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CLINICAL TRIALS

A summary of current clinical trials for Alport syndrome is listed below. For more information visit clinicaltrials.gov.

CARDINAL Study

Conducted by Reata Pharmaceuticals

STATUS

Fully enrolled; recruitment for Phase 3 closed Fall 2018.

STUDY UPDATE

March 30, 2020 UPDATE:  Per Reata Pharmaceutical’s March 30, 2020 press release, “The Company is implementing procedures for the conduct of EAGLE that are similar to those being used in CARDINAL to ensure continued access to bardoxolone and appropriate safety monitoring.” Patients enrolled in these studies are being encouraged to contact their individual study site coordinators for specific instructions during the COVID-19 pandemic.

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First year data from this study was released November 2019. Primary and secondary endpoints were met. This study remains in progress. Following is a press release with additional information about the first year data.

FIRST YEAR DATA

SUMMARY

The CARDINAL Study is investigating bardoxolone methyl given as a once-daily oral medication that promotes normal mitochondrial function and potentially reduces kidney inflammation. Reata Pharmaceuticals has already completed prior studies of the drug for patients with chronic kidney disease (CKD) caused by type 2 diabetes, with favorable results. Currently the effectiveness of bardoxolone methyl is being tested on patients with Alport syndrome.

The Phase 2 CARDINAL Study, a two-year commitment by patients, is investigating how the drug affects the chronic inflammation and decline in kidney function that are key features of Alport syndrome. The Phase 2 portion of the study enrolled 30 patients who received the active drug.

The Phase 3 CARDINAL Study continues to evaluate the drug bardoxolone methyl in Alport syndrome. This international study enrolled 150+ patients, who are receiving the active drug or placebo. Participants receive compensation and/or reimbursement for study-related time and travel, including flight or lodging costs.

HERA Phase 2 Study (Continued)

Conducted by Sanofi-Genzyme

STATUS

Currently Enrolling

SUMMARY

As of November 2019, the HERA clinical trial has been officially re-initiated.

The HERA study was re-initiated after the study was put on hold in 2018 by Regulus Therapeutics for non-safety and non-regulatory reasons. Sanofi has taken over the development of SAR339375 and re-initiated the clinical study after the protocol design was re-assessed and optimized. Sanofi is seeking Alport patients who meet the inclusion criteria in the US, Europe, China and Australia.

The effectiveness and safety of SAR339375, an anti-miR21 compound, is being investigated in a two years study consisting of different periods. In the double-blind period (first year), Sanofi is comparing the investigational drug with a placebo (which looks like the investigational drug, but contains no active medicine). The second year is an open-label period where every patient receives the active investigational medicine. After two years of treatment, there is a 6-week follow-up period.

Patients will have weekly visits, either at the study center or at home. At a minimum, patients will need to visit the study center every 3 months. There will be no cost to the patient while participating in this trial. Travel and lodging will be reimbursed. The investigational drug (and placebo) are provided as an injection under the skin using a small gauge needle. Periodic tests and assessments are to be done, such as blood and urine tests, hearing assessments, ECG, genetic sequencing. Biopsies are not required during the study.

The study is looking for male and female patients who:
· Are 18-55 years of age (inclusive)
· Have a confirmed diagnosis of Alport syndrome
· Have a certain value of kidney filtration rate (eGFR >35 ml/min/1.73m2 and <90 ml/min/1.73m2)
· Have not received a kidney transplant
· Are currently not on dialysis
· Are less than 110kg (242 pounds)
· Do not have diabetes mellitus

More information on this study can be found on the ClinicalTrials.gov website, including
contact information. Currently only one site is active in Cleveland, Ohio, however, more US sites will gradually be added in 2020 (possibly New York, California, Minnesota, Utah). Updated information will be updated on this site as information becomes available.

Freedom-1 Phase 3 Study

Conducted by Talaris Therapeutics

STATUS

Currently Enrolling

SUMMARY

The FREEDOM-1 Study is a clinical research study now enrolling adults who plan to receive a kidney transplant from a living donor. The purpose of this study is to learn more about whether a novel cell therapy called FCR001 can prevent the rejection of living donor kidney transplants without the need for lifelong anti-rejection drugs.

Although anti-rejection medicines are very effective at preventing rejection of kidneys, particularly in the first several years following the transplant, multiple side effects and tolerability issues can affect recipients’ ability or willingness to take these medicines, ultimately adversely affecting long-term outcomes for donated kidneys and recipients.

FREEDOM-1 is a Phase 3 clinical trial that will compare the efficacy and safety of FCR001 treatment to standard anti-rejection treatment. This study is now enrolling at sites around the U.S. Find out where the study is being offered here. To learn more about the study and determine if you are eligible to participate, click here.

An archive of past clinical trials for Alport syndrome is listed below. For more information visit clinicaltrials.gov.

Kidney Response Phase 1 Study

Conducted by Regulus Therapeutics

SUMMARY

When active, the Phase 1 kidney response study was recruiting male and female participants with a diagnosis of Alport syndrome. When recruiting, all participants in this study were receiving the investigational drug. Travel and lodging assistance, along this in-home visits, were available.

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