Disease · MONDO_0018570
hypophosphatasia
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. hypophosphatasia. Diseases, record MONDO_0018570. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0018570
On this page
Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Other names
- HPP, Rathburn disease, deficiency of alkaline phosphatase (disorder) [ambiguous], phosphoethanolaminuria
- Open Targets
- Highest stage of its drugs
- Approval, for 4 of its 11 drugs, from ClinicalTrials.gov, DailyMed, EMA Human Drugs, PMDA and TTD
- Open Targets
Drugs11
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| ASFOTASE ALFA | CHEMBL2108311 | Enzyme | Approval | ClinicalTrials.gov, DailyMed, EMA Human Drugs and PMDA | 17 | 14 | 14 | Drugs and targets |
| CALCIUM ACETATE | CHEMBL1200800 | Small molecule | Approval | TTD | 1 | 0 | 0 | Drugs and targets |
| LANTHANUM CARBONATE | CHEMBL2096647 | Small molecule | Approval | TTD | 1 | 0 | 0 | Drugs and targets |
| SEVELAMER HYDROCHLORIDE | CHEMBL1201492 | Small molecule | Approval | TTD | 1 | 0 | 0 | Drugs and targets |
| EFZIMFOTASE ALFA | CHEMBL5314736 | Enzyme | Phase 3 | ClinicalTrials.gov | 6 | 4 | 4 | Drugs and targets |
| BIXALOMER | CHEMBL4297202 | Small molecule | Phase 3 | TTD | 1 | 0 | 0 | Drugs and targets |
| BUROSUMAB | CHEMBL3707326 | Antibody | Phase 3 | TTD | 1 | 0 | 0 | Drugs and targets |
| SBR-759 | CHEMBL5095380 | Phase 3 | TTD | 1 | 0 | 0 | Drugs and targets | |
| SETRUSUMAB | CHEMBL4297918 | Antibody | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ILOFOTASE ALFA | CHEMBL4594490 | Enzyme | Phase 1/2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
1 to 10 of 11
See all 11 drugsTargets2
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| FGF23 | Fibroblast growth factor 23 | 1 | Fibroblast growth factor 23 inhibitor | Phase 3 | https://conductscience.org/library/genes/8074 | Drugs and targets |
| SOST | Sclerostin | 1 | Sclerostin inhibitor | Phase 2 | https://conductscience.org/library/genes/50964 | Drugs and targets |
Trials20
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT01176266 | Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP) | Phase 2/3 | 69 | 2018-02-26 | Hypophosphatasia | ASFOTASE ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT02797821 | Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP) | Phase 2 | 27 | 2018-07-10 | Hypophosphatasia | ASFOTASE ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT01163149 | Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP) | Phase 2 | 19 | 2017-09-18 | Hypophosphatasia | ASFOTASE ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT04980248 | Study of ALXN1850 in Participants With Hypophosphatasia (HPP) | Phase 1 | 15 | 2024-12-27 | Hypophosphatasia | EFZIMFOTASE ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT00952484 | Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP) | Phase 2 | 13 | 2011-07-26 | Hypophosphatasia (HPP) | ASFOTASE ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT02531867 | Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan | Phase 4 | 13 | 2017-05-08 | Hypophosphatasia | ASFOTASE ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT01203826 | Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP) | Phase 2 | 12 | 2017-07-26 | Hypophosphatasia (HPP) | ASFOTASE ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT05890794 | Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia | Phase 1/2 | 12 | 2025-02-27 | Hypophosphatasia | ILOFOTASE ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT00744042 | Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP) | Phase 1/2 | 11 | 2011-09-30 | Hypophosphatasia (HPP) | ASFOTASE ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT01205152 | Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP) | Phase 2 | 10 | 2017-11-17 | Hypophosphatasia | ASFOTASE ALFA | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 20
See all 20 trialsComparisons in its trials1
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial |
|---|---|---|---|---|
| Asfotase Alfa 2 mg/kg vs Asfotase Alfa 3 mg/kg: serious adverse events | 0 of 6 | 0 of 7 | 13 | NCT00952484 |
Source Trial comparisons
Papers from its trials13
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
| PMID | Listed as | Trials listing it | Source |
|---|---|---|---|
| 41378916 | DERIVED | 1 | PubMed |
| 39135540 | DERIVED | 1 | PubMed |
| 33822385 | DERIVED | 1 | PubMed |
| 32987199 | DERIVED | 1 | PubMed |
| 32762706 | DERIVED | 3 | PubMed |
| 32417537 | DERIVED | 3 | PubMed |
| 30969260 | DERIVED | 1 | PubMed |
| 30811537 | DERIVED | 1 | PubMed |
| 30558909 | DERIVED | 1 | PubMed |
| 28436937 | DERIVED | 1 | PubMed |
1 to 10 of 13
See all 13 papers from its trialsData dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0018570
GET https://conductscience.org/api/diseases/v1/MONDO_0018570/drugs