Clinical Trials

Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B

June 6, 2019

https://clinicaltrials.gov/ct2/show/NCT02141074?type=Intr&cond=hemophilia&lupd_s=05%2F23%2F2019&lupd_d=14

Conditions : Congenital Bleeding Disorder; Haemophilia B

Intervention : Drug: nonacog beta pegol

Sponsor : Novo Nordisk A/S

Recruiting

Safety and Efficacy of Nonacog Beta Pegol (N9-GP) in Previously Untreated Patients With Haemophilia B

NCT02141074

Mon, 19 May 2014 12:00:00 EDT

Last Update Posted: 06/06/19 07:30AM

Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

June 4, 2019

https://clinicaltrials.gov/ct2/show/NCT03974113?type=Intr&cond=hemophilia&lupd_s=05%2F21%2F2019&lupd_d=14

Condition : Hemophilia

Intervention : Drug: Fitusiran

Sponsor : Genzyme, a Sanofi Company

Not yet recruiting

Fitusiran Prophylaxis in Male Pediatric Subjects Aged 1 to Less Than 12 Years With Hemophilia A or B

NCT03974113

Tue, 04 Jun 2019 12:00:00 EDT

Last Update Posted: 06/04/19 06:53AM

Lentiviral FVIII Gene Therapy for Hemophilia A [YUVA-GT-F801]

May 23, 2019

https://clinicaltrials.gov/ct2/show/NCT03217032?type=Intr&cond=hemophilia&lupd_s=05%2F09%2F2019&lupd_d=14

Condition : Hemophilia A

Intervention : Biological: YUVA-GT-F801

Sponsor : Shenzhen Geno-Immune Medical Institute

Not yet recruiting

Lentiviral FVIII Gene Therapy

NCT03217032

Thu, 13 Jul 2017 12:00:00 EDT

Last Update Posted: 05/23/19 07:43AM

A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors

May 22, 2019

https://clinicaltrials.gov/ct2/show/NCT03196284?type=Intr&cond=hemophilia&lupd_s=05%2F08%2F2019&lupd_d=14

Conditions : Congenital Bleeding Disorder; Haemophilia A With Inhibitors; Haemophilia B With Inhibitors

Interventions : Drug: Concizumab; Drug: Eptacog alfa

Sponsor : Novo Nordisk A/S

Active, not recruiting

A Trial Evaluating the Efficacy and Safety of Prophylactic Administration of Concizumab in Haemophilia A and B Patients With Inhibitors

NCT03196284

Thu, 22 Jun 2017 12:00:00 EDT

Last Update Posted: 05/22/19 07:39AM

A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors

May 22, 2019

https://clinicaltrials.gov/ct2/show/NCT03196297?type=Intr&cond=hemophilia&lupd_s=05%2F08%2F2019&lupd_d=14

Conditions : Haemostasis; Haemophilia A

Interventions : Drug: Concizumab; Drug: Turoctocog alfa

Sponsor : Novo Nordisk A/S

Active, not recruiting

A Trial Evaluating Efficacy and Safety of Prophylactic Administration of Concizumab in Patients With Severe Haemophilia A Without Inhibitors

NCT03196297

Thu, 22 Jun 2017 12:00:00 EDT

Last Update Posted: 05/22/19 07:39AM

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

May 15, 2019

https://clinicaltrials.gov/ct2/show/NCT03417245?type=Intr&cond=hemophilia&lupd_s=05%2F01%2F2019&lupd_d=14

Conditions : Hemophilia A; Hemophilia B

Interventions : Drug: fitusiran; Drug: factor VIII or factor IX

Sponsor : Genzyme, a Sanofi Company

Recruiting

A Study of Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

NCT03417245

Wed, 31 Jan 2018 12:00:00 EST

Last Update Posted: 05/15/19 07:45AM

Clinical Trial Comparing a Biosimilar Eptacog Alfa With Novoseven, in Patients With Hemophilia With Inhibitors

May 2, 2019

https://clinicaltrials.gov/ct2/show/NCT03935334?type=Intr&cond=hemophilia&lupd_s=04%2F18%2F2019&lupd_d=14

Condition : Hemophilia A or B With Inhibitor

Interventions : Biological: Biosimilar eptacog alfa, activated (AryoSeven); Biological: Eptacog alfa, activated (Novoseven)

Sponsor : AryoGen Pharmed Co.

Recruiting

Clinical Trial Comparing a Biosimilar Eptacog Alfa With Novoseven, in Patients With Hemophilia With Inhibitors

NCT03935334

Thu, 02 May 2019 12:00:00 EDT

Last Update Posted: 05/02/19 07:49AM

Study to evaluate safety, immunogenicity and hemostatic efficacy of PEGylated Recombinant Factor VIII (BAX 855) in previously untreated patients with severe hemophilia A

April 25, 2019

https://clinicaltrials.gov/ct2/show/NCT02615691?type=Intr&cond=hemophilia&lupd_s=04%2F11%2F2019&lupd_d=14

Condition : Hemophilia A

Intervention : Biological: PEGylated Recombinant Factor VIII

Sponsor : Baxalta now part of Shire

Recruiting

BAX 855 Previously Untreated Patient (PUP)

NCT02615691

Thu, 26 Nov 2015 12:00:00 EST

Last Update Posted: 04/25/19 07:35AM

Long-term Safety and Efficacy Study of SPK-9001 in Individuals With Hemophilia B

April 24, 2019

https://clinicaltrials.gov/ct2/show/NCT03307980?type=Intr&cond=hemophilia&lupd_s=04%2F10%2F2019&lupd_d=14

Condition : Hemophilia B

Intervention : Biological: SPK-9001

Sponsor : Pfizer

Recruiting

Long-term Safety and Efficacy Study of SPK-9001 in Individuals With Hemophilia B

NCT03307980

Thu, 12 Oct 2017 12:00:00 EDT

Last Update Posted: 04/24/19 07:45AM

Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A

April 23, 2019

https://clinicaltrials.gov/ct2/show/NCT03818763?type=Intr&cond=hemophilia&lupd_s=04%2F09%2F2019&lupd_d=14

Condition : Hemophilia A

Intervention : Biological: Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII

Sponsor : Parameswaran Hari

Not yet recruiting

Gene Therapy Trial for Platelet Derived Factor VIII Production in Hemophilia A

NCT03818763

Mon, 28 Jan 2019 12:00:00 EST

Last Update Posted: 04/23/19 08:32AM

Gene Therapy Study in Severe Haemophilia A Patients With Antibodies Against AAV5

April 8, 2019

https://clinicaltrials.gov/ct2/show/NCT03520712?type=Intr&cond=hemophilia&lupd_s=03%2F25%2F2019&lupd_d=14

Conditions : Haemophilia A; Gene Therapy; Clotting Disorders; Blood Disorder

Intervention : Biological: Valoctocogene Roxaparvovec

Sponsor : BioMarin Pharmaceutical

Enrolling by invitation

Gene Therapy Study in Severe Haemophilia A Patients With Antibodies Against AAV5

NCT03520712

Thu, 10 May 2018 12:00:00 EDT

Last Update Posted: 04/08/19 08:25AM

A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment (verITI-8 Study)

April 3, 2019

https://clinicaltrials.gov/ct2/show/NCT03093480?type=Intr&cond=hemophilia&lupd_s=03%2F20%2F2019&lupd_d=14

Condition : Hemophilia A With Inhibitors

Intervention : Biological: rFVIIIFc

Sponsors : Bioverativ Therapeutics Inc.; Swedish Orphan Biovitrum

Recruiting

A Study to Evaluate Efficacy of rFVIIIFc for Immune Tolerance Induction (ITI) in Severe Hemophilia A Participants With Inhibitors Undergoing the First ITI Treatment (verITI-8 Study)

NCT03093480

Tue, 28 Mar 2017 12:00:00 EDT

Last Update Posted: 04/03/19 07:43AM

Study to Determine the Safety and Efficacy of rFIXFc in Untreated Males With Severe Hemophilia B

April 3, 2019

https://clinicaltrials.gov/ct2/show/NCT02234310?type=Intr&cond=hemophilia&lupd_s=03%2F20%2F2019&lupd_d=14

Condition : Hemophilia B

Intervention : Biological: rFIXFc

Sponsors : Bioverativ Therapeutics Inc.; Swedish Orphan Biovitrum

Active, not recruiting

Study to Determine the Safety and Efficacy of rFIXFc in Untreated Males With Severe Hemophilia B

NCT02234310

Tue, 09 Sep 2014 12:00:00 EDT

Last Update Posted: 04/03/19 07:43AM

Evaluation of Safety Following Immune Tolerance Induction Treatment With Turoctocog Alfa in Patients With Haemophilia A Following Inhibitor Development in NN7170-4213 Trial

April 1, 2019

https://clinicaltrials.gov/ct2/show/NCT03588741?type=Intr&cond=hemophilia&lupd_s=03%2F18%2F2019&lupd_d=14

Condition : Haemophilia A

Intervention : Drug: Turoctocog alfa

Sponsor : Novo Nordisk A/S

Active, not recruiting

Evaluation of Safety Following Immune Tolerance Induction Treatment With Turoctocog Alfa in Patients With Haemophilia A Following Inhibitor Development in NN7170-4213 Trial

NCT03588741

Tue, 17 Jul 2018 12:00:00 EDT

Last Update Posted: 04/01/19 08:54AM

Hemophilia Inhibitor Prevention and Eradication Clinical Trials (INHIBIT) Platform

March 26, 2019

https://clinicaltrials.gov/ct2/show/NCT02196207?type=Intr&cond=hemophilia&lupd_s=03%2F12%2F2019&lupd_d=14

Condition : Severe Hemophilia A

Interventions : Drug: rFVIIIFc; Drug: rFVIII; Drug: Emicizumab; Drug: “Winner”: rFVIIIFc or rFVIII; Drug: Eloctate and Emicizumab; Drug: Eloctate

Sponsor : Margaret Ragni

Not yet recruiting

Hemophilia Inhibitor Prevention and Eradication Clinical Trials (INHIBIT) Platform

NCT02196207

Mon, 21 Jul 2014 12:00:00 EDT

Last Update Posted: 03/26/19 09:35AM

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B

March 26, 2019

https://clinicaltrials.gov/ct2/show/NCT02396342?type=Intr&cond=hemophilia&lupd_s=03%2F12%2F2019&lupd_d=14

Condition : Hemophilia B

Intervention : Genetic: AAV5-hFIX

Sponsor : UniQure Biopharma B.V.

Active, not recruiting

Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B

NCT02396342

Tue, 24 Mar 2015 12:00:00 EDT

Last Update Posted: 03/26/19 09:35AM

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

March 25, 2019

https://clinicaltrials.gov/ct2/show/NCT03754790?type=Intr&cond=hemophilia&lupd_s=02%2F27%2F2019&lupd_d=14

Condition : Hemophilia

Intervention : Drug: Fitusiran

Sponsor : Genzyme, a Sanofi Company

Recruiting

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX

NCT03754790

Tue, 27 Nov 2018 12:00:00 EST

Last Update Posted: 03/13/19 10:19AM

A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A

March 22, 2019

https://clinicaltrials.gov/ct2/show/NCT03528551?type=Intr&cond=hemophilia&lupd_s=03%2F08%2F2019&lupd_d=14

Conditions : Congenital Bleeding Disorder; Haemophilia A

Intervention : Drug: Turoctocog alfa pegol

Sponsor : Novo Nordisk A/S

Enrolling by invitation

A Research Study Looking at How a Factor VIII Medicine Called Turoctocog Alfa Pegol (N8-GP) Works in People With Haemophilia A

NCT03528551

Fri, 18 May 2018 12:00:00 EDT

Last Update Posted: 03/22/19 09:19AM

A Gene Therapy (SPK-9001) Study in Subjects with Hemophilia B

March 22, 2019

https://clinicaltrials.gov/ct2/show/NCT02484092?type=Intr&cond=hemophilia&lupd_s=03%2F08%2F2019&lupd_d=14

Condition : Hemophilia B

Intervention : Biological: SPK-9001

Sponsor : Pfizer

Active, not recruiting

A Gene Therapy Study for Hemophilia B

NCT02484092

Mon, 29 Jun 2015 12:00:00 EDT

A Phase 1/2, Open-Label, Non-Randomized, Dose-Escalation Study of SPK-9001 in Subjects with Hemophilia B

Last Update Posted: 03/22/19 09:19AM

Trial to Evaluate Safety and Efficacy of BAY81-8973 in Children With Severe Hemophilia A Under Prophylaxis Therapy

March 21, 2019

https://clinicaltrials.gov/ct2/show/NCT01311648?type=Intr&cond=hemophilia&lupd_s=03%2F07%2F2019&lupd_d=14

Condition : Haemophilia A

Intervention : Biological: Recombinant Factor VIII (BAY81-8973)

Sponsor : Bayer

Recruiting

BAY81-8973 Pediatric Safety and Efficacy Trial

NCT01311648

Wed, 09 Mar 2011 12:00:00 EST

Last Update Posted: 03/21/19 09:55AM

Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A

March 18, 2019

https://clinicaltrials.gov/ct2/show/NCT01775618?type=Intr&cond=hemophilia&lupd_s=03%2F04%2F2019&lupd_d=14

Condition : Hemophilia A

Intervention : Biological: BAY94-9027

Sponsor : Bayer

Active, not recruiting

Safety and Efficacy of BAY94-9027 in Previously Treated Male Children With Haemophilia A

NCT01775618

Fri, 25 Jan 2013 12:00:00 EST

Last Update Posted: 03/18/19 10:44AM

A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A

March 15, 2019

https://clinicaltrials.gov/ct2/show/NCT01580293?type=Intr&cond=hemophilia&lupd_s=03%2F01%2F2019&lupd_d=14

Condition : Hemophilia A

Intervention : Biological: BAY94-9027

Sponsor : Bayer

Active, not recruiting

A Trial Investigating Safety and Efficacy of Treatment With BAY94-9027 in Severe Hemophilia A

NCT01580293

Thu, 19 Apr 2012 12:00:00 EDT

Last Update Posted: 03/15/19 11:40AM

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

March 14, 2019

https://clinicaltrials.gov/ct2/show/NCT03569891?type=Intr&cond=hemophilia&lupd_s=02%2F28%2F2019&lupd_d=14

Condition : Hemophilia B

Intervention : Genetic: AAV5-hFIXco-Padua

Sponsor : UniQure Biopharma B.V.

Recruiting

HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients

NCT03569891

Tue, 26 Jun 2018 12:00:00 EDT

Last Update Posted: 03/14/19 09:49AM

FEIBA Reconstitution Volume Reduction and Faster Infusion Study (FEIBA STAR)

March 13, 2019

https://clinicaltrials.gov/ct2/show/NCT02764489?type=Intr&cond=hemophilia&lupd_s=02%2F27%2F2019&lupd_d=14

Condition : Hemophilia A or B With Inhibitors

Intervention : Biological: FEIBA

Sponsors : Baxalta now part of Shire; Baxalta Innovations GmbH, now part of Shire

Recruiting

FEIBA Reconstitution Volume Reduction and Faster Infusion Study (FEIBA STAR)

NCT02764489

Fri, 06 May 2016 12:00:00 EDT

Last Update Posted: 03/13/19 10:19AM

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

March 13, 2019

https://clinicaltrials.gov/ct2/show/NCT03734588?type=Intr&cond=hemophilia&lupd_s=02%2F27%2F2019&lupd_d=14

Conditions : Adeno-Associated Virus (AAV); Blood Coagulation Disorder; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Factor VIII (FVIII); Factor VIII (FVIII) Deficiency; Factor VIII (FVIII) Gene; Factor VIII (FVIII) Protein; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Gene Therapy; Gene Transfer; Hematologic Diseases; Hemorrhagic Disorders; Recombinant; Vector; Inhibitors

Intervention : Genetic: SPK-8016

Sponsor : Spark Therapeutics

Recruiting

Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors

NCT03734588

Thu, 08 Nov 2018 12:00:00 EST

Last Update Posted: 03/13/19 10:19AM

Human-cl-rhFVIII in Previously Untreated Patients

March 13, 2019

https://clinicaltrials.gov/ct2/show/NCT01712438?type=Intr&cond=hemophilia&lupd_s=02%2F27%2F2019&lupd_d=14

Condition : Severe Hemophilia A

Intervention : Biological: Human cl rhFVIII

Sponsor : Octapharma

Active, not recruiting

Human-cl-rhFVIII in Previously Untreated Patients

NCT01712438

Tue, 23 Oct 2012 12:00:00 EDT

Last Update Posted: 03/13/19 10:19AM

A Gene Transfer Study for Hemophilia A

March 13, 2019

https://clinicaltrials.gov/ct2/show/NCT03003533?type=Intr&cond=hemophilia&lupd_s=02%2F27%2F2019&lupd_d=14

Condition : Hemophilia A

Intervention : Genetic: SPK-8011

Sponsor : Spark Therapeutics

Recruiting

A Gene Transfer Study for Hemophilia A

NCT03003533

Wed, 28 Dec 2016 12:00:00 EST

Last Update Posted: 03/13/19 10:19AM

Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors

March 12, 2019

https://clinicaltrials.gov/ct2/show/NCT03597022?type=Intr&cond=hemophilia&lupd_s=02%2F26%2F2019&lupd_d=14

Condition : Hemophilia A and B

Intervention : Drug: BAY1093884

Sponsor : Bayer

Recruiting

Multiple Escalating Dose Study of BAY1093884 in Adults With Hemophilia A or B With or Without Inhibitors

NCT03597022

Tue, 24 Jul 2018 12:00:00 EDT

Last Update Posted: 03/12/19 09:58AM

Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants

March 11, 2019

https://clinicaltrials.gov/ct2/show/NCT03315455?type=Intr&cond=hemophilia&lupd_s=02%2F25%2F2019&lupd_d=14

Condition : Hemophilia A

Intervention : Drug: Emicizumab

Sponsor : Hoffmann-La Roche

Active, not recruiting

Efficacy, Safety, and Pharmacokinetic Study of Prophylactic Emicizumab Versus No Prophylaxis in Hemophilia A Participants

NCT03315455

Fri, 20 Oct 2017 12:00:00 EDT

Last Update Posted: 03/11/19 10:08AM

A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors

March 8, 2019

https://clinicaltrials.gov/ct2/show/NCT03191799?type=Intr&cond=hemophilia&lupd_s=02%2F22%2F2019&lupd_d=14

Condition : Hemophilia A

Intervention : Drug: Emicizumab

Sponsor : Hoffmann-La Roche

Active, not recruiting

A Study to Evaluate the Safety and Tolerability of Prophylactic Emicizumab in Hemophilia A Patients With Inhibitors

NCT03191799

Mon, 19 Jun 2017 12:00:00 EDT

Last Update Posted: 03/08/19 11:54AM

Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A

March 5, 2019

https://clinicaltrials.gov/ct2/show/NCT03061201

A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B (BENEGENE-2)

March 4, 2019

https://clinicaltrials.gov/ct2/show/NCT03861273

Factor VIII Gene Therapy Study in Patients With Hemophilia A

February 28, 2019

https://clinicaltrials.gov/ct2/show/NCT03588299?type=Intr&cond=hemophilia&lupd_s=02%2F20%2F2019&lupd_d=14

Condition : Hemophilia A

Intervention : Drug: BAY2599023 (DTX201)

Sponsors : Bayer; Ultragenyx Pharmaceutical Inc

Recruiting

Factor VIII Gene Therapy Study in Patients With Hemophilia A

NCT03588299

Tue, 17 Jul 2018 12:00:00 EDT

Last Update Posted: 03/06/19 06:39PM

A Study to Investigate the Safety, Tolerability, PK and PD of MG1113 in Healthy Subjects and Hemophilia Patients

February 27, 2019

https://clinicaltrials.gov/ct2/show/NCT03855696

PF-06741086 Long-term Treatment in Severe Hemophilia

February 27, 2019

https://clinicaltrials.gov/ct2/show/NCT03363321

Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX (ATLAS-OLE)

February 18, 2019

https://clinicaltrials.gov/ct2/show/NCT03754790

Hemophilia Inhibitor Prevention (INHIBIT) Trial (INHIBIT)

February 6, 2019

https://clinicaltrials.gov/ct2/show/NCT02196207