I know a lot of my friends have Ushers. Thought I would pass this along to see if anyone was interested in participating in the study. Study of UshStat in Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B This study is currently recruiting participants. (see Contacts and Locations) Verified June 2014 by Sanofi Sponsor: Sanofi Information provided by (Responsible Party): Sanofi ClinicalTrials.gov Identifier: NCT01505062 First received: January 4, 2012 Last updated: June 24, 2014 Last verified: June 2014 History of Changes · Full Text View · Tabular View · No Study Results Posted · Disclaimer · How to Read a Study Record Purpose The purpose of this first in man study is to examine the safety of an experimental gene transfer agent, UshStat designed to treat retinitis pigmentosa associated with Usher Syndrome Type 1B. Condition Intervention Phase Usher Syndrome Retinitis Pigmentosa Genetic: UshStat Phase 1 Phase 2 Study Type: Interventional Study Design: Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment Official Title: A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected UshStat, Administered to Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B Resource links provided by NLM: Genetics Home Reference related topics: Lenz microphthalmia syndrome nonsyndromic deafness oculofaciocardiodental syndrome Pendred syndrome retinitis pigmentosa Usher syndrome MedlinePlus related topics: Usher Syndrome Genetic and Rare Diseases Information Center resources: Cone-rod Dystrophy Cone-rod Dystrophy 2 Pigmentary Retinopathy Retinitis Pigmentosa Retinitis Pigmentosa-deafness Syndrome Usher Syndrome U.S. FDA Resources Further study details as provided by Sanofi: Primary Outcome Measures: · The incidence of adverse events [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ] The number and percentage of patients with treatment emergent adverse events. Secondary Outcome Measures: · To determine delay in retinal degeneration. [ Time Frame: 48 weeks ] [ Designated as safety issue: No ] Changes in function relative to the contralateral eye utilizing retinal analytical techniques. Estimated Enrollment: 18 Study Start Date: January 2012 Estimated Study Completion Date: January 2017 Estimated Primary Completion Date: January 2017 (Final data collection date for primary outcome measure) Intervention Details: Genetic: UshStat Single dose, 3 ascending dose cohorts, followed by two maximum tolerated dose cohorts Detailed Description: Following screening procedures the gene transfer agent will be injected once only under one retina by an opthalmic surgeon under anesthesia. Patients will then have regular follow up visits where general health examinations, blood tests and ophthalmic examinations including best corrected visual acuity, slit lamp examination, intraocular pressure, fundoscopy, autofluorescence, Optical Coherence Tomography, perimetry and Electroretinogram will be undertaken. Eligibility Ages Eligible for Study: 18 Years and older Genders Eligible for Study: Both Accepts Healthy Volunteers: No Criteria Inclusion Criteria: · Clinical and molecular diagnosis of Retinitis Pigmentosa associated with Usher Syndrome type 1B. With at least one pathogenic mutation in the MYO7A gene on each chromosome. · Suitable verbal, auditory,written and/or tactile sign language communication as to allow informed consent to be obtained. · Women of childbearing potential must agree to use two forms of contraception. · Males must agree to use two forms of contraception if their study partner is of child-bearing potential for three months after treatment. · Affiliated with the French social security healthcare system (french patients only) Exclusion Criteria: · Presence of significant ocular abnormalities that would preclude surgery, effective study follow up or interfere with study endpoints. · Concomitant systemic diseases that can alter visual function. · Contraindication to pupil dilation, anesthesia or eye surgery. · Periocular steroids in the last four months prior to screen. · Known allergies to the components of the delivery vehicle or diagnostic agents such as dilation drops. · Life-threatening illness or a history of malignancy within 5 years · Laboratory test abnormalities that would make the patient unsuitable for participation in the study · Intercurrent illnesses or infections 28 days prior to treatment · Concurrent anti-retroviral or immunosuppressant therapy · Men or women who do not agree to use contraception as specified in the inclusion criteria. · Pregnant or breastfeeding women · Enrollment in any other clinical study during the 48 week study period · Treatment with anticoagulants · Past history of HIV or hepatitis A, B or C · Inability to comply with the demands of the study · Any ocular surgery in the study eye within 6 months 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: NCT01505062 Contacts Contact: Richard Weleber, MD 503-494-7887 Locations United States, Oregon Oregon Health & Science University Recruiting Portland, Oregon, United States, 97239-4197 Principal Investigator: Richard Weleber, MD France Central Hospitalier Nationale dOpthalmolgie des Quinze-Vingts Recruiting Paris, France, 75571 Principal Investigator: Jose Sahel, MD Sponsors and Collaborators Sanofi Investigators Principal Investigator: Richard Weleber, MD Casey Eye Institute, Portland, Oregon Principal Investigator: Jose-Alain Sahel, MD, PhD Hopital Nationale des Quinze-Vingt, Paris France More Information No publications provided by Sanofi Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number): Zallocchi M, Binley K, Lad Y, Ellis S, Widdowson P, Iqball S, Scripps V, Kelleher M, Loader J, Miskin J, Peng YW, Wang WM, Cheung L, Delimont D, Mitrophanous KA, Cosgrove D. EIAV-based retinal gene therapy in the shaker1 mouse model for usher syndrome type 1B: development of UshStat. PLoS One. 2014 Apr 4;9(4):e94272. doi: 10.1371/journal.pone.0094272. eCollection 2014. Responsible Party: Sanofi ClinicalTrials.gov Identifier: NCT01505062 History of Changes Other Study ID Numbers: TDU13600, US1/001/10 Study First Received: January 4, 2012 Last Updated: June 24, 2014 Health Authority: United States: Food and Drug Administration Keywords provided by Sanofi: Usher Syndrome Retinitis Pigmentosa Usher Syndrome associated Retinitis Pigmentosa Additional relevant MeSH terms: Syndrome Usher Syndromes Retinitis Retinitis Pigmentosa Abnormalities, Multiple Blindness Congenital Abnormalities Deaf-Blind Disorders Deafness Disease Ear Diseases Eye Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Hearing Disorders Hearing Loss Hearing Loss, Sensorineural Nervous System Diseases Neurologic Manifestations Otorhinolaryngologic Diseases Pathologic Processes Retinal Degeneration Retinal Diseases Retinal Dystrophies Sensation Disorders Signs and Symptoms Vision Disorders ClinicalTrials.gov processed this record on November 17, 2014
Posted on: Sun, 23 Nov 2014 00:34:54 +0000
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