QR-010 Clinical trials: Study 001

Phase 1b "Safety and Tolerability trial"
Clinical trial PQ-010-001 was an international Phase 1b, randomized, double-blind, placebo-controlled, safety and tolerability trial of QR-010 in 70 adults with cystic fibrosis carrying two copies of the F508del CFTR gene mutation (homozygotes). QR-010 is an investigational drug being developed as a regularly inhaled therapy. The trial was conducted at 23 hospitals in 10 countries in North America and Europe.

The trial consisted of two parts: the single ascending dose (SAD) and the multiple ascending dose (MAD) part. In the SAD part, each participant was in the trial for 1 week and received 1 dose of QR-010 or placebo. In the MAD part, each participant was in the trial for 8 weeks and received 12 doses of QR-010 or placebo (3 doses per week for 4 consecutive weeks).

Trial results
The trial showed that QR-010 was safe and well-tolerated across all dose levels and we saw encouraging signals that people with CF can benefit from taking QR-010. Most subjects in the trial who received QR-010 reported having a reduction in CF respiratory symptoms (as measured by an increase in the Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score, or CFQ-R RSS). The placebo group did not see a similar improvement in symptoms. A supportive trend was observed in improved lung function (as measured by percent predicted forced expiratory volume in 1 second, or ppFEV1) compared to placebo. No change was observed on sweat chloride and weight gain.

A more detailed explanation of the preliminary top-line trial results were published in a press release on September 25, 2017.

The study results were presented at the North American CF Conference (NACFC) in November 2017.

What is a Phase 1 trial?
In Phase 1 trials, the main goal is to assess safety and to identify possible side effects of different doses, also known as a dose escalation trial. Phase 1 trials are necessary to progress to other types of trials (Phase 2 & 3) designed to evaluate the safety and efficacy of an investigational drug over a longer duration.

Contact and further information

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

Additional details about QR-010 clinical trials are available at www.clinicaltrials.gov

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