Primary outcomeCount of participants· 6 months
Number of Participants Experienced a Relapse of Nephrotic Syndrome
| Group | Value (Participants) |
|---|---|
| Adrenocorticotropic Hormone (ACTH) | 14 |
| No Treatment | 15 |
| Rescue Therapy | not posted |
Trial · Interventional
CompletedPhase 3Results postedRegistry dataAdrenocorticotropic Hormone (ACTH) for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome
Overview
- Status
- Completed
- Phase
- Phase 3
- Enrollment
- 31 participants (actual)
- Sponsor
- Emory University · Other
- Design
- Randomized · Open label (no masking)
- Countries
1 country
United States
- Start
- May 2014
- Primary completion
- Jan 2018
- Completion
- Mar 2018
- First posted
- 7 May 2014 (estimated)
- Last update posted
- 29 May 2019
- Conditions
- Nephrotic Syndrome
Timeline
1 trial · 1 completed · 1 with posted results
- Completed
- Ongoing
- Not yet recruiting
- Stopped
- Estimated dates (est.)
- Results posted
201420152016201720182019
- Ph 3
Adrenocorticotropic Hormone (ACTH) for Frequently Relapsing and Steroid Dependent Nephrotic Syndrome
NCT02132195May 2014 to Mar 2018
Registry summary
Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.
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.
Interventions
- ACTHDrug
Arms3
Active comparator
Adrenocorticotropic hormone (ACTH)
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).
No intervention
No treatment
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.
Active comparator
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).
Primary outcomes1
Outcomes the sponsor listed as primary.
Number of Participants Experienced a Relapse of Nephrotic Syndrome
Time frame · 6 months
Registry description
Number of participants experienced a relapse of nephrotic syndrome during the initial 6 months of the study.
Secondary outcomes(2)
Number of Participants Experiencing Relapses After Dose Reduction of ACTH
Time frame · 6 to 12 months
Registry description
The dose of ACTH will be reduced by 50% after 6 months and the rate of relapse during this period will be evaluated.
Number of Adverse Events
Time frame · 12 months
Registry description
Adverse events will be collected (SAEs and AEs)
Posted results
Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.
Primary outcome results
Secondary and other outcome results(2)
Secondary outcomeCount of participants· 6 to 12 months
Number of Participants Experiencing Relapses After Dose Reduction of ACTH
| Group | Value (Participants) |
|---|---|
| Adrenocorticotropic Hormone (ACTH) | 0 |
| No Treatment | not posted |
| Rescue Therapy | 0 |
Secondary outcomeNumber· 12 months
Number of Adverse Events
| Group | Value (number of events) |
|---|---|
| Adrenocorticotropic Hormone (ACTH) | 12 |
| No Treatment | 4 |
| Rescue Therapy | 17 |
Adverse events, as posted
| Group | Deaths | Serious events | Other events |
|---|---|---|---|
| Adrenocorticotropic Hormone (ACTH)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. | 0 / 15 | 3 / 15 | 4 / 15 |
| No TreatmentPatients 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. | 0 / 16 | 2 / 16 | 2 / 16 |
| Rescue TherapyPatients 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 | 0 / 13 | 2 / 13 | 9 / 13 |
Each cell reads participants affected / participants at risk, as the registry posted them.
Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works
Studies0
Studies that cite this trial.
No study cites this trial.
Other registry references
References listed at the registry that are not in PepBank. They link to PubMed.
- DerivedLarkins 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. PMID 39513526
Eligibility
Who could join, as posted.
- Sex
- All
- Age
- from 2 Years up to 20 Years
- Healthy volunteers
- Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria:
1. Age \>1 year at onset of nephrotic syndrome
2. Age 2-20 years at time of randomization
3. Estimated glomerular filtration rate (GFR) \> 50 ml/min/1.73 m2 at most recent measure prior to randomization (Schwartz formula)
4. Steroid responsive nephrotic syndrome throughout clinical course (never required a second agent to attain remission of a relapse of nephrotic syndrome)
5. 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).
6. 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).
Exclusion Criteria:
1. Prior treatment with ACTH.
2. Cyclophosphamide or rituximab within the last 4 months.
3. Lactation, pregnancy, or refusal of birth control in females with child-bearing potential
4. Planned treatment with live or live-attenuated vaccines once enrolled in the study.
5. Participation in another therapeutic trial concurrently or 30 days prior to randomization
6. Active/serious infection (including, but not limited to Hepatitis B or C, HIV)
7. Malignancy concurrently or within the last 2 years.
8. Blood pressure \>95% for age/height while receiving maximal doses of 3 or more medications.
9. Prior diagnosis of diabetes mellitus (Type I or II) or fasting glucose \>200mg/dL
10. Organ transplantation
11. 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
12. Secondary cause of nephrotic syndrome (e.g., SLE)
13. Biopsy demonstrating a diagnosis other than minimal change, focal segmental glomerulosclerosis (FSGS) or a variant (mesangial proliferation, Immunoglobulin M nephropathy)
14. Inability to consent/assent -
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