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 | Completed Phase 3 Results FDA Drug

• 2 other identifiers: C03710042017-001271-23
• Study Type: interventional
• Target: 212
• Locations: 18 countries
• Sites: 75

Brief Summary

To establish baseline prospective efficacy data of current FIX prophylaxis replacement therapy in the usual care setting of hemophilia B subjects, who are negative for nAb to AAV-Spark100, prior to the Phase 3 gene therapy study. To establish baseline prospective efficacy data of current FVIII prophylaxis replacement therapy in the usual care setting of hemophilia A subjects, who are negative for nAb to AAV6, prior to the Phase 3 gene therapy study. The enrollment for hemophilia A participants is completed. At this time participants are only being enrolled for hemophilia B cohort.

Conditions

Hemophilia B; Hemophilia A

Outcome Measures

Primary Outcomes (10)

• Annualized Bleeding Rate (ABR) for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Efficacy Analysis Set

  ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study multiplication(\*)365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia B participants per efficacy analysis set was reported. Abbreviations used: AAV6 = adeno-associated virus 6; AAV-Spark100 = Bioengineered AAV capsid, derived from a naturally occurring AAV serotype; nAb = neutralizing antibodies.

  During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

• ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Per-protocol Analysis Set

  ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia B participants per protocol analysis set was reported.

  During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

• ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set

  ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia B participants per protocol amendment 5 analysis set was reported.

  During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

• ABR for Treated Bleeds and All Bleeds During Retrospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set

  ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during retrospective data collection period in hemophilia B participants per protocol amendment 5 analysis set was reported.

  During retrospective data collection period (12 months before screening collected in the hemophilia history case report form [CRF])

• ABR for Treated Bleeds and All Bleeds From the Combined Retrospective and Prospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set

  ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during retrospective and prospective data collection period in hemophilia B participants per protocol amendment 5 analysis set was reported.

  From start of retrospective data collection period (12 months before screening collected in hemophilia history CRF) up to end of prospective data collection follow-up of period (maximum follow-up:1269 days), for a total of approximately 4.5 years

• ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Efficacy Analysis Set

  ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia A participants per efficacy analysis set was reported.

  During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

• ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Per-protocol Analysis Set

  ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia A participants per protocol analysis set was reported.

  During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

• ABR for Treated Bleeds and All Bleeds During Prospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set

  ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during prospective data collection period in hemophilia A participants per protocol amendment 5 analysis set was reported.

  During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 948 days])

• ABR for Treated Bleeds and All Bleeds During Retrospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set

  ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during retrospective data collection period in hemophilia A participants per protocol amendment 5 analysis set was reported.

  During retrospective data collection period (12 months before screening collected in the hemophilia history CRF)

• ABR for Treated Bleeds and All Bleeds From the Combined Retrospective and Prospective Data Collection Period in Hemophilia A Participants: Protocol Amendment 5 Analysis Set

  ABR per participant was calculated as number of bleeds over number of days from baseline visit (Day 1 of the study) to end of study\*365.25 days. All bleeds included treated and untreated bleeds. A treated bleed was defined as an event necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. An untreated bleed was defined as an event not necessitating administration of coagulation factor within 72 hours of signs or symptoms of bleeding. In this outcome measure, ABR for all and treated bleeds during retrospective and prospective data collection period in hemophilia A participants per protocol amendment 5 analysis set was reported.

  From start of retrospective data collection period (12 months before screening collected in hemophilia history CRF) up to end of prospective data collection follow-up of period (maximum follow-up:948 days), for a total of approximately 3.6 years

Secondary Outcomes (24)

• Annualized Infusion Rate (AIR) During Prospective Data Collection Period in Hemophilia B Participants: Efficacy Analysis Set

  During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

• AIR During Prospective Data Collection Period in Hemophilia B Participants: Per-protocol Analysis Set

  During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

• AIR During Prospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set

  During prospective data collection period (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

• AIR During Retrospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set

  During retrospective data collection period (12 months before screening collected in the hemophilia history CRF)

• AIR From the Combined Retrospective and Prospective Data Collection Period in Hemophilia B Participants: Protocol Amendment 5 Analysis Set

  From start of retrospective data collection period (12 months before screening collected in hemophilia history CRF) up to end of prospective data collection follow-up of period (maximum follow-up:1269 days), for a total of approximately 4.5 years

Other Outcomes (2)

• Number of Participants With Serious Adverse Events (SAEs)

  During prospective data collection period: Day 1 through end of study visit (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

• Number of Participants With Adverse Events of Special Interest (AESIs)

  During prospective data collection period: Day 1 through end of study visit (from baseline visit of the study [Day 1] up to end of study follow-up [maximum follow-up: 1269 days])

Study Arms (2)

Standard of Care FIX replacement therapy

OTHER

Drug: Standard of Care FIX Replacement therapy

Standard of Care FVIII replacement therapy

OTHER

Drug: Standard of Care FVIII Replacement therapy

Interventions

Standard of Care FIX Replacement therapy DRUG

There is no investigational product being administered. Subjects will be administering their own standard of care FIX replacement therapy.

Standard of Care FIX replacement therapy

Standard of Care FVIII Replacement therapy DRUG

There is no investigational product being administered. Subjects will be administering their own standard of care FVIII replacement therapy.

Standard of Care FVIII replacement therapy

Eligibility Criteria

• Age: 18 Years - 64 Years
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Hemophilia B Population:
• Evidence of a signed and dated informed consent document indicating that the participant has been informed of all pertinent aspects of the study.
• Willing and able to comply with scheduled visits, FIX prophylaxis treatment plan, laboratory tests and other study procedures.
• Males ≥ 18 and \<65 years of age with moderately severe to severe hemophilia B and documented FIX activity (≤2%) prior to baseline visit.
• Previous experience with FIX therapy (≥50 documented exposure days to a FIX protein product such as recombinant, plasma-derived or extended half-life FIX product).
• Participants on FIX prophylaxis replacement therapy (recombinant, plasma-derived or extended half-life FIX product) must have the intention to remain on FIX prophylaxis replacement therapy for the duration of the study.
• No known hypersensitivity to FIX replacement product.
• No history of FIX inhibitor (clinical or laboratory-based assessment) defined as a titer
• BU/mL, regardless of the laboratory normal range, or any measured Bethesda inhibitor titer greater than the upper limit of normal for the laboratory performing the assay. Clinically, no signs or symptoms of decreased response to FIX administration. Participants will not be required to undergo diagnostic evaluation of inhibitor status to participate in the study.
• Hemophilia A Population:
• Evidence of a signed and dated informed consent document indicating that the participant has been informed of all pertinent aspects of the study.
• Willing and able to comply with scheduled visits, FVIII prophylaxis treatment plan, laboratory tests and other study procedures.
• Males ≥18 and \<65 years of age with moderately severe to severe hemophilia A and documented FVIII activity (≤1%) prior to baseline visit.
• Previous experience with FVIII therapy (≥150 documented exposure days to a FVIII protein product such as recombinant, plasma-derived or extended half-life FVIII product).
• Participants must be on a stable FVIII prophylaxis replacement therapy (recombinant, plasma-derived or extended half-life FVIII product) at study entry and must have the intention to remain on FVIII prophylaxis replacement therapy for the duration of the study. This does not include nonfactor treatments, which are prohibited.

You may not qualify if:

• Anti-AAV-Spark100 neutralizing antibody titer above the established threshold performed by a central laboratory during screening in hemophilia B subjects or Anti-AAV6 neutralizing antibody titer above the established threshold performed by a central laboratory during screening in hemophilia A subjects.
• Lack of participant compliance with documentation of bleeds and/or prophylaxis replacement therapy administration.
• If there is no documentation regarding hepatitis status, as defined below, within the last 12 months prior to screening for hepatitis B and 6 months prior to screening for hepatitis C, then participants will be required to have the following hepatitis testing performed at screening:
• Hepatitis B screening (acute and chronic):
• HBsAg (also referred to as Hepatitis B surface antigen), HBV-DNA viral assay (also referred to as a nucleic acid test for Hepatitis B virus DNA), and Anti-HBc (also referred to as Total Hepatitis B core antibody).
• A participant is not eligible if either HbsAg is positive or HBV-DNA is positive/detectable.
• Anti-HBc must be obtained in all participants for determination of whether the participant had prior hepatitis B. If the anti-HBc is positive and both HBsAg and HBV DNA are negative this would be consistent with a prior infection and the subject would be eligible for the study. Anti-HBc must be obtained in all subjects to discriminate between those with no prior hepatitis B and those with prior infection in the event of reactivation. FDA has noted reactivation of hepatitis B virus exists.
• One documented negative HBV-DNA viral load is sufficient to assess eligibility. A participant who is currently undergoing anti-viral therapy for hepatitis B is not eligible.
• Hepatitis C (acute or chronic):
• A participant who is currently undergoing anti-viral therapy for chronic hepatitis C is not eligible.
• Participants treated with anti-viral therapy for chronic hepatitis C, must have completed anti-viral therapy at least 6 months prior to screening and have a negative HCV-RNA at least 6 months prior to screening.
• All participants (who are not currently undergoing anti-viral therapy for chronic hepatitis C) must have a single HCV-RNA load assay (also referred to as a nucleic acid test \[NAT\] for HCV RNA) obtained during the 6 months preceding screening. This includes participants with prior known chronic hepatitis C who have completed treatment with anti-viral therapy.
• A participant is not eligible if his HCV-RNA load assay result is positive/detectable.
• Currently on antiviral therapy for hepatitis B or C.
• Significant liver disease, as defined by pre-existing diagnosis of portal hypertension, splenomegaly, or hepatic encephalopathy.

Sponsors & Collaborators

• Pfizer: lead

Study Sites (75)

Clinical and Translational Research Unit (CTRU)

Palo Alto, California, 94304, United States

Location

Lucile Packard Childrens Hospital

Palo Alto, California, 94304, United States

Location

University of California, San Francisco - Outpatient Hematology Clinic

San Francisco, California, 94143, United States

Location

Stanford Health Care

Stanford, California, 94305, United States

Location

Hemophilia and Thrombosis Center at the University of Colorado Anschutz Medical Campus

Aurora, Colorado, 80045, United States

Location

Emory University Hospital Midtown

Atlanta, Georgia, 30308, United States

Location

Emory University Hospital

Atlanta, Georgia, 30322, United States

Location

Investigational Drug Service

Atlanta, Georgia, 30322, United States

Location

Indiana Hemophilia & Thrombosis Center, Inc.

Indianapolis, Indiana, 46260, United States

Location

Mississippi Center for Advanced Medicine

Madison, Mississippi, 39110, United States

Location

Alliance for Childhood Diseases

Las Vegas, Nevada, 89135, United States

Location

Penn Blood Disorder Center

Philadelphia, Pennsylvania, 19104, United States

Location

Bloodworks NW

Seattle, Washington, 98104, United States

Location

Royal Prince Alfred Hospital

Camperdown, New South Wales, 2050, Australia

Location

Royal Brisbane and Women's Hospital

Herston, Queensland, 4029, Australia

Location

Royal Adelaide Hospital

Adelaide, South Australia, 5000, Australia

Location

The Alfred Hospital

Melbourne, Victoria, 3004, Australia

Location

Fiona Stanley Hospital

Murdoch, Western Australia, 6150, Australia

Location

Cliniques universitaires Saint-Luc/Unité d'Hématology et Hémostase

Brussels, 1200, Belgium

Location

Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - Hemoes

Vitória, Espírito Santo, 29047-105, Brazil

Location

Centro de Hemoterapia e Hematologia do Para - Fundação HEMOPA

Belém, Pará, 66033-000, Brazil

Location

Centro de Hematologia e Hemoterapia - Hemocentro de Campinas - UNICAMP

Campinas, São Paulo, 13083-878, Brazil

Location

Hospital Das Clínicas Da Faculdade de Medicina de Ribeirao Preto Da Universidade de Sao Paulo

Ribeirão Preto, São Paulo, 14051-140, Brazil

Location

lnstituto Estadual de Hematologia Arthur de Siqueira Cavalcanti - HEMORIO

Rio de Janeiro, 20211-030, Brazil

Location

Hospital das Clinicas da Faculdade de Medicina da Universidade de Sao Paulo

São Paulo, 05403-000, Brazil

Location

McMaster University Medical Centre - Hamilton Health Sciences

Hamilton, Ontario, L8N 3Z5, Canada

Location

St. Michael's Hospital

Toronto, Ontario, M5B 1W8, Canada

Location

CHRU de Brest - La Cavale Blanche

Brest, 29200, France

Location

Hôpital Cardiologique Louis Pradel

Bron, 69677, France

Location

CHU Hôtel-Dieu

Nantes, 44093, France

Location

Hopital Necker, Hematologie Adultes

Paris, 75015, France

Location

Vivantes Klinikum Friedrichshain

Berlin, 10249, Germany

Location

Universitatsklinikum Bonn. Anstalt des oeffentlichen Rechts

Bonn, 53127, Germany

Location

Universitaetsklinikum Giessen

Giessen, 35392, Germany

Location

Universität und Universitätsklinikum des Saarlandes

Homburg/Saar, 66421, Germany

Location

General Hospital of Athens "Hippokration"

Athens, Attica, 11527, Greece

Location

General Hospital of Athens "LAIKO", 2nd Regional Blood Transfusion Center

Athens, Attica, 11527, Greece

Location

The Chaim Sheba Medical Center, The National Hemophilia Center

Tel Litwinsky, 5262000, Israel

Location

IRCCS - AOU di Bologna, Policlinico di Sant'Orsola

Bologna, BO, 40138, Italy

Location

UOC Medicina Interna - Malattie Emorragiche e Trombotiche

Napoli, Naples, 80131, Italy

Location

SODc. Malattie Emorragiche e della Coagulazione Centro di Riferimento Regionale per le

Florence, 50134, Italy

Location

Università degli studi di Roma "La Sapienza"- Policlinico Umberto I

Roma, 00161, Italy

Location

Nagoya University Hospital - Transfusion Medicine

Nagoya, Aichi-ken, 466-8560, Japan

Location

Sapporo Tokushukai Hospital

Sapporo, Hokkaido, 004-0041, Japan

Location

Nara Medical University Hospital

Kashihara, Nara, 634-8522, Japan

Location

Saitama Medical University Hospital

Iruma-gun, Saitama, 350-0495, Japan

Location

National Center for Child Health and Development

Setagaya-ku, Tokyo, 157-8535, Japan

Location

Ogikubo Hospital

Suginami-ku, Tokyo, 167-0035, Japan

Location

King Abdulaziz Medical City

Riyadh, 11426, Saudi Arabia

Location

King Faisal Specialist Hospital & Research Center

Riyadh, Saudi Arabia

Location

Kyungpook National University Hospital

Daegu, 41944, South Korea

Location

Severance Hospital, Yonsei University Health System

Seoul, 03722, South Korea

Location

Kyung Hee University Hospital At Gangdong

Seoul, 05278, South Korea

Location

Hospital Universitario Virgen de la Arrixaca

El Palmar, Murcia, 30120, Spain

Location

Hospital de la Santa Creu i Sant Pau

Barcelona, 08025, Spain

Location

Hospital Universitari Vall d´Hebrón

Barcelona, 08035, Spain

Location

H.U. La Paz.

Madrid, 28046, Spain

Location

H.U. Rio Hortega

Valladolid, 47012, Spain

Location

Skåne University Hospital

Malmo, 205 02, Sweden

Location

Changhua Christian Hospital

Changhua, 500, Taiwan

Location

Kaohsiung Medical University Chung-Ho Memorial Hospital

Kaohsiung City, 807, Taiwan

Location

Chung Shan Medical University Hospital

Taichung, 40201, Taiwan

Location

Taichung Veterans General Hospital

Taichung, 40705, Taiwan

Location

National Taiwan University Hospital

Taipei, 10002, Taiwan

Location

Ege Universitesi Tip Fakultesi Cocuk Sagligi Ve Hastaliklari Anabilim Dali Pediatric Hematoloji

Bornova, İ̇zmir, 35040, Turkey (Türkiye)

Location

Ege Universitesi Tip Fakultesi Hematoloji BD

Bornova, İ̇zmir, 35040, Turkey (Türkiye)

Location

Acibadem Adana Hospital, Department of Pediatric Hematology

Adana, 01130, Turkey (Türkiye)

Location

Akdeniz University Medical Faculty Hospital

Antalya, 07070, Turkey (Türkiye)

Location

Gaziantep University Sahinbey Training and Research Hospital

Gaziantep, 27310, Turkey (Türkiye)

Location

Istanbul University Oncology Institute

Istanbul, 34093, Turkey (Türkiye)

Location

Newcastle upon Tyne Hospitals NHS Foundation Trust

Newcastle upon Tyne, TYNE & WEAR, NE1 4LP, United Kingdom

Location

Non Malignant Haematology Research Unit

Newcastle upon Tyne, TYNE & WEAR, NE1 4LP, United Kingdom

Location

Clinical Research Facility

Glasgow, G31 2ER, United Kingdom

Location

Department of Haematology

Glasgow, G4 0SF, United Kingdom

Location

Guy's and St Thomas' NHS Foundation Trust

London, SE1 7EH, United Kingdom

Location

Related Links

• To obtain contact information for a study center near you, click here.

MeSH Terms

Conditions

Hemophilia B; Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Results Point of Contact

• Title: Pfizer ClinicalTrials.gov Call Center
• Organization: Pfizer Inc.

ClinicalTrials.gov.Inquiries@pfizer.com

1-800-718-1021

Study Officials

• Pfizer CT.gov Call Center

  Pfizer

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: The data obtained from this lead-in study will serve as the control group for the subsequent Phase 3 gene therapy study.

Study Record Dates

• First Submitted: June 29, 2018
• First Posted: July 16, 2018
• Study Start: July 26, 2018
• Primary Completion: December 13, 2024
• Study Completion: December 13, 2024
• Last Updated: November 6, 2025
• Results First Posted: November 6, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will share

Locations

TU (6); GB (5); DE (4); FR (4); SA (2); BR (6); KR (3); BE (1); SE (1); US (13); TW (5); CA (2); JP (6); IL (1); ES (5); AU (5); IT (4); GR (2)