A Study to Evaluate the Effect of Tacrolimus and Corticosteroid Combination Therapy in Patients With Minimal Change Nephrotic Syndrome (T-OPTIMUM)

Open-Label, Randomized, Comparative, Multi-Center Clinical Trial on the Therapeutic Effect of Tacrolimus (Prograf Cap.®) in Combination With Low-Dose Corticosteroid Compared With High-Dose Corticosteroid Alone in Patients With Minimal-Change Nephrotic Syndrome (MCNS)

To compare the therapeutic effect of tacrolimus in combination with low-dose corticosteroid with high-dose corticosteroid alone in patients with minimal-change nephrotic syndrome.

Study Overview

• Status: Completed
• Conditions: Minimal Change Nephrotic Syndrome (MCNS); MCNS
• Intervention / Treatment: Drug: Tacrolimus; Drug: Prednisolone

• Study Type: Interventional
• Enrollment (Actual): 144
• Phase: Phase 4

Contacts and Locations

Study Locations

• Korea, Republic of
  • Seoul, Korea, Republic of

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years to 78 years (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• patients who have been diagnosed with initial or relapsed primary minimal-change nephrotic syndrome
• patients whose urine protein-creatinine ratio (UPCR) is more than 3.0

Exclusion Criteria:

• patients whose eGFR is less than 30 ml/min/1.73 m2
• patients who were treated with immunosuppressants, such as tacrolimus, cyclosporine, cyclophosphamide (Cytoxan), mizoribine (Bredinin), levamisole, azathioprine, mycophenolate mofetil, or rituximab, within two weeks before the study
• patients to whom more than 10 mg prednisolone or an equivalent dose of steroid was administered daily within two weeks before the study
• patients who are pregnant, breastfeeding, or planning to be pregnant or to breastfeed within six months after the study completion, or who cannot or do not want to use any contraceptive method
• patients who are hypersensitive to the investigational drug or to macrolide, such as azithromycin, clarithromycin, or roxithromycin
• patients who were treated with a live vaccine within four weeks before the study
• patients whose liver panel laboratory test result is three times the normal range, or acute hepatitis patients whose serum bilirubin has been clinically significantly higher than 3.6 mg/dL for more than 1 month
• patients who have a significant general disease that makes it inappropriate for them to participate in this study as adjudged by the investigator (e.g., cardiovascular-acute myocardial infarction, heart failure [classified as more than New York Heart Association {NYHA} class III], hepatic/gastrointestinal/neurologic disease, blood disorder, cancer, infection, renal disorder other than minimal-change nephrotic syndrome, rheumatic arthritis with pneumonia interstitials)
• patients who have genetic problems such as galactose intolerance, Lapp lactose deficiency, or glucose-galactose malabsorption
• patients to whom another investigational drug was administered within 30 days from the enrollment in the study
• patients who participated in the past phases of this study

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Tacrolimus with low-dose corticosteroid; Oral	Drug: Tacrolimus; Oral; Other Names: FK506; Prograf Capsule; Drug: Prednisolone; Oral
Active Comparator: High-dose corticosteroid alone; Oral	Drug: Prednisolone; Oral

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
The percentage of subjects who show a decreased UPCR (Urine Protein Creatinine Rate) of less than 0.2	up to 8 weeks after treatment

Secondary Outcome Measures

Outcome Measure	Time Frame
The period until the UPCR is decreased below 0.2	up to 8 weeks after treatment
The percentage of subjects who show relapse after the remission	up to 24 weeks
The period until the relapse happens from the complete remission	up to 24 weeks
Safety assessed by the incidence of adverse events, labo-tests, vital signs, ECGs and chest X-rays	up to 24 weeks

Collaborators and Investigators

• Sponsor: Astellas Pharma Korea, Inc.
• Investigators: Study Director: Medical Director, Astellas Pharma Inc

Publications and helpful links

General Publications

• 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.

Helpful Links

• Link to results on the Astellas Clinical Study Results website

Study record dates

Study Major Dates

• Study Start (Actual): July 16, 2012
• Primary Completion (Actual): May 2, 2017
• Study Completion (Actual): August 21, 2017

Study Registration Dates

• First Submitted: December 6, 2012
• First Submitted That Met QC Criteria: January 7, 2013
• First Posted (Estimated): January 9, 2013

Study Record Updates

• Last Update Posted (Actual): October 31, 2024
• Last Update Submitted That Met QC Criteria: October 29, 2024
• Last Verified: October 1, 2024

More Information

Terms related to this study

• Keywords: Tacrolimus; Glomerulonephritis; Nephrotic syndrome
• Additional Relevant MeSH Terms: Urogenital Diseases; Pathologic Processes; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Disease; Syndrome; Nephrotic Syndrome; Nephrosis; Nephrosis, Lipoid; Antineoplastic Agents; Immunosuppressive Agents; Immunologic Factors; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Anti-Inflammatory Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Enzyme Inhibitors; Calcineurin Inhibitors; Prednisolone; Tacrolimus
• Other Study ID Numbers: PRGNS-11-02-KOR

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: Access to anonymized individual participant level data will not be provided for this trial. Further details on Astellas' data sharing policy can be found at https://www.clinicaltrials.astellas.com/transparency/.

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No