E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Patients with Cystic Fibrosis-Related Exocrine Pancreatic Insufficiency |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10011766 |
E.1.2 | Term | Cystic fibrosis pancreatic |
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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 |
To determine the efficacy of ALTU-135 for the treatment of fat malabsorption in patients with CF-related exocrine PI.
Primary: • Mean change in CFA from Baseline Period to Double-Blind Treatment Period. Secondary: • Mean change in CNA from Baseline Period to Double-Blind Treatment Period. • Proportion of patients who have a maximum increase in blood glucose of ≥ 10 mg/dl in the SCT (non-diabetic patients only) during the Double-Blind Treatment Period. Mean changes in stool weight and frequency from Baseline Period to Double-Blind Treatment Period. Tertiary: • Mean difference in maximum change in blood glucose level following starch challenge from Baseline Period to Double-Blind Treatment Period. • Mean percent change in CFA and CNA from Baseline Period to Double-Blind Treatment Period. • Mean relative improvement in CFA and CNA from Baseline Period to Double-Blind Treatment Period.
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E.2.2 | Secondary objectives of the trial |
• To determine the efficacy of ALTU-135 for the treatment of protein malabsorption in patients with CF-related exocrine PI. • To determine the efficacy of ALTU-135 treatment in improving the absorption of carbohydrates in patients with CF-related exocrine PI. • To determine the efficacy of ALTU-135 treatment in decreasing stool weight and frequency in patients with CF-related exocrine PI. Safety Endpoints: • Safety endpoints include frequency and severity of treatment-emergent AEs; mean changes in clinical laboratory parameters and vital signs; toxicity shifts in LFT parameters; proportion of patients with abnormal clinical laboratory and vital signs results.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Each patient must meet the following criteria to be enrolled in this study. 1. ≥ 7 years of age. 2. Females of childbearing potential must be willing to use birth control (IUD; oral, transdermal or parenteral contraceptives; abstinence). 3. Diagnosis of CF based upon the following criteria: a. two clinical features consistent with CF; and b. either genotype with two identifiable mutations consistent with CF, c. or sweat chloride > 60 mEq/L by quantitative pilocarpine iontophoresis. 4. Clinically stable with no evidence of acute upper or lower respiratory tract infection. 5. PI determined by fecal elastase < 100 µg/g stool measured at the Screening Visit. 6. Able to take pancreatic enzyme supplementation in the form of capsules. 7. Able to perform the testing (e.g., stool collections) and inpatient stays required for this study, as judged by the Investigator. 8. Willing and able to provide informed consent or assent. 9. Baseline CFA ≤ 80%.
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E.4 | Principal exclusion criteria |
Patients who meet any of the following criteria will be excluded from the study. 1. < 7 years of age. 2. CFA > 80% at Baseline. 3. Pregnancy, breastfeeding or of childbearing potential and not willing to use birth control (IUD; oral, transdermal or parenteral contraceptives; abstinence) during the study. 4. History of fibrosing colonopathy. 5. History of liver transplant, lung transplant or significant surgical resection of the bowel. Note: Significant resection of the bowel is defined as any resection of the terminal ileum, or ileo-cecal valve. Patients who have had qualitative, long-term changes in nutritional status after any other bowel resection (e.g., increased, or new, need for supplementation compared to pre-op in order to maintain the same nutritional status) should also be excluded. 6. Any acute or chronic diarrheal illness unrelated to PI (e.g. infectious gastroenteritis, sprue, lactose intolerance, inflammatory bowel disease). 7. Unable to discontinue enteral tube feedings during the study. 8. Known hypersensitivity to food additives. 9. Inability to consume the PP diets, in the judgment of the Investigator. 10. Participation in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to the Screening Visit. 11. ALT or AST level > 5x ULN, or total bilirubin level > 1.5x ULN at the Screening Visit or at Baseline (except for patients with Gilbert Syndrome). 12. Signs and/or symptoms of liver cirrhosis or portal hypertension (e.g., splenomegaly, ascites, esophageal varices), or documented liver disease unrelated to CF‡. 13. DIOS in the last six months prior to the Screening Visit. 14. Unable to discontinue the use of pancreatic enzymes. 15. Any condition that the Investigator believes would interfere with the intent of this study or would make participation not in the best interest of the patient. 16. Patient is unlikely to complete the study, as determined by the Investigator.
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E.5 End points |
E.5.1 | Primary end point(s) |
Mean change in CFA from Baseline Period to Double-Blind Treatment Period. to determine the efficacy of ALTU-135 for the treatment of fat malabsorption in patients with CF-related exocrine PI. |
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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 | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
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 | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
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.8.3 |
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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 17 |
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 | Information not present in EudraCT |
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 |
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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 | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 1 |