RecruitingRare disease

A Study Testing the Safety and Tolerability of ALE1 in Healthy Adults and Adults with Hypophosphatasia

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 study is looking at a condition called Hypophosphatasia, which is a rare inherited disorder that affects the development of bones and teeth. The treatment being tested is ALE1, which is given as a hard capsule taken by mouth. Some people in the study will receive ALE1 while others will receive placebo. The study will include both healthy adults and adults who have Hypophosphatasia.

The purpose of the study is to investigate the safety and how well people can tolerate ALE1 when it is given in increasing amounts. The study will also look at how the body processes the medicine by measuring how much of it is found in the blood over time and how it affects certain markers in the blood. Additionally, the study will examine whether taking the medicine with or without food makes any difference in how the body handles it.

The study is divided into two parts. The first part will involve giving single doses of the medicine to healthy adults, starting with lower amounts and gradually increasing them. The second part will involve giving multiple doses over a period of time to both healthy adults and adults with Hypophosphatasia. Throughout the study, participants will be closely monitored for any unwanted effects and will have blood samples taken to measure how the medicine works in the body. Different dose strengths of ALE1 will be tested to help identify which doses are suitable for further research.

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
Single dose administration period
During this period, you will receive a single dose of either ALE1 or placebo (an inactive substance that looks like the study medication but contains no active ingredient) by mouth.
The ALE1 medication comes in the form of hard capsules that are taken orally.
The dose you receive will be determined by the study protocol and may vary depending on which group you are assigned to.
In some cases, the medication may be given with or without food to assess how food affects the way your body processes the medication.
Step 2
Monitoring and safety assessments after single dose
After receiving the single dose, you will be monitored for any side effects or adverse reactions.
Blood samples will be collected at specified times to measure the levels of ALE1 in your bloodstream and to assess certain markers in your blood.
These measurements help determine how your body absorbs, distributes, and eliminates the medication.
The medical team will also evaluate your overall safety and tolerability of the medication during this period.
Step 3
Multiple dose administration period
Following the single dose period, you will enter a phase where you receive multiple doses of ALE1 or placebo over a period of time.
The medication will continue to be administered orally in the form of hard capsules.
The exact dosage, frequency, and duration of this treatment period will be determined by the study protocol and the dose level assigned to your group.
Throughout this period, the doses may be increased gradually to assess how your body responds to different amounts of the medication.
Step 4
Ongoing monitoring during multiple dose period
During the multiple dose period, you will continue to be monitored regularly for any side effects or changes in your condition.
Blood samples will be collected at regular intervals to measure medication levels and assess biological markers that indicate how the medication is working in your body.
The medical team will track any adverse events or reactions you may experience.
Your safety and how well you tolerate the medication will be continuously evaluated throughout this phase.
Step 5
Final assessments and follow-up
After completing the multiple dose period, you will undergo final assessments to evaluate the overall effects of the medication.
Additional blood samples may be collected to complete the analysis of how the medication worked in your body.
The medical team will review all safety data and any side effects you experienced during the study.
A follow-up period may be required to ensure your continued safety after the medication has been discontinued.

Who can join the trial?

4 criteria

You must be between 18 and 50 years old when you sign the consent form
You must be in good overall health based on a medical check-up
You should not have any ongoing medical conditions or important past health problems, as decided by the study doctor
You must have a confirmed change in the ALPL gene, which is the gene that causes Hypophosphatasia (a condition that affects bones and teeth)

Who cannot join the trial?

1 criterion

The exclusion criteria information is not available in the provided study data

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Verified sites

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Verified
University Medical Center Hamburg-Eppendorf
Hamburg, Germany

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Investigated drugs

ALE1
is an investigational medication being tested in this study. It is given by mouth (orally) to see if it is safe and well-tolerated in healthy adults and in adults with a condition called hypophosphatasia. The study will look at how the body processes this medication and how it affects the body after taking single doses and multiple doses over time.
Placebo
is an inactive substance that looks like the real medication but contains no active ingredients. It is used for comparison to help researchers understand if any effects seen in the study are due to the actual medication or other factors.

ALE1

What is already known about the treatment

ALE1 – ALE1 is an investigational oral medication currently being studied in early-phase clinical trials for the treatment of hypophosphatasia, a rare genetic disorder that affects bone and tooth development. The drug is administered by mouth and is being tested to understand how safe it is, how the body processes it, and whether it can help patients with this condition. As an experimental treatment, ALE1 is not yet approved for general medical use and is only available through controlled research studies to determine its potential benefits and risks.

Investigated diseases

Hypophosphatasia – Hypophosphatasia is a rare inherited metabolic disorder that affects the development of bones and teeth. The condition occurs when the body does not produce enough of an enzyme called alkaline phosphatase, which is essential for proper mineralization of bones and teeth. People with this disease may experience soft or weak bones that are prone to fractures, premature loss of baby teeth, and skeletal deformities. The severity of hypophosphatasia varies widely, ranging from mild forms that appear in adulthood to severe forms that begin before birth or in infancy. In adults, the disease may cause bone pain, repeated fractures, and dental problems. The condition progresses differently in each person depending on how much enzyme activity remains in their body.