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Showing 1–24 of 25 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Factor VIII (FVIII) Gene Clear all
Withdrawn Phase 3 Interventional

Study of a Gene Therapy Treatment for Hemophilia A

NCT06297486
View directory record Official record
Conditions
Hemophilia A
Interventions
SPK-8011
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2035
U.S. locations
27
States / cities
Phoenix, Arizona • Loma Linda, California • Los Angeles, California + 23 more
Source: ClinicalTrials.gov public record
Updated Dec 12, 2024 · Synced Jul 22, 2026, 8:18 PM EDT
Recruiting Phase 1Phase 2 Interventional

A Gene Therapy Study of SPK-8011QQ in Adults With Severe or Moderately Severe Hemophilia A

NCT07226206
View directory record Official record
Conditions
Hemophilia A
Interventions
SPK-8011QQ
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
2
States / cities
Orange, California • Vallejo, California
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Jul 22, 2026, 8:18 PM EDT
Completed Phase 1Phase 2 Interventional Results available

A Gene Transfer Study for Hemophilia A

NCT03003533
View directory record Official record
Conditions
Hemophilia A
Interventions
SPK-8011
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2023
U.S. locations
11
States / cities
Sacramento, California • Gainesville, Florida • Boston, Massachusetts + 7 more
Source: ClinicalTrials.gov public record
Updated Dec 29, 2024 · Synced Jul 22, 2026, 8:18 PM EDT
Active, not recruiting Phase 2 Interventional

Long-term Safety and Efficacy Study and Dose-Escalation Substudy of PF 06838435 in Individuals With Hemophilia B

NCT03307980
View directory record Official record
Conditions
Hemophilia B
Interventions
PF-06838435 (formerly SPK-9001)
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
21 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2029
U.S. locations
17
States / cities
Sacramento, California • Metairie, Louisiana • New Orleans, Louisiana + 4 more
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced Jul 22, 2026, 8:18 PM EDT
Completed Phase 3 Interventional Results available

Study to Evaluate the Efficacy and Safety of Valoctocogene Roxaparvovec, With Prophylactic Steroids in Hemophilia A

NCT04323098
View directory record Official record
Conditions
Hemophilia A
Interventions
valoctocogene roxaparvovec
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older · Male only
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2025
U.S. locations
3
States / cities
Sacramento, California • St Louis, Missouri • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Oct 7, 2025 · Synced Jul 22, 2026, 8:18 PM EDT
Active, not recruiting Phase 3 Interventional Results available

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
View directory record Official record
Conditions
Hemophilia B
Interventions
PF-06838435/ fidanacogene elaparvovec
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
51 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2031
U.S. locations
17
States / cities
San Francisco, California • Aurora, Colorado • Indianapolis, Indiana + 3 more
Source: ClinicalTrials.gov public record
Updated May 31, 2026 · Synced Jul 22, 2026, 8:18 PM EDT
Active, not recruiting Phase 3 Interventional Results available

Study to Evaluate the Efficacy and Safety of PF-07055480 / Giroctocogene Fitelparvovec Gene Therapy in Moderately Severe to Severe Hemophilia A Adults

NCT04370054
View directory record Official record
Conditions
Hemophilia A
Interventions
PF-07055480 (giroctocogene fitelparovec): Recombinant AAV2/6 Human Factor VIII Gene Therapy
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 64 Years · Male only
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
19
States / cities
Mountain View, California • Oakland, California • Palo Alto, California + 5 more
Source: ClinicalTrials.gov public record
Updated May 17, 2026 · Synced Jul 22, 2026, 8:18 PM EDT
Recruiting Phase 3 Interventional

Safety and Effectiveness of Giroctocogene Fitelparvovec or Fidanacogene Elaparvovec in Patients With Hemophilia A or B Respectively

NCT05568719
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Testing of hepatic AAV Vector integration
Diagnostic Test
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older · Male only
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2040
U.S. locations
14
States / cities
Rancho Cordova, California • Sacramento, California • San Francisco, California + 5 more
Source: ClinicalTrials.gov public record
Updated May 26, 2026 · Synced Jul 22, 2026, 8:18 PM EDT
Enrolling by invitation No phase listed Observational

A Long-Term Follow-Up Study in Severe Hemophilia A Subjects Who Received BMN 270 in a Prior BioMarin Clinical Trial (270-401)

NCT05768386
View directory record Official record
Conditions
Hemophilia A
Interventions
Not listed
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older · Male only
Enrollment
172 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2040
U.S. locations
13
States / cities
Los Angeles, California • Sacramento, California • San Diego, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jan 11, 2026 · Synced Jul 22, 2026, 8:18 PM EDT
Not yet recruiting No phase listed Observational

Liver Biopsy Following Gene Therapy For Hemophilia

NCT05932914
View directory record Official record
Conditions
Hemophilia A, Hemophilia B
Interventions
Liver Biopsy
Procedure
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
18 Years to 80 Years · Male only
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Jun 7, 2026 · Synced Jul 22, 2026, 8:18 PM EDT
Completed Phase 2 Interventional Results available

A Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 (PF-07055480) in Subjects With Severe Hemophilia A

NCT03061201
View directory record Official record
Conditions
Hemophilia A
Interventions
SB-525 (PF-07055480)
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older · Male only
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
22
States / cities
Little Rock, Arkansas • Duarte, California • Sacramento, California + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 29, 2025 · Synced Jul 22, 2026, 8:18 PM EDT
Recruiting Phase 1Phase 2 Interventional

ASC618 Gene Therapy in Hemophilia A Patients

NCT04676048
View directory record Official record
Conditions
Hemophilia A
Interventions
ASC618
Biological
Lead sponsor
ASC Therapeutics
Industry
Eligibility
18 Years and older · Male only
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2026
U.S. locations
1
States / cities
Little Rock, Arkansas
Source: ClinicalTrials.gov public record
Updated Jan 31, 2023 · Synced Jul 22, 2026, 8:18 PM EDT
Terminated Phase 1Phase 2 Interventional Results available

Safety & Efficacy of Encapsulated Allogeneic FVIII Cell Therapy in Haemophilia A

NCT04541628
View directory record Official record
Conditions
Hemophilia A
Interventions
SIG-001
Combination Product
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2023
U.S. locations
3
States / cities
Indianapolis, Indiana • Boston, Massachusetts • Seattle, Washington
Source: ClinicalTrials.gov public record
Updated Sep 3, 2024 · Synced Jul 22, 2026, 8:18 PM EDT
Active, not recruiting No phase listed Observational

Long-Term Safety and Efficacy of Spark-Sponsored Gene Therapies in Males With Hemophilia A

NCT03432520
View directory record Official record
Conditions
Hemophilia A
Interventions
SPK-8011, SPK-8016
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
29 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2032
U.S. locations
9
States / cities
Boston, Massachusetts • Madison, Mississippi • Kansas City, Missouri + 5 more
Source: ClinicalTrials.gov public record
Updated Nov 28, 2024 · Synced Jul 22, 2026, 8:18 PM EDT
Completed Phase 3 Interventional Results available

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients at a Dose of 4E13 vg/kg

NCT03392974
View directory record Official record
Conditions
Hemophilia A
Interventions
Valoctocogene Roxaparvovec
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Oct 2, 2023 · Synced Jul 22, 2026, 8:18 PM EDT
Completed Phase 1Phase 2 Interventional Results available

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

NCT03734588
View directory record Official record
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
Interventions
SPK-8016
Genetic
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
11
States / cities
Los Angeles, California • Peoria, Illinois • Ann Arbor, Michigan + 7 more
Source: ClinicalTrials.gov public record
Updated Feb 22, 2024 · Synced Jul 22, 2026, 8:18 PM EDT
Active, not recruiting Phase 1 Interventional

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

NCT03818763
View directory record Official record
Conditions
Hemophilia A
Interventions
Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII
Biological
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
18 Years and older · Male only
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2033
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Jul 22, 2026, 8:18 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy for Haemophilia A.

NCT03001830
View directory record Official record
Conditions
Hemophilia A
Interventions
AAV2/8-HLP-FVIII-V3
Biological
Lead sponsor
University College, London
Other
Eligibility
18 Years and older · Male only
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2029
U.S. locations
3
States / cities
Boise, Idaho • Lexington, Kentucky • Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Mar 4, 2025 · Synced Jul 22, 2026, 8:18 PM EDT
Completed Phase 3 Interventional Results available

A Study to Evaluate Prospective Efficacy and Safety Data of Current FIX Prophylaxis Replacement Therapy in Adult Hemophilia B Subjects (FIX:C≤2%) or Current FVIII Prophylaxis Replacement Therapy in Adult Hemophilia A Subjects (FVIII:C≤1%)

NCT03587116
View directory record Official record
Conditions
Hemophilia B, Hemophilia A
Interventions
Standard of Care FIX Replacement therapy, Standard of Care FVIII Replacement therapy
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 64 Years · Male only
Enrollment
212 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
13
States / cities
Palo Alto, California • San Francisco, California • Stanford, California + 7 more
Source: ClinicalTrials.gov public record
Updated Nov 5, 2025 · Synced Jul 22, 2026, 8:18 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

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
View directory record Official record
Conditions
Hemophilia A
Interventions
BAY2599023 (DTX201)
Drug
Lead sponsor
Bayer
Industry
Eligibility
18 Years and older · Male only
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2026
U.S. locations
3
States / cities
Little Rock, Arkansas • Ann Arbor, Michigan • Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Jul 5, 2026 · Synced Jul 22, 2026, 8:18 PM EDT
Terminated Phase 1 Interventional

Hemophilia B Gene Therapy With AAV8 Vector

NCT01620801
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV8-hFIX19
Biological
Lead sponsor
Spark Therapeutics, Inc.
Industry
Eligibility
18 Years and older · Male only
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2016
U.S. locations
2
States / cities
Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 11, 2019 · Synced Jul 22, 2026, 8:18 PM EDT
Completed Phase 3 Interventional Results available

Single-Arm Study To Evaluate The Efficacy and Safety of Valoctocogene Roxaparvovec in Hemophilia A Patients (BMN 270-301)

NCT03370913
View directory record Official record
Conditions
Hemophilia A
Interventions
valoctocogene roxaparvovec
Biological
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
18 Years and older · Male only
Enrollment
144 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
15
States / cities
Los Angeles, California • Sacramento, California • San Diego, California + 12 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2025 · Synced Jul 22, 2026, 8:18 PM EDT
Completed Phase 3 Interventional Results available

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

NCT03569891
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV5-hFIXco-Padua, Factor IX (FIX)
Genetic · Biological
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older · Male only
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
19
States / cities
Phoenix, Arizona • Little Rock, Arkansas • Los Angeles, California + 14 more
Source: ClinicalTrials.gov public record
Updated Mar 26, 2026 · Synced Jul 22, 2026, 8:18 PM EDT
Completed Phase 2 Interventional Results available

Dose Confirmation Trial of AAV5-hFIXco-Padua

NCT03489291
View directory record Official record
Conditions
Hemophilia B
Interventions
AAV5-hFIXco-Padua (AMT-061)
Genetic
Lead sponsor
CSL Behring
Industry
Eligibility
18 Years and older · Male only
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
4
States / cities
Phoenix, Arizona • Sacramento, California • San Diego, California + 1 more
Source: ClinicalTrials.gov public record
Updated Jun 27, 2024 · Synced Jul 22, 2026, 8:18 PM EDT