Clinical trials: Calcium Deficiency
- Condition Calcium Deficiency
- Funder type Industry (pharma, biotech, devices)
- Age group Children (under 18)
Hypophosphatasia, Bone Disease
Non-interventional Substudy of ALX-HPP-502 to Assess Natural History of Patients With Juvenile-onset HPP Who Served as Historical Controls in ENB-006-09
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP who served as historical controls in ENB-006-09.
- Trial name
- NCT02235493
- Testing
- Retrospective Case Only
- Where
- United States
- Volunteers
- 6 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.
DiGeorge Anomaly, Complete DiGeorge Anomaly
Phase I/II Thymus Transplantation With Immunosuppression #950
The study purpose is to determine if cultured thymus tissue implantation (CTTI) (previously described as transplantation) with tailored immunosuppression based on the recipient's pre-implantation T cell population is a safe and…
- Conditions
- DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete Atypical DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- #950 (NCT00579527)
- Testing
- Cultured Thymus Tissue for Implantation (CTTI), Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation, Blood Draw
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
X-linked Hypophosphatemia
Study of KRN23 (Burosumab), a Recombinant Fully Human Monoclonal Antibody Against Fibroblast Growth Factor 23 (FGF23), in Pediatric Subjects With X-linked Hypophosphatemia (XLH)
The objectives of the study are to: Identify a dose and dosing regimen of burosumab, based on safety and pharmacodynamic (PD) effect, in pediatric XLH participants Establish the safety profile of burosumab for the treatment of…
- Trial name
- NCT02163577
- Testing
- burosumab
- Where
- 9 sites in United States, France and 2 more
- Volunteers
- 52 people
- Sponsor
- Kyowa Kirin, Inc.
Rickets
Specturi Device - Feasibility Study
On a daily basis many (prematurely born) newborn are subjected to different urine collecting techniques to study biochemical abnormalities.
- Trial name
- NCT03105557
- Testing
- Urinary collecting device
- Where
- Netherlands
- Volunteers
- 20 people
- Sponsor
- Capturin Distribution B.V.
X-linked Hypophosphatemia, Tumor-Induced Osteomalacia
Expanded Access to Burosumab
Individual patient expanded access requests may be considered for patients who have no other treatment options
- Trial name
- NCT03775187
- Testing
- Burosumab
- Sponsor
- Kyowa Kirin Co., Ltd.
DiGeorge Anomaly, DiGeorge Syndrome
Thymus Transplantation Dose in DiGeorge #932
One purpose of this study is to determine whether the amount of cultured thymus tissue implanted into DiGeorge anomaly infants has any effect on the immune outcome.
- Conditions
- DiGeorge Anomaly, DiGeorge Syndrome, Complete DiGeorge Anomaly, Complete DiGeorge Syndrome
- Trial name
- NCT00576836
- Testing
- Cultured Thymus Tissue Implantation (CTTI), Cultured Thymus Tissue Implantation with Parathyroid Transplantation
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Sumitomo Pharma Switzerland GmbH
Hypophosphatasia
Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP)
This clinical trial was conducted to study hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone.
- Trial name
- NCT01163149
- Testing
- asfotase alfa
- Where
- 3 sites in United States, Canada
- Volunteers
- 19 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
X-Linked Hypophosphatemia
Long-Term Extension Study of KRN23 in Adult Subjects With X-Linked Hypophosphatemia (XLH)
The primary objectives of this study are to: Assess the long-term safety of KRN23 subcutaneous (SC) administration in adult subjects with XLH Assess the proportion of subjects achieving serum phosphorus levels in the normal range…
- Trial name
- NCT02312687
- Testing
- KRN23
- Where
- 5 sites in United States
- Volunteers
- 20 people
- Sponsor
- Kyowa Kirin, Inc.
Hypophosphatasia (HPP)
Retrospective, Non-interventional Natural History of Patients With Juvenile-onset Hypophosphatasia (HPP)
The purpose of this study is to characterize the natural history of HPP in patients with Juvenile-onset HPP.
- Trial name
- NCT02104219
- Where
- 19 sites in United States, Australia and 6 more
- Volunteers
- 32 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
X-Linked Hypophosphataemia
A UK Multicentre, Health-Related Quality of Life Study for Children and Adolescents With XLH
This study aims to provide Health-related Quality of Life (HRQoL) data from children and adolescents with growing skeletons in the United Kingdom (UK), including those treated with burosumab or alternative XLH treatment, as part…
- Trial name
- NCT04819490
- Where
- 6 sites in United Kingdom
- Volunteers
- 32 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
X-linked Hypophosphatemia (XLH)
Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age
A Phase 1/2, Open-label, Multicenter, Non-randomized Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Paediatric Patients from Birth to Less than 1 Year of Age with X-linked Hypophosphatemia…
- Trial name
- NCT04188964
- Testing
- Burosumab
- Where
- 9 sites in Austria, France and 4 more
- Volunteers
- 16 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Infantile Malignant Osteopetrosis
A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis
The primary objective of this Phase 1 study is to evaluate the therapeutic safety and feasibility of the investigational product (IP), RP-L401.
- Trial name
- NCT04525352
- Testing
- RP-L401
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Rocket 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
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
Extension Study of Protocol ENB-002-08 - Study of Asfotase Alfa in Infants and Young Children With Hypophosphatasia (HPP)
This clinical trial studied the long term safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP who completed study ENB-002-08 (NCT00744042).
- Trial name
- NCT01205152
- Testing
- asfotase alfa
- Where
- 8 sites in United States, United Arab Emirates and 1 more
- Volunteers
- 10 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
X-Linked Hypophosphatemia
Study of Longitudinal Observation for Patient With X-linked Hypophosphatemic Rickets/Osteomalacia in Collaboration With Asian Partners
Through observation of patients with X-linked hypophosphatemic rickets/osteomalacia (XLH) for up to 10 years, the study intends to collect data that allow achievement of the following objectives: 1.
- Trial name
- SUNFLOWER (NCT03745521)
- Testing
- no intervention
- Where
- Japan
- Volunteers
- 226 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Primary Hyperoxaluria Type 1
A Study of DCR-PH1 in Patients With Primary Hyperoxaluria Type 1 (PH1)
A phase 1 study of DCR-PH1 in patients with primary hyperoxaluria type 1 (PH1) to determine the safety, tolerability, pharmacokinetic (PK) and pharmacodynamics (PD) effects of DCR-PH1 administered via-intravenous infusion (IV)
- Trial name
- NCT02795325
- Testing
- DCR-PH1
- Where
- 2 sites in Germany, Netherlands
- Volunteers
- 41 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
X-linked Hypophosphatemia, Hypophosphatemic Rickets
X-linked Hypophosphatemia Disease Monitoring Program
The objectives of this observational study are to characterize XLH disease presentation and progression and to assess long-term effectiveness and safety of burosumab.
- Trial name
- NCT03651505
- Testing
- No intervention
- Where
- 38 sites in United States, Argentina and 4 more
- Volunteers
- 782 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Hypophosphatasia (HPP)
Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)
This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09 (NCT00952484).
- Trial name
- NCT01203826
- Testing
- Asfotase Alfa
- Where
- 2 sites in United States, Canada
- Volunteers
- 12 people
- Sponsor
- Alexion Pharmaceuticals, Inc.