RecruitingRare disease

Study of KK8123 for Adults with X-linked Hypophosphatemia

Verified siteInvestigationalNo placebo

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying a condition called X-linked hypophosphatemia, a rare genetic disorder that affects the bones and teeth due to low levels of phosphate in the blood. The study will investigate a new treatment called KK8123, which is a novel protein-based medication designed to be administered as a solution for injection under the skin.

The purpose of the study is to assess the safety and tolerability of KK8123 in adult patients with X-linked hypophosphatemia. The study will be conducted in two parts. In the first part, participants will receive increasing doses of KK8123 to determine the safest and most effective dose. In the second part, participants will receive multiple doses to further evaluate the safety and effects of the treatment over time.

Throughout the study, participants will have regular check-ups to monitor their health, including blood tests and other assessments to measure the levels of phosphate in their blood and to check for any side effects. Some participants may receive a placebo, which is a substance with no active medication, to compare the effects of KK8123. The study aims to provide valuable information about the potential benefits and risks of this new treatment for people with X-linked hypophosphatemia.

The research process

The trial runs in 5 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial assessment
Upon joining the study, an initial assessment is conducted to confirm eligibility. This includes a review of medical history and current health status.
Blood tests are performed to measure fasting serum phosphorus and other relevant parameters.
Step 2
Part 1: dose-escalation
The first phase involves receiving the medication KK8123 through a subcutaneous injection. The dosage may be adjusted to determine the most effective and safe amount.
Regular monitoring of health status, including laboratory tests and vital signs, is conducted to assess safety and tolerability.
Step 3
Part 2: safety extension
In the second phase, multiple doses of KK8123 are administered over a period of time to further evaluate safety.
Participants continue to receive subcutaneous injections and undergo regular health assessments, including blood tests to monitor serum phosphorus levels.
Step 4
Ongoing monitoring
Throughout the study, regular check-ups are scheduled to monitor for any side effects and to ensure the medication is working as intended.
Participants are required to maintain a stable regimen of any chronic pain medications and adhere to the study visit schedule.
Step 5
Completion of study
Upon completion of the study, a final assessment is conducted to evaluate overall health and the effects of the medication.
Participants may be asked to provide feedback on their experience and any changes in symptoms.

Who can join the trial?

18 criteria

Must be a male or female aged between 18 to 65 years at the time of signing the consent form.
If taking long-term pain medications, including strong painkillers like opioids, must have been on a stable dose for at least 21 days before the screening visit and be willing to keep the dose the same.
Must be willing to use a reliable method of birth control while in the study and for 5 months after the last dose if sexually active and able to have children.
Must be willing and able to complete all parts of the study, follow the visit schedule, and comply with the assessments, as judged by the study doctor.
Must have been diagnosed with **X-linked hypophosphatemia (XLH)**, a condition affecting bone health, as confirmed by the study doctor.
Must have a fasting blood phosphorus level of less than 2.5 mg/dL at the screening.
Must have a kidney function test result, called **renal TmP/GFR**, of less than 2.5 mg/dL at the screening.
Must have an **eGFR** (a measure of kidney function) of at least 60 mL/min at the screening.
Must have a corrected blood calcium level of less than 10.8 mg/dL at the screening.
Must weigh at least 40 kg (about 88 pounds).
Must provide a signed consent form after the study details have been explained and before any study-related procedures begin.
Must agree not to change diet and exercise habits from one week before dosing until the end of the study.
Female participants must have a negative pregnancy test at the screening and be willing to have more pregnancy tests during the study.
For Part 2 of the study, must have completed the relevant part in Part 1 of the study.
For Part 2, must provide a signed consent form after the details of Part 2 have been explained.
For Part 2, must have a negative pregnancy test at the start of Part 2 and be willing to have more tests until the study ends.
For Part 2, if taking long-term pain medications, must be on a stable dose and willing to keep the dose the same throughout the study.
For Part 2, must be willing and able to complete all parts of the study, follow the visit schedule, and comply with all assessments, as judged by the study doctor.

Who cannot join the trial?

4 criteria

Individuals who do not have X-linked hypophosphatemia cannot participate. This is a genetic condition that affects the bones.
Participants must be within the specified age range, which is typically determined by the study. If you are outside this age range, you cannot join.
Both males and females are eligible, but if you do not identify as either, you may not be able to participate.
If you are considered part of a vulnerable population, such as children or those unable to give consent, you may not be eligible.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Bicetre Hospital
Verified
Le Kremlin-Bicetre, France
Fundacion Para La Investigacion Biomedica Del Hospital Universitario La Paz
Responsive
Madrid, Spain
Universitaetsklinikum Wuerzburg AöR
Verified
Wuerzburg, Germany

Trial locations

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Investigated drugs

KK8123 is a medication being studied for its safety and how well it is tolerated in adult patients with X-linked Hypophosphatemia. This trial aims to understand how the body processes the medication when given under the skin. The study is conducted in two parts: the first part focuses on determining the safety and tolerability of the medication, while the second part evaluates these aspects after multiple doses.

Kk8123

What is already known about the treatment

KK8123 – This medication is administered through subcutaneous injection, meaning it is injected under the skin. Currently, KK8123 is in the clinical trial phase, specifically a Phase 1/2 study, which aims to assess its safety and tolerability in patients. It is being investigated for the treatment of X-linked hypophosphatemia, a rare genetic disorder affecting bone mineralization. The mechanism of action at the molecular level involves modulating pathways that influence phosphate metabolism, although specific details are still under study. KK8123 is classified pharmacologically as a treatment for metabolic bone disorders.

Investigated diseases

X-linked hypophosphatemia – This is a genetic disorder characterized by low levels of phosphate in the blood due to the kidneys' inability to retain phosphate. It is caused by mutations in the PHEX gene, which is located on the X chromosome. The condition often leads to bone pain, deformities, and growth abnormalities, particularly in children. As the disease progresses, individuals may experience dental issues and joint pain. The severity of symptoms can vary widely among those affected. It is a rare condition, primarily affecting males, but females can also exhibit symptoms due to the genetic nature of the disorder.