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Could your body fight back against this rare disease drug?

NCT ID NCT05234567

Knowledge-focused 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 study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 follows 30 people with hypophosphatasia (HPP) who are taking the drug asfotase alfa. Researchers want to see if the immune system can make the drug less effective or cause serious allergic reactions. Participants will be monitored for at least 5 years to track these risks.

What this could mean

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

Active substance
asfotase alfa (Strensiq)
What this could lead to
If successful, this study could help doctors understand when and why the drug stops working, leading to better monitoring or alternative treatments for hypophosphatasia.
What could go wrong
This is a small observational study, not a treatment trial. It only watches what happens and cannot prove whether the drug works or not. Results may not apply to all patients.

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.

Participants

About 30 people

The number the study aims to enrol. It can still change while the study runs.

Started

Aug 2022

Expected to finish

Jul 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.

Who is studied

Patients with HPP

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

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: * Any age or sex with a confirmed diagnosis of pediatric-onset HPP (that is, first HPP sign or symptom presented at \< 18 years of age). * Currently receiving asfotase alfa treatment at Enrollment (not treatment-naïve) or the Physician has decided to resume (not treatment-naïve) or start (treatment-naïve) the participant's asfotase alfa treatment within 6 months after Enrollment. * Participant must have documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, and a documented ALPL gene mutation (Note: An exception is made for infants with clinical features of HPP plus low ALP who need to start asfotase alfa treatment right away, at the Physician's discretion, but do not yet have a genetic result. In this case, ALPL gene documentation is not required at the time of sub-study enrollment but should be documented within 6 months after Enrollment). * Participant or participant's parent/legally authorized representative is able to read and/or understand the informed consent and study questionnaires in the local language. * Participant or participant's parent/legally authorized representative must be willing and able to give signed informed consent for this sub-study, and the participant must be willing to give written informed assent, if appropriate and required by local regulations. Exclusion Criteria: * Currently participating in an Alexion-sponsored interventional clinical study. Participants who have concluded participation in an Alexion-sponsored asfotase alfa clinical study are eligible to enroll in this sub-study.

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    12 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Clinical Trial Site

    RECRUITING

    Hartford, Connecticut, 06106, United States

  • Clinical Trial Site

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Clinical Trial Site

    RECRUITING

    Boston, Massachusetts, 02122, United States

  • Clinical Trial Site

    RECRUITING

    Kansas City, Missouri, 64108, United States

  • Clinical Trial Site

    RECRUITING

    Mineola, New York, 11501, United States

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Cincinnati, Ohio, 45229, United States

  • Clinical Trial Site

    RECRUITING

    Columbus, Ohio, 43203, United States

  • Clinical Trial Site

    NOT_YET_RECRUITING

    Pittsburgh, Pennsylvania, 15224, United States

  • Clinical Trial Site

    RECRUITING

    Nashville, Tennessee, 37112, United States

  • Clinical Trial Site

    RECRUITING

    Salt Lake City, Utah, 84108, United States

  • Clinical Trial Site

    RECRUITING

    Charlottesville, Virginia, 22903, United States

  • Clinical Trial Site

    RECRUITING

    Madison, Wisconsin, 53792, United States

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