You are here

Our science / Clinical Trials / Find a Trial / NCT03061201

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

Back to Search Print Save as PDF Bookmark Trial

NCT03061201

Last updated on March 31, 2020
Back to Search Print Save as PDF Bookmark Trial

FOR MORE INFORMATION
Study Location
Arkansas Children's Hospital
Little Rock, Arkansas, 72202 United States
See other locations
Contact
1-800-718-1021
[email protected]
Related Links
Eligibility criteria
Condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Hemophilia A
Sex
Male
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
18 + years
Inclusion criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

- Male ≥18 years of age

- Severe hemophilia A (past evidence of circulating FVIII activity of

- Treated or exposed to FVIII concentrates or cryoprecipitate for at least 150 exposure
days

- ≥12 bleeding episodes if receiving on-demand therapy over the preceding 12 months

- Agree to use double barrier contraceptive until at least 3 consecutive semen samples
are negative for AAV 2/6 after SB-525 infusion

Exclusion criteria
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details

- Presence of neutralizing antibodies

- Current inhibitor, or history of FVIII inhibitor (except for transient low titer
inhibitor detected in childhood)

- History of hypersensitivity response to FVIII

- History of Hepatitis B or HIV-1/2 infection

- History of Hepatitis C, unless viral assays in two samples, collected at least 6
months apart, are negative

- Evidence of any bleeding disorder in addition to hemophilia A

- Markers of hepatic inflammation or overt or occult cirrhosis

- History of chronic renal disease or creatinine ? 1.5 mg/dL

- Presence of liver mass on magnetic resonance imaging (MRI), or, positive alpha
fetoprotein

- Presence of > grade 2 liver fibrosis on elastography for subjects with history of
treated Hepatitis C or suspicion of chronic liver disease

NCT03061201
Pfizer
Recruiting
Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A

NEED INFO?

Questions about a trial? Call or email to reach a Pfizer Clinical Trial Contact Center Representative

Pfizer Clinical Trials Contact Center

1-800-718-1021

[email protected]

call now

Try a new search

Search for Clinical Trials by condition, keyword or trial number. Share your location or enter your city or zip code to find studies near you.
Advanced Search

Based on your search, you may also be interested in

Hemophilia A, Hemophilia B
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Patients With Severe Hemophilia A or B
NCT03938792
Males
12+
Years
Multiple Sites
Hemophilia B, Hemophilia A
Six Month lead-in 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 (FIX:C≤1%)
NCT03587116
Males
18+
Years
Multiple Sites
Hemophilia A
Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A
NCT03061201
Males
18+
Years
Multiple Sites
Hemophilia A or B
PF-06741086 Long-term Treatment in Severe Hemophilia
NCT03363321
Males
12+
Years
Multiple Sites
Descriptive Information
Brief Title  ICMJE Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A
Official Title  ICMJE A Phase 1/2, Open-Label, Adaptive, Dose-Ranging Study to Assess the Safety and Tolerability of SB-525 (Recombinant AAV2/6 Human Factor 8 Gene Therapy) in Adult Subjects With Severe Hemophilia A
Brief Summary The purpose of the study is to evaluate the safety, tolerability and time-course profile of FVIII activity levels with adaptive doses of SB-525.
Detailed Description

The objective of the study is to reduce or eliminate the need for FVIII replacement therapy. The proposed SB-525 clinical study uses a recombinant adeno-associated virus 2/6 (AAV2/6) vector encoding the cDNA for the B-domain deleted human F8 (hF8). The secreted FVIII has the same amino acid sequence as approved recombinant anti hemophilic factors (Refacto® and Xyntha®). The SB-525 vector encodes a liver-specific promotor module and AAV2/6 exhibits liver tropism, thus providing the potential for long-term hepatic production of FVIII in hemophilia A subjects.

The constant production of FVIII after a single SB-525 administration may provide potential benefit in durable protection against bleeding and the complications thereof without lifelong repetitive IV factor replacement administration.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Intervention Model: Parallel Assignment
Intervention Model Description:
Dose selection based on safety and observed FVIII levels.

Masking: None (Open Label)
Masking Description:

Open Label

Primary Purpose: Treatment

Condition  ICMJE Hemophilia A
Intervention  ICMJE Biological: SB-525
Single dose of investigational product SB-525
Study Arms  ICMJE Experimental: Sequential dose escalation
SB-525 is administered as a single infusion
Intervention: Biological: SB-525
Publications * Not Provided


*   Includes publications given by the data provider as well as publications
identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 25, 2020) 		13
Original Estimated Enrollment  ICMJE
 (submitted: February 17, 2017) 		20
Estimated Study Completion Date  ICMJE July 24, 2024
Estimated Primary Completion Date July 24, 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • Male ?18 years of age
  • Severe hemophilia A (past evidence of circulating FVIII activity of < 1% normal)
  • Treated or exposed to FVIII concentrates or cryoprecipitate for at least 150 exposure days
  • ?12 bleeding episodes if receiving on-demand therapy over the preceding 12 months
  • Agree to use double barrier contraceptive until at least 3 consecutive semen samples are negative for AAV 2/6 after SB-525 infusion

Exclusion Criteria:

  • Presence of neutralizing antibodies
  • Current inhibitor, or history of FVIII inhibitor (except for transient low titer inhibitor detected in childhood)
  • History of hypersensitivity response to FVIII
  • History of Hepatitis B or HIV-1/2 infection
  • History of Hepatitis C, unless viral assays in two samples, collected at least 6 months apart, are negative
  • Evidence of any bleeding disorder in addition to hemophilia A
  • Markers of hepatic inflammation or overt or occult cirrhosis
  • History of chronic renal disease or creatinine ? 1.5 mg/dL
  • Presence of liver mass on magnetic resonance imaging (MRI), or, positive alpha fetoprotein
  • Presence of > grade 2 liver fibrosis on elastography for subjects with history of treated Hepatitis C or suspicion of chronic liver disease
Sex/Gender  ICMJE
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Medical Pfizer CT.gov Call Center 1-800-718-1021 [email protected]
Listed Location Countries  ICMJE United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03061201
Other Study ID Numbers  ICMJE SB-525-1603
C3731001 ( Other Identifier: Alias Study Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD: Yes
Plan Description: Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/da....
URL: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/da...
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director: Pfizer CT.gov Call Center Pfizer
PRS Account Pfizer
Verification Date February 2020

ICMJE     Data element required by the

International Committee of Medical Journal Editors and the
World Health Organization ICTRP

FOR MORE INFORMATION

Contact a representative by phone, email, or visiting the study website. Please see the references below:

BY PHONE

Pfizer Clinical Trials Contact Center

1-800-718-1021

BY EMAIL

Contact

[email protected]

Call Now