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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

An update on the CARDINAL Study was shared as part of a Reata Pharmaceuticals Press Release published on April 26, 2021: “Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” or “we”), a clinical-stage biopharmaceutical company, today announced that the U.S. Food and Drug Administration (“FDA”) accepted for filing the New Drug Application (“NDA”) for bardoxolone methyl (“bardoxolone”) for the treatment of patients with chronic kidney disease (“CKD”) caused by Alport syndrome.”

VIEW THE FULL APRIL 26, 2021 PRESS RELEASE

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 receive the active drug or placebo. Participants receive compensation and/or reimbursement for study-related time and travel, including flight or lodging costs. See “Study Update” at left for a March 2021 update.


EAGLE is an extended access study that will assess the long-term safety and tolerability of bardoxolone methyl in qualified patients with chronic kidney disease (CKD) who previously participated in one of the qualifying clinical studies with bardoxolone methyl. Patients will remain in the study until bardoxolone methyl is available through commercial channels or until patient withdrawal, whichever is sooner.

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 lademirsen (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 10-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 HERA Clinical Trial website and the ClinicalTrials.gov website. As of May 25, 2021, there are five active investigational sites in the United States (Los Angeles, California; Minneapolis, Minnesota; New York City, New York; Cleveland, Ohio; Salt Lake City, Utah). Updated site information will be posted as it becomes available.

ASF invited a medical expert at Sanofi-Genzyme who designed the HERA clinical trial to speak to the larger Alport community about this ongoing study and to answer patients’ questions as part of a webinar held on June 30, 2021. You can watch a closed-captioned recording of the webinar at this link.

The AFFINITY Study

Conducted by Chinook Therapeutics U.S., Inc. 

STATUS

Currently Enrolling.

April 7, 2021 Update

Chinook Therapeutics, Inc., a biopharmaceutical company focused on the discovery, development and commercialization of precision medicines for kidney diseases, announced that the first patient has been dosed in the AFFINITY Study, a phase 2 clinical trial evaluating the efficacy and safety of atrasentan, a potent and selective inhibitor of the endothelin A receptor, in patients with proteinuric glomerular disease who are at risk of progressive loss of renal function. Read the full press release.

SUMMARY

The AFFINITY study is a phase 2 clinical trial testing an investigational medication called atrasentan. Atrasentan has the potential to provide benefit in Alport Syndrome and other glomerular diseases by reducing the amount of protein in the urine and preserving kidney function.

  • The goal of the study is to evaluate the effect of atrasentan on proteinuria in patients with Alport Syndrome, IgA nephropathy, focal segmental glomerulosclerosis, and in patients with diabetic kidney disease who are taking an SGLT2 inhibitor.
  • Approximately 20 patients will be enrolled in each group, including 20 patients with Alport Syndrome (~80 patients total). Patients in this study will receive atrasentan as an oral tablet once a day for approximately 1 year.
  • To facilitate study participation, where allowed by local regulations, options for remote study visits using telemedicine and home health may be offered. Patients will be reimbursed for study-related expenses, such as travel, flight, or lodging costs.
  • Enrollment begins in the United States, Australia, South Korea, Italy, Spain, and the United Kingdom in early 2021. A list of active study sites as of September 7, 2021 can be viewed here.

More information on this study can be found on the clinicaltrials.gov website. To inquire about screening for the study in early 2021 contact: [email protected]

ASF hosted an educational webinar, featuring Q&A, about the AFFINITY Study on Friday, March 19, 2021. Click here to watch a recording of the webinar.

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.

Additionally, you can watch the recording of a 1-hour, closed captioned, educational webinar about living organ donation and the FREEDOM-1 Study at this link.

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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