RecruitingPHASE1, PHASE2INTERVENTIONAL

A First-in-human Study of KK8123 in Adults With X-linked Hypophosphatemia

This research is looking into a new medicine called KK8123 for people aged 18 to 65 who have X-linked Hypophosphatemia (XLH). This is the very first time this medicine is being tested in humans. The main goal is to find out if KK8123 is safe, what the right dose might be, and how it behaves in the body. Researchers will also be checking if it helps improve the condition. The study involves different stages, including an initial health check, a period where participants receive the medicine, and an optional longer follow-up period. It's an important step in developing new treatments for XLH, aiming to understand the medicine better before it can be studied in larger groups.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Kyowa Kirin Co., Ltd.

Enrolment target

Start

09 Oct 2024

Estimated completion

10 May 2028

X-linked Hypophosphatemia

What is this study about?

This clinical trial is an important step in understanding a new medicine called KK8123, which is being developed for people with a condition called X-linked Hypophosphatemia (often shortened to XLH). XLH is a rare genetic disorder that affects how the body handles phosphate, a mineral vital for strong bones and teeth. People with XLH often have low levels of phosphate in their blood, which can lead to weakened bones, pain, and other health issues.

The study is called a 'first-in-human' study, which means it's the very first time this particular medicine is being given to people. Because of this, it's divided into two main parts (Phase 1 and Phase 2). In these early phases, the primary focus is not on whether the medicine cures XLH, but on making sure it's safe for people to take. Researchers will carefully check for any side effects and work out the best dose to give.

By taking part, individuals are helping doctors and scientists learn more about XLH and explore potential new treatments. The information gathered from this study will be crucial for deciding whether KK8123 can be tested in larger groups of people later on, with the hope that it might eventually become a new way to help manage XLH.

Key takeaways

This is a 'first-in-human' study for a new XLH medicine, KK8123.
The primary goals are to check the medicine's safety, side effects, and how it acts in the body.
Participants must be adults (18-65) with XLH and good kidney function.
The study involves a screening period, a treatment period (32-44 weeks), and an optional extension.
Close medical monitoring will be provided throughout the study.
You have the right to withdraw from the study at any time.

Who may be eligible?

To be considered for this study, you need to be an adult between 18 and 65 years old. You must have a confirmed diagnosis of X-linked Hypophosphatemia (XLH) and blood tests showing low phosphate levels. Your kidney function also needs to be healthy enough to take part.

There are some other important requirements. You must weigh at least 6 stone 4 pounds (40 kg) and be able to give your informed consent. If you are female and could become pregnant, you'll need to have a negative pregnancy test and agree to use effective contraception during and for some time after the study. The same applies to male participants who could father a child. If you take pain medication, it needs to be at a stable dose. You also need to be willing to keep your diet and exercise routines consistent throughout the study.

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 between 18 and 65 years old?
Do you have a diagnosis of X-linked Hypophosphatemia (XLH)?
Are your blood phosphate levels currently low?
Do you weigh at least 6 stone 4 pounds (40 kg)?
Are you able to use contraception if you could become pregnant or father a child?
Are you willing to stick to your usual diet and exercise routines during the study?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the first step is a 'screening period' which can last up to 28 days. During this time, you'll have various checks and tests to make sure you meet all the requirements for the study. If you qualify, you'll enter the 'treatment period'. This part involves taking the study medicine, KK8123, and attending regular appointments so the research team can monitor your health, check for any side effects, and see how the medicine is working. This treatment and observation period will last between 32 and 44 weeks. After this, some participants might be offered the chance to continue in an 'extension period' for further monitoring if they wish. Throughout the study, you'll be giving blood samples and having other medical assessments.

Potential risks and benefits

Taking part in any clinical trial involves potential benefits and risks. For this study, potential benefits aren't certain, as the main goal is to understand the medicine's safety and how it works, rather than directly treating your XLH. However, you will receive close medical monitoring. Potential risks include side effects from the new medicine, which are currently unknown as this is the first time it will be given to humans. There may also be discomfort from blood tests and other procedures. It's very important to remember that you can choose to leave the study at any time, for any reason, without it affecting your usual medical care.

Locations (9)

University of California - San Francisco
Verified postcode
San Francisco, United States· Recruiting
Yale Center for XLH/ Yale University School of Medicine
Verified postcode
New Haven, United States· Recruiting
Indiana University School of Medicine University Hospital
Verified postcode
Indianapolis, United States· Recruiting
Mayo Clinic
Verified postcode
Rochester, United States· Not yet recruiting
Vanderbilt University Medical Center
Verified postcode
Nashville, United States· Recruiting
Hoptial Bictre
Verified postcode
Le Kremlin-Bicêtre, France· Recruiting
Institute of Osteology and Biomechanics (IOBM)
Verified postcode
Hamburg, Germany· Recruiting
Universitaetsklinikum Wurzburg
Verified postcode
Würzburg, Germany· Recruiting
Hospital Universitario La Paz
Verified postcode
Madrid, Spain· Recruiting

Common questions

What is X-linked Hypophosphatemia (XLH)?

XLH is a rare inherited condition that causes low levels of phosphate in the blood, leading to problems like soft bones, pain, and dental issues.

What is the purpose of KK8123?

KK8123 is a new medicine being developed to potentially help manage XLH. This study is the first time it's being tested in people to check its safety and how it works.

How long does the study last?

The initial screening period can be up to 28 days. If you qualify, the main treatment part of the study will last between 32 and 44 weeks, with an option to continue in an extension period.

Will I receive KK8123 if I join?

Yes, if you meet the study requirements and are enrolled, you will receive the study medicine, KK8123, as this is a dose-escalation study where participants receive the active drug.

What happens if I change my mind during the study?

You are free to leave the study at any time, for any reason. Your decision will not affect your ongoing medical care.

How to find out more

Kyowa Kirin

kkd.clintrial.82@kyowakirin.com 609-919-1100 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.