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.
Acquired Factor X Deficiency
A Study Investigating Coagadex in the Treatment AFXD Associated With AL Amyloidosis
The purpose of this study is to evaluate the efficacy and safety of Coagadex in treating active bleeds and to manage peri-operative bleeding in participants with acquired factor X deficiency associated with AL amyloidosis.
- Trial name
- NCT06963216
- Testing
- Coagadex(R)
- Sponsor
- Kedrion S.p.A.
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
Hemophilia B
Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B
In this study a modified virus called adeno-associated virus (AAV) will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B (AAV human Factor IX vector).
- Trial name
- NCT00076557
- Testing
- Adeno-Associated Viral with Human Factor IX
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- Avigen
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.
Hemophilia B
Open-Label Single Ascending Dose of Adeno-associated Virus Serotype 8 Factor IX Gene Therapy in Adults With Hemophilia B
The purpose of this study is to evaluate the safety of single ascending IV doses of a Factor IX (FIX) Gene Therapy in up to 16 Adults with Hemophilia B.
- Trial name
- NCT01687608
- Testing
- AskBio009
- Where
- 15 sites in United States
- Volunteers
- 30 people
- Sponsor
- Baxalta now part of Shire
Healthy Participants, Hemophilia A
A Study of Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SR604 in Two Participants Groups (Part A: Healthy Participants, and Part B: Participants With Hemophilia A or Hemophilia B or Factor VII Deficiency)
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamic (PD) of SR604 in healthy participants (Part A) and to evaluate the safety, tolerability, PK, PD, and efficacy of SR604…
- Conditions
- Healthy Participants, Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT06349473
- Testing
- SR604
- Where
- 10 sites in United States, Canada
- Volunteers
- 31 people, healthy volunteers welcome
- Sponsor
- Equilibra Bioscience LLC
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
HIV Infections, Hemophilia A
Effect of Indinavir Plus Two Other Anti-HIV Drugs on Blood Clotting in HIV-Positive Males With Hemophilia
The purpose of this study is to see if indinavir plus two other anti-HIV drugs affect blood clotting in HIV-positive patients with hemophilia.
- Trial name
- NCT00002386
- Testing
- Indinavir sulfate, Lamivudine, Stavudine
- Where
- 8 sites in United States, Canada
- Volunteers
- 55 people
- Sponsor
- Merck Sharp & Dohme LLC
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.
Acquired Coagulation Factor Deficiency
Prothrombin Complex Concentrate for Anticoagulant Reversal
Patients on oral anticoagulants need rapid reversal of the anticoagulant effect in case of acute bleeding or emergency surgery. Prothrombin Complex Concentrates are known to provide a fast reversal of the anticoagulant effect…
- Trial name
- NCT00168077
- Testing
- Prothrombin Complex Concentrate
- Where
- 15 sites in Austria, Germany and 6 more
- Volunteers
- 40 people
- Sponsor
- CSL Behring
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
Hemophilia B Gene Therapy With AAV8 Vector
Hemophilia B is a bleeding disease in males due to very low levels of coagulation factor IX (FIX) in the blood. The current treatment is intravenous injection of FIX clotting factor concentrates, in response to bleeding.
- Trial name
- NCT01620801
- Testing
- AAV8-hFIX19
- Where
- 3 sites in United States, Australia
- Volunteers
- 4 people
- Sponsor
- Spark Therapeutics, Inc.
Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited
Lead-in Study to Collect Prospective Efficacy and Safety Data of Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Participants
The aim of this prospective, observational study is to establish a dataset on the frequency of bleeding events, as well as other characteristics of bleeding events and FVIII infusions, in patients with clinically severe…
- Conditions
- Blood Coagulation Disorder, Blood Coagulation Disorders, Inherited, Coagulation Protein Disorders, Hemophilia A
- Trial name
- NCT03876301
- Testing
- Standard of Care FVIII Replacement therapy
- Where
- 11 sites in United States, Australia and 2 more
- Volunteers
- 25 people
- Sponsor
- Spark Therapeutics, Inc.
Hemophilia A, Hemophilia B
Study of Prophylaxis, ACtivity and Effectiveness (SPACE) in Hemophilia Patients Currently Treated With ADVATE or RIXUBIS
This 6-month prospective outcomes study addresses the association between timing of infusion, patient activity levels, and bleeding episodes through patient-reported measurements.
- Trial name
- SPACE (NCT02190149)
- Testing
- ADVATE (Antihemophilic Factor [Recombinant]), RIXUBIS (Coagulation Factor IX [Recombinant])
- Where
- 17 sites in United States
- Volunteers
- 64 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Hemophilia B
An Open-label, Multicenter Phase I/II Clinical Trial to Evaluate the Safety, Tolerability, Efficacy, and Pharmacokinetic/Pharmacodynamic (PK/PD) Characteristics of SR604 Injection in Patients With Hemophilia A/B and Congenital Factor VII Deficiency
The purpose of this study is to evaluate the safety, tolerability, immunogenicity , PK, and PD of a single dose of SR604 in participants with Hemophilia A or Hemophilia B, with or without inhibitors （Part A）and to evaluate the…
- Conditions
- Hemophilia A, Hemophilia B, Factor VII Deficiency
- Trial name
- NCT07644832
- Testing
- SR604
- Where
- 9 sites in China
- Volunteers
- 76 people
- Sponsor
- Shanghai RAAS Blood Products Co., Ltd.
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
Prothrombin Complex Factor Deficiency
Efficacy and Safety of Prothromplex Total (Prothrombin Complex Concentrate) in Oral Anticoagulant Reversal
The purpose of the study is to assess the efficacy and safety of Prothromplex Total as a treatment for the immediate reversal of oral anticoagulant therapy with vitamin K antagonists in patients with acquired deficiency of…
- Trial name
- NCT01159210
- Testing
- Prothrombin complex concentrate (coagulation factors IX, II, VII and X in combination)
- Where
- 6 sites in Austria, Hungary
- Volunteers
- 61 people
- Sponsor
- Baxalta now part of Shire