Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector For Gene Transfer in Hemophilia B
|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. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT00979238|
Recruitment Status : Active, not recruiting
First Posted : September 17, 2009
Last Update Posted : May 15, 2023
- Study Details
- Tabular View
- No Results Posted
- How to Read a Study Record
|Condition or disease||Intervention/treatment||Phase|
|Hemophilia B||Genetic: Gene Transfer Drug: scAAV2/8-LP1-hFIXco||Phase 1|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||14 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||An Open Label Dose-Escalation Study Of A Self Complementary Adeno-Associated Viral Vector (scAAV 2/8-LP1-hFIXco) For Gene Transfer in Hemophilia B|
|Actual Study Start Date :||February 22, 2010|
|Estimated Primary Completion Date :||June 12, 2032|
|Estimated Study Completion Date :||December 31, 2032|
All participants who meet the eligibility requirements.
Intervention: Gene Transfer and drug (scAAV2/8-LP1-hFIXco).
Genetic: Gene Transfer
Peripheral vein infusion of scAAV2/8-LP1-hFIXco vector once per participant
Peripheral vein infusion of scAAV2/8-LP1-hFIXco vector once per participant.
Other Name: vector
- To assess the safety of systemic administration of a novel self-complementary AAV vector in adults with severe hemophilia B at up to four different dosage levels. [ Time Frame: 15 years ]Outcome measures are descriptive in nature but data collected in a longitudinal manner will also be analyzed (as and when appropriate) using longitudinal methods such as mixed effect model which takes into account the correlation among the observation taken at various time points within a participant.
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, Learn About Clinical Studies.
|Ages Eligible for Study:||18 Years and older (Adult, Older Adult)|
|Sexes Eligible for Study:||Male|
|Accepts Healthy Volunteers:||No|
- Males ≥ 18 years of age with established severe HB (FIX:C<1u/dl),
- Treated/exposed to FIX products (e.g., concentrates or fresh frozen plasma) for at least 10 years or 50 exposure days.
- A minimum of an average of 3 bleeding episodes per year requiring FIX infusions or prophylactic FIX infusions because of frequent prior bleeding episodes
- Able to give informed consent and comply with requirements of the trial
- Currently free of inhibitor and have no history of inhibitors to FIX protein
- A negative family history for the development of an inhibitor,
- Willing to practice a reliable barrier method of contraception until 3 sequential samples are negative for vector genomes using our PCR assay.
- Evidence of active infection with Hepatitis B or C virus as reflected by HBsAg or NCV RNA positivity, respectively. To be considered negative for active infection, two negative assays at a minimum of a six month interval are required.
- Exposure to Hepatitis B or C who are currently on antiviral therapy.
Serological evidence of HTLV or active HIV infection. Individuals who are effectively being treated with antiretroviral therapy are eligible. Specific criteria for effectiveness of treatment include the following:
- Documented CD4+ T-cell count of > 350 cells/mm^3.
- HIV-1 RNA viral load < 400 copy/ml for at least the past 12 months, including at least 2 viral load test results of < 400 copy/ml during the immediate 12 month interval prior to screening.
- Screening HIV-RNA viral load < 400 copies/ml.
- Stable HAART regimen (drugs of at least 2 different classes) for at least 12 months prior to study entry. Treatment regimen changes for dosing convenience and in response to toxicity are permitted.
- Documented and confirmed (repeated) viral loads of ≥ 400 copies/ml during the 12 month time interval prior to screening are bases for exclusion although a single, unconfirmed, "glimpse" of ≥ 400 copies/ml are permitted.
- Significant liver dysfunction as defined by an abnormal ALT (alanine transaminase), bilirubin, alkaline phosphatase or INR. Potential participants who have had a liver biopsy in the past 3 years will be excluded if they have significant fibrosis of 3 or 4 as rated on a scale of 0-4.
- Coronary artery disease as a co-morbid condition
- Platelet count of <50 x 10^9/l
- Creatinine ≥ 1.5 mg/dl
- Hypertension with systolic blood pressure (BP) ≥ 140 mmHg or diastolic BP ≥ 90 mmHg
- History of active tuberculosis, fungal disease or other chronic infection
- History of chronic disease that would adversely affect performance other than hemophilic arthropathy
- Detectable antibodies reactive with AAV8
- Subjects who are unwilling to provide the required semen samples
- Poor performance status (WHO performance status score >1) or
- Received an AAV vector or any other gene transfer agent in the previous 6 months
- Presence of lung nodule(s) suspicious of malignancy on screening chest tomography
- Presence of liver abnormalities suspicious of malignancy on screening liver ultrasound
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): NCT00979238
|United States, California|
|Stanford Medical School|
|Stanford, California, United States, 94305|
|United States, Kentucky|
|University of Kentucky|
|Lexington, Kentucky, United States, 40536|
|United States, Oregon|
|Oregon Health and Science University|
|Portland, Oregon, United States, 97239|
|United States, Pennsylvania|
|Hemophilia Center of Western Pennsylvania|
|Pittsburgh, Pennsylvania, United States, 15213|
|United States, Tennessee|
|St. Jude Children's Research Hospital|
|Memphis, Tennessee, United States, 38119|
|United States, Texas|
|University of Texas Southwestern|
|Dallas, Texas, United States, 75390-9063|
|Scott and White Memorial Hospital|
|Temple, Texas, United States, 76508|
|Katharine Dormandy Haemophilia Centre and Haemostasis Unit, University College of London|
|London, United Kingdom|
|Principal Investigator:||Ulrike Reiss, MD||St. Jude Children's Research Hospital|
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
|Responsible Party:||St. Jude Children's Research Hospital|
|Other Study ID Numbers:||
5R01HL094396 ( U.S. NIH Grant/Contract )
|First Posted:||September 17, 2009 Key Record Dates|
|Last Update Posted:||May 15, 2023|
|Last Verified:||May 2023|
|Studies a U.S. FDA-regulated Drug Product:||Yes|
|Studies a U.S. FDA-regulated Device Product:||No|
Factor IX Gene
Factor IX Protein
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Coagulation Protein Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked