Hypophosphatasia clinical trials
The registry lists 46 studies for Hypophosphatasia across more than 10 countries. 11 are recruiting or about to start. 44 of them are mainly about Hypophosphatasia (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-04.
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Status
Who runs them
Alexion Pharmaceuticals, Inc. (23) · Sheffield Teaching Hospitals NHS Foundation Trust (3) · AstraZeneca (2) · University of Bonn (2) · RTI International (1) · Emine Hafize (1) · AgNovos Healthcare, LLC (1) · Wuerzburg University Hospital (1)
What is being tested here
Drugs under test in Hypophosphatasia studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 5
- Nov 2026Early Check: Expanded Screening in Newborns · RTI International
- Jan 2027A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Adults With Hypophosphatasia in Order to Identify Suitable Doses of ALE1 · Alesta Therapeutics
- Apr 2027GRACE II (General Retrospective Analysis of Commercial Experience With AGN1 LOEP) · AgNovos Healthcare, LLC
- Sep 2027Natural History Study of Patients With Hypophosphatasia (HPP) · Duke University
- Sep 2027Characteristics of Hypophosphatasia in Adult Patients in Rheumatology and Their Value in Developing an Algorithm to HPP-diagnosis - the COHIR Multi-center Study · University of Bonn
Who these studies take
Of the 9 Hypophosphatasia studies recruiting now, 8 (89%) set no upper age limit and 1 (11%) stop at 75 or younger. 5 (56%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (11%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 5 (56%) | 5 (56%) |
| 18 | 9 (100%) | 9 (100%) |
| 30 | 9 (100%) | 9 (100%) |
| 40 | 9 (100%) | 9 (100%) |
| 50 | 9 (100%) | 9 (100%) |
| 65 | 8 (89%) | 8 (89%) |
| 75 | 8 (89%) | 8 (89%) |
| 85 | 8 (89%) | 8 (89%) |
Across all 46 studies, recruiting or not: 33% stop at 75 or younger and 30% at 65; 65% take children; 0% are for women only and 0% for men only; 13% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 13, 2026.
Newly registered1
- Clarify HPP: Disease Burden of HPP Patients in ChinaOct 29, 2026NCT07905287
Studies
Recruiting first, then most recently updated. Showing 46 of 46. search them.
- Registry of Patients With HypophosphatasiaEnrolling by invitationNCT0230672013 countries
- GRACE II (General Retrospective Analysis of Commercial Experience With AGN1 LOEP)Enrolling by invitationNCT075946391 country
- Clarify HPP: Disease Burden of HPP Patients in ChinaRecruitingNCT079052871 country
- Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at SanfordRecruitingNCT017931681 country
- The Effect of Monoallelic Variants in the ALPL Gene on the Natural Course of Hypophosphatasia in RussiaRecruitingNCT073902401 country
- Prospective, Longitudinal, Observational Registry of Adult Patients With Hypophosphatasia (REG-HYPO)RecruitingNCT055965391 country
- A Study to Assess Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ALE1 in Healthy Adults and Adults With Hypophosphatasia in Order to Identify Suitable Doses of ALE1RecruitingNCT07179640Phase 1/23 countries
- Natural History Study of Patients With Hypophosphatasia (HPP)RecruitingNCT022376251 country
- A Prospective Sub-Study of the Global Hypophosphatasia RegistryRecruitingNCT052345671 country
- Characteristics of Hypophosphatasia in Adult Patients in Rheumatology and Their Value in Developing an Algorithm to HPP-diagnosis - the COHIR Multi-center StudyRecruitingNCT065742821 country
- Characteristics of Hypophosphatasia in Adult Patients in RheumatologyRecruitingNCT061611421 country
- Early Check: Expanded Screening in NewbornsActive, not recruitingNCT036552231 country
- Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase AlfaActive, not recruitingNCT06079281Phase 317 countries
- Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPPActive, not recruitingNCT06079359Phase 316 countries
- United States Hypophosphatasia Molecular Research CenterCompletedNCT050626291 country
- Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase AlfaActive, not recruitingNCT06079372Phase 37 countries
- Mitigate Immune-Mediated Loss of Therapeutic Response to Asfotase Alfa (STRENSIQ®) for HypophosphatasiaWithdrawnNCT06015750Phase 4
- Pilot Trial of Single Dose Ilofotase Alfa in HypophosphatasiaCompletedNCT05890794Phase 1/21 country
- Study of ALXN1850 in Participants With Hypophosphatasia (HPP)CompletedNCT04980248Phase 11 country
- Serum Alkaline Phosphatase Levels In Healthy Turkish ChildrenCompletedNCT06689709Not applicable
- Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)CompletedNCT041957631 country
- Characterisation of Adult-Onset HypophosphatasiaCompletedNCT027968851 country
- Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)WithdrawnNCT00894075Phase 2
- Circulating miRNAs and Bone Microstructure in Adults With HypophosphatasiaStatus unknownNCT040182871 country
- The PORTRAIT StudyStatus unknownNCT042224521 country
- Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP)CompletedNCT01406977Phase 21 country
- Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for HypophosphatasiaCompletedNCT034183891 country
- Unraveling Genetics of HypoPhosPhatasia (HPP Genetics)CompletedNCT049258041 country
- Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP)Status unknownNCT041811641 country
- Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic DiseaseWithdrawnNCT04189315Phase 4
- Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)CompletedNCT02797821Phase 22 countries
- A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)CompletedNCT014190287 countries
- Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)CompletedNCT00952484Phase 22 countries
- Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)CompletedNCT021042198 countries
- Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)CompletedNCT00744042Phase 1/24 countries
- Safety Study of Human Recombinant Tissue Non-Specific Alkaline Phosphatase Fusion Protein Asfotase Alfa in Adults With Hypophosphatasia (HPP)CompletedNCT00739505Phase 12 countries
- Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)Approved for marketingNCT024966892 countries
- Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP)CompletedNCT01176266Phase 2/312 countries
- Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)CompletedNCT01203826Phase 22 countries
- Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)CompletedNCT01163149Phase 22 countries
- Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)CompletedNCT01205152Phase 23 countries
- Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09CompletedNCT022354931 country
- Health Burden of HypophosphatasiaCompletedNCT027518011 country
- Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in JapanCompletedNCT02531867Phase 4
- Burden of Disease in Hypophosphatasia (HPP)CompletedNCT022914971 country
- Safety and Efficacy of Asfotase Alfa in Patients With Hypophosphatasia (HPP)CompletedNCT02456038Phase 21 country