Clinical trials: Hemophilia B, Severe
Hemophilia B
HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients
This is an open-label, single-dose, multi-center, multinational trial to demonstrate the efficacy of AMT-061 and to further describe its safety profile. The study drug is identified as AAV5-hFIXco-Padua (AMT- 061).
- Trial name
- NCT03569891
- Testing
- AAV5-hFIXco-Padua, Factor IX (FIX)
- Where
- 33 sites in United States, Belgium and 6 more
- Volunteers
- 67 people
- Sponsor
- CSL Behring
Haemophilia B
A Study to Compare the Pharmacokinetics and Safety of Replenine®-VF, Replenine® or Other Factor IX in Haemophilia B
The purpose of this study is: to compare the pharmacokinetics of Replenine®-VF and Replenine® or any other high purity Factor IX concentrate, when given as a bolus dose of 75IU/kg.
- Trial name
- NCT02263456
- Testing
- Replenine®-VF (High Purity Factor IX)
- Where
- 7 sites in United Kingdom
- Sponsor
- Bio Products Laboratory
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Haemophilia B
An Open Study to Investigate the Safety and Efficacy of Replenine®-VF in Severe Haemophilia B Patients
To compare the first and second recovery assessments on Replenine®-VF and to evaluate recovery of different batches if patients changed batches during the study.
- Trial name
- NCT02231944
- Testing
- Replenine®-VF (High Purity Factor IX)
- Where
- 7 sites in Poland, United Kingdom
- Sponsor
- Bio Products Laboratory
Hemophilia B
Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B
This is an open-label, extension study enrolling patients who have successfully completed all assessments in Study CT-AMT-060-01 (Years 1-5).
- Trial name
- NCT05360706
- Testing
- AAV5-hFIX
- Where
- 6 sites in Germany, Netherlands
- Volunteers
- 9 people
- Sponsor
- CSL Behring
Hemophilia
In Vitro Correction of Thrombin Generation by Concizumab (Anti-TFPI) for Severe Hemophilia Patients
Studies have shown, in haemophilia patients, the effectiveness of Anti TFPI antibody (aTFPIAb) to prevent joint and muscular bleeding (tissues poor in Tissue Factor (TF)).
- Trial name
- NCT05617209
- Testing
- thrombin generation measurement
- Where
- France
- Volunteers
- 20 people
- Sponsor
- Centre Hospitalier Universitaire de Saint Etienne
Hemophilia A or B
PF-06741086 Long-term Treatment in Severe Hemophilia
This study is designed to evaluate the safety, tolerability and efficacy of long-term treatment with PF-06741086 in subjects with severe hemophilia who participated in the 3-month Phase 1b/2 B7841002 study.
- Trial name
- NCT03363321
- Testing
- PF-06741086
- Where
- 8 sites in United States, Brazil and 5 more
- Volunteers
- 20 people
- Sponsor
- Pfizer
Hemophilia B
Dose Confirmation Trial of AAV5-hFIXco-Padua
This is an open-label, single-dose, single-arm, multi-center trial, with a screening, a treatment + post-treatment follow-up phase, and a long-term follow-up phase.
- Trial name
- NCT03489291
- Testing
- AAV5-hFIXco-Padua (AMT-061)
- Where
- 4 sites in United States
- Volunteers
- 3 people
- Sponsor
- CSL Behring
Hemophilia
The Effectiveness of Recombinant Coagulation Factor IX With Recombinant Albumin (rIX-FP) in Severe Hemophilia B Patients
The pivotal clinical data support prolonged dosing intervals of up to 14 days for routine prophylaxis in hemophilia B patients. The majority of adult and pediatric patients using Idelvion for routine prophylaxis had a median…
- Trial name
- NCT04108260
- Testing
- Albutrepenonacog Alfa 1 UNT [IDELVION]
- Where
- Taiwan
- Volunteers
- 5 people
- Sponsor
- National Taiwan University Hospital
Haemophilia B
A Study to Investigate the Safety and Efficacy of Replenine®-VF in Haemophilia B Patients Under the Age of 6 Years
The objective of this study was to assess the safety and efficacy of Replenine®-VF in children enrolled in the study, under the age of six years, with severe haemophilia B.
- Trial name
- NCT02263469
- Testing
- Replenine®-VF (High Purity Factor IX)
- Where
- 4 sites in Poland, Ukraine
- Sponsor
- Bio Products Laboratory
Hemophilia A, Hemophilia B
The Hemophilia Ultrasound Project
To evaluate the prevalence of subclinical arthropathy in children with severe hemophilia undergoing a prophylaxis regimen and without evidence of target joints, using a validated ultrasound scoring method.
- Trial name
- HUP (NCT02807753)
- Testing
- Ankle and Knee Ultrasound Joint Assessment, Ankle and Knee Magnetic Resonance Imaging
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- University of Miami
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
Hemophilia A, Hemophilia B
Study of FVIIa Variant BAY86-6150 (B0189) in Subjects With Moderate or Severe Hemophilia Types A or B With or Without Inhibitors
This is the first in humans study of BAY86-6150 (B0189) in non-bleeding subjects with moderate or severe congenital hemophilia A or B with or without inhibitors.
- Trial name
- MATCHBOX (NCT01921855)
- Testing
- BAY Factor VII (BAY86-6150)
- Where
- 4 sites in Poland, South Africa and 1 more
- Volunteers
- 16 people
- Sponsor
- Bayer
Hemophilia B
BAX 326 Surgery Study in Hemophilia B Patients
The purpose of the study is to assess the hemostatic efficacy and safety of BAX 326 in subjects with severe (FIX level < 1%) or moderately severe (FIX level 1-2%) hemophilia B undergoing major or minor elective or emergency…
- Trial name
- NCT01507896
- Testing
- Recombinant factor IX
- Where
- 14 sites in Argentina, Bulgaria and 8 more
- Volunteers
- 30 people
- Sponsor
- Baxalta now part of Shire
Hemophilia A, Hemophilia B
A Single Escalating Dose and Multiple Dose Study of BAY 1093884 in Subjects With Severe Hemophilia Types A or B, With or Without Inhibitors
Investigate the safety, tolerability and pharmacokinetics of BAY1093884 after Intravenous (IV) and subcutaneous (SC) administration of increasing single doses and SC administration of multiple doses.
- Trial name
- NCT02571569
- Testing
- BAY1093884
- Where
- 10 sites in Bulgaria, Germany and 3 more
- Volunteers
- 32 people
- Sponsor
- Bayer
Hemophilia B
Nonacog Alfa Prophylaxis And Treatment Of Bleeding Episodes In Previously Treated Patients With Hemophilia B
Nonacog alfa is indicated for the control and prevention of hemorrhagic episodes and for routine and surgical prophylaxis in patients with hemophilia B.
- Trial name
- NCT04286412
- Testing
- Nonacog alfa
- Where
- 5 sites in India
- Volunteers
- 25 people
- Sponsor
- Pfizer
Severe Hemophilia B
Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B
The primary objectives of the study were: to evaluate the safety and tolerability of rFIXFc; to evaluate the efficacy of rFIXFc in all treatment arms; to evaluate the effectiveness of prophylaxis over on-demand (episodic) therapy…
- Trial name
- NCT01027364
- Testing
- Factor IX (rFIXFc), rFIX
- Where
- 51 sites in United States, Australia and 15 more
- Volunteers
- 123 people
- Sponsor
- Bioverativ Therapeutics Inc.
Hemophilia a and B
KN057 Multiple Dose Study in Patients with Hemophilia a or Hemophilia B with or Without Inhibitors
The goal of this clinical trial is to evaluate the safety and efficacy of KN057 in adult patients with severe Hemophilia A (coagulation factor FVIII activity <1%) or moderate-to-severe Hemophilia B (FIX activity ≤2%).
- Trial name
- NCT06747416
- Testing
- KN057
- Volunteers
- 24 people
- Sponsor
- Suzhou Alphamab Co., Ltd.
Haemophilia A, Haemophilia B
Impact of Conservative Treatment by Custom-made Orthoses in Patients With Haemophilic Ankle Arthropathy
The purpose of this study intend to evaluate the effectiveness of functional orthoses (including custom-made insoles and shoes) for preventing and controlling repetitive haemarthrosis in patients suffering of haemophilic ankle…
- Trial name
- NCT00638001
- Testing
- custom-molded insoles and custom-molded shoes
- Where
- Belgium
- Volunteers
- 70 people
- Sponsor
- Cliniques universitaires Saint-Luc- Université Catholique de Louvain
Hemophilia B
Study Comparing On-Demand Treatment With Two Prophylaxis Regimens Of BeneFIX In Patients With Severe Hemophilia B
This study is designed to evaluate BeneFIX infused as prophylaxis regimens, compared with BeneFIX administered as an on-demand regimen only. In the trial, subjects will participate in 4 study periods.
- Trial name
- NCT00364182
- Testing
- Recombinant Coagulation Factor IX (BeneFIX)
- Where
- 19 sites in United States, Canada and 7 more
- Volunteers
- 50 people
- Sponsor
- Pfizer