Clinical trials: Factor X Deficiency
X-linked Hypophosphatemia
Study of KRN23 in Adults With X-linked Hypophosphatemia (XLH)
The primary efficacy objective of this study is to establish the effect of burosumab treatment compared with placebo on increasing serum phosphorus levels in adults with XLH.
- Trial name
- NCT02526160
- Testing
- burosumab
- Where
- 25 sites in United States, France and 5 more
- Volunteers
- 134 people
- Sponsor
- Kyowa Kirin, Inc.
Neovacular Age-related Macular Degeneration
UNcovering the Difference Between Ranibizumab and Aflibercept, Focusing on Systemic Anti-vascular Endothelial Growth Factor (VEGF) Effects in Patients With neovascuLar Age-related Macular Degeneration (AMD)
This study assessed systemic vascular endothelial growth factor (VEGF) level in patients with neovascular Age-related Macular Degeneration following treatment with Ranibizumab or Aflibercept.
- Trial name
- UNRAVEL (NCT01988662)
- Testing
- Neovascular Age-related Macular Degeneration
- Where
- 24 sites in Israel, Malaysia and 5 more
- Volunteers
- 205 people
- Sponsor
- Novartis Pharmaceuticals
X-linked Hypophosphatemia
Open Label Study of KRN23 on Osteomalacia in Adults With X-linked Hypophosphatemia (XLH)
The primary objective of this study is to establish the effect of KRN23 treatment on improvement in XLH-associated osteomalacia as determined by osteoid volume (osteoid volume/bone volume, OV/BV).
- Trial name
- NCT02537431
- Testing
- burosumab
- Where
- 14 sites in United States, Canada and 4 more
- Volunteers
- 14 people
- Sponsor
- Kyowa Kirin, Inc.
Adeno-Associated Virus (AAV), Blood Coagulation Disorder
Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors
SPK-8016 is in development for the treatment of patients with inhibitors to FVIII. This Phase 1/2, open-label, non-randomized, dose-finding study to evaluate the safety, efficacy, and tolerability of SPK-8016 in adult males with…
- Conditions
- Adeno-Associated Virus (AAV), Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders
- Trial name
- NCT03734588
- Testing
- SPK-8016
- Where
- 11 sites in United States
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
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.
Hemophilia A
A Gene Transfer Study for Hemophilia A
This clinical research study is being conducted by Spark Therapeutics, Inc. to determine the safety and efficacy of the factor VIII gene transfer treatment with SPK-8011 in individuals with hemophilia A.
- Trial name
- NCT03003533
- Testing
- SPK-8011
- Where
- 16 sites in United States, Australia and 3 more
- Volunteers
- 25 people
- Sponsor
- Spark Therapeutics, Inc.
Hemophilia B
A Gene Therapy Study for Hemophilia B
A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B.
- Trial name
- NCT02484092
- Testing
- SPK-9001
- Where
- 10 sites in United States, Australia
- Volunteers
- 15 people
- Sponsor
- Pfizer