Steroid-Resistant Nephrotic SyndromePhase 4ADRENLNCT03408405
ACTHAR Gel for Drug REsistant Nephrotic Syndrome in Children
A Phase 4 study testing Acthar Gel 80 UNT/ML Injectable Solution in people with Steroid-Resistant Nephrotic Syndrome, run by Rhode Island Hospital.
Trial summary
We propose to study the use of purified porcine Acthar Gel (ACTHAR, Mallinckrodt Pharmaceuticals) for treatment of steroid resistance nephrotic syndrome (SRNS) in a prospective pilot study. We plan to enroll 25 children between the ages of 2 to 21 years. Read more about this study
- Who is it for?
- Ages 2 to 21 years
- Conditions the study is for
- Steroid-Resistant Nephrotic Syndrome
- What is being tested?
- Acthar Gel 80 UNT/ML Injectable Solution
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Jun 2018to Jul 2021
- Phase
- Phase 4
- Are there results?
- None posted in the registry
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 21 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients between the ages of 2-21 years who fail a minimum of 12 weeks of cumulative therapy with prednisone OR one other alternate immunosuppressive agent for treatment of nephrotic syndrome, will be eligible for inclusion. Nephrotic syndrome is defined as: Presence of edema, Edema, UP/C ≥2, ≥300mg/dl or 3+ protein on Albustix, and hypoalbuminemia ≤2.5 g/dL
- Calculated GFR (eGFR) using modified Schwartz formula of > 50 mls/min/m2. (Modified Schwartz formula = 0.413 x height (cms) ÷ serum creatinine mg/dL)
- A clinical or biopsy diagnosis of nephrotic syndrome within the last 3 years prior to enrollment in the study.
- Renal biopsy (if available) consistent with a diagnosis of Minimal Change Disease, IgM nephropathy, Mesangioproliferative Glomerulonephritis, Primary Focal Segmental Glomerulosclerosis or C1q Nephropathy
Who cannot join
- Patients with an inherited or genetic disorder presenting with nephrotic syndrome (eg: NPHS 1 \& 2 defects, WT-1 mutations, α actinin 4 mutation, TRP-6 mutation).
- Presence of diabetes or severe (stage 2) uncontrolled hypertension.
- Any metabolic condition that specifically precludes the use of Acthar Gel for treatment.
- Pregnancy or unwilling to agree to contraception which may include abstinence.
- eGFR <50 mls/min/m2
About this study
Official title: ADRENL - ACTHAR Gel for Drug REsistant Nephrotic Syndrome in Children, Pilot Study
We propose to study the use of purified porcine Acthar Gel (ACTHAR, Mallinckrodt Pharmaceuticals) for treatment of steroid resistance nephrotic syndrome (SRNS) in a prospective pilot study. We plan to enroll 25 children between the ages of 2 to 21 years. Children fulfilling strict inclusion criteria, whose parents agree to written informed consent after institutional IRB approval for the study, will be enrolled. Purified porcine Acthar Gel will be administered SQ to all children using a defined treatment protocol for a period of six months. Renal function, urine protein excretion, serum albumin levels, blood pressure and growth parameters will be monitored closely on all patients. Baseline urine protein excretion will be compared to end of treatment levels to determine successful response to therapy. There will be an 18 month enrollment period, 6 month treatment period and a 12 month follow-up period.
Read the detailed description
Treatment Protocol:
Patients who fulfill inclusion criteria and who agree to participate after signing informed consent and assent forms will be treated with the following protocol:
Acthar Gel will be dosed by body surface area (BSA) using the Dubois Method. The dose of Acthar Gel will be 80 units/1.73 m2 per dose administered subcutaneously (SQ) twice a week on Mondays and Thursdays.
Week 1 to 2:
During week 1, patients will receive 50% of initial calculated dose twice a week.
During week 2, patients will receive 75% of initial calculated dose twice a week.
Week 3 to 6 Months:
Patients will receive the full dose of Acthar Gel (80 units/1.73 m2 per dose) administered subcutaneously (SQ) twice a week on Mondays and Thursdays.
During this period, the treating physician will have the discretion of reducing the dose of Acthar Gel by 25-50% based on response with respect to reduction in proteinuria and tolerability of side-effects (uncontrolled hypertension, excessive weight gain, severe acne, hyperglycemia, etc.)
Patients will have the option of remaining on Acthar Gel after the 6 month study period based on clinical response at discretion of the co-PI. Study drug will be provided free of cost to patients in the first 6 months only.
Concomitant Treatments:
- Amlodipine 0.1 mg/kg PO in one or two divided doses will be given to all patients who develop elevated blood pressure (>95th percentile) starting after 14 days of initiation of Acthar Gel therapy and will continue till 6 months. Dose can be increased up to 0.5 mg/kg divided bid as needed to achieve goal BP <95th percentile. If Amlodipine is not covered by the participants insurance, then an alternate calcium channel blocker will be started at the clinical discretion of the PI/co-PI that is covered by insurance. If the participant is not covered by any insurance the participant will have to pay out of pocket
- If BP remains elevated despite maximum calcium channel therapy as described in the study protocol above, the choice and dose of additional anti-hypertensive agents will be at the discretion of the study investigator on an individualized and as needed basis.
- Patients on prednisone on initiation of study will be tapered off over 2-4 weeks. Non-steroidal immunosuppression may be tapered off over four weeks after the start of Acthar Gel therapy at the discretion of the PI/Co-PI.
- Additional standard of care therapy (statins, vitamin D, antacids, etc.) can be continued and modified based on the discretion and clinical judgment of the study PI/Co-PIs.
Quality of Life Assessment:
Quality of life will be measured using the patient and parent self-report versions of the Pediatric Quality of Life Inventory, ESRD, Acute Version. The PedsQL 3.0 ESRD Scales measure physical, emotional, social and role (i.e. school) dimensions. Scores are derived for physical functioning and psychological functioning, in addition to a total score, which enables comparison against healthy populations. Scales are rated on a 5-point Likert scale (0 = never, 1= almost never, 2 = sometimes, 3 = often, 4 = almost always), with scores linearly transformed to range from 0 - 100, with higher scores indicating fewer problems or symptoms.
Surveys will be administered by trained study personnel at enrollment (baseline), six months, 12 months, and 18 months.
Laboratory Testing:
Baseline and then once a month till the end of the treatment period (6 months): Serum electrolytes, BUN, creatinine, albumin, ACTH level, glucose, random cortisol, calcium, magnesium, phosphorus, ALT, AST, total and direct bilirubin, fasting cholesterol, triglycerides, HDL, LDL, urine protein to creatinine ratio. During the 12 month follow-up period laboratory evaluation will be performed every third month. (11 total blood draws). A urine pregnancy test shall be sent on all female patients of child-bearing potential at the beginning and end of treatment period.
All available biopsy slides will be reviewed by a blinded renal pathologist to insure accuracy and to confirm the initial histologic diagnosis.
Telephone Contact One, two and three weeks after initial dose. Phone contacts will screen for: adverse events, and review dosing of ACTHAR GEL.
- Conditions
- Steroid-Resistant Nephrotic Syndrome
- Being tested
- Acthar Gel 80 UNT/ML Injectable Solution
- Medical subject headings
- Nephrotic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Acthar Gel 80 UNT/ML Injectable SolutionDrug
Also called H.P. Acthar gel
Participant will self inject 'Acthar Gel 80 UNT/ML Injectable Solution' 2 x per week for six months.
| Study group | Role | What they receive |
|---|---|---|
| Acthar Gel treatment group | Experimental | Participants will be treated with 'Acthar Gel 80 UNT/ML Injectable Solution'. Initial dose for week 1 will be 50% of 80 units, injected twice per week. Week 2 is 75% of 80 units, week 3 and throughout treatment period (6 months in total) will be 80 units/ml twice per week. Receives: Drug: Acthar Gel 80 UNT/ML Injectable Solution |
What is being measured
The primary outcome is the main question the study is designed to answer.
Remission of proteinuria
Partial remission: Greater than or equal to 50% reduction in proteinuria when compared to baseline.
Complete remission: Greater than or equal to 90% reduction in proteinuria when compared to baseline or Urine Protein Creatinine ratio less than 0.5.
Measured: 18 months
Secondary outcomes (6)
Blood pressure
Blood pressure during study visit as measured in mm/Hg
Measured: 18 months
Serum Creatinine
Serum value in mg/dL
Measured: 18 months
BMI
Weight (kgs) and height (cms) will be combined to report BMI in kg/m^2
Measured: 18 months
Serum Glucose
Serum value in mg/dL
Measured: 18 months
Serum Lipids
Serum Value in mg/dL
Measured: 18 months
Quality of life measures obtained via survey
PedsQL 3.0 will be used as the standardized survey tool
Measured: 18 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2018
- Study startJun 2018 (estimated)
- Primary completionJan 2021 (estimated)
- Study completionJul 2021 (estimated)
Why it stopped: Withdrawal of funding from primary sponsor
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (8)Papers the sponsor has linked to this study.
- Other trials for Steroid-Resistant Nephrotic SyndromeStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Diagnosis
- Intervention model
- Sequential
- Model description
- Inclusion Criteria: 1. Patients between the ages of 2-21 years who fail a minimum of 12 weeks of cumulative therapy with prednisone OR one other alternate immunosuppressive agent for treatment of nephrotic syndrome, will be eligible for inclusion. Nephrotic syndrome is defined as: Presence of edema, Edema, UP/C ≥2, ≥300mg/dl or 3+ protein on Albustix, and hypoalbuminemia ≤2.5 g/dL 2. Calculated GFR (eGFR) using modified Schwartz formula of \> 50 mls/min/m2. (Modified Schwartz formula = 0.413 x height (cms) ÷ serum creatinine mg/dL) 3. A clinical or biopsy diagnosis of nephrotic syndrome within the last 3 years prior to enrollment in the study. 4. Renal biopsy (if available) consistent with a diagnosis of Minimal Change Disease, IgM nephropathy, Mesangioproliferative Glomerulonephritis, Primary Focal Segmental Glomerulosclerosis or C1q Nephropathy
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rhode Island Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Mallinckrodt, University of Minnesota, Medalytics
- Responsible party
- Principal investigator, Mohammed Khurram Faizan, Rhode Island Hospital
- Investigators
- Mohammed K Faizan, MD, Principal investigator, Rhode Island Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03408405
- Sponsor's protocol ID
- 1050945-5
- Acronym
- ADRENL
- Status last verified
- Apr 2019
- First submitted
- 8 Jan 2018
- Last updated
- 23 Apr 2019
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Coppo R. Non-steroidal and non-cytotoxic therapies for nephrotic syndrome. Nephrol Dial Transplant. 2008 Jun;23(6):1793-6. doi: 10.1093/ndt/gfn211. Epub 2008 Apr 25. No abstract available.
PubMed 18441003Mackay MT, Weiss SK, Adams-Webber T, Ashwal S, Stephens D, Ballaban-Gill K, Baram TZ, Duchowny M, Hirtz D, Pellock JM, Shields WD, Shinnar S, Wyllie E, Snead OC 3rd; American Academy of Neurology; Child Neurology Society. Practice parameter: medical treatment of infantile spasms: report of the American Academy of Neurology and the Child Neurology Society. Neurology. 2004 May 25;62(10):1668-81. doi: 10.1212/01.wnl.0000127773.72699.c8.
PubMed 15159460Rauen T, Michaelis A, Floege J, Mertens PR. Case series of idiopathic membranous nephropathy with long-term beneficial effects of ACTH peptide 1-24. Clin Nephrol. 2009 Jun;71(6):637-42. doi: 10.5414/cnp71637.
PubMed 19473632Ponticelli C, Passerini P, Salvadori M, Manno C, Viola BF, Pasquali S, Mandolfo S, Messa P. A randomized pilot trial comparing methylprednisolone plus a cytotoxic agent versus synthetic adrenocorticotropic hormone in idiopathic membranous nephropathy. Am J Kidney Dis. 2006 Feb;47(2):233-40. doi: 10.1053/j.ajkd.2005.10.016.
PubMed 16431252Berg AL, Arnadottir M. ACTH-induced improvement in the nephrotic syndrome in patients with a variety of diagnoses. Nephrol Dial Transplant. 2004 May;19(5):1305-7. doi: 10.1093/ndt/gfh110. No abstract available.
PubMed 15102969Bomback AS, Tumlin JA, Baranski J, Bourdeau JE, Besarab A, Appel AS, Radhakrishnan J, Appel GB. Treatment of nephrotic syndrome with adrenocorticotropic hormone (ACTH) gel. Drug Des Devel Ther. 2011 Mar 14;5:147-53. doi: 10.2147/DDDT.S17521.
PubMed 21448451Bomback AS, Canetta PA, Beck LH Jr, Ayalon R, Radhakrishnan J, Appel GB. Treatment of resistant glomerular diseases with adrenocorticotropic hormone gel: a prospective trial. Am J Nephrol. 2012;36(1):58-67. doi: 10.1159/000339287. Epub 2012 Jun 19.
PubMed 22722778LAUSON HD, FORMAN CW, McNAMARA H, MATTAR G, BARNETT HL. The effect of corticotropin (ACTH) on glomerular permeability to albumin in children with the nephrotic syndrome. J Clin Invest. 1954 Apr;33(4):657-64. doi: 10.1172/JCI102936. No abstract available.
PubMed 13152205
Study at a glance
From the registry record, last updated by the sponsor on 23 Apr 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ADRENL - ACTHAR Gel for Drug REsistant Nephrotic Syndrome in Children, Pilot Study
- ClinicalTrials.gov ID
- NCT03408405
- Acronym / sponsor ID
- ADRENL / 1050945-5
- Conditions
- Steroid-Resistant Nephrotic Syndrome
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Withdrawal of funding from primary sponsor
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Rhode Island HospitalUniversity, hospital or other
- Collaborators
- Mallinckrodt, University of Minnesota, Medalytics
- Study start
- Jun 2018 (estimated)
- Primary completion
- Jan 2021 (estimated)
- Study completion
- Jul 2021 (estimated)
- First posted
- 24 Jan 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
We propose to study the use of purified porcine Acthar Gel (ACTHAR, Mallinckrodt Pharmaceuticals) for treatment of steroid resistance nephrotic syndrome (SRNS) in a prospective pilot study. We plan to enroll 25 children between the ages of 2 to 21 years. Children fulfilling strict inclusion criteria, whose parents agree to written informed consent after institutional IRB approval for the study, will be enrolled. Purified porcine Acthar Gel will be administered SQ to all children using a defined treatment protocol for a period of six months. Renal function, urine protein excretion, serum albumin levels, blood pressure and growth parameters will be monitored closely on all patients. Baseline urine protein excretion will be compared to end of treatment levels to determine successful response to therapy. There will be an 18 month enrollment period, 6 month treatment period and a 12 month follow-up period.
Detailed description
Treatment Protocol:
Patients who fulfill inclusion criteria and who agree to participate after signing informed consent and assent forms will be treated with the following protocol:
Acthar Gel will be dosed by body surface area (BSA) using the Dubois Method. The dose of Acthar Gel will be 80 units/1.73 m2 per dose administered subcutaneously (SQ) twice a week on Mondays and Thursdays.
Week 1 to 2:
During week 1, patients will receive 50% of initial calculated dose twice a week.
During week 2, patients will receive 75% of initial calculated dose twice a week.
Week 3 to 6 Months:
Patients will receive the full dose of Acthar Gel (80 units/1.73 m2 per dose) administered subcutaneously (SQ) twice a week on Mondays and Thursdays.
During this period, the treating physician will have the discretion of reducing the dose of Acthar Gel by 25-50% based on response with respect to reduction in proteinuria and tolerability of side-effects (uncontrolled hypertension, excessive weight gain, severe acne, hyperglycemia, etc.)
Patients will have the option of remaining on Acthar Gel after the 6 month study period based on clinical response at discretion of the co-PI. Study drug will be provided free of cost to patients in the first 6 months only.
Concomitant Treatments:
- Amlodipine 0.1 mg/kg PO in one or two divided doses will be given to all patients who develop elevated blood pressure (>95th percentile) starting after 14 days of initiation of Acthar Gel therapy and will continue till 6 months. Dose can be increased up to 0.5 mg/kg divided bid as needed to achieve goal BP <95th percentile. If Amlodipine is not covered by the participants insurance, then an alternate calcium channel blocker will be started at the clinical discretion of the PI/co-PI that is covered by insurance. If the participant is not covered by any insurance the participant will have to pay out of pocket
- If BP remains elevated despite maximum calcium channel therapy as described in the study protocol above, the choice and dose of additional anti-hypertensive agents will be at the discretion of the study investigator on an individualized and as needed basis.
- Patients on prednisone on initiation of study will be tapered off over 2-4 weeks. Non-steroidal immunosuppression may be tapered off over four weeks after the start of Acthar Gel therapy at the discretion of the PI/Co-PI.
- Additional standard of care therapy (statins, vitamin D, antacids, etc.) can be continued and modified based on the discretion and clinical judgment of the study PI/Co-PIs.
Quality of Life Assessment:
Quality of life will be measured using the patient and parent self-report versions of the Pediatric Quality of Life Inventory, ESRD, Acute Version. The PedsQL 3.0 ESRD Scales measure physical, emotional, social and role (i.e. school) dimensions. Scores are derived for physical functioning and psychological functioning, in addition to a total score, which enables comparison against healthy populations. Scales are rated on a 5-point Likert scale (0 = never, 1= almost never, 2 = sometimes, 3 = often, 4 = almost always), with scores linearly transformed to range from 0 - 100, with higher scores indicating fewer problems or symptoms.
Surveys will be administered by trained study personnel at enrollment (baseline), six months, 12 months, and 18 months.
Laboratory Testing:
Baseline and then once a month till the end of the treatment period (6 months): Serum electrolytes, BUN, creatinine, albumin, ACTH level, glucose, random cortisol, calcium, magnesium, phosphorus, ALT, AST, total and direct bilirubin, fasting cholesterol, triglycerides, HDL, LDL, urine protein to creatinine ratio. During the 12 month follow-up period laboratory evaluation will be performed every third month. (11 total blood draws). A urine pregnancy test shall be sent on all female patients of child-bearing potential at the beginning and end of treatment period.
All available biopsy slides will be reviewed by a blinded renal pathologist to insure accuracy and to confirm the initial histologic diagnosis.
Telephone Contact One, two and three weeks after initial dose. Phone contacts will screen for: adverse events, and review dosing of ACTHAR GEL.
Study design
- Primary purpose
- Diagnosis
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Inclusion Criteria: 1. Patients between the ages of 2-21 years who fail a minimum of 12 weeks of cumulative therapy with prednisone OR one other alternate immunosuppressive agent for treatment of nephrotic syndrome, will be eligible for inclusion. Nephrotic syndrome is defined as: Presence of edema, Edema, UP/C ≥2, ≥300mg/dl or 3+ protein on Albustix, and hypoalbuminemia ≤2.5 g/dL 2. Calculated GFR (eGFR) using modified Schwartz formula of > 50 mls/min/m2. (Modified Schwartz formula = 0.413 x height (cms) ÷ serum creatinine mg/dL) 3. A clinical or biopsy diagnosis of nephrotic syndrome within the last 3 years prior to enrollment in the study. 4. Renal biopsy (if available) consistent with a diagnosis of Minimal Change Disease, IgM nephropathy, Mesangioproliferative Glomerulonephritis, Primary Focal Segmental Glomerulosclerosis or C1q Nephropathy
| Arm | Type | Receives |
|---|---|---|
| Acthar Gel treatment group | Experimental | Drug: Acthar Gel 80 UNT/ML Injectable SolutionParticipants will be treated with 'Acthar Gel 80 UNT/ML Injectable Solution'. Initial dose for week 1 will be 50% of 80 units, injected twice per week. Week 2 is 75% of 80 units, week 3 and throughout treatment period (6 months in total) will be 80 units/ml twice per week. |
Who may qualify
- Ages
- 2 to 21 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Patients between the ages of 2-21 years who fail a minimum of 12 weeks of cumulative therapy with prednisone OR one other alternate immunosuppressive agent for treatment of nephrotic syndrome, will be eligible for inclusion. Nephrotic syndrome is defined as: Presence of edema, Edema, UP/C ≥2, ≥300mg/dl or 3+ protein on Albustix, and hypoalbuminemia ≤2.5 g/dL
- Calculated GFR (eGFR) using modified Schwartz formula of > 50 mls/min/m2. (Modified Schwartz formula = 0.413 x height (cms) ÷ serum creatinine mg/dL)
- A clinical or biopsy diagnosis of nephrotic syndrome within the last 3 years prior to enrollment in the study.
- Renal biopsy (if available) consistent with a diagnosis of Minimal Change Disease, IgM nephropathy, Mesangioproliferative Glomerulonephritis, Primary Focal Segmental Glomerulosclerosis or C1q Nephropathy
Key exclusion criteria (5 listed)
- Patients with an inherited or genetic disorder presenting with nephrotic syndrome (eg: NPHS 1 \& 2 defects, WT-1 mutations, α actinin 4 mutation, TRP-6 mutation).
- Presence of diabetes or severe (stage 2) uncontrolled hypertension.
- Any metabolic condition that specifically precludes the use of Acthar Gel for treatment.
- Pregnancy or unwilling to agree to contraception which may include abstinence.
- eGFR <50 mls/min/m2
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 |
|---|---|---|
| Acthar Gel 80 UNT/ML Injectable SolutionAlso: H.P. Acthar gelParticipant will self inject 'Acthar Gel 80 UNT/ML Injectable Solution' 2 x per week for six months. | Drug | Acthar Gel treatment group |
Primary outcome
Remission of proteinuria
Time frame: 18 months
Partial remission: Greater than or equal to 50% reduction in proteinuria when compared to baseline. Complete remission: Greater than or equal to 90% reduction in proteinuria when compared to baseline or Urine Protein Creatinine ratio less than 0.5.
6 secondary outcomes
Blood pressure
Time frame: 18 months
Serum Creatinine
Time frame: 18 months
BMI
Time frame: 18 months
Serum Glucose
Time frame: 18 months
Serum Lipids
Time frame: 18 months
Quality of life measures obtained via survey
Time frame: 18 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Apr 2019.
- Background: PubMed 18441003Coppo R. Non-steroidal and non-cytotoxic therapies for nephrotic syndrome. Nephrol Dial Transplant. 2008 Jun;23(6):1793-6. doi: 10.1093/ndt/gfn211. Epub 2008 Apr 25. No abstract available.
- Background: PubMed 15159460Mackay MT, Weiss SK, Adams-Webber T, Ashwal S, Stephens D, Ballaban-Gill K, Baram TZ, Duchowny M, Hirtz D, Pellock JM, Shields WD, Shinnar S, Wyllie E, Snead OC 3rd; American Academy of Neurology; Child Neurology Society. Practice parameter: medical treatment of infantile spasms: report of the American Academy of Neurology and the Child Neurology Society. Neurology. 2004 May 25;62(10):1668-81. doi: 10.1212/01.wnl.0000127773.72699.c8.
- Background: PubMed 19473632Rauen T, Michaelis A, Floege J, Mertens PR. Case series of idiopathic membranous nephropathy with long-term beneficial effects of ACTH peptide 1-24. Clin Nephrol. 2009 Jun;71(6):637-42. doi: 10.5414/cnp71637.
- Background: PubMed 16431252Ponticelli C, Passerini P, Salvadori M, Manno C, Viola BF, Pasquali S, Mandolfo S, Messa P. A randomized pilot trial comparing methylprednisolone plus a cytotoxic agent versus synthetic adrenocorticotropic hormone in idiopathic membranous nephropathy. Am J Kidney Dis. 2006 Feb;47(2):233-40. doi: 10.1053/j.ajkd.2005.10.016.
- Background: PubMed 15102969Berg AL, Arnadottir M. ACTH-induced improvement in the nephrotic syndrome in patients with a variety of diagnoses. Nephrol Dial Transplant. 2004 May;19(5):1305-7. doi: 10.1093/ndt/gfh110. No abstract available.
- Background: PubMed 21448451Bomback AS, Tumlin JA, Baranski J, Bourdeau JE, Besarab A, Appel AS, Radhakrishnan J, Appel GB. Treatment of nephrotic syndrome with adrenocorticotropic hormone (ACTH) gel. Drug Des Devel Ther. 2011 Mar 14;5:147-53. doi: 10.2147/DDDT.S17521.
- Background: PubMed 22722778Bomback AS, Canetta PA, Beck LH Jr, Ayalon R, Radhakrishnan J, Appel GB. Treatment of resistant glomerular diseases with adrenocorticotropic hormone gel: a prospective trial. Am J Nephrol. 2012;36(1):58-67. doi: 10.1159/000339287. Epub 2012 Jun 19.
- Background: PubMed 13152205LAUSON HD, FORMAN CW, McNAMARA H, MATTAR G, BARNETT HL. The effect of corticotropin (ACTH) on glomerular permeability to albumin in children with the nephrotic syndrome. J Clin Invest. 1954 Apr;33(4):657-64. doi: 10.1172/JCI102936. No abstract available.
- 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.
Mohammed K Faizan, MD principal investigator
Rhode Island Hospital
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Acthar Gel 80 UNT/ML Injectable Solution in people with Steroid-Resistant Nephrotic Syndrome, run by Rhode Island Hospital.
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 4 inclusion and 5 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: withdrawn, as of the sponsor's last update on 23 Apr 2019. Status can change between updates, so confirm with the study contact before making plans.
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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
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?
Remission of proteinuria (time frame: 18 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 NCT03408405, which also lists 8 linked publications.
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 23 Apr 2019.