Study to evaluate the safety of QR-010 in Cystic fibrosis patients

• Research type

  Research Study

• Full title

  Phase 1b, Randomized, Double-blind, Placebo-controlled, Dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of QR-010 in Subjects With Homozygous ΔF508 Cystic Fibrosis

• IRAS ID

  170872

• Contact name

  Joseph Elborn

• Sponsor organisation

  ProQR Therapeutics BV

• Eudract number

  2014-004352-57

• Clinicaltrials.gov Identifier

  NCT02532764

• Duration of Study in the UK

  1 years, 0 months, 1 days

• Research summary

  The Study is to evaluate the safety of a new drug called QR-010, a drug being developed for participants with CF that have two copies of the ΔF508 CF gene mutation (homozygous) and is given as an inhaled medication. QR-010 has not been tested in people before. Information gathered will be used to determine which dose levels are safe to be used in future research studies. Participants will be assigned to one of 8 dosing groups, with 8 participants in each group. This Study will be done at approximately 21 sites in Belgium, Czech Republic, France, Germany, Italy, Netherlands, UK and US and will involve about 64 participants.
  Four of the groups will receive 1 dose of drug (Single Dose Groups), and the other 4 groups will receive 12 doses of drug (Multiple Dose Groups).
  Participants will be assigned to the Single Dose Groups, starting with the lowest dose level, and then later to the Multiple Dose Groups.
  Six participants in each multiple dosing group will receive study drug, and two will receive the placebo randomly, or a 1 out of 3 chance of receiving placebo. The placebo used in this study is normal saline (salt water). Participant, the study doctors and nurses will not know what participant will be receiving unless there is a medical emergency.
  Participants will have the option of enrolling in whichever group they prefer.
  The Single Dose Groups Study will last 5 days and 4 nights in the clinical research unit and a visit on day 8. The total duration of the study will be 8 days. Multiple Dose Groups will be required a total of 5 days and 4 nights, and 9 more times for dosing over 4 weeks, and 2 times for follow-up visits, for a total of 8 weeks.
  

• REC name

  HSC REC A

• REC reference

  15/NI/0017

• Date of REC Opinion

  31 Mar 2015

• REC opinion

  Further Information Favourable Opinion