RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa. Clinical Trial on X-Linked Retinitis Pigmentosa: rAAV2tYF ... Epub 2020 Feb 24. Natural History of the Progression of X-Linked Retinitis Pigmentosa. Association between the number of visual fields and the ... Am J Ophthalmol 2004; 137:704-18. Early results from a first-in-human retinal gene therapy trial for X-linked retinitis pigmentosa indicate that, at an intermediate dose, AAV8-RPGR is safe and in a subset of patients can lead to . Title: AAV-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa: 6-month Results From a Phase 1/2 Clinical Trial Presenter: Michel Michaelides, UCL Institute of Ophthalmology . Applied Genetic Technologies Corporation (AGTC) reported positive data from its ongoing phase 1/2 clinical program in patients with X-linked retinitis pigmentosa (XLRP). This is a Phase 1/2 multicenter study with two parallel parts: an observational natural history cohort and an open-label, prospective interventional trial in males with non-syndromic X-linked retinitis pigmentosa (XLRP) due to mutations in retinitis pigmentosa GTPase regulator (RPGR).. Clinical Trials Registry. The SCENIC Clinical Trials: Now Enrolling. A total of 18 patients took part in the six-month Phase I/II dose escalation clinical trial for X-linked retinitis pigmentosa (RP) caused by the RPGR gene mutation, which blocks production of a . All ages Under 18 Over 18. The purpose of this study is to learn about a new investigational gene therapy that may help patients . Conditions: X-Linked Retinitis Pigmentosa. X-Linked Retinitis Pigmentosa Caused by Non-Canonical Splice Site Variants in. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). 70% of XLRP cases are due to mutations in the retina-specific isoform of the gene encoding retinitis pigmentosa GTPase regulator (RPGR ORF15).Despite successful RPGR ORF15 gene replacement with adeno-associated viral (AAV) vectors being established in a . Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene. Promising Results From Ongoing Phase 1/2 Clinical Trial for X-Linked Retinitis Pigmentosa (XLRP) The Retina Foundation of the Southwest is one of four U.S. centers conducting a Phase 1/2 dose escalation, gene replacement … AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Most affected males show symptomatic night blindness before the age of 10 years, are often myopic and show fundus abnormalities and ERG changes in early childhood. A randomized, placebo-controlled clinical trial of docosahexaenoic acid supplementation for X-linked retinitis pigmentosa. Expert opinion. Retinitis Pigmentosa clinical trials at UCSF . X-Linked Retinitis Pigmentosa (RPGR) Gene Therapy Trial. - AGTC to regain rights to five clinical and preclinical development programs; Biogen terminates collaboration agreement - - AGTC on schedule to advance its Phase 1/2 clinical studies in achromatopsia and X-linked retinitis pigmentosa; three data releases expected in 2019 - - AGTC to host conference call and webcast today at 5:00 p.m. ET- X-linked Retinitis Pigmentosa (XLRP) The Company is currently conducting the Skyline expanded Phase 1/2 XLRP and Vista Phase 2/3 XLRP clinical trials to support a potential filing of a Biologics License Application (BLA). Visionary science for life-changing cures. Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR. Gene therapies for X-linked retinitis pigmentosa. Purpose: A clinical trial of AAV5-RPGR vector for patients with X-linked retinitis pigmentosa (XLRP) Site approved (Likely to be enrolling in the next 6 months) Achromatopsia (Genes: CNGA3, CNGB3) New trial for blindness rewrites the genetic code. With the RPGR gene therapy clinical trials still in the early stages, the confirmation of the safety, tolerability and potency of the therapy is . Read about the causes, diagnosis, and treatment of retinitis pigmentosa and about current research. Conditions: X-Linked Retinitis Pigmentosa. NCT02556736. Women have two X-chromosomes and so a normal RPGR gene on . All ages Under 18 Over 18. Visual sensitivity, visual acuity and safety data were reported for 12-month timepoints for Groups 2 and 4, and 6-month time points for Groups 5 and 6. open to eligible people ages 7 years and up . Keywords X-Linked Retinitis Pigmentosa RP GTPase regulator (RPGR) Degenerative retinal disease XLRP Natural history Rare disease Retinitis Retinitis Pigmentosa Other: Assessments Subgroup 1 Subgroup 2 Nat Med. In the present study, we report the first-in-human phase 1/2, dose-escalation clinical trial for X-linked RP caused by mutations i … AGTC | 4,005 followers on LinkedIn. Active, not recruiting. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHMB3 and ACHMA3). Company reiterates that its favorable safety profile and its advanced manufacturing and analytics capabilities enable rapid clinical development. 4D-125 was . | AGTC is a clinical-stage biotechnology company that uses its proprietary gene therapy platform to develop products . The purpose of this study is to evaluate subjects with X-linked retinitis pigmentosa caused by RPGR-ORF15 mutations in a clinical setting to fully characterize their condition, measure testing variability, and estimate rates of progression of clinical parameters.. Clinical Trials Registry. Natural History of the Progression of X-Linked Retinitis Pigmentosa. 2020 Mar;26(3):354-359. doi: 10.1038/s41591-020-0763-1. 7 in progress, 3 open to eligible people . Gene therapy is the process of injecting a healthy gene to replace a damaged or mutated one. A randomized, placebo-controlled clinical trial of docosahexaenoic acid supplementation for X-linked retinitis pigmentosa. The Natural History of the Progression of X-Linked Retinitis Pigmentosa (XOLARIS) study focuses on patients with retinitis pigmentosa (RP) inherited in an X-linked fashion (XLRP). open to eligible people ages 7 years and up . Active, not recruiting. Three doses of the gene therapy will be tested. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Recruiting. Gene therapy is the process of injecting a healthy gene to replace a damaged or mutated one. Retinitis pigmentosa (RP) is a group of rare, inherited disorders that involve a breakdown and loss of cells in the retina. Retinal gene therapy has shown great promise in treating retinitis pigmentosa (RP), a primary photoreceptor degeneration that leads to severe sight loss in young people 1,2,3,4,5,6.Here we report the first in human Phase I/II dose escalation clinical trial for X-linked RP caused by mutations in the RP GTPase regulator (RPGR) gene 7 in 18 patients up to 6 months follow-up (Clinicaltrials.gov . Background: In a 4-year placebo-controlled trial to elevate blood docosahexaenoic acid levels in patients with X-linked retinitis pigmentosa (XLRP), the goal was to assess the potential benefit of docosahexaenoic acid supplementation in altering disease progression. Hughbanks-Wheaton DK, Birch DG, Fish GE, Spencer R, Pearson NS, Takacs A, Hoffman DR. Safety assessment of docosahexaenoic acid in X-linked retinitis pigmentosa: the 4-year DHAX trial. 2014 Jul 11;55(8):4958-66. doi: 10.1167/iovs.14-14437. ICH GCP. NCT04794101. The mode of inheritance in a particular family is determined by evaluating the family history and, in some instances, by molecular genetic testing.There are many potential complications in interpreting the family history, so in some cases, identifying the responsible gene with genetic . On September 26th, the clinical trial sponsor, Applied Genetic Technologies Corporation (AGTC) announced the initial results. About X-linked Retinitis Pigmentosa (XLRP) XLRP is an inherited condition that causes boys to develop night blindness by the time they are ten and progresses to legal blindness by their early forties. 45 NCT03116113 A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa BIIB-112 Recruiting 63 AAV8 Yes Yes Subretinal Ophthalmology Phase 2/3 01/08/2020 46 NCT01344798 Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C AAV1-gamma-sarcoglycan The clinical characteristics of XLRP include diminished visual acuity, progressive night blindness in early childhood due to loss of rod photoreceptor function, tunnel vision . The goals of this study were to elevate blood DHA levels and determine the effect on the rate of disease progression. The Retina Foundation of the Southwest is one of four U.S. centers conducting a Phase 1/2 dose escalation, gene replacement trial for x-linked retinitis pigmentosa (XLRP). This 2-year, observational study provides extremely valuable information on the clinical features, disease progression rates, and demographic and physiological . Int J Mol Sci. Our pre-clinical programs build on our industry leading AAV manufacturing technology and expertise. Gene therapy rescues photoreceptor blindness in dogs and paves the way for treating human X-linked retinitis pigmentosa. NCT02556736. AGTC has active clinical trials in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHMB3 and ACHMA3). X-linked retinitis pigmentosa (XLRP) is an inherited retinal disease causing significant vision loss, sometimes complete blindness, in males. Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Approximately 15 patients will be enrolled in the study, which is primarily evaluating safety. What is X-linked Retinitis Pigmentosa? 4D Molecular Therapeutics Presents Interim Results from the Ongoing 4D-125 Phase 1/2 Clinical Trial in Patients with Advanced X-linked Retinitis Pigmentosa at the ASRS Annual Meeting. 11 in progress, 5 open to eligible people . The investigational gene therapy (AGTC-501) is designed to replace the mutated RPGR . In XLRP, both rods and cones function poorly, leading to degeneration of the retina and total blindness. X-linked retinitis pigmentosa (XLRP) is an orphan retinal degenerative disease with a frequency in the population of approximately 1 in 100 000, 1,2 resulting in about 3000 affected individuals in the United States. Seventy-eight male patients diagnosed as having X-linked retinitis pigmentosa were randomized to DHA or placebo. X-linked Retinitis Pigmentosa (XLRP) is a severe form of RP with early onset of nyctalopia and progression to legal blindness by the 3rd to 4th decade. US Clinical Trials Registry; Clinical Trials Nct Page; Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene Follow-up Phase 3 Randomized, Controlled Study of AAV5-RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene This condition is caused by a mutation in the RPGR gene and typically affects men. Initially focusing on ophthalmology, our goal is to preserve or, hopefully, be able to improve vision in some cases. July 22, 2020 at 7:00 AM EDT PDF Version. Conditions: X-Linked Retinitis Pigmentosa. Design, setting, and participants: A 4-year, single-site, randomized, placebo-controlled, double-masked phase 2 clinical trial at a research center specializing in medical retina. AGTC has active clinical trials in X-linked retinitis pigmentosa and achromatopsia (ACHM CNGB3 & ACHM CNGA3). With the RPGR gene therapy clinical trials still in the NEW YORK and LONDON, Aug. 24, 2017 /PRNewswire/ -- MeiraGTx, a New York and London based gene therapy company, announced today the first patient in its clinical study for X-Linked Retinitis . In October, 2020, sponsorship of the trial was transferred to Biogen. Am J Ophthalmol 2004; 137:704-18. However, docosahexaenoic acid (22:6omega3) is a highly unsaturated fatty acid and considered a target molecule for free-radical . Click to Access Audio Press Release. Purpose: Low docosahexaenoic acid (DHA) in X-linked retinitis pigmentosa (XLRP) may influence retinal function. RARITAN, NJ, July 17, 2020 - The Janssen Pharmaceutical Companies of Johnson & Johnson announced today six-month data from the ongoing Phase 1/2 trial (NCT03252847) of its investigational gene therapy for the treatment of inherited retinal disease X-linked retinitis pigmentosa (XLRP).The interim data showed that low and intermediate doses of the . Retinitis Pigmentosa clinical trials at University of California Health . AGTC Announces Updated Development Plan for its X-Linked Retinitis Pigmentosa Clinical Program, Including Q1 2021 Start of Planned Phase 2/3 Trial. Codon optimisation has overcome the challenge of designing an RPGR vector without mutations, and with a therapeutic effect in different animal models. ICH GCP. An Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1- RPGR) in Subjects with X-linked Retinitis Pigmentosa caused by RPGR-ORF15 mutations. RPGR. 2021 Jan 16;22 (2):850. doi: 10.3390/ijms22020850. Its pre-clinical programs build on the Company's industry leading AAV manufacturing . A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With X-linked Retinitis Pigmentosa Caused by RPGR Mutations A Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated . RST-001 Phase I/II Trial for Advanced Retinitis Pigmentosa. This is a Phase 1/2 multicenter study with two parallel parts: an observational natural history cohort and an open-label, prospective interventional trial in males with non-syndromic X-linked retinitis pigmentosa (XLRP) due to mutations in retinitis pigmentosa GTPase regulator (RPGR). X-linked retinitis pigmentosa (XLRP) is an incurable genetic disease that causes blindness in men, and affects approximately one in 15,000 people.The disease is caused by a defect in the RPGR gene which is located on the X-chromosome, and this is why the disease affects men and women differently. Common symptoms include trouble seeing at night and a loss of side (peripheral) vision. Retinitis pigmentosa (RP) can be inherited in an autosomal dominant, autosomal recessive, or X-linked manner. In about 10% of RP cases, the non-working gene is passed down from the mother to her children resulting in a form of RP known as X-Linked RP (XLRP). Retinitis pigmentosa (RP) describes a group of rare genetic eye diseases that damage light-sensitive cells in the retina, leading to loss of sight over time. Title: Phase 1/2 Clinical Trial of AAV-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa: 9-month Results Presenter : Michel Michaelides, BSc MB BS MD(Res) FRCOphth FACS The Company's most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. The mutation accounts for approximately 70 percent of all cases of X-linked retinitis pigmentosa, which causes night blindness in early childhood followed by progressive daytime vision loss. A Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR Gene Recruiting. Title: Phase 1/2 Clinical Trial of AAV-RPGR Gene Therapy for RPGR-Associated X-Linked Retinitis Pigmentosa: 9-month Results Presenter: Michel Michaelides, BSc MB BS MD(Res) FRCOphth FACS Date and . Inherited Eye Diseases October 1, 2019. All Female Male . Applied Genetic Technologies Corporation (AGTC) is now recruiting for its Phase 1 / 2 gene therapy clinical trial for males with X-linked retinitis pigmentosa (XLRP) caused by mutations in the gene RPGR. Its preclinical programs build on the Company's industry leading AAV manufacturing . The European Union Clinical Trials Register allows you to search for protocol and results information on: interventional clinical trials that are conducted in the European Union (EU) and the European Economic Area (EEA); clinical trials conducted outside the EU / EEA that are linked to European . Conditions: X-Linked Retinitis Pigmentosa. Codon optimisation has overcome the challenge of designing an RPGR vector without mutations, and with a therapeutic effect in different animal models. Natural History of the Progression of X-Linked Retinitis Pigmentosa (XOLARIS) The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. About X-Linked Retinitis Pigmentosa (XLRP) XLRP is the most severe form of retinitis pigmentosa (RP), a group of inherited retinal diseases characterized by progressive retinal degeneration and vision loss. Overview. Poster presentation highlights safety and efficacy potential of AGTC-501 as a treatment for X-linked retinitis pigmentosa (XLRP) GAINESVILLE, Fla. and CAMBRIDGE, Mass., Nov. 12, 2021 (GLOBE NEWSWIRE) -- Applied Genetic Technologies Corporation (Nasdaq: AGTC), a biotechnology company conducting human clinical trials of adeno-associated virus (AAV)-based gene therapies for the treatment of rare . AGTC's most advanced clinical programs leverage its best-in-class technology platform to potentially improve vision for patients with an inherited retinal disease. Clinical trials for retinitis pigmentosa. Twelve Month Analysis of Macular Structure using Optical Coherence Tomography (OCT) from a Phase 1/2 Clinical Study of Subretinal Gene Therapy Drug AGTC-501 for XLinked Retinitis Pigmentosa. A patient with X-linked retinitis pigmentosa (XLRP) is hoping to save his vision after an innovative gene therapy procedure at Bascom Palmer Eye Institute at the University of Miami Miller School of Medicine. 10.1016/j.ajo.2003.10.045 [Google Scholar] All Female Male . X-linked retinitis pigmentosa (Gene: RPGR) Title: Analysis of retinal structure and function to assess disease progression. Information about the Phase 1/2 clinical trial in XLRP can be found at ClinicalTrials.gov under trial identifier number NCT03314207. Mutations affecting the Retinitis Pigmentosa GTPase Regulator (RPGR) gene are the commonest cause of X-linked and recessive retinitis pigmentosa (RP), accounting for 10%-20% of all cases of RP.The phenotype is one of the most severe amongst all causes of RP, characteristic for its early onset and rapid progression to blindness in young people. Credit: Shutterstock. Researchers have started a new gene therapy clinical trial to treat X-linked retinitis pigmentosa (XLRP), the most common . "The results from this initial trial showed no significant safety concerns after gene therapy surgery," said Dr. Davis. X-Linked Retinitis Pigmentosa (XLRP)- Phase 2/3 Study oversight Retinitis pigmentosa (RP) describes a group of rare genetic eye diseases that damage light-sensitive cells in the retina, leading to . X-Linked Retinitis Pigmentosa. Females are often considered to be unaffected carriers of the condition, with a 50 percent chance of passing XLRP to their sons. 10.1016/j.ajo.2003.10.045 [Google Scholar] Its initial focus is in the field of ophthalmology, where it has active clinical trial programs in X-linked retinitis pigmentosa (XLRP) and achromatopsia (ACHM CNGB3 & ACHM CNGA3). Showing trials for . X-linked retinitis pigmentosa is an amenable disease to be treated by gene therapy. X-linked retinitis pigmentosa (XLRP) is generally a severe form of retinitis pigmentosa, a neurodegenerative, blinding disorder of the retina. The investigational gene therapy (AGTC-501) is designed to replace the mutated RPGR . 28 MeiraGTx Announces Data from Ongoing Clinical Trial of AAV-RPGR for the Treatment of X-Linked Retinitis Pigmentosa to be Presented at AAO 2020 Virtual Meeting 12-Month Dose Escalation Data from . Showing trials for . Expert opinion: X-linked retinitis pigmentosa is an amenable disease to be treated by gene therapy. The SCENIC clinical trials are studying an investigational (not yet FDA-approved) gene therapy called AGTC-501 for patients with X-linked retinitis pigmentosa (XLRP). RARITAN, N.J., November 13, 2020 - The Janssen Pharmaceutical Companies of Johnson & Johnson announced today new 12-month data from the ongoing Phase 1/2 trial (NCT03252847) of its investigational gene therapy for inherited retinal disease X-linked retinitis pigmentosa (XLRP). Search by clinical trial title XIRIUS Study: A Dose Escalation, Phase 1/2 Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR) RARITAN, NJ, July 17, 2020 - The Janssen Pharmaceutical Companies of Johnson & Johnson announced today six-month data from the ongoing Phase 1/2 trial (NCT03252847) of its investigational gene therapy for the treatment of inherited retinal disease X-linked retinitis pigmentosa (XLRP).The interim data showed that low and intermediate doses of the . NCT04794101. a There was a 14% event rate for the placebo group in the phase 1 X-linked retinitis pigmentosa clinical trial. Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gene. 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