Condition (MeSH)
Hypophosphatasia
ClinicalTrials.gov lists 45 trials for Hypophosphatasia or a narrower condition beneath it. Alexion Pharmaceuticals, Inc. has the most among industry and government lead sponsors, with 23.
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Trials per phase (log scale)
1 more section with rows
45 of the 45 trials are tagged with Hypophosphatasia itself; the rest are tagged with a narrower condition beneath it.
When did Hypophosphatasia trials start? (19)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Phase 4
- Not applicable
- Observational
Top of scale: 5
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 2 |
| 2025 | 2 |
| 2024 | 4 |
| 2023 | 3 |
| 2022 | 1 |
| 2021 | 4 |
| 2020 | 1 |
| 2019 | 2 |
| 2018 | 2 |
| 2017 | 1 |
Showing 10 of 19 earlier years.
No forecast is made for this condition: it averages 2.4 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: 1 trial still running at the 2026-10-09 snapshot, fewer than the 5 needed for a median. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Hypophosphatasia trials in? (9)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 2 |
| Phase 1/2 | 3 |
| Phase 2 | 8 |
| Phase 2/3 | 1 |
| Phase 3 | 3 |
| Phase 4 | 3 |
| Not applicable | 1 |
| Observational | 23 |
| Expanded access | 1 |
How many Hypophosphatasia trials are recruiting? (8)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Not yet recruiting | 1 |
| Recruiting | 7 |
| Enrolling by invitation | 1 |
| Active, not recruiting | 4 |
| Completed | 24 |
Showing 5 of 8 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Hypophosphatasia trials? (6)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Alexion Pharmaceuticals, Inc. | 23 |
| AM-Pharma | 1 |
| AgNovos Healthcare, LLC | 1 |
| Alesta Therapeutics | 1 |
| AstraZeneca | 1 |
Showing 5 of 6 sponsors.
Which drugs are being tested for Hypophosphatasia? (6)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| Asfotase Alfa | 16 |
| ALXN1850 | 4 |
| RITUXIMAB | 1 |
| Folic (or folinic) acid | 1 |
| Ilofotase alfa | 1 |
Showing 5 of 6 drugs.
Interventions as named in the registry (8)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Biochemical analysis of different bone markers. | 1 |
| data collection | 1 |
| No Intervention -- Observational study | 1 |
| ALE1 | 1 |
| Bortezomib SC | 1 |
Showing 5 of 8 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Hypophosphatasia trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Craniosynostosis | 20 | 3 |
| Pneumonia | 13 | 3 |
| Convulsion | 7 | 3 |
| Pyrexia | 6 | 1 |
| Intracranial pressure increased | 5 | 3 |
Showing 5 of 10 adverse events.
Which publications cover Hypophosphatasia? (33)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
| Publication | Journal | Year | Major topic |
|---|---|---|---|
| Circulating Micro-RNAs in Patients With Hypophosphatasia: Results of the First Micro-RNA Analysis in HPP. | The Journal of clinical endocrinology and metabolism | 2025 | Yes |
| Disease burden by <i>ALPL</i> variant number in patients with non-life-threatening hypophosphatasia in the Global HPP Registry. | Journal of medical genetics | 2025 | Yes |
| Clinical characterization and impact of hypophosphatasia in Spain: An observational analysis of the Spanish cohort included in the Global HPP Registry. | Medicina clinica | 2025 | Yes |
| The Global Hypophosphatasia Registry: lessons learned from a decade of real-world data. | Orphanet journal of rare diseases | 2025 | Yes |
| Clinical Profiles of Children with Hypophosphatasia prior to Treatment with Enzyme Replacement Therapy: An Observational Analysis from the Global HPP Registry. | Hormone research in paediatrics | 2024 | Yes |
Showing 5 of 33 publications.
Prevalence (6)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Hypophosphatasia | Point prevalence | Unknown | Worldwide | Not stated | Exact |
| Hypophosphatasia | Point prevalence | Unknown | Worldwide | Not stated | Exact |
| Hypophosphatasia | Point prevalence | Unknown | Worldwide | Not stated | Unspecified |
| Hypophosphatasia | Point prevalence | <1 / 1 000 000 | China | Not stated | Exact |
| Hypophosphatasia | Point prevalence | <1 / 1 000 000 | China | Not stated | Unspecified |
Showing 5 of 6 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Hypophosphatasia | 436 | Exact | Orphadata Science knowledge files, Product 1 |
| Hypophosphatasia | 436 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.