Clinical trials: Phosphate Deficiency
Urea Cycle Disorder
Observational Study That Will Collect Information on Patients With Urea Cycle Disorders (UCDs)
THRIVE is an observational study that will collect information on patients with UCDs. THRIVE will follow enrolled participants for up to 10 years.
- Trial name
- THRIVE (NCT01948427)
- Where
- United States
- Volunteers
- 203 people
- Sponsor
- Amgen
Malaria
G6PD (Glucose-6-phosphate Dehydrogenase) Study to Evaluate Hemolysis Potential of TFQ (Tafenoquine)
SB-252263 (Tafenoquine, TQ) is an 8-aminoquinoline (8-AQ) antimalarial drug being developed by GlaxoSmithKline (GSK), the U.S. Army Medical Research and Materiel Command (USAMRMC) and Medicines for Malaria Venture (MMV).
- Trial name
- NCT01205178
- Testing
- Chloroquine, Primaquine, Tafenoquine
- Where
- 2 sites in Thailand
- Volunteers
- 192 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Ataxia Telangiectasia, Genetic Syndrome
Extension Treatment Using EryDex System in Patients With AT Who Participated in the ATTeST-IEDAT-02-2015 Study
Primary Objective To monitor and evaluate the long-term safety and tolerability of EDS-EP in AT patients. Secondary Objective To evaluate the long-term effect of EDS-EP on health-related Quality of Life (QoL; EQ-5D-5L scale).
- Trial name
- OLE-IEDAT (NCT03563053)
- Testing
- EryDex System
- Where
- 17 sites in United States, Australia and 9 more
- Volunteers
- 104 people
- Sponsor
- Quince Therapeutics S.p.A.
Pyridox(am)Ine 5'-Phosphate Oxidase Deficiency
Oral Pyridoxal 5'-Phosphate for the Treatment of Patients With PNPO Deficiency
The proposed clinical study is intended to evaluate oral P5P for the treatment of patients confirmed to have Pyridox(am)ine 5'-Phosphate Oxidase (PNPO) deficiency via genetic analysis.
- Trial name
- MEND-PNPO (NCT04706013)
- Testing
- Pyridoxal Phosphate
- Where
- 7 sites in United States, Australia
- Volunteers
- 15 people
- Sponsor
- Medicure
X-Linked Hypophosphatemia
Efficacy and Safety of Burosumab Versus Oral Phosphate and Active Vitamin D Treatment in Pediatric Patients With XLH
The primary objective of this study is to evaluate the effect of KRN23 (burosumab) therapy in improving rickets in children with XLH compared with active control (oral phosphate/active vitamin D).
- Trial name
- NCT02915705
- Testing
- burosumab, Oral Phosphate Supplement, active vitamin D
- Where
- 16 sites in United States, Australia and 5 more
- Volunteers
- 61 people
- Sponsor
- Kyowa Kirin, Inc.