Registered clinical trials of gene therapy in haemophilia
The following clinical trials of gene therapy in haemophilia are currently registered on ClinicalTrials.gov and/or the EU Clinical Trials Register
| Title
|
ClinicalTrials.gov Identifier
|
Phase | Haemophilia | Vector/Transgene | Status* | Sponsor | Dates | Patients |
| An open label dose-escalation study of a self-complementary adeno-associated viral vector (scAAV 2/8-LP1-hFIXco) for gene transfer in hemophilia B
(N Engl J Med 2011;365:2357-65; N Engl J Med 2014;371:1994-2004) |
NCT00979238 | I | B | scAAV 2/8-LP1-hFIXco | Active, not recruiting | St Jude Children’s Research Hospital | Start Feb 2010
Completion Dec 2018 (2032 for secondary outcomes)
|
N = 14 |
| A Phase I/II, open-label, uncontrolled, single-dose, dose-ascending, multi-centre trial investigating an adeno-associated viral vector containing a codon-optimized human factor IX gene (AAV5-hFIX) administered to adult patients with severe or moderately severe hemophilia B
(Blood 2018;131:1022-1031) |
NCT02396342 | I/II | B | AAV5-hFIX
(AMT-060) |
Active, not recruiting | UniQure | Start May 2015
Completion 2021 |
N = 10 |
| A Phase 1/2, dose-escalation safety, tolerability and efficacy study of BMN 270, an adenovirus-associated virus vector-mediated gene transfer of human factor VIII in patients with severe haemophilia A
(N Engl J Med 2017;377:2519-30) |
NCT02576795 | I/II | A | valoctocogene roxaparvovec | Active, not recruiting | Biomarin | Start August 2015
Completion 2022 |
N = 15 |
| Gene therapy, open-label, dose-escalation study of SPK-9001 [adeno-associated viral vector with human factor IX gene] in subjects with hemophilia B
(N Engl J Med 2017;377:2215-27) |
NCT02484092 | II | B | fidanacogene elaparvovec
(SPK-9001) |
Active, not recruiting | Spark | Start November 2015
Completion 2019 |
N = 15 |
| Dose-finding Study of SPK-8016 Gene Therapy in Patients With Hemophilia A to Support Evaluation in Individuals With FVIII Inhibitors | NCT03734588 | I/II | A | SPK-8016 | Recruiting | Spark
|
Start 2016
Completion 2019 |
N = 30 |
| A Phase I/II open-label safety and dose-finding study of adeno-associated virus (AAV) rh10-mediated gene transfer of human factor IX in adults with moderate/severe to severe hemophilia B
|
NCT02618915 | I/II | B | DTX101
(now known as BAY2599023) |
Terminated
(not due to safety concerns) |
Dimension | Start 2016
Ended 2017 |
N = 6 elderly |
|
GO-8: Gene therapy for haemophilia A using a novel serotype 8 capsid pseudotyped adeno-associated viral vector encoding factor VIII-V3
(product is licensed to Biomarin) |
NCT03001830 | I | A | AAV2/8-HLP-FVIII-V3 | Recruiting | University College, London
St Jude Children’s Research Hospital
|
Start 2017
Completion 2019 (2034 for secondary outcomes)
|
N = 18 |
| Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A (also known as Alta)
|
NCT03061201 | II | A | giroctocogene fitelparvovec
(SB-525, PF-07055480) |
Recruiting | Pfizer | Start 2017
Completion July 2024 |
N = 13 |
| A Factor IX Gene Therapy Study (FIX-GT, also known as B-AMAZE)
|
NCT03369444 | I | B | FLT180a
(AAVS3 vector) |
Recruiting | University College, London
Freeline
|
Start 2017
Completion 2021
|
N = 24 |
| A Phase 3 open-label, single-arm study to evaluate the efficacy and safety of BMN 270, an adeno-associated virus vector-mediated gene transfer of human factor VIII in hemophilia A patients with residual FVIII levels ≤ 1 IU/dL receiving prophylactic FVIII infusions(also known as GENEr8-1,BMN 270-301) ) | NCT03370913 | III | A | valoctocogene roxaparvovec
6E13 vg/kg |
Recruiting | Biomarin | Start Dec 2017
Completion 2022 (2023 for secondary outcomes) |
N = 134 |
| Phase 3 study to evaluate efficacy/safety of valoctocogene roxaparvovec an AAV vector-mediated gene transfer of hFVIII at a dose of 4E13vg/kg in hemophilia A patients with residual FVIII levels ≤1IU/dL receiving prophylactic FVIII infusions
(also known as GENEr8-2, BMN270-302)
|
NCT03392974 | III | A | valoctocogene roxaparvovec
4E13vg/kg |
Recruiting
|
Biomarin | Start March 2018
Completion 2022 (2024 for secondary outcomes)
|
N = 40 |
| Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A (GENEr8-3)
|
NCT04323098 | III | A | valoctocogene roxaparvovec
6E13 vg/kg with prophylactic corticosteroids
|
Not yet recruiting | Biomarin | Start June 2020
Completion December 2025 |
N = 20 |
| A Phase 1/2 safety, tolerability, and efficacy study of valoctocogene roxaparvovec, an adeno-associated virus vector-mediated gene transfer of human factor VIII in hemophilia A patients with residual FVIII levels ≤ 1 IU/dL and pre-existing antibodies against AAV5
|
NCT03520712 | I/II | A | valoctocogene roxaparvovec
6E13 vg/kg |
Enrolling by invitation | Biomarin | Start May 2018
Completion 2024 |
N = 10 |
| Phase III, open-label, single-dose, multi-center, multinational trial investigating a serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene (AAV5-hFIXco-Padua, AMT-061) administered to adult subjects with severe or moderately severe hemophilia B (HOPE-B)
|
NCT03569891 | III | B | etranacogene dezaparvovec
(AAV5-hFIXco-Padua, AMT-061) |
Recruiting | UniQure | Start June 2018 (first dose early 2019)
Completion 2020 (2024 for secondary outcomes) |
N = 56 |
| Phase IIb, open-label, single-dose, single-arm, multi-center trial to confirm the factor IX activity level of the serotype 5 adeno-associated viral vector containing the Padua variant of a codon-optimized human factor IX gene (AAV5-hFIXco-Padua, AMT-061) administered to adult subjects with severe or moderately severe hemophilia B | NCT03489291 | IIb | B | etranacogene dezaparvovec
(AAV5-hFIXco-Padua, (AMT-061) |
Active, not recruiting | UniQure | Start July 2018
Completion Oct 2018 (2023 for secondary outcomes) |
N = 3 |
| A long-term follow-up study of haemophilia B patients who have undergone gene therapy | NCT03641703 | II/III | B | FLT180a | Recruiting | Freeline | Start July 2018
Complete 2035 |
N = 50 |
| A multi-center evaluation of the long-term safety and efficacy of SPK-8011 [adeno-associated viral vector with B-domain deleted human factor VIII gene] in males with hemophilia A | NCT03432520 | Observational cohort | A | SPK-8011 | Enrolling by invitation | Spark | Start Aug 2018
Completion 2022 |
N = 100 |
| Dose-finding study of SPK-8016 gene therapy in patients with hemophilia A to support evaluation in individuals with FVIII inhibitors | NCT03734588 | I/II | A | SPK-8016 | Active, not recruiting | Spark | Start Nov 2018
Completion 2020 |
N = 30 |
| Study to test the safety and how well patients with severe hemophilia a respond to treatment with BAY 2599023 (DTX 201), a drug therapy that delivers a healthy version of the defective factor viii gene into the nucleus of liver cells using an altered, non-infectious virus (AAV) as a “shuttle”. | NCT03588299 | I/II | A | BAY2599023 (DTX201) | Recruiting | Bayer | Start Nov 2018
Completion 2022 (2026 for secondary outcomes)
|
N = 30 |
| Study to Evaluate the Efficacy and Safety of PF-07055480 in Moderately Severe to Severe Hemophilia A Adults (AFFINE) | NCT04370054 | III | A | giroctocogene fitelparvovec
(SB-525, PF-07055480) |
Recruiting | Pfizer | Started August 2020
Completion October 2026
|
N = 63 |
| BENEGENE 2. 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. | NCT03861273 | III | B | fidanacogene elaparvovec | Recruiting | Pfizer, Spark | Started July 2019 | N = 55 |
| Hematopoietic stem cell transplantation gene therapy for treatment of severe hemophilia A. (ET3-201) | NCT04418414 | I | A | CD68-ET3 lentiviral vector | Not yet recruiting (Sept 2020) | Expression Therapeutics | February 2021 | N = 7 |