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

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What is this study about?

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.

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.

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.

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.

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.

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 Study?

General Requirements:

You must be between 18 and 50 years old when you sign the consent form

For Part 1 of the study (Healthy Participants):

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

For Part 2 of the study (Patients with Hypophosphatasia):

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 Study?

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

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country	Status
Koenig-Ludwig-Haus Orthopaedische Klinik	Wuerzburg	Germany
Udvymynleb Mnzvwbq Csvilf Hnmdjrdmizzlgrefr	Hamburg	Germany

Trial status

Country	Status	Recruitment Start
Germany	Not yet recruiting	01.12.2025

Trial locations

Investigated drugs:

ALE1

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.

Investigated diseases:

Hypophosphatasia

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.

Trial ID:

2025-522378-36-00

Protocol code:

ALE1-101

NCT ID:

NCT07179640

Trial Phase:

Phase I and Phase II (Integrated) – First administration to humans

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?