Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
QBW276 · 1 trial · 1 indication
Adverse events were summarized by the number of patients having any adverse event overall and presented in the safety section. Study was prematurely terminated
Blood collection will be used to observe the maximum plasma concentration (Cmax) following administration of QBW276. Pharmacokinetic blood samples were collected at the time points. In Cohorts 1 and 2 this consisted of multiple samples through 6 hours after inhalation on Days 1 and 7 in Cohort 1 and Days 1 and 14 in Cohort 2 with predose samples on selected days. The Cmax, was determined using the actual recorded sampling times and noncompartmental methods. Since steady state was likely reached by Day 7, Cmax on Days 7 or 14 correspond to Cmax,ss
Blood collection will be used to observe the maximum plasma concentration (Tmax) following administration of QBW276. Pharmacokinetic blood samples were collected at the time points. In Cohorts 1 and 2 this consisted of multiple samples through 6 hours after inhalation on Days 1 and 7 in Cohort 1 and Days 1 and 14 in Cohort 2 with predose samples on selected days. The Tmax, was determined using the actual recorded sampling times and noncompartmental methods. Since steady state was likely reached by Day 7, Tmax on Days 7 or 14 correspond to Tmax,ss
Blood collection will be used to observe the maximum plasma concentration (AUCtau) following administration of QBW276. Pharmacokinetic blood samples were collected at the time points. In Cohorts 1 and 2 this consisted of multiple samples through 6 hours after inhalation on Days 1 and 7 in Cohort 1 and Days 1 and 14 in Cohort 2 with predose samples on selected days. The AUCtau, was determined using the actual recorded sampling times and noncompartmental methods. Since steady state was likely reached by Day 7, AUCtau on Days 7 or 14 correspond to AUClast,ss
The accumulation ratio (Racc) will be reported using blood samples taken on days 1 -7 in cohort 1 and days 1-14 in cohort 2. Accumulation ratio (Racc) for QBW276 and metabolites was not calculated by PK software for patients where BLOQ values were observed for all blood samples in their PK profile.
| Arm | Type | Description |
|---|---|---|
| CQBW276 | EXPERIMENTAL | Cohorts 1 and 2 will enroll 8 patients each (6:2 QBW276 and placebo, respectively). Cohort 1: dose is 3 mg bid (6 mg daily) QBW276 or placebo for 7 days. Cohort 2: dose and frequency will be confirmed after cohort 1 is complete. The duration is 14 days. Cohort 3: dose and frequency will be confirmed after cohort 2 is complete. The duration is approximately 4 months. Patients will be randomized to one of two treatment sequences: QBW276 in Period 1 and Placebo in Period 2 or Placebo in Period 1 and QBW276 in Period 2. Twenty four patients are required to complete cohort 3. |
| Placebo | PLACEBO_COMPARATOR | Cohorts 1 and 2 will enroll 8 patients each (6:2 QBW276 and placebo, respectively). Cohort 1: dose is 3 mg bid (6 mg daily) QBW276 or placebo for 7 days. Cohort 2: dose and frequency will be confirmed after cohort 1 is complete. The duration is 14 days. Cohort 3: dose and frequency will be confirmed after cohort 2 is complete. The duration is approximately 4 months. Patients will be randomized to one of two treatment sequences: QBW276 in Period 1 and Placebo in Period 2 or Placebo in Period 1 and QBW276 in Period 2. Twenty four patients are required to complete cohort 3. |
| Name | Type | Description |
|---|---|---|
| Placebo | OTHER | Placebo |
| QBW276 | DRUG | 0.3 mg and 1.5 mg strengths |
Inclusion Criteria: * Cohorts 1 and 2 = any genotype on any standard of care treatment * Cohort 3 = F508del homozygotes on standard of care at that time * FEV₁between 40 and 100% * LCI2.5 ≥ 8 if FEV₁is more than 80% Exclusion Criteria: * Adrenal or electrolyte abnormalities * Lung transplant * Au...
QBW276 is an investigational small molecule developed by Novartis AG for pulmonary cystic fibrosis. It is a Phase 1 candidate in the respiratory therapeutic area and has been studied in a single completed clinical trial. It is not an approved therapy and remains in clinical development.
QBW276 is being studied for pulmonary cystic fibrosis, a respiratory condition. The completed Phase 1 trial enrolled 16 adult patients with cystic fibrosis in the United States and Germany. Because the program is still in Phase 1, QBW276 is investigational and has no approved use in cystic fibrosis or any other indication.
QBW276 is developed by Novartis AG, which trades under the ticker NVS. Novartis sponsored the Phase 1 study of the inhaled small molecule in patients with cystic fibrosis. No other developer or partner is identified for this asset.
QBW276 is in Phase 1 clinical development. Its only recorded trial, NCT02566044, was a Phase 1 study that has been completed. There are no active trials listed for QBW276, and the drug is not approved by the FDA or any other regulator.
QBW276 has been evaluated in one clinical trial, NCT02566044, titled Safety, Pharmacokinetics and Pharmacodynamics Study of Inhaled QBW276 in Patients With Cystic Fibrosis. The Phase 1 trial was placebo controlled, double blind, enrolled 16 patients aged 18 years and older, and took place in the United States and Germany. It is completed.
Yes. The Phase 1 trial of QBW276, NCT02566044, was biomarker selected, meaning enrollment was guided by a biomarker. The study was placebo controlled and double blind, enrolled 16 adults with pulmonary cystic fibrosis, and has been completed. It was not randomized and did not include healthy volunteers.