New pill could ease rare bone disease

NCT ID NCT07179640

First seen Jun 27, 2026 · Last updated Jun 27, 2026

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for HYPOPHOSPHATASIA (HPP) are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

hypophosphatasia

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

    Contact Phone: •••-•••-••••

  • New Zealand Clinical Research

    RECRUITING

    Grafton, Auckland, 1010, New Zealand

    Contact Phone: •••-•••-•••• Email: •••••@•••••