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New hope for kids with rare bone disease: experimental drug enters final testing

NCT ID NCT06079359

Disease control Sponsor: Alexion Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Summary

This study tests a new drug called ALXN1850 in children aged 2 to 12 with hypophosphatasia, a rare genetic condition that weakens bones. The trial compares the drug to a placebo to see if it improves bone health and movement. About 30 children who have not received prior treatment for this condition will take part.

What this could mean

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

Active substance
ALXN1850
What this could lead to
If it works, this could provide a new treatment option for children with hypophosphatasia, improving bone strength and physical function.
What could go wrong
This is an early-stage Phase 3 trial with only 30 participants, so results may not apply to all patients. The drug may not work better than placebo or could cause side effects.

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

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

30 people

The number who actually took part.

Started

May 2024

Expected to finish

Aug 2028

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

2 to 11 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Diagnosis of HPP documented in the medical records, and the following criteria fulfilled without other probable cause than HPP: 1. Presence of HPP-related rickets on skeletal X-rays during the Screening Period, with a minimum Rickets Severity Score (RSS) of 1.0 AND 2. Serum ALP activity below the age- and sex-adjusted normal range during the Screening Period as measured by the Central Laboratory OR 2 documented serum ALP activity results, at least 15 days apart, below the age- and sex-adjusted local laboratory normal range during the 24 months before the Day 1 Visit. Note: Local laboratories need to be Clinical Laboratory Improvement Amendments (CLIA) or ISO 15189 certified, or have other local equivalent laboratory certification with Alexion's approval. * Must meet 1 of the following criteria: 1. Documented ALPL gene variant (pathogenic, likely pathogenic, or variant of unknown significance) from a CLIA certified laboratory (Section 8.7) 2. Plasma PLP above the upper limit of normal (ULN) during the Screening Period (central or local laboratory results allowed per local regulations) * Tanner stage 2 or less during the Screening Period Exclusion Criteria: * History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrinological, hematological, neurological disorders, or any other disorders that are capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study intervention; or interfering with the interpretation of data as determined by the Investigator * Diagnosis of primary or secondary hyperparathyroidism * Hypoparathyroidism, unless secondary to HPP * Any new fracture within 12 weeks before Day 1 (excluding pseudofractures) * Planned surgical intervention which may impact the results of study assessments (in the opinion of the Investigator) during the Randomized Evaluation Period * History of allergy or hypersensitivity to any ingredient contained in ALXN1850 or the placebo comparator

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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.

Hypophosphatasia

Contacts and locations

Locations

  • Research Site

    Baltimore, Maryland, 21287, United States

  • Research Site

    Kansas City, Missouri, 64108, United States

  • Research Site

    Durham, North Carolina, 27705, United States

  • Research Site

    Nedlands, 6009, Australia

  • Research Site

    Parkville, 3052, Australia

  • Research Site

    Brussels, 1020, Belgium

  • Research Site

    Brasília, 71625-009, Brazil

  • Research Site

    Porto Alegre, 90610-261, Brazil

  • Research Site

    Recife, 50740-465, Brazil

  • Research Site

    Salvador, 40050-410, Brazil

  • Research Site

    São Paulo, 01409-902, Brazil

  • Research Site

    São Paulo, 05403-900, Brazil

  • Research Site

    Calgary, Alberta, T2E 7H7, Canada

  • Research Site

    Winnepeg, Manitoba, R3E 3P4, Canada

  • Research Site

    Beijing, 100045, China

  • Research Site

    Guangzhou, 510623, China

  • Research Site

    Shanghai, 2000127, China

  • Research Site

    Shenzhen, 518053, China

  • Research Site

    Helsinki, 00290, Finland

  • Research Site

    Ashkelon, 7830604, Israel

  • Research Site

    Chihuahua City, 31238, Mexico

  • Research Site

    Lodz, 93-338, Poland

  • Research Site

    Bucharest, 011863, Romania

  • Research Site

    Madrid, 28046, Spain

  • Research Site

    Vitoria-Gasteiz, 01009, Spain

  • Research Site

    Stockholm, 17176, Sweden

  • Research Site

    Taipei, 100, Taiwan

  • Research Site

    Ankara, 06560, Turkey (Türkiye)

  • Research Site

    Bursa, 16059, Turkey (Türkiye)

  • Research Site

    Erzurum, 25240, Turkey (Türkiye)

  • Research Site

    Istanbul, 34899, Turkey (Türkiye)

  • Research Site

    Birmingham, B4 6NH, United Kingdom

More trials for these conditions

Other studies related to the condition(s) this trial covers.