Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation

Phase 1 Completed 18 enrolled ProQR Therapeutics European Commission · collab

Non-randomizedParallel-groupSingle-blindTreatment

Summary

Exploratory proof of concept study to determine whether intranasal administration of QR-010 in subjects with cystic fibrosis, homozygous or compound heterozygous for the ΔF508 mutation, can increase the function of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR).

Timeline

Start: 2015-09
Primary completion: 2016-09
Completion: 2016-09

Outcome

Sources disagree · no verdict is set; each source's reading is shown below

release “clinical study PQ-010-002, a proof-of-concept study of nasal potential difference (NPD), demonstrated that QR-010 restored CFTR function” reads as mixed primary results proqr.com ↗

release “The study met its primary endpoint in this cohort as measured by a change in total chloride response” reads as mixed primary results proqr.com ↗

release “In the compound heterozygous ∆F508 cohort, no meaningful difference was found.” reads as mixed primary results proqr.com ↗

release “Clinical trial PQ-010-002 top-line data shows significant improvement of CFTR function as measured by NPD in subjects homozygous for the F508del mutation” reads as met primary endpoint proqr.com ↗

Publications

Miah KM, Hyde SC, Gill DR. Emerging gene therapies for cystic fibrosis. Expert Rev Respir Med. 2019 Aug;13(8):709-725. doi: 10.1080/17476348.2019.1634547. Epub 2019 Jun 27.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	ELUFORSEN	Antisense oligonucleotide	10 mg	Intranasal