Clinical trial NCT01197040: Evaluation of Low Dose Corticosteroids Efficiency, Associated With Myfortic ® in the Treatment of Nephrotic Syndrome
Official title: Low Steroid Dose Combined With Mycophenolic Acid (Myfortic) Compared With High Dose Steroid for Minimal Change Nephrotic Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Nephrotic Syndrome
Keywords: Minimal Change Nephrotic Syndrome
Complete remission
Myfortic
Minimal Change Disease
Lead sponsor: Assistance Publique - Hôpitaux de Paris
Sponsor class: OTHER
Collaborators: name: Novartis
First posted: 2010-09-09
Enrollment: 117
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: A multicenter, randomized, study will be performed to evaluate the efficacy of low dose steroid combined with mycophenolic acid (MyforticR) versus high dose steroid in inducing remission in adults with minimal change nephrotic syndrome (MCNS). One hundred and fourteen patients (CPP decision 2009-04-02-a5) will be included in this study. They will be randomly assigned to an open label treatment with either prednisone

1 mg/kg/day (arm A) or 0,5 mg/kg/day plus myforticR 1440 mg/day (arm B) for four weeks. The outcome will be compared during one-year follow up

Detailed description: The treatment for minimal change nephrotic syndrome (MCNS) is empirically based on high dose steroid. However, the side effects in adult patients are often significant and induce a lot of complications. This prospective study aimed to compare low dose steroid combined with mycophenolic acid (MyforticR) versus high dose steroid in the treatment of the first episode of MCNS.

Treatment Plan

After baseline evaluation including clinical biological and histological analyses, all eligible patients will be an open label assigned to two groups:

Group A: patients will receive prednisone 1 mg/kg/day for 4 weeks. Then, the dose will be progressively tapered if the remission will be achieved. For patients who exhibit incomplete remission at this time, high dose steroid will be continued for 4 weeks again before the tapering.

Group B: patients will receive prednisone 0.5 mg/kg/day combined with mycophenolic acid (MyforticR) 1440 mg/day. The management of steroid therapy will be identical in both groups, while MyforticR will be continued for six months.

In both groups, patients who will not achieve remission after 8 weeks of steroid therapy at full dose will be excluded from the study.

Statistical Analysis In this multicenter, randomized trial, the primary and secondary end points will be the rate of complete remission within 4 and 8 weeks of the start of induction therapy, respectively.

Eligibility criteria: Inclusion Criteria:

* Idiopathic nephrotic syndrome
* Flare of idiopathic syndrome without treatment from one year
* Confirming by Renal Biopsy

Exclusion Criteria:

* Secondary nephrotic syndrome
* Pregnancy
* Focal Segmental Glomerular sclerosis lesion in the Biopsy
* Neutropenia \< 2000/mm3
* Hb\<9gr/dl

Primary outcomes:
measure: complete remission
timeFrame: within 4 weeks
Secondary outcomes:
measure: complete remission
timeFrame: within 8 weeks
measure: partial remission
timeFrame: within 4 and 8 weeks
measure: Adverse effects in both arms
timeFrame: 1 year
measure: Number of flare in both arms
timeFrame: at 1 year
Other outcomes:
Locations:
city: Creteil
country: France
facility: Henri Mondor Hospital
state: Val de Marne
Study officials:
affiliation: Assistance Publique - Hôpitaux de Paris
name: Philippe REMY, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: Azukaitis K, Palmer SC, Strippoli GF, Hodson EM. Interventions for minimal change disease in adults with nephrotic syndrome. Cochrane Database Syst Rev. 2022 Mar 1;3(3):CD001537. doi: 10.1002/14651858.CD001537.pub5.
citation: Remy P, Audard V, Natella PA, Pelle G, Dussol B, Leray-Moragues H, Vigneau C, Bouachi K, Dantal J, Vrigneaud L, Karras A, Pourcine F, Gatault P, Grimbert P, Ait Sahlia N, Moktefi A, Daugas E, Rigothier C, Bastuji-Garin S, Sahali D; MSN Trial Investigators. An open-label randomized controlled trial of low-dose corticosteroid plus enteric-coated mycophenolate sodium versus standard corticosteroid treatment for minimal change nephrotic syndrome in adults (MSN Study). Kidney Int. 2018 Dec;94(6):1217-1226. doi: 10.1016/j.kint.2018.07.021. Epub 2018 Oct 29.