 0 participants 13 participants 13 participants
9.15
(4.84 to 11.9)
9.15
(4.84 to 11.9)
[1]
Measure Analysis Population Description: Of 31 patients enrolled: 15 were randomized to receive ACTH, and 16 were randomized to No-treatment. There was an option for patients in No-treatment arm to elect to be placed on ACTH (rescue therapy). 13 patients in No-treatment arm used this option, and received ACTH as rescue therapy.
Sex: Female, Male   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Initial assignment Number Analyzed 15 participants 16 participants 0 participants 31 participants
Female
5
  33.3%
4
  25.0%
0
9
  29.0%
Male
10
  66.7%
12
  75.0%
0
22
  71.0%
Rescue therapy Number Analyzed 0 participants 0 participants 13 participants 13 participants
Female
3
  23.1%
3
  23.1%
Male
10
  76.9%
10
  76.9%
[1]
Measure Analysis Population Description: Of 31 patients enrolled: 15 were randomized to receive ACTH, and 16 were randomized to No-treatment. There was an option for patients in No-treatment arm to elect to be placed on ACTH (rescue therapy). 13 patients in No-treatment arm used this option, and received ACTH as rescue therapy.
Race (NIH/OMB)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Initial assignment Number Analyzed 15 participants 16 participants 0 participants 31 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
0
   0.0%
1
   6.3%
1
   3.2%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
0
   0.0%
Black or African American
2
  13.3%
1
   6.3%
3
   9.7%
White
13
  86.7%
13
  81.3%
26
  83.9%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
1
   6.3%
1
   3.2%
Rescue therapy Number Analyzed 0 participants 0 participants 13 participants 13 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
Asian
1
   7.7%
1
   7.7%
Native Hawaiian or Other Pacific Islander
0
   0.0%
0
   0.0%
Black or African American
0
   0.0%
0
   0.0%
White
12
  92.3%
12
  92.3%
More than one race
0
   0.0%
0
   0.0%
Unknown or Not Reported
0
   0.0%
0
   0.0%
[1]
Measure Analysis Population Description: Of 31 patients enrolled: 15 were randomized to receive ACTH, and 16 were randomized to No-treatment. There was an option for patients in No-treatment arm to elect to be placed on ACTH (rescue therapy). 13 patients in No-treatment arm used this option, and received ACTH as rescue therapy.
Region of Enrollment  
Measure Type: Count of Participants
Unit of measure:  Participants
United States Number Analyzed 15 participants 16 participants 13 participants 44 participants
Initial assignment
15
 100.0%
16
 100.0%
0
   0.0%
31
  70.5%
Rescue therapy
0
   0.0%
0
   0.0%
13
 100.0%
13
  29.5%
1.Primary Outcome
Title Number of Participants Experienced a Relapse of Nephrotic Syndrome
Hide Description Number of participants experienced a relapse of nephrotic syndrome during the initial 6 months of the study.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
The primary outcome measure was accessed during the initial 6 months based on initial assignment, rescue therapy group was not part of the analysis.
Arm/Group Title Adrenocorticotropic Hormone (ACTH) No Treatment Rescue Therapy
Hide Arm/Group Description:

Patients will receive ACTH twice weekly subcutaneously The initial dosing will be based on body surface area (BSA): 80 IU/1.73 m2

The patients will receive the initial dose for 6 months. At 6 months, the dose will be reduced by 50%. Patients who have side effects may have the dose reduced by 50% during the initial 6 months. A second dose reduction would still occur at 6 months (25% of initial dose).

ACTH: Patients will receive ACTH twice weekly for 6 months, with a 50% dose reduction allowed for side effects. The dose will be reduce by 50% at 6 months and continued for an additional 6 months.

Patients in this treatment arm will receive no treatment to prevent relapses of nephrotic syndrome. A relapse, if it occurs, will be treated with prednisone and the patient will leave the no treatment arm of the study. There is an option for the patient to elect to be placed in the active treatment arm of the trial (rescue therapy).

Patients moved from No-treatment group to receive rescue ACTH therapy twice weekly for 6 months, with a 50% dose reduction allowed for side effects. The dose will be reduce by 50% at 6 months and continued for an additional 6 months.

Other Names:

Acthar Adrenocorticotropic hormon

Overall Number of Participants Analyzed 15 16 0
Measure Type: Count of Participants
Unit of Measure: Participants
14
  93.3%
15
  93.8%
2.Secondary Outcome
Title Number of Participants Experiencing Relapses After Dose Reduction of ACTH
Hide Description The dose of ACTH will be reduced by 50% after 6 months and the rate of relapse during this period will be evaluated.
Time Frame 6 to 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
Three participants who completed 6 months of ACTH Full Dose therapy and then 6 months of Reduced Dose ACTH - inclusive of one subject initially randomized to ACTH and two subjects who received ACTH as rescue treatment
Arm/Group Title Adrenocorticotropic Hormone (ACTH) No Treatment Rescue Therapy
Hide Arm/Group Description:

Patients will receive ACTH twice weekly subcutaneously The initial dosing will be based on body surface area (BSA): 80 IU/1.73 m2

The patients will receive the initial dose for 6 months. At 6 months, the dose will be reduced by 50%. Patients who have side effects may have the dose reduced by 50% during the initial 6 months. A second dose reduction would still occur at 6 months (25% of initial dose).

ACTH: Patients will receive ACTH twice weekly for 6 months, with a 50% dose reduction allowed for side effects. The dose will be reduce by 50% at 6 months and continued for an additional 6 months.

Patients in this treatment arm will receive no treatment to prevent relapses of nephrotic syndrome. A relapse, if it occurs, will be treated with prednisone and the patient will leave the no treatment arm of the study. There is an option for the patient to elect to be placed in the active treatment arm of the trial (rescue therapy).

Patients moved from No-treatment group to receive rescue ACTH therapy twice weekly for 6 months, with a 50% dose reduction allowed for side effects. The dose will be reduce by 50% at 6 months and continued for an additional 6 months.

Other Names:

Acthar Adrenocorticotropic hormon

Overall Number of Participants Analyzed 1 0 2
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
3.Secondary Outcome
Title Number of Adverse Events
Hide Description Adverse events will be collected (SAEs and AEs)
Time Frame 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
15 pts randomized to receive ACTH, and 16 pts randomized to No-treatment. 13 patients in No-treatment arm elected to receive ACTH as rescue therapy.
Arm/Group Title Adrenocorticotropic Hormone (ACTH) No Treatment Rescue Therapy
Hide Arm/Group Description:

Patients will receive ACTH twice weekly subcutaneously The initial dosing will be based on body surface area (BSA): 80 IU/1.73 m2

The patients will receive the initial dose for 6 months. At 6 months, the dose will be reduced by 50%. Patients who have side effects may have the dose reduced by 50% during the initial 6 months. A second dose reduction would still occur at 6 months (25% of initial dose).

ACTH: Patients will receive ACTH twice weekly for 6 months, with a 50% dose reduction allowed for side effects. The dose will be reduce by 50% at 6 months and continued for an additional 6 months.

Patients in this treatment arm will receive no treatment to prevent relapses of nephrotic syndrome. A relapse, if it occurs, will be treated with prednisone and the patient will leave the no treatment arm of the study. There is an option for the patient to elect to be placed in the active treatment arm of the trial (rescue therapy).

Patients moved from No-treatment group to receive rescue ACTH therapy twice weekly for 6 months, with a 50% dose reduction allowed for side effects. The dose will be reduce by 50% at 6 months and continued for an additional 6 months.

Other Names:

Acthar Adrenocorticotropic hormon

Overall Number of Participants Analyzed 15 16 13
Measure Type: Number
Unit of Measure: number of events
12 4 17
Time Frame 1 year
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Adrenocorticotropic Hormone (ACTH) No Treatment Rescue Therapy
Hide Arm/Group Description

Patients will receive ACTH twice weekly subcutaneously The initial dosing will be based on body surface area (BSA): 80 IU/1.73 m2

The patients will receive the initial dose for 6 months. At 6 months, the dose will be reduced by 50%. Patients who have side effects may have the dose reduced by 50% during the initial 6 months. A second dose reduction would still occur at 6 months (25% of initial dose).

ACTH: Patients will receive ACTH twice weekly for 6 months, with a 50% dose reduction allowed for side effects. The dose will be reduce by 50% at 6 months and continued for an additional 6 months.

Patients in this treatment arm will receive no treatment to prevent relapses of nephrotic syndrome. A relapse, if it occurs, will be treated with prednisone and the patient will leave the no treatment arm of the study.

Patients moved from No-treatment group to receive rescue ACTH therapy twice weekly for 6 months, with a 50% dose reduction allowed for side effects. The dose will be reduce by 50% at 6 months and continued for an additional 6 months.

Other Names:

Acthar Adrenocorticotropic hormon

All-Cause Mortality
Adrenocorticotropic Hormone (ACTH) No Treatment Rescue Therapy
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/15 (0.00%)      0/16 (0.00%)      0/13 (0.00%)    
Show Serious Adverse Events Hide Serious Adverse Events
Adrenocorticotropic Hormone (ACTH) No Treatment Rescue Therapy
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   3/15 (20.00%)      2/16 (12.50%)      2/13 (15.38%)    
General disorders       
Hospitalization for renal disease relapse affecting multiple systems *  3/15 (20.00%)  5 2/16 (12.50%)  2 2/13 (15.38%)  2
*
Indicates events were collected by non-systematic assessment
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Adrenocorticotropic Hormone (ACTH) No Treatment Rescue Therapy
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   4/15 (26.67%)      2/16 (12.50%)      9/13 (69.23%)    
Endocrine disorders       
Increase in Cushingoid symptoms *  0/15 (0.00%)  0 1/16 (6.25%)  1 3/13 (23.08%)  4
Infections and infestations       
Flu and strep infections *  0/15 (0.00%)  0 0/16 (0.00%)  0 3/13 (23.08%)  3
Rash under arms *  0/15 (0.00%)  0 1/16 (6.25%)  1 0/13 (0.00%)  0
Injury, poisoning and procedural complications       
Swelling, redness, rash at injection site *  1/15 (6.67%)  1 0/16 (0.00%)  0 4/13 (30.77%)  5
Nervous system disorders       
Sleep disturbances *  2/15 (13.33%)  2 0/16 (0.00%)  0 2/13 (15.38%)  2
Social circumstances       
Behavioral changes *  2/15 (13.33%)  2 0/16 (0.00%)  0 1/13 (7.69%)  1
Vascular disorders       
Increase in blood pressure *  1/15 (6.67%)  2 0/16 (0.00%)  0 0/13 (0.00%)  0
*
Indicates events were collected by non-systematic assessment
Certain Agreements
All Principal Investigators ARE employed by the organization sponsoring the study.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Greenbaum
Organization: Emory University
Phone: 404-712-6374
EMail: LGREEN6@emory.edu
Layout table for additonal information
Responsible Party: Larry Greenbaum, MD, PhD, Emory University
ClinicalTrials.gov Identifier: NCT02132195     History of Changes
Other Study ID Numbers: IRB00068101
EmoryPedNeph-002 ( Other Identifier: Emory Division of Pediatric Nephrology )
First Submitted: December 12, 2013
First Posted: May 7, 2014
Results First Submitted: March 4, 2019
Results First Posted: May 10, 2019
Last Update Posted: May 29, 2019