Ongoing, recruitingPhase I and Phase II (Integrated)- OtherInterventional

A Phase 1/2a Dose-Escalating Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of ARO-C3 in Adult Healthy Volunteers and in Adult Patients With Complement-Mediated Renal Disease

This research study is looking at a new medication called ARO-C3. It's designed for adult patients who have a specific kidney disease where their immune system, called the 'complement system,' isn't working properly. The main goals of this study are to check if ARO-C3 is safe to use and how well people can tolerate it (meaning, if it causes any uncomfortable side effects). Researchers will also be looking at how the body processes the medication and if it has any helpful effects on the kidney condition. This is an early-stage study, starting with healthy volunteers and then moving on to patients, to carefully learn more about this new treatment.

At a glance

Status

Ongoing, recruiting

Phase

Phase I and Phase II (Integrated)- Other

Sponsor

Arrowhead Pharmaceuticals Inc.

Enrolment target

Start

07 Dec 2023

complement-mediated renal disease

What is this study about?

This study is about a new medicine, ARO-C3, for adults who have a kidney problem where a part of their immune system, called the 'complement system,' isn't working as it should. This can cause damage to the kidneys. The main purpose of this research is to find out if ARO-C3 is safe for people to take and if they experience any side effects.

Researchers will also be looking at what happens to the medicine once it's in the body – how it's absorbed, distributed, used up, and eventually leaves the body. This helps them understand the best way to give the medicine. Another important part of the study is to see if ARO-C3 affects the misplaced activity of the complement system, which could potentially help improve the kidney condition.

This study starts with a small group of healthy volunteers to make sure the medicine is safe in people who don't have the kidney condition. Then, it will gradually involve adult patients with the kidney condition. By doing this carefully, researchers can learn as much as possible about ARO-C3 and decide if it's a promising treatment for this type of kidney disease.

Key takeaways

This study is testing a new medicine called ARO-C3 for a specific kidney disease.
The main aim is to check if ARO-C3 is safe and well-tolerated.
Researchers will also see how the body handles the medicine and if it helps the kidneys.
It's an early-stage study, starting with healthy volunteers and then patients.
Participants must be 18 years or older, both men and women.
Close monitoring for side effects is a key part of participating.

Who may be eligible?

This study is open to adults aged 18 and over. There is no upper age limit, meaning older adults can also take part. Both men and women are welcome to participate in this study.

Initially, the study will involve healthy people. After checking for safety, it will gradually include adult patients who have been diagnosed with a specific type of kidney disease where their immune system, called the 'complement system,' is causing the problem.

Specific medical checks will be done to make sure you are suitable for the study. For example, your overall health, kidney function, and other conditions will be reviewed by the study doctors to determine if you can safely take part.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you 18 years old or older?
Do you have a kidney disease caused by your immune system's 'complement system'?
Are you able to attend regular clinic appointments for checks and blood tests?
Are you willing to potentially receive a new, untested medicine?
Are you generally in good health apart from the kidney condition (if a patient)?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you would receive the new medicine, ARO-C3. This would involve regular visits to the study clinic for assessments. During these visits, doctors and nurses would check your health, ask about any side effects you might be experiencing, and take blood or urine samples. These samples help them understand how your body is reacting to the medicine, how much medicine is in your system, and if it's having any effect on your kidney condition. The total length of your participation, including the period you take the medication and follow-up checks after you stop, will be clearly explained by the study team. You would also have regular contact with the study team to monitor your well-being.

Potential risks and benefits

Taking part in this study means you could be among the first to try a new medicine that might help with your kidney condition. However, because this is a new medicine, we don't know all its effects yet. There might be side effects, some of which could be unexpected or serious. The study team will monitor you very closely for any issues. You are free to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (1)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

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Unverified
Germany

Common questions

What is 'complement-mediated renal disease'?

It's a kidney disease where a part of your immune system, called the 'complement system,' becomes overactive and mistakenly attacks and damages your kidneys.

What is ARO-C3?

ARO-C3 is a new medicine being tested. It's designed to calm down the overactive complement system that causes kidney damage in this particular disease.

What does 'Phase 1/2a' mean?

This means it's an early-stage study. Phase 1 focuses heavily on safety in healthy people, and Phase 2a starts to look at safety and early signs of effectiveness in patients with the condition.

Will I definitely get the new medicine?

Yes, if you meet all the study requirements, you will receive ARO-C3. This study isn't comparing it to a dummy treatment (placebo).

How long will I be in the study?

The study team will tell you the exact duration, including how long you'd take the medicine and how long you'd have follow-up visits after stopping treatment.