New pill could ease rare bone disease

NCT ID NCT07179640

First seen Jun 27, 2026 · Last updated Aug 04, 2026 · Updated 2 times

Summary

This early-stage trial tests an oral drug called ALE1 for hypophosphatasia (HPP), a rare genetic bone disease. The study will first check safety and how the drug moves through the body in healthy volunteers, then in adult HPP patients. It is a small, placebo-controlled study to find the right dose and see if the drug is safe enough to test further.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
ALE1 (an oral drug)
What this could lead to
If successful, this could point toward a new oral treatment option for hypophosphatasia, potentially reducing the need for enzyme replacement therapy.
What could go wrong
This is a very early Phase 1/2a trial with only 120 participants, focused on safety and dosing. It is too soon to know if ALE1 will be effective or safe long-term.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Fortrea Clinical Research Unit

    RECRUITING

    Leeds, United Kingdom

  • New Zealand Clinical Research

    RECRUITING

    Grafton, Auckland, 1010, New Zealand

  • Universitätsklinikum Würzburg

    RECRUITING

    Würzburg, 97074, Germany

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