E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cystic Fibrosis
Cystic Fibrosis is a genetic disorder which results in thick mucus formation on the airways leading to increased lung infections, fibrosis of the lungs and digestive tract and abnormal immune function. |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Diseases [C] - Respiratory Tract Diseases [C08] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10011762 |
E.1.2 | Term | Cystic fibrosis |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
Evaluate the safety [vital signs, physical examination, adverse events, blood and urine laboratory safety tests, QT/QTc intervals, and psychotrophic activity] and tolerability of JBT-101 in subjects with cystic fibrosis. |
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E.2.2 | Secondary objectives of the trial |
Evaluate JBT-101 plasma concentrations and metabolites in cystic fibrosis;
Evaluate efficacy of JBT-101 in cystic fibrosis, measuring changes from baseline and trends for efficacy in forced expiratory volume in one second, Lung Clearance Index (optional by site), and Cystic Fibrosis Questionnaire - Revised Respiratory Symptoms score in subjects with cystic fibrosis.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Documented diagnosis of cystic fibrosis 2. ≥18 and < 65 years of age at the time the Informed Consent Form is signed 3. FEV1 ≥ 40% predicted, either pre- or post bronchodilator. 4. Stable treatment of cystic fibrosis for 14 days before Visit 1.
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E.4 | Principal exclusion criteria |
1. Severe or unstable cystic fibrosis. 2. Any of the following values for laboratory tests at Screening a. A positive pregnancy test (also Visit 1) b. Hemoglobin < 10 g/dL c. Neutrophils < 1.0 x 109/L d. Platelets < 500 cells/µL e. Creatinine clearance < 50 ml/min according to modified Cockcroft-Gault equation; f. Serum transaminases > 2.5 x upper normal limit g. Total bilirubin ≥ 1.5 x upper limit of normal 3. Any other condition that, in the opinion of the site investigator, are clinically significant and may put the subject at greater safety risk, influence response to study product, or interfere with study assessments.
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety and tolerability, comparing all subjects who received JBT-101 at any time during the trial to subjects who received placebo throughout the trial.
• Safety will be assessed by percentage and number of subjects who experience treatment-emergent adverse events, including serious adverse events. Adverse events will be identified through verbal reports by the subject and clinical assessment of vital signs, physical examination, laboratory safety tests, electrocardiograms including QT/QTc intervals, and Addiction Research Center inventory-Marijuana scale.
• Tolerability will be assessed by the number and proportion of subjects that withdraw from the trial during the treatment period because of treatment-related adverse events
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
• Safety will be assessed from baseline on Day 1 (Visit 1) prior to study product administration through 28 +/- 3 days after the last dose of study product (Day 113 +/- 3, Visit 7). Subject-reported adverse events and vital signs will be collected at every visit. The other clinical assessment will be done at various visits. In addition, non-treatment related adverse events will be captured from the screening up to the first dose of study product.
•Tolerability will be assessed from the first dose of study product (Day 1, Visit 1) through the end of active treatment (Day 85 +/- 3, Visit 6), with an 84 day active treatment period planned.
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E.5.2 | Secondary end point(s) |
Endpoint 1: Levels of JBT-101 and its metabolites in plasma, in all subjects who received JBT-101 during the trial Endpoint 2: Change in FEV1, percent predicted and absolute value, comparing all subjects who received JBT-101 at any time during the trial to subjects who received placebo throughout the trial Endpoint 3: Change in lung clearance index, comparing all subjects who received JBT-101 at any time during the trial to subjects who received placebo throughout the trial Endpoint 4: Cystic Fibrosis Questionnaire–Revised Respiratory Symptom Score, comparing all subjects who received JBT-101 at any time during the trial to subjects who received placebo throughout the trial |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Endpoint 1: Prior to study product administration (Day 1, Visit 1), 3 hours after study product administration on Day 1, Day 15 +/- 3 (Visit 2), Day 29 +/- 3 (Visit 3) both prior to study product administration and 3 hours after study product administration, Day 43 +/- 3 (Visit 4), and Day 85 +/- 3 (Visit 6, end of active treatment) Endpoint 2: Day 1 (Visit 1) prior to study product administration through Day 85 +/- 3 (Visit 6, end of active treatment) Endpoint 3: Day 1 (Visit 1) prior to study product administration through Day 85 +/- 3 (Visit 6, end of active treatment) Endpoint 4: Day 1 (Visit 1) prior to study product administration through Day 85 +/- 3 (Visit 6, end of active treatment) |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Includes dose escalation on JBT-101 and switching 10 placebo subjects to JBT-101. |
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E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 7 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 13 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Belgium |
France |
Germany |
Israel |
Italy |
Poland |
United Kingdom |
United States |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 1 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 3 |