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QR-010 Clinical trials: Study 001

Phase 1b "Single and multiple doses 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 administered by inhalation using a nebulizer.

Study 001 consists of two parts i.e. 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, 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 will participate in Study 001. The trial is conducted at hospitals in the USA, Canada and several countries in Europe.

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.
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.

Requirements for Taking Part in this Study

To participate in this trial, you must meet, amongst others, the following requirements:

  • Are diagnosed with cystic fibrosis
  • Have 2 copies of the F508del mutation (homozygous)
  • Are between 18 and 60 years of age
  • Have a FEV1 of ≥ 70%
  • Have a Body Mass Index (BMI) ≥ 17 kg/m2
  • Do not have any acute infections
  • Are not taking lumacaftor or ivacaftor
  • Have not had a lung transplant
For questions, please contact ProQR Therapeutics Patient Advocacy via the contact form or email patient@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. Below is a list of participating hospitals.

Study Locations

Study 001 is currently being conducted at the following hospitals:

North America
  • Penn State Milton S. Hershey Medical Center, Hershey, PA
  • University of Texas Southwestern Medical Center, Dallas, TX
  • University of Washington Medical Center, Seattle, WA
  • Nationwide Children’s Hospital, Columbus, OH
  • Stanford University, Palo Alto Stanford, CA
  • Medical University of South Carolina, Charleston, SC
  • University of Kansas Medical Center Research Institute, Kansas City, KS
  • Massachusetts General Hospital Cystic Fibrosis Clinical Research Center, Boston, MA
  • Boston Children’s Hospital, Boston, MA
  • University of Calgary Health Sciences Centre, Calgary, Alberta, Canada
  • Northwestern University Depart of Medicine – Pulmonary and Critical Care, Chicago, IL
  • University of Southern California USC - Keck School of Medicine, Los Angeles, CA
  • Washington University School of Medicine, Saint Louis, MO
Europe
  • Hôpital Necker – Enfants Malades, Paris, France
  • CHU de Nantes – Hôpital Nord Laennec (HGRL) – Pneumologie, Nantes, France
  • Universitair Ziekenhuis Leuven, Leuven, Belgium
  • Universitair Ziekenhuis Brussel, Brussel, Belgium
  • Trust-Wolfson Northern Ireland Clinical Research Facility and Queens University Belfast, Belfast, Ireland
  • Royal Brompton Hospital – Adult Cystic Fibrosis Centre, London, England
  • Fakultní nemocnice v Motole, Praha, Czech Republic
  • Bispebjerg Universitetshospital, København, Danmark
  • Klinikum der Universität München, Germany
  • Charite Universitätsmedizin Berlin, Germany
  • Centro di Ricerche Cliniche - Azienda Ospedaliera Universitaria Integrata di Verona, Verona, Italy
  • Medizinische Hochschule Hannover, Hannover, Germany
  • Hospital Vall D'Hebron, Barcelona, Spain
  • Southampton General Hospital, Southampton, United Kingdom

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