Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR

• Research type

  Research Study

• Full title

  Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Protocol MGT-RPGR-021

• IRAS ID

  286986

• Contact name

  James Bainbridge

• Contact email

  james.bainbridge1@nhs.net

• Sponsor organisation

  MeiraGTx UK II Limited

• Eudract number

  2020-002873-88

• ISRCTN Number

  ISRCTN00000000

• Clinicaltrials.gov Identifier

  NCT00000000

• Clinicaltrials.gov Identifier

  EDMS-ERI-205374434, 4.0, EDMS number; IND 17, 866, IND

• Duration of Study in the UK

  1 years, 3 months, 0 days

• Research summary

  Research Summary:
  The purpose of this study is to see if the investigational gene therapy, AAV5-hRKp.RPGR, is safe and useful for improving vision in people with X-linked Retinitis Pigmentosa (XLRP).

  Approximately 36-48 individuals will participate worldwide. As part of this study, Potential participants will be invited to consent to 2 closely-related studies. Potential participants will review the information for both studies and decide whether they wish to participate in both. In the first study (MGT-RPGR-021) which is outlined in this consent, participants will be in the study for up to 15 months. Once participants finish their participation in this study, they will continue into the second study (MGT-RPGR-022), for an additional 4 to 5 years.

  All participants will be administered the investigational gene therapy in both eyes. To enable its impact to be measured in comparison with the untreated course of the condition, two thirds of participants will have intervention shortly after enrolment (as part of the MGT-RPGR-021 study) and one third of participants will be have intervention after 12 months (as part of the MGT-RPGR-022 study). For each participant the timing of intervention will be determined by their treatment allocation.

  During both studies, the sponsor may learn new information about the investigational gene therapy, the risks, and additional information. Participants' doctor/staff will tell participants in a timely manner if there is any new information that might make participants change their mind about being in the studies.

  Lay summary of study results:
  https://www.trialsummaries.com/Study/StudyDetails?id=25688&tenant=MT_JNJ_9011

• REC name

  London - West London & GTAC Research Ethics Committee

• REC reference

  20/LO/1175

• Date of REC Opinion

  11 Feb 2021

• REC opinion

  Further Information Favourable Opinion