Disease · MONDO_0010619
X-linked dominant hypophosphatemic rickets
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. X-linked dominant hypophosphatemic rickets. Diseases, record MONDO_0010619. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010619
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
X-linked hereditary hypophosphatemic rickets, X-linked hypophosphatemia, X-linked hypophosphatemic rickets, XLH, hereditary hypophosphatemic rickets, X-linked, hypophosphatemic rickets, X-linked, hypophosphatemic rickets, X-linked dominant, X-linked dominant, rickets, vitamin D-resistant, vitamin D-resistant rickets, X-linked
- Open Targets
Drugs4
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 |
|---|---|---|---|---|---|---|---|---|
| BUROSUMAB | CHEMBL3707326 | Antibody | Approval | EMA Human Drugs | 16 | 15 | 15 | Drugs and targets |
| CALCITRIOL | CHEMBL846 | Small molecule | Early phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| PHOSPHORIC ACID | CHEMBL1187 | Small molecule | Early phase 1 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| CALCITONIN | CHEMBL4594242 | Protein | Unknown | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
Targets3
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 |
|---|---|---|---|---|---|---|
| CALCR | Calcitonin receptor | 1 | Calcitonin receptor agonist | Unknown | https://conductscience.org/library/genes/799 | Drugs and targets |
| FGF23 | Fibroblast growth factor 23 | 1 | Fibroblast growth factor 23 inhibitor | Approval | https://conductscience.org/library/genes/8074 | Drugs and targets |
| VDR | Vitamin D3 receptor | 1 | Vitamin D receptor agonist | Early phase 1 | https://conductscience.org/library/genes/7421 | Drugs and targets |
Trials17
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. 1 more is not shown, since the checks do not confirm it.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT02526160 | Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH) | Phase 3 | 134 | 2021-02-05 | X-linked Hypophosphatemia | BUROSUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT02915705 | Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With XLH | Phase 3 | 61 | 2019-04-11 | X-Linked Hypophosphatemia | BUROSUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT02163577 | Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH) | Phase 2 | 52 | 2019-05-22 | X-linked Hypophosphatemia | BUROSUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT01340482 | A Repeated Study of KRN23 in Adults With X-Linked Hypophosphatemia | Phase 1/2 | 29 | 2020-10-26 | X-linked Hypophosphatemia | BUROSUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT01571596 | An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia | Phase 1/2 | 23 | 2021-02-17 | X-linked Hypophosphatemia | BUROSUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT01652573 | Calcitonin for Treating X-linked Hypophosphatemia | Not applicable | 21 | 2017-05-31 | Hypophosphatemic Rickets, X Linked Dominant | CALCITONIN | Its record names the disease | ClinicalTrials.gov |
| NCT02312687 | Long-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH) | Phase 2 | 20 | 2019-12-16 | X-Linked Hypophosphatemia | BUROSUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT02537431 | Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH) | Phase 3 | 14 | 2018-09-25 | X-linked Hypophosphatemia | BUROSUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT02750618 | Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH) | Phase 2 | 13 | 2018-06-19 | X-Linked Hypophosphatemia | BUROSUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT04146935 | Examining the Effect of Burosumab on Muscle Function | Phase 4 | 10 | 2023-08-24 | X-linked Hypophosphatemia | BUROSUMAB | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 17
See all 17 trialsComparisons in its trials16
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 | Source |
|---|---|---|---|---|---|
| Burosumab Q2W vs Burosumab Q4W Then Q2W: deaths from any cause | 0 of 26 | 0 of 26 | 52 | NCT02163577 | Trial comparisons |
| Burosumab Q2W vs Burosumab Q4W Then Q2W: serious adverse events | 0 of 26 | 1 of 26 | 52 | NCT02163577 | Trial comparisons |
| Nasal Calictonin vs Saline Nasal Spray: Number of Participants With Nasal Ulceration at 1 Month | 0 of 10 | 2 of 11 | 21 | NCT01652573 | Trial comparisons |
| Nasal Calictonin vs Saline Nasal Spray: Number of Participants With Nasal Congestion at 1 Month | 3 of 10 | 2 of 11 | 21 | NCT01652573 | Trial comparisons |
| Nasal Calictonin vs Saline Nasal Spray: Number of Participants With Allergic Reactions at 1 Month | 0 of 10 | 0 of 11 | 21 | NCT01652573 | Trial comparisons |
| Nasal Calictonin vs Saline Nasal Spray: Number of Patients With Nasal Congestion at Baseline | 0 of 10 | 0 of 11 | 21 | NCT01652573 | Trial comparisons |
| Nasal Calictonin vs Saline Nasal Spray: Number of Participants With Allergic Reactions at Baseline | 0 of 10 | 0 of 11 | 21 | NCT01652573 | Trial comparisons |
| Nasal Calictonin vs Saline Nasal Spray: Number of Participants With Nasal Ulcerations at Baseline | 0 of 10 | 0 of 11 | 21 | NCT01652573 | Trial comparisons |
| Nasal Calictonin vs Saline Nasal Spray: deaths from any cause | 0 of 10 | 0 of 11 | 21 | NCT01652573 | Trial comparisons |
| Nasal Calictonin vs Saline Nasal Spray: serious adverse events | 0 of 10 | 0 of 11 | 21 | NCT01652573 | Trial comparisons |
1 to 10 of 16
See all 16 comparisons in its trialsPapers from its trials22
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data 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_0010619
GET https://conductscience.org/api/diseases/v1/MONDO_0010619/papers?page=2