E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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 | 10011765 |
E.1.2 | Term | Cystic fibrosis pancreas |
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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 evaluate the long-term safety and tolerability of ALTU-135 treatment in patients with Cystic Fibrosis related exocrine Pancreatic Insufficiency |
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E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
≥ 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 either ◦ A genotype with two identifiable mutations consistent with CF, 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 measured at the Screening Visit unless previously documented 6. Able to take pancreatic enzyme supplementation in the form of capsules 7. Able to perform the testing and procedures required for this study, as judged by the Investigator 8. Willing and able to provide informed consent or assent
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E.4 | Principal exclusion criteria |
1. < 7 years of age 2. Pregnancy, breastfeeding or of childbearing potential and not willing to use birth control (IUD; oral, transdermal or parenteral contraceptives; abstinence) during the study 3. Participation in an investigational study of a drug, biologic, or device not currently approved for marketing within 30 days prior to the Screening Visit except for the Phase III Efficacy Study (Protocol 0000726) 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 chronic diarrheal illness unrelated to PI (e.g., sprue or inflammatory bowel disease) 7. ALT or AST level > 5 x ULN, or total bilirubin level > 1.5x ULN at the Screening Visit or at Baseline (except for patients with Gilbert Syndrome) 8. Signs and/or symptoms of liver cirrhosis or portal hypertension (e.g., splenomegaly, ascites, esophageal varices), or documented liver disease unrelated to CF 9. Unable to discontinue enteral tube feedings during the study 10. 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. 11. Baseline CFA ≥ 93% from the Phase III efficacy study (Protocol 0000726) 12. 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) |
To evaluate the long-term safety and tolerability of ALTU-135 treatment in patients with CYstic Fibrosis related exocrine Pancretic Insufficiency |
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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 | No |
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 | Yes |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
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 | No |
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 | 10 |
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 | 10 |