Phase 2
Completed N=36
Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients
Source: ClinicalTrials.gov NCT02532764 ↗Enrolled (actual)
36
Serious AEs
2.9%
Results posted
Feb 2019
Primary outcomePrimary: Incidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study — 3; 2; 7; 4 Participants
Summary
A randomized, double-blind, placebo-controlled study of single and multiple ascending doses of QR-010 in adults homozygous for ΔF508 Cystic Fibrosis.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Incidence of Subjects Experiencing Treatment Emergent Adverse Events From Baseline Through End of Study |
3; 2; 7; 4; 2; 5 | — |
| PRIMARY Severity of Treatment Emergent Adverse Events From Baseline Through End of Study |
3; 2; 5; 4; 2; 5 | — |
| PRIMARY Incidence of Subjects Experiencing Dose-Limiting Toxicities (DLT) in Each Dose Cohort From Baseline Through End of Study Visit. |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Subjects With Abnormalities Reported Regarding Laboratory Parameters, Vital Signs, ECG, Spirometry, and Physical Findings. |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Maximum Serum Concentration |
2.9; 4.53; NA; 3.63 | — |
| SECONDARY Time to Maximum Serum Concentration |
0.6; 0.5; NA; 1.0 | — |
| SECONDARY Terminal Half-life (T1/2) |
— | — |
| SECONDARY Area Under the Curve to Final Sample [AUC(0-last)] |
17.2; 19.0; NA; 17.0 | — |
| SECONDARY Area Under the Curve to Infinity [AUC(0-∞)] |
— | — |
| SECONDARY Serum Clearance (CL) |
— | — |
Eligibility Criteria
Inclusion Criteria
- Confirmed diagnosis of CF as defined by iontophoretic pilocarpine sweat chloride test (sweat chloride) of > 60 mmol/L
- Confirmation of CFTR gene mutations homozygous for the ΔF508 mutation
- Body mass index (BMI) ≥ 17 kg/m2
- Non-smoking for a minimum of two years
- FEV1 ≥70% of predicted normal for age, gender, and height, at Screening
- Stable lung function
- Adequate hepatic and renal function
Exclusion Criteria
- Breast-feeding or pregnant
- Use of lumacaftor or ivacaftor
- Use of any investigational drug or device
- History of lung transplantation
- Hemoptysis
Data sourced from ClinicalTrials.gov (NCT02532764). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.