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Trial record 4 of 8 for:    AGTC

Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03314207
Recruitment Status : Recruiting
First Posted : October 19, 2017
Last Update Posted : December 11, 2019
Sponsor:
Information provided by (Responsible Party):
Applied Genetic Technologies Corp

Tracking Information
First Submitted Date October 11, 2017
First Posted Date October 19, 2017
Last Update Posted Date December 11, 2019
Actual Study Start Date December 1, 2017
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: October 17, 2017)
Disease progression in subjects with XLRP [ Time Frame: Day 0 - Month 36 ]
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: October 18, 2017)
  • Disease progression using visual acuity testing [ Time Frame: Day 0 - Month 36 ]
  • Disease progression using perimetry [ Time Frame: Day 0 - Month 36 ]
  • Disease progression using OCT [ Time Frame: Day 0 - Month 36 ]
  • Disease progression using electroretinography [ Time Frame: Day 0 - Month 36 ]
  • Disease progression using the National Eye Institute Visual Functioning Questionnaire-25 (VFQ-25) quality of life questionnaire [ Time Frame: Day 0 - Month 36 ]
Original Secondary Outcome Measures
 (submitted: October 17, 2017)
  • Disease progression using visual acuity testing [ Time Frame: Day 0 - Month 36 ]
  • Disease progression using perimetry [ Time Frame: Day 0 - Month 36 ]
  • Disease progression using OCT [ Time Frame: Day 0 - Month 36 ]
  • Disease progression using electroretinography [ Time Frame: Day 0 - Month 36 ]
  • Disease progression using the VFQ-25 quality of life questionnaire [ Time Frame: Day 0 - Month 36 ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Clinical Evaluation of Patients With X-linked Retinitis Pigmentosa (XLRP)
Official Title Clinical Evaluation of Individuals With X-linked Retinitis Pigmentosa (XLRP) Caused by RPGR-ORF15 Mutations
Brief Summary 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.
Detailed Description Males with a clinical diagnosis of X-linked retinitis pigmentosa (XLRP) caused by RPGR-ORF15 mutations will be asked to provide informed consent and will have a single blood or saliva sample obtained for DNA sequence analysis of genes known to cause XLRP, including the RPGR-ORF15 gene. All participants will be informed of the results of testing for these mutations. Those with qualifying mutations in the RPGR-ORF15 gene will be evaluated every 6 months for 3 years using a variety of non-invasive visual function tests to more fully characterize their clinical condition. Testing will include routine ophthalmic examinations and tests of visual acuity, perimetry, OCT, fundus imaging, and completion of quality of life questionnaires.
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
Sampling Method Non-Probability Sample
Study Population Males with a clinical diagnosis of X-linked Retinitis Pigmentosa (XLRP)
Condition X-Linked Retinitis Pigmentosa
Intervention Not Provided
Study Groups/Cohorts Not Provided
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: October 17, 2017)
45
Original Estimated Enrollment Same as current
Estimated Study Completion Date December 2021
Estimated Primary Completion Date December 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria

Inclusion Criteria:

  • Male subjects with a clinical diagnosis of XLRP and a documented molecular diagnosis from a CLIA-certified laboratory of mutation within the ORF15 exon of the RPGR gene;
  • At least 6 years of age;
  • Willing and able to perform study procedures;
  • Signed informed consent(s) obtained (and child assent where applicable).

Exclusion Criteria:

  • Pre-existing eye conditions that would interfere with interpretation of study endpoints (e.g. glaucoma, corneal or lenticular opacities, diabetic retinopathy, history of retinal detachment);
  • Participating in an interventional research study of drugs or devices for treatment of XLRP or other retinal diseases;
  • Monoocular participants
  • Any condition which leads the investigator to believe that the participant cannot comply with the protocol requirements or that may place the participant at an unacceptable risk for participation.
Sex/Gender
Sexes Eligible for Study: Male
Gender Based Eligibility: Yes
Ages 6 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts
Contact: Jill Dolgin, PharmD 833-770-2862 advocacy@agtc.com
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT03314207
Other Study ID Numbers AGTC-XLRP-001
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party Applied Genetic Technologies Corp
Study Sponsor Applied Genetic Technologies Corp
Collaborators Not Provided
Investigators
Study Director: Theresa Heah, MD Applied Genetics Technologies Corporation
PRS Account Applied Genetic Technologies Corp
Verification Date December 2019