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Gene Therapy Trial Report

Summary

AC6 Gene Transfer for CHF


NCTID NCT00787059 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Congestive Heart Failure
Disease Ontology Term DOID:9651
Compound Name RT-100
Compound Description Ad5.CMV.hAC6
Sponsor Hammond, H. Kirk, M.D.
Funder Type Indiv
Recruitment Status
Completed
Enrollment Count 56 (ACTUAL)
Results Posted Not Available

Therapy Information


Target Gene/Variant ADCY6
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Overexpression of protective allele/gene
Route of Administration Intracoronary
Drug Product Type Viral vector
Target Tissue/Cell Myocardium
Delivery System Viral transduction
Vector Type Ad5
Dose 1 3.2E9 vp (n=6)
Dose 2 3.2E10 vp (n=6)
Dose 3 1E10 vp (n=6)
Dose 4 3.2E11 vp (n=12)
Dose 5 1E12 vp (n=12)

Study Record Dates


Current Stage Phase1, Phase2
Submit Date 2008-11-06
Completion Date 2017-11-16
Last Update 2018-02-09

Participation Criteria


Eligible Age 18 Years - 80 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria 1. Male or non-pregnant female patients aged 18-80 years of age 2. ≥3-month history of heart failure 3. Compensated (stable) CHF not on intravenous inotropes, vasodilators or diuretics, on optimal medical and device therapy as defined by AHA/ACC Guidelines 4. LV ejection fraction (on optimal therapy) no greater than 40% 5. Implanted cardiac defibrillator 6. At least one major coronary artery (or graft) with \<50% proximal obstruction 7. Patients unable to walk (spinal injury, orthopedic problems) can be enrolled if all other criteria are met. 8. Women of child-bearing capacity must have a negative pregnancy test within 2 days of test substance administration, and female and male patients must be willing to use birth control during sex for 12w after test substance administration if the female partner is of child-bearing capacity. 9. Subjects willingly provide informed consent consistent with ICH-GCP guidelines Exclusion Criteria 1. Unstable or Class IV angina 2. Coronary revascularization planned or predicted in next 6 months 3. Ischemic myocardium in 3 or more regions of a single perfusion bed, as assessed by stress echocardiography or jeopardized viable myocardium \>15% on perfusion imaging. 4. ≥50% occlusion of an "unprotected" left main coronary artery. If arterial or venous conduits provide blood flow to the distal left coronary circulation (ie, patent bypass grafts) then left main disease is "protected" and such patients are not excluded. The cardiologist performing the cardiac catheterization will make these decisions. 5. 2° AV Block (Mobitz 2) or 3° AV block unless pacemaker is present 6. Hospitalization for CHF requiring intravenous inotropes or vasodilators in the past 4 weeks 7. History of biopsy proven myocarditis 8. Myocardial infarction in previous 6 months 9. Restrictive, hypertrophic or infiltrative cardiomyopathy or chronic pericarditis 10. Previous or planned organ transplant recipient or donor. 11. Thrombocytopenia (\<100,000 platelets/µl) or bleeding diathesis 12. COPD requiring supplemental oxygen at home 13. AST \> 2 times upper limit of normal or chronic liver disease such as cirrhosis or Hepatitis C Virus (HCV). Patients with HCV are eligible only if both of two conditions are met: a) liver function tests are normal; AND b) liver biopsy is normal or shows only mild fibrosis. 14. Current or predicted hemodialysis within 12 months or estimated glomerular filtration rate (EGFR) \<30 ml/min. On online EGFR calculator that uses sex, age, body weight and serum creatinine is available at: www.kidney.org/professionals/kdoqi/gfr\_calculator.cfm. Use the higher of two EGFR results, which are based upon MDRD and CKD-EPI formulas. 15. CVA or TIA \<6 months prior to enrollment 16. Patients who are immunosuppressed by medicines (corticosteroids, methotrexate, cyclophosphamide, cyclosporine), illnesses (AIDS, HIV), or neutrophil count \<1000/mm3 17. Patients receiving other investigational drug therapy within 30 days of enrollment including gene transfer 18. Patients with diseases other than CHF that, in the opinion of the investigator, put the subject at risk or adversely affect the results
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 7
Locations United States

Regulatory Information


Has US IND True
FDA Designations Fast Track
Recent Updates Company is preparing to submit a plan to the FDA for the Phase 2b/3 study

Resources/Links