EUCTR2016-003967-21-GB进行中（未招募）1 期
An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy of adults and children with X-linked Retinitis Pigmentosa owing to defects in Retinitis Pigmentosa GTPase Regulator (RPGR) - Gene Therapy Trial for People with Retinitis Pigmentosa: RPGR
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 71
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
入选标准
- •Inclusion in the trial will be limited to individuals who meet the following criteria:
- •1. Are males aged 5 years or older
- •2. Have X-linked retinitis pigmentosa confirmed by a retinal specialist (CI or PI)
- •3. Have relatively symmetrical retinal disease - both structurally and functionally defined as < 15 letters [Best Corrected Visual Acuity (BCVA)] difference between eyes
- •4. Have a predicted disease-causing missense or null mutation in RPGR confirmed in an
- •accredited laboratory
- •5. Evidence of relative preservation of retinal structure at the macula
- •6. Evidence of impaired navigation in dim illumination on Mobility assessment in both eyes monocularly; defined by either an inability to complete the visual mobility maze at the 1 lux level, or the time taken to complete the maze at either 1 or 4 lux being greater or equal to 12 seconds.
- •7. Are able to give informed consent or assent, with the guidance of their parent/guardian where appropriate: children aged 5-6 years will not be asked to provide assent
- •8. Are able to undertake age appropriate clinical assessments at the trial sites as specified in the protocol
- •9. Are willing to use barrier and spermicide form of contraception or will be sexually abstinent (when this is in line with the participant’s preferred and usual lifestyle) for at least 12 months following ATIMP administration
- •10. Are willing to give consent for the use of blood and blood components collected through the trial for the investigation of immune responses to the ATIMP
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 23
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 48
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Individuals will be excluded if they meet any of the following criteria:
- •1. Have a known allergy to any of the non-investigational drugs to be used in the trial as defined in Section 5.4.1
- •2. Have participated in another research study involving an investigational medicinal therapy for ocular disease within the last 6 months
- •3. Have any other condition that the CI/PI considers makes them inappropriate for entry into the trial, inclusive of but not limited to a history of the following:
- •o Uncontrolled hypertension defined as a systolic value =160mmHg or diastolic value =100mmHg.
- •o Uncontrolled diabetes mellitus defined as an HbA1c =9% (75mmol/mol) at screening.
- •o Uncontrolled heart failure (NYHA class II-IV).
- •o Any history of tuberculosis
- •o Chronic kidney disease (defined as eGFR =60ml/min calculated using Cockroft Gault or MDRD equations.
- •o Immunocompromised state (including long term immunosuppressant therapy).
- •o Osteoporosis (defined as presence of 1 or more non-traumatic fragility” fractures or proven BMD of 2.5SD less than anticipated as demonstrated on DEXA scan).
- •o Active peptic ulcer disease or uncontrolled gastro-oesophageal reflux. o Severe affective disorder or past history of drug induced psychosis
- •4. Use of high dose regular non-steroidal anti-inflammatory drugs at the time of screening.
- •5. Have an ocular or systemic disorder that may preclude subretinal surgery and/or interfere with interpretation of the study results
- •6. Have had intraocular surgery within 3 months of screening
- •7. Have recently (within 4 weeks of last dose of concomitant immunosuppressive therapy) received a live vaccine
- •8. Have a condition that requires glucocorticoid replacement therapy, such as in endocrine diseases, if this would interfere with the immunosuppressive regime.
- •9. Have known chronic hepatitis B infection as indicated by detectable surface antigen. Entry with negative surface antigens but positive hepatitis core antibody will be allowed however additional monitoring throughout the trial may be required.
- •10. Are unwilling to consider the possibility of entry into a subsequent longer term follow up study
研究者
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