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