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Trial record 3 of 18 for:    sangamo

Ascending Dose Study of Genome Editing by Zinc Finger Nuclease Therapeutic SB-FIX in Subjects With Severe Hemophilia B

This study is currently recruiting participants.
See Contacts and Locations
Verified January 2017 by Sangamo Therapeutics
Sponsor:
Information provided by (Responsible Party):
Sangamo Therapeutics
ClinicalTrials.gov Identifier:
NCT02695160
First received: February 24, 2016
Last updated: January 25, 2017
Last verified: January 2017
  Purpose
The purpose of the study is to evaluate the safety, tolerability and effect on FIX antigen and activity levels of ascending doses of SB-FIX. SB-FIX is an intravenously delivered Zinc Finger Nuclease (ZFN) Therapeutic for genome editing. It inserts a correct copy of the Factor 9 gene into the albumin locus in hepatocytes with the goal of lifelong therapeutic production of the Factor IX clotting factor.

Condition Intervention Phase
Hemophilia B Biological: SB-FIX Phase 1

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: A Phase I, Open-Label, Ascending Dose Study to Assess the Safety and Tolerability of AAV2/6 Factor IX Gene Therapy Via Zinc Finger Nuclease (ZFN) Mediated Targeted Integration of SB-FIX in Adult Subjects With Severe Hemophilia B

Resource links provided by NLM:


Further study details as provided by Sangamo Therapeutics:

Primary Outcome Measures:
  • Treatment related Adverse Events in subjects who received SB-FIX as assessed by Common Terminology Criteria for Adverse Events (CTCAE) [ Time Frame: Up to 36 months after the SB-FIX infusion ]

Secondary Outcome Measures:
  • Change from baseline in FIX antigen and activity levels [ Time Frame: up to 36 months after the SB-FIX infusion ]
  • Presence and shedding of AAV2/6 vector DNA by PCR in plasma, saliva, urine, stool and semen [ Time Frame: Follow up until 2 consecutive measurements are negative of AAV ]

Estimated Enrollment: 12
Actual Study Start Date: June 2016
Estimated Study Completion Date: January 2021
Estimated Primary Completion Date: January 2019 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Cohort 1
SB-FIX: Low Dose
Biological: SB-FIX
Single dose of each of the 3 components of SB-FIX: ZFN1, ZFN2 and cDNA Donor.
Experimental: Cohort 2
SB-FIX: Medium Dose
Biological: SB-FIX
Single dose of each of the 3 components of SB-FIX: ZFN1, ZFN2 and cDNA Donor.
Experimental: Cohort 3
SB-FIX: High Dose
Biological: SB-FIX
Single dose of each of the 3 components of SB-FIX: ZFN1, ZFN2 and cDNA Donor.

Detailed Description:
The objective of the study is to provide long term expression of Factor IX in subjects with severe hemophilia B. SB-FIX is a therapeutic for ZFN-mediated genome editing which will be delivered by adeno-associated virus (AAV)-derived vectors. SB-FIX is intended to function by placement of a corrective copy of the Factor IX transgene into the genome of the subject's own hepatocytes, under the control of the highly expressed endogenous albumin locus, and is expected to provide permanent, liver-specific expression of Factor IX for the lifetime of a hemophilia B subject.
  Eligibility

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 B (native circulating FIX activity <1%, with or without cross reactive material)

Exclusion Criteria:

  • Presence of neutralizing antibodies
  • History of hypersensitivity response or an allergic reaction to FIX or FIX products
  • Currently receiving long acting FIX replacement therapy
  • FIX mutations known to be associated with FIX inhibitors
  • Polymorphisms in the ZFN target region
  • Presence of any liver mass on MRI, or elevated alpha-fetoprotein (AFP)
  • Any contraindication to the use of corticosteroids for immunosuppression
  • Currently receiving antiviral therapy for hepatitis B or C or with history or active hepatitis B or hepatitis C or HIV-1 or HIV1/2 antibody positive.
  • Chronic anemia, leukopenia, or thrombocytopenia
  • Past medical history of active tuberculosis or significant fungal disease
  • Symptomatic cardiovascular disease as a co-morbid condition
  • Markers of hepatic inflammation or overt or occult cirrhosis
  • History of chronic renal disease or creatinine ≥ 1.5 mg/dL
  • Systemic (iv or oral) immunomodulatory agent or steroid use (topical treatment is allowed)
  • History of chronic infection or other chronic disorder considered an unacceptable risk
  • History of malignancy except for treated basal cell or squamous cell carcinoma
  • History of alcohol or substance abuse
  • Previously received gene therapy product
  • Participation in prior investigational drug or medical device study within the previous 3 months
  • History of therapeutic non-adherence
  • Any other reason that, in the opinion of the Investigator or Medical Monitor, would render the subject unsuitable for participation in the study
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02695160

Contacts
Contact: Medical Monitor clinicaltrials@sangamo.com

Locations
United States, California
Recruiting
Duarte, California, United States, 91010
Recruiting
Los Angeles, California, United States, 90007
Recruiting
San Francisco, California, United States, 94115
United States, Michigan
Recruiting
Detroit, Michigan, United States, 48202
Sponsors and Collaborators
Sangamo Therapeutics
Investigators
Study Director: Medical Monitor Sangamo BioSciences, Inc.
  More Information

Responsible Party: Sangamo Therapeutics
ClinicalTrials.gov Identifier: NCT02695160     History of Changes
Other Study ID Numbers: SB-FIX-1501
Study First Received: February 24, 2016
Last Updated: January 25, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked

ClinicalTrials.gov processed this record on July 21, 2017