Clinical trials: Hypophosphatasia, active
Hypophosphatasia
The Effect of Monoallelic Variants in the ALPL Gene on the Natural Course of Hypophosphatasia in Russia
The effect of monoallelic variants in the ALPL gene on the natural course of hypophosphatasia (HPP) in children and adults in Russia (ATLANTIS)
- Trial name
- ATLANTIS (NCT07390240)
- Where
- 4 sites in Russia
- Volunteers
- 55 people
- Sponsor
- AstraZeneca
Hypophosphatasia (HPP)
Registry of Patients With Hypophosphatasia
In this prospective, observational, long term registry patients of all ages with a diagnosis of hypophosphatasia (HPP) are followed at participating sites in multiple countries.
- Trial name
- NCT02306720
- Where
- 66 sites in United States, Australia and 10 more
- Volunteers
- 1,571 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Prospective, Longitudinal, Observational Registry of Adult Patients With Hypophosphatasia (REG-HYPO)
The purpose of this study is to assess medical events during follow-up of adult patients having hypophosphatasia and consulting rheumatologists.
- Trial name
- REG-HYPO (NCT05596539)
- Testing
- Data collection
- Where
- 11 sites in France
- Volunteers
- 200 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Hypophosphatasia (HPP)
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 ALE1
This is a phase 1/2a randomised, placebo controlled, double-blind study investigating the safety, tolerability, pharmacokinetics, and pharmacodynamics of ALE1 on healthy adult subjects and adult patients with Hypophosphatasia…
- Trial name
- NCT07179640
- Testing
- ALE1
- Where
- 3 sites in Germany, New Zealand and 1 more
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Alesta Therapeutics
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Hypophosphatasia
Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa
The primary objective of this study is to assess the efficacy of ALXN1850 versus placebo on functional outcomes in adolescent and adult participants with HPP who have not previously been treated with asfotase alfa.
- Trial name
- HICKORY (NCT06079281)
- Testing
- ALXN1850
- Where
- 65 sites in United States, Argentina and 15 more
- Volunteers
- 124 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP
The primary purpose of this study is to evaluate the efficacy of ALXN1850 versus placebo on radiographic outcomes in pediatric participants with HPP who have not previously been treated with asfotase alfa.
- Trial name
- MULBERRY (NCT06079359)
- Testing
- ALXN1850
- Where
- 32 sites in United States, Australia and 14 more
- Volunteers
- 30 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Osteoporosis
GRACE II (General Retrospective Analysis of Commercial Experience With AGN1 LOEP)
The study is designed as a retrospective, multicenter study for subjects previously treated with the AGN1 LOEP Kit in the proximal femur as part of standard hospital practice. This will be a non-randomized and non-blinded study.
- Trial name
- NCT07594639
- Testing
- AGN1 LOEP
- Where
- Belgium
- Volunteers
- 305 people
- Sponsor
- AgNovos Healthcare, LLC
Hypophosphatasia
Phase 3 Study of ALXN1850 in Pediatric Participants With HPP Previously Treated With Asfotase Alfa
The primary purpose of this study is to assess the safety and tolerability of ALXN1850 versus asfotase alfa in pediatric participants with HPP previously treated with asfotase alfa.
- Trial name
- CHESTNUT (NCT06079372)
- Testing
- ALXN1850, asfotase alfa
- Where
- 21 sites in United States, Argentina and 5 more
- Volunteers
- 43 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Natural History Study of Patients With Hypophosphatasia (HPP)
Hypophosphatasia (HPP) is a rare inherited metabolic disorder characterized by defective bone and teeth mineralization caused by mutations of the ALPL gene, which encodes for the tissue-nonspecific alkaline phosphatase (TNSALP)…
- Trial name
- NatHisHPP (NCT02237625)
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Duke University
Hypophosphatasia
A Prospective Sub-Study of the Global Hypophosphatasia Registry
In this prospective observational sub-study, participants with pediatric-onset hypophosphatasia (HPP) (perinatal/infantile- or juvenile-onset) of any age will be followed for a minimum of 5 years at sites in the United States and…
- Trial name
- NCT05234567
- Testing
- Asfotase Alfa
- Where
- 12 sites in United States
- Volunteers
- 30 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- 2 sites in United States, Australia
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Hypophosphatasia
Characteristics of Hypophosphatasia in Adult Patients in Rheumatology and Their Value in Developing an Algorithm to HPP-diagnosis - the COHIR Multi-center Study
Non-interventional, prospective, multi-center investigation with exploratory data analysis to assess the proportion of patients with hypophosphatasia presenting at departments of rheumatology and to establish an algorithm to HPP…
- Trial name
- COHIRnational (NCT06574282)
- Testing
- Second alkaline phosphatase measurement
- Where
- Germany
- Volunteers
- 720 people
- Sponsor
- University of Bonn