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A Study of RG-012 in Subjects With Alport Syndrome

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ClinicalTrials.gov Identifier: NCT03373786
Recruitment Status : Active, not recruiting
First Posted : December 14, 2017
Last Update Posted : January 29, 2019
Sponsor:
Information provided by (Responsible Party):
Regulus Therapeutics Inc.

Brief Summary:
This is a Phase 1, open-label, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome.

Condition or disease Intervention/treatment Phase
Alport Syndrome Drug: RG012 Phase 1

Detailed Description:
This is a Phase 1, multi-center study of the safety, pharmacodynamics, and pharmacokinetics of RG-012 administered to subjects with Alport syndrome. During this open-label study, all eligible subjects will receive RG-012. The study consists of two parts (Part A and Part B). During Part A, half of the participants will receive a single dose of RG-012 and half will receive 4 doses of RG-012 (one dose every other week for 6 weeks). All subjects will undergo two renal biopsies, one before and one after receiving RG-012, to assess the effect of RG-012 on the kidney. After completing Part A, subjects will be able to enter Part B of the study. During Part B, all subjects will receive RG-012 every other week for 48 weeks.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 4 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 1, Open-Label Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of RG-012 for Injection, Including Its Effect on Renal microRNA-21, in Subjects With Alport Syndrome
Actual Study Start Date : December 22, 2017
Estimated Primary Completion Date : March 2019
Estimated Study Completion Date : March 2019

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: RG-012 Single Dose
1.5 mg/kg RG012 subcutaneous injection
Drug: RG012
RG012 in 0.3% sodium chloride

Experimental: RG012 Every Other Week
1.5 mg/kg RG012 subcutaneous injections every other week
Drug: RG012
RG012 in 0.3% sodium chloride




Primary Outcome Measures :
  1. Safety - Adverse Events [ Time Frame: 8 weeks ]
    Incidence and severity of adverse events

  2. Effect of RG-012 on renal microRNA-21 (miR-21) [ Time Frame: 8 weeks ]
    Change in miR-21 expression in renal tissue


Secondary Outcome Measures :
  1. Pharmacokinetic (PK) parameter - Cmax [ Time Frame: 8 weeks ]
    Maximum observed plasma concentration

  2. Pharmacokinetic (PK) parameter - Tmax [ Time Frame: 8 weeks ]
    Time to maximum observed plasma concentration

  3. Pharmacokinetic (PK) parameter - AUC [ Time Frame: 8 weeks ]
    Area under the plasma concentration vs. time curve



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Males or females, ages 18 to 65 years
  2. Confirmed diagnosis of Alport syndrome
  3. eGFR between 40 and 90 mL/min/1.73m2
  4. Proteinuria of at least 300 mg protein/g creatinine
  5. For subjects taking an ACE inhibitor or an ARB, the dosing regimen should be stable for at least 30 days prior to screening
  6. Willing to comply with contraception requirements

Exclusion Criteria:

  1. Causes of chronic kidney disease aside from Alport syndrome (such as diabetic nephropathy, hypertensive nephropathy, lupus nephritis, or IgA nephropathy)
  2. End stage renal disease (ESRD) as evidenced by ongoing dialysis therapy or history of renal transportation
  3. Any other condition that may pose a risk to the subject's safety and well-being
  4. Female subjects who are pregnant or lactating

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03373786


Locations
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United States, California
eStudySite
La Mesa, California, United States, 91942
Academic Medical Research Institute
Los Angeles, California, United States, 90022
Apex Research of Riverside
Riverside, California, United States, 92505
United States, Florida
Eminence Medical & Clinical Research
Tampa, Florida, United States, 33604
United States, Texas
Houston Nephrology Research
Cypress, Texas, United States, 77429
United States, Utah
Utah Kidney Research Institute
Salt Lake City, Utah, United States, 84107
United States, Wisconsin
Allegiance Research Specialists, LLC
Wauwatosa, Wisconsin, United States, 53226
Sponsors and Collaborators
Regulus Therapeutics Inc.
Investigators
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Study Director: Mark Deeg, M.D. Regulus Therapeutics

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Responsible Party: Regulus Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT03373786     History of Changes
Other Study ID Numbers: RG012-06
First Posted: December 14, 2017    Key Record Dates
Last Update Posted: January 29, 2019
Last Verified: January 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Regulus Therapeutics Inc.:
Kidney disease
Nephritis
Hereditary kidney disease
Hereditary nephritis

Additional relevant MeSH terms:
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Syndrome
Nephritis, Hereditary
Disease
Pathologic Processes
Urogenital Abnormalities
Nephritis
Kidney Diseases
Urologic Diseases
Congenital Abnormalities
Collagen Diseases
Connective Tissue Diseases