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Withdrawn study aimed to counteract antibodies blocking hypophosphatasia drug

NCT ID NCT06015750

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
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 Results may not be published yet.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 This study
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This status has not been confirmed recently, so it may be out of date.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 planned to test a combination of immunosuppressive drugs (methotrexate, rituximab, bortezomib, IVIg, and folic acid) in people with hypophosphatasia whose bodies had stopped responding to asfotase alfa due to antibodies. The goal was to see if suppressing the immune system could restore the drug's effectiveness. However, the trial was withdrawn before any participants were enrolled.

What this could mean

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

Active substance
methotrexate, rituximab, bortezomib, IVIg, folic acid
What this could lead to
If successful, this approach could help restore the effectiveness of asfotase alfa in patients whose bodies have developed antibodies against it.
What could go wrong
The trial was withdrawn before enrolling any participants, so no data were collected. Immunosuppressive therapies carry risks of infection and other 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 4

Runs after approval, following long-term safety and how well the treatment works in everyday use.

Expected to start

Jul 2026

An estimate. Start dates often move.

Expected to finish

Mar 2030

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 18 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: * Reoccurrence or worsening of rickets for at least the past 3 months in participants who showed an initial efficacy response to asfotase alfa after at least 6 months of continuous treatment and currently receiving asfotase alfa. RSS will be used to determine severity at Baseline. * Presence of ADAs, with or without NAbs, irrespective of their titers. * Confirmation by the TMB that both the clinical evidence and immunogenicity-mediated association noted above are present. * Female participants of childbearing potential and male participants with partners of childbearing potential must follow protocol-specified contraception guidance as described in Section 10.5. * Participant, or participant's legal guardian, is capable of signing informed consent or assent as described in Section 10.1.3, which includes compliance with the requirements and restrictions listed in the informed consent or assent form and in this protocol. Exclusion Criteria: * Known history of human immunodeficiency virus (HIV) infection (evidenced by HIV type 1 or type 2 \[HIV 1, HIV 2\] antibody) or hepatitis B or C viral infection. * Known or suspected history of drug or alcohol abuse or dependence within 1 year prior to Screening. * Inability of the participant, or the participant's legal guardian, to provide informed consent. * Pregnant, breastfeeding, or intending to conceive during the course of the study. * Inability to travel to the clinic for specified visits during the Treatment Period caused by disease per se or logistics (does not apply to external travel restrictions). * The participant is at risk of reactivation or has an active significant viral infection such as hepatitis B, cytomegalovirus, herpes simplex, human polyomavirus (also known as John Cunningham \[JC\] virus), parvovirus, or Epstein Barr virus. * The participant is at risk of reactivation of tuberculosis or has regular contact (eg, in the household) with individuals who are being actively treated for tuberculosis. * The participant has had or is required to have any live vaccination within 1 month prior to enrollment.

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Conditions

The condition(s) this trial relates to.

As listed by the trial registrant

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