Clinical trials: Hypophosphatasia
Hypophosphatasia
Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan
This is a multicenter study in Japan. Eleven sites which have already participated in the investigator-initiated clinical study (Early Access Program) will participate in this study.The objective of this study is to gain further…
- Trial name
- NCT02531867
- Testing
- Asfotase Alfa
- Volunteers
- 13 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia
The goal of this clinical trial is to compare the effectiveness of two doses of ilofotase alfa, an enzyme replacement treatment, in patients with hypophosphatasia (HPP).
- Trial name
- NCT05890794
- Testing
- Ilofotase Alfa, 0.8 mg/kg, Ilofotase Alfa, 3.2 mg/kg
- Where
- Germany
- Volunteers
- 12 people
- Sponsor
- AM-Pharma
Hypophosphatasia
Study of ALXN1850 in Participants With Hypophosphatasia (HPP)
This is an open-label, dose-escalating study to assess safety, tolerability, pharmacokinetic (PK), pharmacodynamic (PD), and immunogenicity of ALXN1850 when given intravenous (IV) and subcutaneous (SC) to adults with HPP.
- Trial name
- NCT04980248
- Testing
- ALXN1850
- Where
- 4 sites in United States
- Volunteers
- 15 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia (HPP)
Evaluation of Bone Architecture and Bone Strength in Adults With Hypophosphatasia (HPP)
The study aims to evaluate the bone architecture and bone strength in adults with Hypophosphatasia (HPP).
- Trial name
- BABS (NCT04181164)
- Testing
- Microindentation, High resolution peripheral quantitative computed tomography (HRpQCT), Biochemical analysis of different bone markers.
- Where
- Denmark
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Hvidovre University Hospital
Hypophosphatasia
Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)
This observational study will evaluate the treatment effect of Strensiq (asfotase alfa) on Patient Reported Outcomes (PROs) in participants diagnosed with pediatric-onset hypophosphatasia (HPP) registered in the patient support…
- Trial name
- NCT04195763
- Testing
- asfotase alfa
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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.
Hypophosphatasia (HPP)
A Retrospective Study of the Natural History of Patients With Severe Perinatal and Infantile Hypophosphatasia (HPP)
This study aims to characterize the natural history of patients with severe perinatal or infantile onset HPP.
- Trial name
- NCT01419028
- Where
- 12 sites in United States, Australia and 5 more
- Volunteers
- 48 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
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
Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia
Hypophosphatasia is a rare inherited metabolic disorder due to inactivating mutations of the ALPL-Gene. Particularly among adult patients, clinical manifestation exhibits a broad range of signs and symptoms, most commonly…
- Trial name
- EmPATHY (NCT03418389)
- Where
- Germany
- Volunteers
- 23 people
- Sponsor
- Dr. Lothar Seefried
Hypophosphatasia (HPP)
Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)
This clinical trial studied the safety and efficacy of asfotase alfa in children with HPP compared to a historical control group.
- Trial name
- NCT00952484
- Testing
- asfotase alfa
- Where
- 2 sites in United States, Canada
- Volunteers
- 13 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
The PORTRAIT Study
Clinical Consequences of Adults Presenting with Hypophosphatasia with Special Focus on Gait, Bone Microstructure and Cognition: The PORTRAIT study Hypophosphatasia (HPP) is an inherited condition that leads to weak bones.
- Trial name
- PORTRAIT (NCT04222452)
- Where
- United Kingdom
- Volunteers
- 14 people, healthy volunteers welcome
- Sponsor
- Sheffield Teaching Hospitals NHS Foundation Trust
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
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 (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
Health Burden of Hypophosphatasia
Hypophosphatasia (HPP) is a genetic disorder caused by mutation in the tissue-non-specific alkaline phosphatase gene (TNSALP). It causes impaired bone mineralisation, fractures, tooth loss, muscle weakness and possibly other…
- Trial name
- NCT02751801
- Testing
- no intervention
- Where
- United Kingdom
- Volunteers
- 28 people
- Sponsor
- Sheffield Teaching Hospitals NHS Foundation Trust
Hypophosphatasia
Relieving Burden of Hypophosphatasia in Adults With Functional Impairment Due to Chronic Disease
The study will evaluate safety, tolerability, efficacy, pharmacokinetics, and pharmacodynamics of 2 different doses (approved dose and lower dose) of asfotase alfa in adult participants with pediatric-onset hypophosphatasia (HPP).
- Trial name
- REBUILD (NCT04189315)
- Testing
- asfotase alfa
- Sponsor
- Alexion Pharmaceuticals, Inc.
Hypophosphatasia
Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)
This clinical trial is being conducted in Hypophosphatasia, a bone disorder caused by gene mutation(s) resulting in bone defects. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT02496689
- Testing
- asfotase alfa
- Where
- 5 sites in United States, France
- 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
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege