E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Cystic Fibrosis Related Diabetes (CFRD) |
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E.1.1.1 | Medical condition in easily understood language |
Diabetes of Cystic Fibrosis |
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E.1.1.2 | Therapeutic area | Body processes [G] - Physiological processes [G07] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10022468 |
E.1.2 | Term | Insulin |
E.1.2 | System Organ Class | 100000004848 |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
There is a body of evidence in Type 1 and Type 2 Diabetes that “resting” the pancreas by giving insulin in the early stages of the disease allows the pancreatic beta cells to recover, thereby delaying progression of disease. This has not been looked at in CF and our study aims to test this.
Hence, the main objective of this study is to test the folowing hypothesis:
Supplementation with small doses of insulin in individuals with CF and altered glucose tolerance leads to an improvement in the first phase responses in beta-cell functioning |
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E.2.2 | Secondary objectives of the trial |
1. To study whether supplementation of insulin deficient CF patients, with small doses of insulin, leads to an increase in β-cell sensitivity in the dynamic state
2. To evaluate if beta-cell rest improves the overall glycaemic profile in an individual with CF and the duration this is sustained for?
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Male or female with a confirmed diagnosis of cystic fibrosis defined by
a) Clinical features consistent with a diagnosis of CF AND
b) Sweat chloride ≥60mmol/L by pilocarpine ionotophoresis; OR
c) Genotypic confirmation of CFTR mutation
2. Aged 18 – 50 years
3. Outpatients from the regional adult unit in Liverpool
4. Currently not on insulin
5. Clinically stable over the preceding 4 weeks i.e. no indication for iv antibiotics, steroids or hospital admissions
6. CGM result: At least 4.5% of time spent ≥7.8 % (This indicates altered glucose handling implying insulin insufficiency) |
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E.4 | Principal exclusion criteria |
1. Patients on glucose lowering medications (insulin, oral agents)
2. Ongoing acute illness
3. Those on long term oral steroids
4. Pregnant women
5. Those on immunosuppressive treatment
6. History of, or planned organ transplant
7. Known clinically significant abnormal findings on haematology or clinical chemistry
8. Subjects with documented or suspected, clinically significant, alcohol or drug abuse. The determination of clinical significance will be determined by the investigator.
9. Current malignant disease
10. Any serious or active medical or psychiatric illness, which in the opinion of the investigator, would interfere with subject treatment, assessment, or compliance with the protocol. |
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E.5 End points |
E.5.1 | Primary end point(s) |
This is an observational physiological study. Completion of whole study, change of clinical status between or during the study days, commencement of insulin for a clinical need, patient withdrawing consent or death. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | No |
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Physiological Study
Our study does not evaluate the efficacy of insulin to create pancreatic rest (this is already known), but it does seek to evaluate the effect of resting the pancreas (by using insulin as a physiological probe) on its subsequent ability to respond to a glucose load. |
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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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
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 | Yes |
E.8.1.7.1 | Other trial design description |
Observational Physiological Study |
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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 | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | No |
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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LVLS.
However, these patients are all seen routinely in clinic as part of their standard CF care. |
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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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |