Nephrotic SyndromePhase 3NCT02132195
Adrenocorticotropic Hormone (ACTH) for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome
A Phase 3 study testing ACTH in people with Nephrotic Syndrome, run by Emory University at 16 sites in United States.
Trial summary
In childhood nephrotic syndrome, the kidneys leak protein, causing body swelling and a variety of possible complications such as infection, blood clots, and kidney failure. The first-line treatment for nephrotic syndrome is corticosteroids. Read more about this study
- Who is it for?
- Ages 2 to 20 years
- Conditions the study is for
- Nephrotic Syndrome
- What is being tested?
- ACTH
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 31 participants
- Where
- 16 sitesUnited States
- Study dates
- May 2014to Mar 2018
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Age >1 year at onset of nephrotic syndrome
- Age 2-20 years at time of randomization
- Estimated glomerular filtration rate (GFR) > 50 ml/min/1.73 m2 at most recent measure prior to randomization (Schwartz formula)
- Steroid responsive nephrotic syndrome throughout clinical course (never required a second agent to attain remission of a relapse of nephrotic syndrome)
- History of frequently relapsing or steroid dependent nephrotic syndrome (defined as 2 or more relapses within 6 months after initial therapy or 4 or more relapses in any 12 month period OR relapse during taper or within 2 weeks of discontinuing prednisone).
- Patient is currently in relapse of nephrotic syndrome or had a relapse within the last 4 months (defined as an increase in the first morning urine protein to creatinine ratio ≥2 or Albustix reading of ≥2 for 3 or 5 consecutive days).
Who cannot join
- Prior treatment with ACTH.
- Cyclophosphamide or rituximab within the last 4 months.
- Lactation, pregnancy, or refusal of birth control in females with child-bearing potential
- Planned treatment with live or live-attenuated vaccines once enrolled in the study.
- Participation in another therapeutic trial concurrently or 30 days prior to randomization
- Active/serious infection (including, but not limited to Hepatitis B or C, HIV)
Show all 14Show fewer
- Malignancy concurrently or within the last 2 years.
- Blood pressure >95% for age/height while receiving maximal doses of 3 or more medications.
- Prior diagnosis of diabetes mellitus (Type I or II) or fasting glucose >200mg/dL
- Organ transplantation
- Contraindications to Acthar: scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of or the presence of peptic ulcer, congestive heart failure, uncontrolled hypertension, primary adrenocortical insufficiency, or adrenal cortical hyperfunction
- Secondary cause of nephrotic syndrome (e.g., SLE)
- Biopsy demonstrating a diagnosis other than minimal change, focal segmental glomerulosclerosis (FSGS) or a variant (mesangial proliferation, Immunoglobulin M nephropathy)
- Inability to consent/assent -
Trial locations and contacts
16 sites in 1 country.
16 sites in 1 country
United States16 sites
Emory University
Atlanta, Georgia, 30322
Pediatric Nephrology of Alabama, PC.
Birmingham, Alabama, 35205
Boston Children's Hopsital
Boston, Massachusetts, 02115
Driscoll Children's Hospital
Corpus Christi, Texas, 78411
Duke Children's Health Center
Durham, North Carolina, 27704
Helen DeVos Children's Hospital at Spectrum Health
Grand Rapids, Michigan, 49503
East Carolina University
Greenville, North Carolina, 27834
Texas Children's Hospital
Houston, Texas, 77030
Riley Hospital for Children
Indianapolis, Indiana, 46202
Children's Mercy
Kansas City, Missouri, 64109
University of California, Los Angeles
Los Angeles, California, 90095
Nemours Children's Hospital
Orlando, Florida, 32827
Children's Hospital of Richmond at VCU
Richmond, Virginia, 23298
Mayo Clinic
Rochester, Minnesota, 55905
Children's Hospital at Montefiore
The Bronx, New York, 10467
Nemours/AI duPont Hospital for Children
Wilmington, Delaware, 19803
About this study
In childhood nephrotic syndrome, the kidneys leak protein, causing body swelling and a variety of possible complications such as infection, blood clots, and kidney failure. The first-line treatment for nephrotic syndrome is corticosteroids. Many children respond to prednisone treatment, but the disease comes back (relapses) when the prednisone is stopped or the dose is reduced. Children with frequently relapsing or steroid dependent nephrotic syndrome are at risk for toxicity from frequent exposure to corticosteroids.
Currently, the standard treatment for frequently relapsing and steroid dependent nephrotic syndrome involves a variety of medications that suppress the immune system, which can produce serious side effects. We propose a study to examine the effects of a different medication, ACTH, on nephrotic syndrome. ACTH is a hormone naturally found in the body. Recently, in adult studies, ACTH has been shown to be effective for the treatment of nephrotic syndrome. It has also been shown to have mild and reversible side effects. ACTH is potentially an attractive therapeutic alternative for the treatment of frequently relapsing and steroid dependent nephrotic syndrome in children. Our study will randomly assign patients with frequently relapsing or steroid dependent nephrotic syndrome to either ACTH treatment or no treatment. This will allow us to study the effects of ACTH on this disease and its side effects, by comparing how patients do on ACTH treatment versus no treatment. We hypothesize that ACTH gel is superior to no treatment in maintaining remission in children with frequently relapsing or steroid dependent nephrotic syndrome.
Read the detailed description
Our hypotheses are the following:
Hypothesis 1: ACTH gel is superior to no treatment in maintaining remission in children with frequently relapsing or steroid dependent nephrotic syndrome (NS).
Hypothesis 2: Relapses in children with frequently relapsing or steroid dependent nephrotic syndrome receiving ACTH gel will increase when the dose of ACTH gel is reduced by 50%.
Hypothesis 3: ACTH gel will increase the percentage of children with frequently relapsing or steroid dependent nephrotic syndrome that remain relapse free off medication.
Primary end-points:
The primary end-point related to Hypothesis 1 is the proportion of patients in each arm with a relapse during the initial 6 months of treatment.
The primary end-point related to Hypothesis 2 is the proportion of relapse-free patients during the first 6 months and second 6 months of treatment with ACTH. This will include patients initially randomized to ACTH and patients who receive ACTH as rescue therapy following their initial relapse in patients randomized to no treatment.
The primary end-point related to Hypothesis 3 is the proportion of patients who have relapses in the 6 months following completion of one year of ACTH. This will be compared to the proportion of patients with relapses during the initial 6 months in the patients randomized to no treatment.
Secondary end-points:
Our secondary end-points are the following:
- The total prednisone exposure over the initial 12 months in the two groups.
- The number of relapses over the initial 6 months in the two groups.
- The change in body mass index (BMI), height standard deviation score (SDS), and cholesterol over the study period for both groups Study Design and Methods The experimental design is a multi-center, prospective, controlled open label, randomized trial comparing ACTH gel and no treatment in preventing relapses in pediatric patients with frequently relapsing or steroid dependent nephrotic syndrome.
Patients will be randomized in a 1:1 ratio to either no treatment or treatment with ACTH gel. The primary outcome will be the presence of a relapse within 6 months of starting ACTH gel or no treatment.
After initial recruitment, enrollment will begin with a screening visit to determine eligibility and obtain informed consent and assent. Randomization and weaning of all other medications for the treatment of NS will begin after remission has been achieved for those with active relapse. There will be a 2 week overlap of ACTH and current immunosuppressive medications:
- ACTH will be initiated 2 weeks prior to the completion of the prednisone taper for patients who are receiving prednisone at the time of consent.
- ACTH will be initiated 2 weeks prior to stopping preventive medications such as tacrolimus, cyclosporin, and mycophenolate.
Patients randomized to no treatment will be followed for up to 6 months or until disease relapse, whichever occurs first. Patients who relapse within 6 months will be given the option of reassignment to the ACTH treatment group after remission has been achieved using conventional corticosteroid therapy.
Patients randomized to ACTH treatment will be given ACTH for 12 months. During the second 6 months, the ACTH dose will be reduced to 50% of the starting dose. The outcome of interest is the presence of relapses after dose reduction. Follow-up will occur throughout the 12 months of therapy, and also for 6 months following the completion of ACTH therapy. The outcome of interest is the percentage of patients with relapses in the 6 months after completing a 12 month course of ACTH treatment.
The primary end-point of the study and on which the statistical power is based is the proportion of patients who have a relapse in the 6 months following randomization to either ACTH or no ACTH.
We hypothesize that the 6 month relapse rate for patients receiving no treatment is 70%. In order to detect a 6 month relapse rate of 30% for patients receiving the ACTH gel, we will randomize 30 patients in each arm using a two sided z test with alpha=.05. Our statistical power to detect such a difference is 91% which assumes two interim analyses at 50% and 100% of accumulated information. That is, once 30 patients have 6 month relapse data, we will conduct the first interim analysis. The last one will be completed once we have relapse data on all 60 patients. The table below gives the operating characteristics for such a design:
Number of Patients with 6 Months Data Boundary p-value 30 0.006 60 0.045
Using the method of Lan-DeMets, we list the boundary p-value for this sequential design. Thus, after our first interim look, we will reject the null hypothesis of equal 6 month relapse rates between ACTH gel and no treatment if our test statistic renders a p-value < .006. According to the intent to treat principle, patients will be analyzed according to the treatment they have been assigned to during the randomization procedure. The odds ratio of ACTH versus no ACTH, plus the Wald 95% confidence interval, will be also be calculated.
The primary end-point related to specific aim 2 is the proportion of relapse-free patients during the first 6 months and second 6 months of treatment with ACTH. This will include patients initially randomized to ACTH and patients who receive ACTH as rescue therapy following their initial relapse. We will estimate 6 month and 12 month relapse-free rate using the method of Kaplan-Meier and compare treatments using a log-rank test.
The primary end-point related to specific aim 3 is the proportion of patients who have relapses in the 6 months following completion of one year of ACTH. This will be compared to the proportion of patients with relapses during the initial 6 months in the patients randomized to no treatment using a z test statistic. Odds ratios of ACTH vs no ACTH will also be calculated.
We will also compare patients as randomized by secondary endpoints such as total prednisone exposure in 12 months, number of relapses, cholesterol and change in BMI with two sample t-tests. If normality assumptions do not hold, appropriate non-parametric methods will be used.
Growth data collected during the study will be summarized descriptively for each treatment group at each time point. Based on height data collected during the study and published reference height information, the height standard deviation score (SDS, also called z-score) will be computed for each patient at each time point as:
(Height - mean height for that age category) / SD of height for that age category.
Descriptive statistics of this endpoint will be presented by time point and the z-scores will allow identification of potential outliers.
Treatment Assignment and Randomization Treatment assignments will be stratified according to clinical center. The treatment assignments will be generated by the Data Coordinating Center (DCC) with the use of a pseudo-random-number generator with randomly permutated blocks that will be used to ensure balance between the number of subjects assigned to each treatment (ACTH or no ACTH). Before the study starts, the institutional research coordinator at each clinical center will be given a batch of 20 sealed, sequenced, opaque envelopes containing the treatment assignment and will have a unique identification number consisting of the clinical center stratum.
Patients will be assigned to one of the two treatment arms in a ratio of 1:1.
- Conditions
- Nephrotic Syndrome
- Being tested
- ACTH
- Medical subject headings
- Nephrotic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ACTHDrug
Also called Acthar, Adrenocorticotropic hormone
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.
| Study group | Role | What they receive |
|---|---|---|
| Adrenocorticotropic hormone (ACTH) | Active comparator | 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). Receives: Drug: ACTH |
| No treatment | No intervention | 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. |
| Rescue therapy | Active comparator | 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). Receives: Drug: ACTH |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants Experienced a Relapse of Nephrotic Syndrome
Number of participants experienced a relapse of nephrotic syndrome during the initial 6 months of the study.
Measured: 6 months
Secondary outcomes (2)
Number of Participants Experiencing Relapses After Dose Reduction of ACTH
The dose of ACTH will be reduced by 50% after 6 months and the rate of relapse during this period will be evaluated.
Measured: 6 to 12 months
Number of Adverse Events
Adverse events will be collected (SAEs and AEs)
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 May 2014 (estimated)
- Study startMay 2014
- Primary completionJan 2018
- Study completionMar 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Nephrotic SyndromeStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 31 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- Mallinckrodt
- Responsible party
- Principal investigator, Larry Greenbaum, MD, PhD, Emory University
- Investigators
- Larry A Greenbaum, MD, PhD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02132195
- Sponsor's protocol ID
- IRB00068101
- Other IDs
- EmoryPedNeph-002
- Status last verified
- May 2019
- First submitted
- 12 Dec 2013
- Last updated
- 29 May 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Larkins NG, Hahn D, Liu ID, Willis NS, Craig JC, Hodson EM. Non-corticosteroid immunosuppressive medications for steroid-sensitive nephrotic syndrome in children. Cochrane Database Syst Rev. 2024 Nov 8;11(11):CD002290. doi: 10.1002/14651858.CD002290.pub6.
PubMed 39513526
Study at a glance
From the registry record, last updated by the sponsor on 29 May 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Adrenocorticotropic Hormone (ACTH) for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome
- ClinicalTrials.gov ID
- NCT02132195
- Acronym / sponsor ID
- IRB00068101
- Conditions
- Nephrotic Syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 31 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- Mallinckrodt
- Study start
- May 2014 (actual)
- Primary completion
- Jan 2018 (actual)
- Study completion
- Mar 2018 (actual)
- First posted
- 7 May 2014 (estimated)
- Results
- Posted 10 May 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
In childhood nephrotic syndrome, the kidneys leak protein, causing body swelling and a variety of possible complications such as infection, blood clots, and kidney failure. The first-line treatment for nephrotic syndrome is corticosteroids. Many children respond to prednisone treatment, but the disease comes back (relapses) when the prednisone is stopped or the dose is reduced. Children with frequently relapsing or steroid dependent nephrotic syndrome are at risk for toxicity from frequent exposure to corticosteroids.
Currently, the standard treatment for frequently relapsing and steroid dependent nephrotic syndrome involves a variety of medications that suppress the immune system, which can produce serious side effects. We propose a study to examine the effects of a different medication, ACTH, on nephrotic syndrome. ACTH is a hormone naturally found in the body. Recently, in adult studies, ACTH has been shown to be effective for the treatment of nephrotic syndrome. It has also been shown to have mild and reversible side effects. ACTH is potentially an attractive therapeutic alternative for the treatment of frequently relapsing and steroid dependent nephrotic syndrome in children. Our study will randomly assign patients with frequently relapsing or steroid dependent nephrotic syndrome to either ACTH treatment or no treatment. This will allow us to study the effects of ACTH on this disease and its side effects, by comparing how patients do on ACTH treatment versus no treatment. We hypothesize that ACTH gel is superior to no treatment in maintaining remission in children with frequently relapsing or steroid dependent nephrotic syndrome.
Detailed description
Our hypotheses are the following:
Hypothesis 1: ACTH gel is superior to no treatment in maintaining remission in children with frequently relapsing or steroid dependent nephrotic syndrome (NS).
Hypothesis 2: Relapses in children with frequently relapsing or steroid dependent nephrotic syndrome receiving ACTH gel will increase when the dose of ACTH gel is reduced by 50%.
Hypothesis 3: ACTH gel will increase the percentage of children with frequently relapsing or steroid dependent nephrotic syndrome that remain relapse free off medication.
Primary end-points:
The primary end-point related to Hypothesis 1 is the proportion of patients in each arm with a relapse during the initial 6 months of treatment.
The primary end-point related to Hypothesis 2 is the proportion of relapse-free patients during the first 6 months and second 6 months of treatment with ACTH. This will include patients initially randomized to ACTH and patients who receive ACTH as rescue therapy following their initial relapse in patients randomized to no treatment.
The primary end-point related to Hypothesis 3 is the proportion of patients who have relapses in the 6 months following completion of one year of ACTH. This will be compared to the proportion of patients with relapses during the initial 6 months in the patients randomized to no treatment.
Secondary end-points:
Our secondary end-points are the following:
- The total prednisone exposure over the initial 12 months in the two groups.
- The number of relapses over the initial 6 months in the two groups.
- The change in body mass index (BMI), height standard deviation score (SDS), and cholesterol over the study period for both groups Study Design and Methods The experimental design is a multi-center, prospective, controlled open label, randomized trial comparing ACTH gel and no treatment in preventing relapses in pediatric patients with frequently relapsing or steroid dependent nephrotic syndrome.
Patients will be randomized in a 1:1 ratio to either no treatment or treatment with ACTH gel. The primary outcome will be the presence of a relapse within 6 months of starting ACTH gel or no treatment.
After initial recruitment, enrollment will begin with a screening visit to determine eligibility and obtain informed consent and assent. Randomization and weaning of all other medications for the treatment of NS will begin after remission has been achieved for those with active relapse. There will be a 2 week overlap of ACTH and current immunosuppressive medications:
- ACTH will be initiated 2 weeks prior to the completion of the prednisone taper for patients who are receiving prednisone at the time of consent.
- ACTH will be initiated 2 weeks prior to stopping preventive medications such as tacrolimus, cyclosporin, and mycophenolate.
Patients randomized to no treatment will be followed for up to 6 months or until disease relapse, whichever occurs first. Patients who relapse within 6 months will be given the option of reassignment to the ACTH treatment group after remission has been achieved using conventional corticosteroid therapy.
Patients randomized to ACTH treatment will be given ACTH for 12 months. During the second 6 months, the ACTH dose will be reduced to 50% of the starting dose. The outcome of interest is the presence of relapses after dose reduction. Follow-up will occur throughout the 12 months of therapy, and also for 6 months following the completion of ACTH therapy. The outcome of interest is the percentage of patients with relapses in the 6 months after completing a 12 month course of ACTH treatment.
The primary end-point of the study and on which the statistical power is based is the proportion of patients who have a relapse in the 6 months following randomization to either ACTH or no ACTH.
We hypothesize that the 6 month relapse rate for patients receiving no treatment is 70%. In order to detect a 6 month relapse rate of 30% for patients receiving the ACTH gel, we will randomize 30 patients in each arm using a two sided z test with alpha=.05. Our statistical power to detect such a difference is 91% which assumes two interim analyses at 50% and 100% of accumulated information. That is, once 30 patients have 6 month relapse data, we will conduct the first interim analysis. The last one will be completed once we have relapse data on all 60 patients. The table below gives the operating characteristics for such a design:
Number of Patients with 6 Months Data Boundary p-value 30 0.006 60 0.045
Using the method of Lan-DeMets, we list the boundary p-value for this sequential design. Thus, after our first interim look, we will reject the null hypothesis of equal 6 month relapse rates between ACTH gel and no treatment if our test statistic renders a p-value < .006. According to the intent to treat principle, patients will be analyzed according to the treatment they have been assigned to during the randomization procedure. The odds ratio of ACTH versus no ACTH, plus the Wald 95% confidence interval, will be also be calculated.
The primary end-point related to specific aim 2 is the proportion of relapse-free patients during the first 6 months and second 6 months of treatment with ACTH. This will include patients initially randomized to ACTH and patients who receive ACTH as rescue therapy following their initial relapse. We will estimate 6 month and 12 month relapse-free rate using the method of Kaplan-Meier and compare treatments using a log-rank test.
The primary end-point related to specific aim 3 is the proportion of patients who have relapses in the 6 months following completion of one year of ACTH. This will be compared to the proportion of patients with relapses during the initial 6 months in the patients randomized to no treatment using a z test statistic. Odds ratios of ACTH vs no ACTH will also be calculated.
We will also compare patients as randomized by secondary endpoints such as total prednisone exposure in 12 months, number of relapses, cholesterol and change in BMI with two sample t-tests. If normality assumptions do not hold, appropriate non-parametric methods will be used.
Growth data collected during the study will be summarized descriptively for each treatment group at each time point. Based on height data collected during the study and published reference height information, the height standard deviation score (SDS, also called z-score) will be computed for each patient at each time point as:
(Height - mean height for that age category) / SD of height for that age category.
Descriptive statistics of this endpoint will be presented by time point and the z-scores will allow identification of potential outliers.
Treatment Assignment and Randomization Treatment assignments will be stratified according to clinical center. The treatment assignments will be generated by the Data Coordinating Center (DCC) with the use of a pseudo-random-number generator with randomly permutated blocks that will be used to ensure balance between the number of subjects assigned to each treatment (ACTH or no ACTH). Before the study starts, the institutional research coordinator at each clinical center will be given a batch of 20 sealed, sequenced, opaque envelopes containing the treatment assignment and will have a unique identification number consisting of the clinical center stratum.
Patients will be assigned to one of the two treatment arms in a ratio of 1:1.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Adrenocorticotropic hormone (ACTH) | Active comparator | Drug: ACTHPatients 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). |
| No treatment | No intervention | –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. |
| Rescue therapy | Active comparator | Drug: ACTHThere 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). |
Who may qualify
- Ages
- 2 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Age >1 year at onset of nephrotic syndrome
- Age 2-20 years at time of randomization
- Estimated glomerular filtration rate (GFR) > 50 ml/min/1.73 m2 at most recent measure prior to randomization (Schwartz formula)
- Steroid responsive nephrotic syndrome throughout clinical course (never required a second agent to attain remission of a relapse of nephrotic syndrome)
- History of frequently relapsing or steroid dependent nephrotic syndrome (defined as 2 or more relapses within 6 months after initial therapy or 4 or more relapses in any 12 month period OR relapse during taper or within 2 weeks of discontinuing prednisone).
Key exclusion criteria (14 listed)
- Prior treatment with ACTH.
- Cyclophosphamide or rituximab within the last 4 months.
- Lactation, pregnancy, or refusal of birth control in females with child-bearing potential
- Planned treatment with live or live-attenuated vaccines once enrolled in the study.
- Participation in another therapeutic trial concurrently or 30 days prior to randomization
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ACTHAlso: Acthar, Adrenocorticotropic hormonePatients 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. | Drug | Adrenocorticotropic hormone (ACTH), Rescue therapy |
Primary outcome
Number of Participants Experienced a Relapse of Nephrotic Syndrome
Time frame: 6 months
Number of participants experienced a relapse of nephrotic syndrome during the initial 6 months of the study.
2 secondary outcomes
Number of Participants Experiencing Relapses After Dose Reduction of ACTH
Time frame: 6 to 12 months
Number of Adverse Events
Time frame: 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 May 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 39513526Larkins NG, Hahn D, Liu ID, Willis NS, Craig JC, Hodson EM. Non-corticosteroid immunosuppressive medications for steroid-sensitive nephrotic syndrome in children. Cochrane Database Syst Rev. 2024 Nov 8;11(11):CD002290. doi: 10.1002/14651858.CD002290.pub6.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Larry A Greenbaum, MD, PhD principal investigator
Emory University
16 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ACTH in people with Nephrotic Syndrome, run by Emory University at 16 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 29 May 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 May 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants Experienced a Relapse of Nephrotic Syndrome (time frame: 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02132195, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 May 2019.