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 being developed for the treatment of pulmonary cystic fibrosis. It is administered via inhalation and is currently in Phase 1 clinical development. The drug has been studied in a completed clinical trial involving patients with cystic fibrosis.
QBW276 is being developed by Novartis AG, a multinational pharmaceutical company. Novartis is listed on the stock exchange under the ticker symbol NVS. The company has conducted clinical research on this investigational drug for the treatment of pulmonary cystic fibrosis.
QBW276 is in Phase 1 clinical development. It has completed a Phase 1 clinical trial, which was a placebo-controlled, double-blind study. The drug is still investigational and has not been approved by regulatory authorities for commercial use.
QBW276 has been studied in one clinical trial with the identifier NCT02566044. This was a Phase 1 study titled 'Safety, Pharmacokinetics and Pharmacodynamics Study of Inhaled QBW276 in Patients With Cystic Fibrosis.' The trial was completed and enrolled 16 participants in the United States and Germany.
QBW276 is not FDA approved. It is an investigational drug that has completed a Phase 1 clinical trial. The drug is still in clinical development and has not received regulatory approval for the treatment of pulmonary cystic fibrosis.