Withdrawn study aimed to counteract antibodies blocking hypophosphatasia drug
NCT ID NCT06015750
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 However, the trial was withdrawn before any participants were enrolled.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- The trial was withdrawn before enrolling any participants, so no data were collected.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.
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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
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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 Hide 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
The condition terms exactly as the trial's registrant entered them.
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