Clinical Trials

ALPORT SYNDROME CLINICAL TRIALS CURRENTLY RECRUITING


CARDINAL Phase 3 Study

Conducted by Reata Pharmaceuticals

Summary:
The phase 3 portion of CARDINAL will continue to evaluate the drug bardoxolone methyl in Alport syndrome. Bardoxolone methyl is a once-daily oral medication. This international study will enroll up to 150 patients, who will receive the active drug or placebo. Eligible participants may receive compensation and/or reimbursement for study-related time and travel, including flight or lodging costs.

Status: Recruitment for the phase 3 portion of CARDINAL began enrolling patients in August 2017.


HERA Phase 2 Study

Conducted by Regulus Therapeutics

Summary:
The HERA Phase 2 study is recruiting approximately 40 male Alport syndrome patients for HERA, a Phase II study evaluating the safety and efficacy of RG-012 compared to placebo.  Travel and lodging assistance is available as well as in-home visits.

RG-012 is an investigational drug administered weekly through injection using a small injection needle similar to those used to administer insulin to people with diabetes.

Status:
Recruitment began in fall 2017.


Kidney Response Phase 1 Study

Conducted by Regulus Therapeutics

Summary:
The Phase 1 kidney response study is recruiting male and female participants with a diagnosis of Alport syndrome. All participants in this study will receive the investigational drug. Travel and lodging assistance is available, as well as in-home visits.

Status:
Recruitment began in fall 2017.

 

RECENTLY COMPLETED ALPORT SYNDROME CLINICAL TRIALS


ATHENA Natural History Study
Conducted by Regulus Therapeutics

Summary:
The ATHENA trial is a natural history study to collect data to characterize or measure the progress of kidney disease in Alport syndrome patients over the course of 6 to 24 months. No investigational treatment for Alport syndrome is provided in this study and participants may remain on any medications prescribed by their physicians.

Status:
The study is still ongoing, but recruitment ended in June 2017.


Multiple Ascending Dose Study
Conducted by Regulus Therapeutics

Summary:
In response to feedback from European regulatory agencies requesting additional information on how the drug RG-012 works and interacts with the body after repeated injections, Regulus Therapeutics initiated a Phase 1 multiple ascending dose study in healthy volunteers.

RG-012 is an investigational anti-microRNA 21 drug administered weekly through injection using a small injection needle similar to those used to administer insulin to people with diabetes.

Status:
The Multiple Ascending Dose study was completed in mid-2017.  


CARDINAL Phase 2 Study
Conducted by Reata Pharmaceuticals

Summary:
The CARDINAL Phase 2 study tests the drug bardoxolone methyl to see 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 will all receive the active drug.

Bardoxolone methyl is 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. They are now testing its effectiveness on patients with Alport syndrome.

Status:
The Phase 2 CARDINAL study is still ongoing, but recruitment ended in mid-2017.

For more information about clinical trials in Alport syndrome, go to clinicaltrials.gov.

 

CLINICAL TRIALS 101

A clinical trial is a formal medical study using human volunteers that allows researchers to better understand a particular disease. Clinical trials can be conducted for several purposes:

  • Prevention
  • New treatment options
  • New diagnostic alternatives
  • Improve quality of life for patients with a serious disease

Following laboratory research studies, researchers create a plan, or protocol, for how to conduct similar tests on human participants. The protocol provides a detailed outline researchers will follow and helps safeguard the health of individuals participating in the study.

In order to conduct a clinical trial, researchers identify individuals who are affected by the disease they are studying; however, they may also need healthy participants who may or may not have the disease, depending on the protocol requirements. Each clinical trial has different requirements for participation. Researchers carefully screen all volunteer participants to ensure each individual meets the protocol parameters and that they understand the important details of the proposed clinical trial and agree to participate.

Following completion of a clinical trial, researchers evaluate the data collected and determine its value for additional research and treatment. Further clinical trials may or may not be deemed appropriate. However, clinical trials are important because they provide new insight into diseases and new treatment options.

Following completion of a clinical trial, researchers evaluate the data collected and determine its value for additional research and treatment. Further clinical trials may or may not be deemed appropriate. However, clinical trials are important because they provide new insight into diseases and new treatment options.

Whether you’ve taken a medication today to manage a chronic disease or simply to keep a headache at bay, you know that medical innovations have the power to change lives. But these breakthroughs don’t just happen. Developing new medicines is a lengthy and complex process, relying heavily on volunteer participation to evaluate potential therapies for safety and effectiveness in clinical studies. Without the patients who volunteer to participate in clinical trials, the development of new medicines would not be possible.

Clinical trials are conducted in phases that accomplish different purposes. For example, the ATHENA study mentioned above was an observational study designed to learn more about the changes over time in the kidneys of Alport syndrome patients. This study did not involve the use of any investigational drugs. The Multiple Ascending Dose study tested an investigational drug in healthy patients (i.e., people without Alport syndrome) to make sure there were no safety concerns or serious side effects. The CARDINAL and HERA studies will test how specific drugs affect chronic kidney disease in people with Alport syndrome.

Below is a simple explanation of the different stages in the development of new treatments to help patients better understand the research process.

  • Observational study. This early phase allows researchers to better understand the natural progression of the disease they are studying. While experimental drugs are not typically provided during observational studies, participants are often able to continue taking existing medications prescribed by their own physicians.
  • Phase 1 trial. Before new medications are approved for human patients, researchers give the experimental drugs to a small group of healthy volunteers. Researchers determine appropriate dosage and test for safety and side effects.
  • Phase 2 trial. Once an experimental drug is deemed safe for patients, it is administered to a small group of patients to see if it improves health outcomes for the condition being studied and to continue to evaluate safety.
  • Phase 3 trial. Once data demonstrates an experimental treatment is both safe and effective, a larger group of patients are given the drug. Researchers continue to evaluate the safety and determine if the new drug is more or less effective than current treatments.
  • Phase 4 trial. After the new treatment is marketed to patients, researchers continue to assess the drug’s safety and efficacy.

If you are interested in any of the active trials in Alport syndrome listed above, please contact the study administrators directly for eligibility, study locations, and other information about the trials.

Patients, family members, and physicians are invited to join the patient registry. When a new trial is announced, the patient registry will contact any registry participants who meet the study protocol requirements. The Alport Syndrome Treatments and Outcomes Registry (ASTOR) at the University of Minnesota, the US patient registry, can be reached here.

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