QR-010 Clinical trials: Study 002

"Nasal Potential Difference trial"
Clinical Study 002 was an international exploratory proof of concept trial in cystic fibrosis patients that have 1 or 2 copies of the F508del mutation (compound heterozygous or homozygous). The trail explored whether intranasal administration of QR-010 can increase function of the CFTR protein. The effect of QR-010 was assessed using the Nasal Potential Difference (NPD) test. The NPD test measures CFTR protein activity by measuring how well chloride is transported through the nasal cell membranes as well as the sodium channel activity.

Each participant was in the trial for about 8 weeks and received 12 doses of 10 mg (5 mg in each nostril) of QR-010 (3 administrations per week for 4 consecutive weeks). 18 adults were included in the trial (10 homozygous and 8 compound heterozygous). The trial was conducted at 5 specialized hospitals in the USA, France and Belgium.

Trial Results

Initial trial results were presented during the North American CF Conference in October 2016; the trial demonstrated that QR-010 significantly improved CFTR-mediated total chloride response following 4 weeks of treatment with QR-010 in patients that are homozygous for the F508del mutation. This was supported by the change in sodium channel activity as measured by the basal potential difference. In patients that are compound heterozygous for the F508del mutation, no meaningful difference was found. QR-010 was observed to be safe and well-tolerated.

More information on the trials of QR-010 and initial results can be found in this interview with Daniel de Boer and Noreen Henig or the press release issued on October 27, 2016.

Contact and further information

For questions, please contact ProQR Therapeutics Patient Advocacy via the contact form below or email patientinfo@proqr.com.

Additional details about this clinical trial are available at www.clinicaltrials.gov and will be explained in detail by physicians conducting the study.

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