Study Title: Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study of N91115 to Evaluate Efficacy and Safety in Patients With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation Treated With Lumacaftor/Ivacaftor
Wait, what? More context please…
Already a combination therapy, Orkambi was approved by the FDA nine months ago. Orkambi targets the root cause of CF rather than treating the symptoms of CF. It is intended only for people who have 2 copies of the F508del mutation and works to correct the mutated (or the misfolded CFTR protein) genes.
Orkambi’s approval came quickly thanks to the Office of New Drugs, Center for Drug Evaluation and Research. Under it’s priority review program, Orkambi was approved in less than the 10 month standard and won the following FDA designations; breakthrough therapy and orphan drug.
UNC affiliated pulmonologist, Dr. Scott Donaldson, who conducts procedures and research involving Cystic Fibrosis, mucociliary clearance and ion transport. Dr. Donaldson is the principle investigator for Phase II Clinical Trial N91115. Estimated Enrollment: 135 Study Start Date: 11/ 2015 Estimated Study Completion Date: July 2016
Great. Ok that was pretty fast. Kudos FDA approver-s!
So #patientperspective is proving its worthiness to industry ears. These designation status’ are actually expediting the review process of clinical trials. Nivalis Therapeutics announced on Thursday that the FDA designated N91115 as both fast track and orphan drug. Estimated completion date for the study and its primary outcome measure is in July of 2016 – only 5 months away.
ClinicalTrials.gov references N91115 as Identifier NCT02589236. It is an interventional, parallel group, double-blind, randomized and placebo-controlled study. Kicked off on October 26, 2015 the study’s primary objective is to assess the efficacy of N91115 at 12 weeks when added to preexisting treatment (Orkamabi). Public reports were last updated on February 8, 2016.
Drug Application and Approval Process – “Questions and Answers”
Got a question? click here to submit it and maybe your question will be added to the FAQs page of the ONDs site.
Dr. Scott Donaldson of University of North Carolina, Chapel Hill – go @TarHeels ! – is the principle investigator over the 48 study locations, and ClinicalTrial.gov reports that N91115 is currently recruiting participants. Click here to check out the list of Contacts and Locations or reach out directly to the following contacts for more information:
- Raven Kidd 984-974-2964 [email protected]
- Nadia Shive 984-974-2961 [email protected]
