QR-010 Clinical trials: Study 001

Phase 1b "Safety and Tolerability trial"
Clinical Study 001 is an international Phase 1b, randomized, double-blind, placebo-controlled, safety and tolerability trial of QR-010 in cystic fibrosis patients carrying two copies (homozygous) of the F508del CFTR gene mutation. QR-010 is an investigational drug being developed as a regularly inhaled therapy.

Study 001 consists of two parts: the single ascending dose (SAD) and the multiple ascending dose (MAD) part. In the SAD part, which was successfully completed in 2016, each participant was in the trial for 1 week and received 1 dose of QR-010 or placebo. In the MAD part, which is currently ongoing but enrollment is complete, each participant will be in the trial for 8 weeks and will receive 12 doses of QR-010 or placebo (3 doses per week for 4 consecutive weeks). Approximately 64 adults participated in Study 001. The trial is conducted at hospitals in the North America and several countries in Europe.

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

Trial Results

The SAD part of Study 001 was completed and data were presented during the North American CF Conference (NACFC) in October 2016. The MAD part of the study is ongoing but enrollment is complete. Top-line data from the trial will be announced in September 2017. Full data from the study are expected to be presented during the North American CF Conference (NACFC) in November 2017.

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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