Clinical trial NCT06712407: Efficacy and Safety of Extended TARPEYO® Treatment Beyond 9 Months in Adult Patients With Primary IgA Nephropathy
Official title: An Open-label Study to Assess the Efficacy and Safety of Extended TARPEYO® (Delayed-release Budesonide Capsules) Treatment in Adult Patients With Primary IgA Nephropathy Who Have Completed 9 Months of TARPEYO® 16 mg Once Daily Treatment in Real-world Clinical Practice
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE4
Conditions: IgA Nephropathy
Keywords: TARPEYO®
IgAN
urine protein-to-creatinine
ESRD
Nefecon
Lead sponsor: Calliditas Therapeutics AB
Sponsor class: INDUSTRY
Collaborators: name: Worldwide Clinical Trials
First posted: 2024-12-02
Enrollment: 60
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Intervention model description: There are 2 treatment periods with the study drug:

* TARPEYO® 16 mg (4 capsules) once a day for 6 months
* TARPEYO® 8 mg (2 capsules) once a day for 9 months.
Primary purpose: TREATMENT

Brief summary: The goal of this clinical trial is to assess the efficacy and safety of extended TARPEYO® (delayed-release budesonide capsules) treatment in adult patients with primary IgA nephropathy who have completed 9 months of TARPEYO® 16 mg once daily treatment in real-world clinical practice. The main question it aims to answer is:

Is there a treatment benefit of TARPEYO® 16 mg QD extended use?

Participants will

* take part in this study for about 19 months
* Have urine tests done
* Have blood samples taken
* Have physical examinations done

Detailed description: This clinical trial will investigate the efficacy and safety of TARPEYO® treatment extended for an additional 15 months in adult IgAN participants who have completed their 9-month TARPEYO® 16 mg QD commercial treatment regimen. Participants with residual proteinuria will be eligible for enrollment. The Treatment Period will consist of a 6-month Treatment Period with TARPEYO® 16 mg QD, followed by a 9-month Treatment Period with TARPEYO® 8 mg QD. This will be followed by a 3-month Follow-up Period, which includes the first 2 weeks of Tapering Period with TARPEYO® 4 mg QD.

The overall aim of the extended treatment is to improve and maintain the treatment effect with reduced proteinuria and protection of kidney function over a total of 2 years of TARPEYO® treatment.

Eligibility criteria: Inclusion Criteria:

1. Diagnosed IgAN with biopsy verification
2. Female or male participants ≥18 years of age
3. Completion of 9 months of treatment with TARPEYO® 16 mg QD at the Baseline visit
4. Access to retrospective local laboratory assessment data on UPCR and serum creatinine.

   Available retrospective data should include at least 1 assessment timepoint within 3 months prior to the first dose of TARPEYO® commercial treatment
5. Proteinuria at Screening based on 2 consecutive measurements (24-hour urine collection) after informed consent, separated by at least 1 week and calculated by the central laboratory.

   Both samples of the same parameter must show either of the following:
   * Proteinuria ≥0.5 g per day (≥500 mg per day) in 2 consecutive measurements, or
   * UPCR ≥0.3 g/gram in 2 consecutive measurements
6. On stable treatment with renin-angiotensin system (RAS) inhibitor therapy for at least 8 weeks prior to the Baseline visit. A stable dose is defined as a dose within 25% of the dose at Baseline
7. If on current treatment with sodium-glucose cotransporter-2 (SGLT2) inhibitor, the treatment should have been stable for at least 8 weeks prior to the Baseline visit. A stable dose is defined as a dose within 25% of the dose at Baseline

Exclusion Criteria:

1. Participants who have been treated with systemic immunosuppressive medications including glucocorticosteroids (GCS) other than TARPEYO® during the TARPEYO® commercial treatment period. Topical or inhalation products containing GCS or immunosuppressants are allowed
2. Presence of other glomerulopathies (e.g., C3 glomerulopathy and/or diabetes nephropathy)
3. Presence of nephrotic syndrome (i.e., proteinuria \>3.5 g per day and serum albumin \<3.0 g/dL, with or without edema)
4. Presence of medical condition excluding continued TARPEYO® treatment, as assessed by the Investigator
5. On current or planned dialysis.
6. Undergone kidney transplant.
7. Poorly controlled diabetes mellitus or hypertension, as assessed by the Investigator.
8. Participants with known osteoporosis in the medium- or high-risk category according to the 2010 American College of Rheumatology recommendations.
9. Any medical or social circumstance making trial participation and/or TARPEYO® treatment unsuitable, as assessed by the Investigator.
10. Participants with clinically significant infections that put the participant at risk, at the discretion of the Investigator.
11. Participants unwilling or unable to meet the requirements of the protocol.
12. Intake of another investigational drug during trial, or during the preceding 9-month commercial TARPEYO® treatment period.
13. Females who are pregnant, breastfeeding, or plan to become pregnant in the trial period.
14. Participants taking potent inhibitors of cytochrome P450 (CYP) 3A4

Primary outcomes:
measure: Ratio of Urine Protein to Creatine Ratio (UPCR) at 6 months compared to Baseline
timeFrame: 6 months
Secondary outcomes:
measure: Ratio of Urine Protein to Creatine Ratio (UPCR) at 15 months compared to Baseline
timeFrame: 15 months
measure: Ratio of Urine Protein to Creatine Ratio (UPCR) at 6 months compared to prior to start of TARPEYO® commercial treatment (i.e., comparison with retrospective local laboratory data from medical records as available)
timeFrame: 18 months
measure: Ratio of Urine Protein to Creatine Ratio (UPCR) at 15 months compared to prior to start of TARPEYO® commercial treatment (i.e., comparison with retrospective local laboratory data from medical records as available)
timeFrame: 27 months
measure: Ratio of estimated glomerular filtration rate (eGFR) at 6 months compared to Baseline
timeFrame: 6 months
measure: Ratio of estimated glomerular filtration rate (eGFR) at 15 months compared to Baseline
timeFrame: 15 months
measure: Ratio of estimated glomerular filtration rate (eGFR) at 6 months compared to start of TARPEYO® commercial treatment (i.e., comparison with retrospective local laboratory data from medical records as available)
timeFrame: 18 months
measure: Ratio of estimated glomerular filtration rate (eGFR) at 15 months compared to start of TARPEYO® commercial treatment (i.e., comparison with retrospective local laboratory data from medical records as available)
timeFrame: 27 months
measure: Ratio of 24-hour urine proteinuria at 6 months compared to Baseline
timeFrame: 6 months
measure: Ratio of 24-hour urine proteinuria at 15 months compared to Baseline
timeFrame: 15 months
measure: Proportion of participants with microhematuria at 6 months
timeFrame: 6 months
measure: Proportion of participants with microhematuria at 15 months
timeFrame: 15 months
measure: To evaluate the safety (SAEs and AEs) of extended TARPEYO® treatment
timeFrame: 19 months
Other outcomes:
Locations:
city: Glendale
country: United States
facility: Arizona Kidney Disease & Hypertension Centers (AKDHC)
state: Arizona
city: New Haven
country: United States
facility: Yale University Nephrology Clinical Trials Program
state: Connecticut
city: Boca Raton
country: United States
facility: Florida Kidney Physicians
state: Florida
city: Austell
country: United States
facility: Cobb Nephrology Hypertension Associates, PC
state: Georgia
city: Albuquerque
country: United States
facility: Renal Medical Associates
state: New Mexico
city: New York City
country: United States
facility: Chinatown Kidney Care, PLLC
state: New York
city: Dallas
country: United States
facility: Dallas Renal Group
state: Texas
city: El Paso
country: United States
facility: MedResearch Inc
state: Texas
city: Galveston
country: United States
facility: The University of Texas Medical Branch UTMB
state: Texas
city: Houston
country: United States
facility: Memorial Hermann Houston
state: Texas
city: McKinney
country: United States
facility: Dallas Nephrology Associates McKinney
state: Texas
city: Odessa
country: United States
facility: Permian Basin Kidney Center
state: Texas
Study officials:
affiliation: Calliditas Therapeutics
name: Kristin Önnestam
role: STUDY_DIRECTOR
References: