Dose-Exploration Evaluating the Efficacy and Safety of Voclosporin in Subjects With Focal Segmental Glomerulosclerosis

NCT03598036 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: AUR-VCS-2017-03
• Study Type: interventional
• Target: 5
• Locations: 2 countries
• Sites: 23

Brief Summary

Evaluating the Efficacy and Safety of Voclosporin in Achieving Complete or Partial Remission of Proteinuria in Subjects with Focal Segmental Glomerulosclerosis

Conditions

Focal Segmental Glomerulosclerosis

Keywords

calcineurin inhibitors; Focal Segmental Glomerulosclerosis

Outcome Measures

Primary Outcomes (1)

• Proportion of Subjects With Remission of Proteinuria

  Complete remission OR Partial remission

  24 weeks

Secondary Outcomes (19)

• Proportion of Subjects With Complete Remission or Partial Remission of Proteinuria

  Weeks 8 and 12

• Proportion of Subjects With Complete Remission of Proteinuria

  Weeks 8, 12, and 24

• Proportion of Subjects With Reduction of Proteinuria

  Weeks 8, 12, and 24

• Proportion of Subjects With Partial Remission of Proteinuria

  Weeks 8, 12, and 24

• Time to First Occurrence of Complete or Partial Remission of Proteinuria

  Up to 26 weeks

Study Arms (1)

Voclosporin

EXPERIMENTAL

Cohort 1: Maximum dose of 3 capsules (7.9mg) BID Cohort 2 Dosing to be decided based on the safety from the first 5 or 6 subjects in Cohort 1.

Drug: Voclosporin

Interventions

Voclosporin DRUG

Voclosporin softgel capsules. Up to 10 subjects will be enrolled into Cohort 1 and take up to 3 capsules twice daily (BID). The dose of voclosporin for Cohort 2 (at least 10 subjects) will be determined by analysis of efficacy and safety data at Week 12 from the first 5 or 6 subjects in Cohort 1.

Voclosporin

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Primary FSGS diagnosed by renal biopsy.
• Urine protein creatinine ratio (UPCR) ≥2.0 mg/mg, serum albumin ≤3.2 g/dL and can be immunosuppressant treatment-naïve, or receiving treatment with steroids.
• Stable proteinuria, renal function, and BP.

You may not qualify if:

• Clinical or histologic evidence of secondary FSGS.
• Histologic evidence of collapsing variant FSGS.
• eGFR as calculated by the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) equation of ≤30 mL/minute/1.73 m2 at initial screening assessment or ≤45 mL/minute/1.73 m2 at last qualifying assessment.
• Currently requiring renal dialysis (hemodialysis or peritoneal dialysis) or expected to require dialysis during the study period.
• Current or medical history of:
• Congenital or acquired immunodeficiency.
• In the opinion of the Investigator, clinically significant drug or alcohol abuse within 2 years prior to screening.
• Malignancy within 5 years of screening, with the exception of basal and squamous cell carcinomas treated by complete excision.
• Current or past lymphoproliferative disease or previous total lymphoid irradiation.
• Severe viral infection within 3 months of screening, or known HIV infection. Severe viral infection is defined as active disease requiring antiviral therapy.
• Active tuberculosis (TB) or known history of TB/evidence of old TB if not taking prophylaxis with isoniazid.

Sponsors & Collaborators

• Aurinia Pharmaceuticals Inc.: lead

Study Sites (23)

FSGS Investigative Center

Denver, Colorado, 80213, United States

Location

FSGS Investigative Center

Winter Park, Florida, 32789, United States

Location

FSGS Investigative Center

Lawrenceville, Georgia, 30046, United States

Location

FSGS Investigative Center

Chicago, Illinois, 60611, United States

Location

FSGS Investigative Center

Iowa City, Iowa, 52242, United States

Location

FSGS Investigative Center

Louisville, Kentucky, 40202, United States

Location

FSGS Investigative Center

Shreveport, Louisiana, 71101, United States

Location

FSGS Investigative Center

Detroit, Michigan, 48201, United States

Location

FSGS Investigative Center

Minneapolis, Minnesota, 55414, United States

Location

FSGS Investigative Centre

St Louis, Missouri, 63110, United States

Location

FSGS Investigative Center

Newark, New Jersey, 07103, United States

Location

FSGS Investigative Center

New York, New York, 10032, United States

Location

FSGS Investigative Center

Chapel Hill, North Carolina, 27599-7155, United States

Location

FSGS Investigative Center

Charlotte, North Carolina, 28204, United States

Location

FSGS Investigative Center

Columbus, Ohio, 43210, United States

Location

FSGS Investigative Center

Bethlehem, Pennsylvania, 18017, United States

Location

FSGS Investigative Center

Dallas, Texas, 75231, United States

Location

FSGS Investigative Center

Dallas, Texas, 75246, United States

Location

FSGS Investigative Center

El Paso, Texas, 79935, United States

Location

FSGS Investigative Center

Houston, Texas, 77030, United States

Location

FSGS Investigative Site

Salt Lake City, Utah, 84115, United States

Location

FSGS Investigative Centre

Santiago de los Caballeros, 51000, Dominican Republic

Location

FSGS Investigative Center

Santo Domingo, Dominican Republic

Location

Related Publications (4)

• Ling SY, Huizinga RB, Mayo PR, Larouche R, Freitag DG, Aspeslet LJ, Foster RT. Cytochrome P450 3A and P-glycoprotein drug-drug interactions with voclosporin. Br J Clin Pharmacol. 2014 Jun;77(6):1039-50. doi: 10.1111/bcp.12309.

  PMID: 24330024 BACKGROUND

• Ling SY, Huizinga RB, Mayo PR, Freitag DG, Aspeslet LJ, Foster RT. Pharmacokinetics of voclosporin in renal impairment and hepatic impairment. J Clin Pharmacol. 2013 Dec;53(12):1303-12. doi: 10.1002/jcph.166. Epub 2013 Oct 8.

  PMID: 23996158 BACKGROUND

• Mayo PR, Huizinga RB, Ling SY, Freitag DG, Aspeslet LJ, Foster RT. Voclosporin food effect and single oral ascending dose pharmacokinetic and pharmacodynamic studies in healthy human subjects. J Clin Pharmacol. 2013 Aug;53(8):819-26. doi: 10.1002/jcph.114. Epub 2013 Jun 4.

  PMID: 23736966 BACKGROUND

• Busque S, Cantarovich M, Mulgaonkar S, Gaston R, Gaber AO, Mayo PR, Ling S, Huizinga RB, Meier-Kriesche HU; PROMISE Investigators. The PROMISE study: a phase 2b multicenter study of voclosporin (ISA247) versus tacrolimus in de novo kidney transplantation. Am J Transplant. 2011 Dec;11(12):2675-84. doi: 10.1111/j.1600-6143.2011.03763.x. Epub 2011 Sep 22.

  PMID: 21943027 BACKGROUND

MeSH Terms

Conditions

Glomerulosclerosis, Focal Segmental

Interventions

voclosporin

Condition Hierarchy (Ancestors)

Glomerulonephritis; Nephritis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases

Limitations and Caveats

Due to difficulties enrolling appropriate Focal Segmental Glomerulosclerosis (FSGS) patients, Aurinia terminated the clinical trial early. Focal Segmental Glomerulosclerosis (FSGS) is a rare disease with an estimated 40,000 adult patients in the USA. Only five subjects were enrolled into the clinical trial.

Results Point of Contact

• Title: Clinical Trial Support
• Organization: Aurinia Pharmaceuticals Inc.

clinicaltrials@auriniapharma.com

1 (250) 744-2487

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 14, 2018
• First Posted: July 26, 2018
• Study Start: June 21, 2018
• Primary Completion: May 19, 2020
• Study Completion: May 19, 2020
• Last Updated: September 16, 2021
• Results First Posted: September 16, 2021

Record last verified: 2020-06

Data Sharing

• IPD Sharing: Will not share

Locations

DO (2); US (21)