Ongoing Clinical Trials for Hereditary Hypophosphataemic Rickets
This article provides information about ongoing clinical trials for hereditary hypophosphataemic rickets, a rare genetic disorder affecting bone health due to low phosphate levels in the blood. Currently, there is 1 active clinical trial investigating a new treatment approach for adults with this condition. (Also known as: X-linked hypophosphatemia, XLH)
Clinical trial locations
- France
- Germany
- Spain
Study of KK8123 for Adults with X-linked Hypophosphatemia
This trial is investigating a new medication called KK8123 for adults living with X-linked hypophosphatemia. This rare genetic condition affects the bones and teeth because the kidneys cannot retain phosphate properly, leading to low phosphate levels in the blood. KK8123 is a protein-based medication given as an injection under the skin.
Main goal of the trial: The study aims to evaluate how safe KK8123 is and how well adult patients tolerate it. The trial is designed in two parts. In the first part, participants receive increasing doses of the medication to find the safest and most effective amount. The second part involves giving multiple doses over time to further assess safety and effects. Throughout the study, participants undergo regular health monitoring, including blood tests to measure phosphate levels and check for side effects.
Who can participate: This study is looking for adults between 18 and 65 years old who have been diagnosed with X-linked hypophosphatemia. Participants must have a fasting blood phosphate level below 2.5 mg/dL and good kidney function (eGFR of at least 60 mL/min). They must weigh at least 40 kg and have a corrected blood calcium level below 10.8 mg/dL. If taking long-term pain medications, participants must have been on a stable dose for at least 21 days before screening and be willing to maintain that dose. Participants must also agree to use reliable birth control if sexually active and able to have children, and maintain their usual diet and exercise habits during the study.
Who cannot participate: Individuals who do not have X-linked hypophosphatemia cannot join this trial. The study is restricted to adults within the specified age range of 18 to 65 years. Vulnerable populations, such as those unable to give informed consent, are not eligible to participate.
Investigational drug: KK8123 is administered through subcutaneous injection. It is currently in Phase 1/2 clinical trials and works by modulating pathways that influence how the body processes phosphate. The medication is being investigated specifically for its potential to treat metabolic bone disorders related to X-linked hypophosphatemia.
Summary
Currently, there is one active clinical trial for hereditary hypophosphataemic rickets, specifically targeting adults with X-linked hypophosphatemia. This trial is being conducted across three European countries: France, Germany, and Spain. The focus is on evaluating a novel treatment called KK8123, which represents a new approach to managing this rare genetic condition. The study is still in early phases, carefully assessing both safety and how well patients tolerate the medication before it can be considered for wider use. This represents an important step forward in research for this rare bone disorder, which has limited treatment options.