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Trial record 1 of 1 for:    SB-525-1603
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Dose-Ranging Study of Recombinant AAV2/6 Human Factor 8 Gene Therapy SB-525 in Subjects With Severe Hemophilia A

This study is currently recruiting participants.
Verified August 2017 by Sangamo Therapeutics
Sponsor:
ClinicalTrials.gov Identifier:
NCT03061201
First Posted: February 23, 2017
Last Update Posted: October 23, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Sangamo Therapeutics
  Purpose
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.

Condition Intervention Phase
Hemophilia A Biological: SB-525 Phase 1 Phase 2

Study Type: Interventional
Study Design: Intervention Model: Sequential Assignment
Intervention Model Description:
Dose selection based on safety and observed FVIII levels.
Masking: None (Open Label)
Masking Description:
Open Label
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Sangamo Therapeutics:

Primary Outcome Measures:
  • Number of treatment related adverse events as assessed by laboratory assessments and vital signs [ Time Frame: Up to 3 years after SB-525 infusion ]
  • Changes in circulating FVIII activity [ Time Frame: Up to 3 years after SB-525 infusion ]

Estimated Enrollment: 20
Actual Study Start Date: June 2, 2017
Estimated Study Completion Date: January 30, 2022
Estimated Primary Completion Date: November 15, 2021 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Sequential dose escalation
SB-525 is administered as a single infusion
Biological: SB-525
Single dose of investigational product 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.

  Eligibility

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03061201


Contacts
Contact: Medical Monitor 510.307.7266 clinicaltrials@sangamo.com

Locations
United States, Arkansas
Arkansas Children's Hospital Recruiting
Little Rock, Arkansas, United States, 19406
Contact: Jocelyn Wright    501-364-2391    WrightJJ@archildrens.org   
United States, California
City of Hope National Medical Center Recruiting
Duarte, California, United States, 91010
Contact: Janice Kim, CCRP    626-218-4350    jankim@coh.org   
United States, Florida
University of Miami Recruiting
Miami, Florida, United States, 33136
Contact: Leandro Pisani    305-243-6925    lfp34@miami.edu   
United States, Georgia
Emory University, Dept. of Hematology Recruiting
Atlanta, Georgia, United States, 30322
Contact: Stephanie Whitten    404-686-0277    stephanie.whitten@emoryhealthcare.org   
United States, Pennsylvania
Hemophilia Center of Western PA Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Margaret Ragni, MD, MPH    412-209-7263    JKadosh@itxm.org   
United States, Washington
Bloodworks Recruiting
Seattle, Washington, United States, 98104
Contact: Barbara Konkle, MD    206-689-6191    BarbaraK@BloodworksNW.org   
Sponsors and Collaborators
Sangamo Therapeutics
Investigators
Study Director: Medical Monitor Sangamo Therapeutics, Inc.
  More Information

Responsible Party: Sangamo Therapeutics
ClinicalTrials.gov Identifier: NCT03061201     History of Changes
Other Study ID Numbers: SB-525-1603
First Submitted: February 15, 2017
First Posted: February 23, 2017
Last Update Posted: October 23, 2017
Last Verified: August 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants