Clinical Trial: Study to Evaluate QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

Study Status: Not yet recruiting
Recruit Status: Not yet recruiting
Study Type: Interventional

Official Title: An Open-label, Single Arm, Multiple Dose, Dose Escalation Study to Evaluate the Safety and Tolerability of QR-110 in Subjects With Leber's Congenital Amaurosis (LCA) Due t

Brief Summary: The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation.

Detailed Summary: The purpose of this study is to evaluate the safety and tolerability of QR-110 administered via intravitreal injection in subjects with LCA due to the CEP290 p.Cys998X mutation. Subjects will receive QR-110 in one eye every 3 months, for a maximum of 4 doses. Up to 3 dose levels of QR-110 will be evaluated.
Sponsor: ProQR Therapeutics

Current Primary Outcome: Frequency and severity of ocular adverse events in the treatment and contralateral eyes [ Time Frame: 1 year ]

Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Frequency and severity of non-ocular adverse events [ Time Frame: 1 year ]
  • Change in ophthalmic examination findings [ Time Frame: 1 year ]
  • Change in best-corrected visual acuity (BCVA) [ Time Frame: 1 year ]
  • Change in infrared imaging [ Time Frame: 1 year ]
  • Change in optical coherence tomography (OCT) findings [ Time Frame: 1 year ]
  • Changes in safety parameters, including vital sign measurements, physical examination findings, ECG and laboratory parameters [ Time Frame: 1 year ]
  • Area under the curve 0 hour to infinity [AUC(0-∞)] of QR-110 in serum (if measurable) [ Time Frame: 1 year ]
  • Area under the curve 0 hour to time of the last measurable concentration [AUC(0-t)] of QR-110 in serum [ Time Frame: 1 year ]
  • Maximum concentration (Cmax ) of QR-110 in serum [ Time Frame: 1 year ]
  • Minimum concentration (Cmin) of QR-110 in serum [ Time Frame: 1 year ]
  • Time of maximum concentration (Tmax ) of QR-110 in serum [ Time Frame: 1 year ]
  • Terminal half life (T1/2) of QR-110 in serum (if measurable) [ Time Frame: 1 year ]
  • Clearance (CL) of QR-110 in serum (if measurable) [ Time Frame: 1 year ]
  • Volume of distribution (Vd) of QR-110 in serum (if measurable) [ Time Frame: 1 year ]
  • Change in full-field stimulus test (FST) [ Time Frame: 1 year ]
  • Change in pupillary light reflex (PLR) [ Time Frame: 1 year ]
  • Change in mobility course score [ Time Frame: 1 year ]


Original Secondary Outcome: Same as current

Information By: ProQR Therapeutics

Dates:
Date Received: May 1, 2017
Date Started: June 2017
Date Completion: December 2018
Last Updated: May 4, 2017
Last Verified: May 2017