Resmetirom for Kidney Failure

No longer recruiting at 2 trial locations

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: Madrigal Pharmaceuticals, Inc.

Disqualifiers: Gilbert's syndrome, Hepatobiliary disease, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Breakthrough Therapy This drug has been fast-tracked for approval by the FDA given its high promise

Approved in 1 Jurisdiction This treatment is already approved in other countries

Compensation: Varies

Number of Visits: Unknown

Duration: 6 days

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests how the body processes a drug called resmetirom (also known as Rezdiffra or MGL-3196) in individuals with severe kidney problems compared to healthy individuals. Resmetirom may potentially aid in kidney failure, and the trial aims to understand its behavior in the body. Potential participants include those with severe kidney impairment who are not on dialysis and healthy individuals of similar age and body size for comparison. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this potentially beneficial treatment.

Do I have to stop taking my current medications for the trial?

The trial information does not specify if you need to stop taking your current medications. It's best to discuss this with the trial team or your doctor.

Is there any evidence suggesting that resmetirom is likely to be safe for humans?

Research shows that resmetirom has been tested for various health issues, but not specifically in individuals with severe kidney problems. Other studies have found resmetirom to be safe for its intended uses, with common side effects including stomach issues. The FDA has already approved this treatment for other uses, indicating its safety for those conditions. However, since this trial is in an early phase for individuals with severe kidney problems, the researchers are still collecting safety information for this specific group. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Resmetirom is unique because it targets a different mechanism in treating kidney failure. While most treatments focus on managing symptoms or complications, Resmetirom is a thyroid hormone receptor beta-selective agonist. This means it specifically activates certain receptors that could potentially improve kidney function and metabolism. Researchers are excited about Resmetirom because it offers a novel approach that could complement or enhance existing therapies, possibly leading to better outcomes for patients with severe renal impairment.

What evidence suggests that resmetirom might be an effective treatment for kidney failure?

Research has shown that resmetirom has been effective in treating nonalcoholic steatohepatitis (NASH), a liver condition. However, its effectiveness for kidney failure remains unknown. This trial will compare how participants with severe kidney impairment and those with healthy kidneys process resmetirom. While resmetirom works well for NASH, its effects on kidney health are still under investigation. ^{1 4 5 6 7}

Who Is on the Research Team?

Geoff Rezvani

Principal Investigator

Madrigal Pharmaceuticals, Inc.

Are You a Good Fit for This Trial?

This trial is for adults with severe kidney failure and those who are healthy with normal kidney function. Participants will be matched based on factors like age, weight, and gender. People cannot join if they have other significant health issues or are taking medications that could interfere with the study.

Inclusion Criteria

I understand the study details and agree to participate.

I weigh over 50 kg, my BMI is between 18 and 45, and I have severe kidney issues but am otherwise healthy.

I am healthy, with normal kidney function, and my BMI and age match a patient with severe renal impairment.

Exclusion Criteria

I have severe kidney issues with specific thyroid levels, or a history of major health problems.

All Subjects: Gilbert's syndrome, pre-existing condition interfering with normal gastrointestinal anatomy or motility, or hepatic function, TSH < 0.3 or > 7 mIU/L, evidence or history of clinically significant diseases, failed renal transplant, end stage renal disease, abnormal blood pressure or heart rate

I am healthy with no liver, thyroid, significant diseases, or abnormal heart rates.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

1-2 weeks

Treatment

Participants receive 100 mg resmetirom orally once daily for 6 days

6 days

Follow-up

Participants are monitored for safety and effectiveness after treatment

2 days

What Are the Treatments Tested in This Trial?

Interventions

• Resmetirom

Trial Overview The study tests how a drug called Resmetirom behaves in the body when taken orally at a dose of 100 mg daily for six days by people with severe renal impairment compared to healthy individuals.

How Is the Trial Designed?

Group I: Severe renal impairment Experimental Treatment 1 Intervention

Group II: Normal Healthy Match Experimental Treatment 1 Intervention

Matched to severe renal impairment

Resmetirom is already approved in United States for the following indications:

Approved in United States as Rezdiffra for:

• Noncirrhotic nonalcoholic steatohepatitis with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis)

Who Is Running the Clinical Trial?

Madrigal Pharmaceuticals, Inc.

Lead Sponsor

Trials

16

Recruited

5,400+

Citations

1. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC11569717/

Resmetirom - PMC The recommended dosage of resmetirom in patients with mild to moderate renal impairment is the same as in patients with normal kidney function; ...

2. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/nda/2024/217785Orig1s000IntegratedR.pdf

217785Orig1s000 INTEGRATED REVIEW - accessdata.fda.gov Substantial evidence of effectiveness for resmetirom in patients with NASH was established using data ... renal impairment on the PK of REZDIFFRA.

3. rezdiffrahcp.com rezdiffrahcp.com/safety/

Clinical Safety Profile | Rezdiffra® (resmetirom) Rezdiffra has not been studied in patients with severe renal impairment. ... The safety and effectiveness have not been established in patients with cirrhosis.

4. cureus.com cureus.com/articles/390697-translating-trial-to-treatment-a-scoping-review-of-resmetirom-first-year-in-real-world-use-for-moderate-to-advanced-fibrosis-in-metabolic-dysfunction-associated-steatohepatitis.pdf

A Scoping Review of Resmetirom First Year in Real-World ... While beneficial, these are often insufficient for patients with moderate to advanced fibrosis (F2-F3), where the risk of disease progression is ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT06397872

NCT06397872 | A Study to Evaluate the Pharmacokinetics, ... Impaired renal function with estimated glomerular filtration rate (eGFR) < 30 mL/min/1.73 m2, determined using the Chronic Kidney Disease Epidemiology ...

6. accessdata.fda.gov accessdata.fda.gov/drugsatfda_docs/label/2024/217785s000lbl.pdf

full prescribing information for REZDIFFRA - accessdata.fda.gov The safety evaluation of REZDIFFRA also included an analysis of an additional randomized placebo- controlled safety trial which included 969 patients from a ...

7. rezdiffrahcp.com rezdiffrahcp.com/

Official HCP Site | Rezdiffra® (resmetirom) Renal Impairment. Rezdiffra has not been studied in patients with severe renal impairment. Hepatic Impairment. Avoid use in patients with decompensated ...