Metabolic Efficiency of Combined Pancreatic Islet and Lung Transplant for the Treatment of End-Stage Cystic Fibrosis (PIM)
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| ClinicalTrials.gov Identifier: NCT01548729 |
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Recruitment Status :
Completed
First Posted : March 8, 2012
Last Update Posted : March 30, 2021
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Sponsor:
University Hospital, Strasbourg, France
Information provided by (Responsible Party):
University Hospital, Strasbourg, France
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Brief Summary:
Patients with end-stage cystic fibrosis (CF) and severe CF-related diabetes (CFRD) may benefit from combined lung-pancreatic islet transplantation. A recent case series showed that combined bilateral lung and pancreatic islet transplantation is a viable therapeutic option for patients with end-stage CF and CFRD. The use of different organs from a single donor may lead to reduced immunogenicity. As the prevalence of CFRD has increased dramatically with the improved life expectancy of patients with CF, islet transplantation should be considered at the end-stage CF. By restoring metabolic control, the investigators hypothesize that islet transplantation may improve the management of CF patients undergoing lung transplant and decrease the complication rate in the early postoperative period.
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Cystic Fibrosis Diabetes Related Cystic Fibrosis Insulin-dependent Diabetes | Procedure: Combined pancreatic islet and lung transplantation | Phase 1 Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 14 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| Official Title: | Metabolic Efficiency of Combined Pancreatic Islet and Lung Transplant for the Treatment of End-Stage Cystic Fibrosis : a Pilot Study |
| Actual Study Start Date : | February 25, 2012 |
| Actual Primary Completion Date : | December 20, 2019 |
| Actual Study Completion Date : | December 20, 2019 |
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| Arm | Intervention/treatment |
|---|---|
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Experimental: Patient with cystic fibrosis
Patients with end-stage cystic fibrosis
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Procedure: Combined pancreatic islet and lung transplantation
Combined pancreatic islet and lung transplant from the same donor for the treatment of patients with end-stage cystic fibrosis |
Primary Outcome Measures :
- Metabolic efficiency at 1 year [ Time Frame: 1 year ]Combined criteria based on the 4 following criteria: weight increase > 5% compared to inclusion, fasting blood glucose < 1.1 g/l at 12 months post-transplant (beta score criterion), Reducing insulin requirements (expressed in UI/day) compared to inclusion & decreased in HbA1c >= 0.5% (in absolute value) compared to inclusion Success is defined by achieving at least three of these criteria
Secondary Outcome Measures :
- Ratio [C-peptide stimulated T6min/ C-peptide basal T0] [ Time Frame: 1 year after transplant ]Δ C peptide = [ C-peptide stimulated T6min/ C-peptide basal T0] Success if Δ C peptide > 2
- Ratio [C-peptide/Glucose-Creatinine] & ratio [C-peptide/Glucose] [ Time Frame: Every week during the first month, and every month during 1 year ]
- HbA1c [ Time Frame: Every 3 months during 1 year after transplant ]
- C-peptide stimulated by glucagon [ Time Frame: Every 3 months during 1 year after transplant ]
- Ratio [C-peptide stimulated T6min/ C-peptide basal T0] [ Time Frame: Every 3 months during 1 year after transplant ]
- Microalbuminuria & proteinuria [ Time Frame: Every 3 months during 1 year after transplant ]
- Insulin requirements [ Time Frame: Every month during 1 year after transplant ]Unit/day
- Number of minor hypoglycemia [ Time Frame: Every month during 1 year after transplant ]defined by a blood glucose level < 0.6g/L at which the patient is capable of self-sugaring
- Number of major hypoglycemia [ Time Frame: Every month during 1 year after transplant ]
- Glycemic variability (MAGE) [ Time Frame: Every 6 months during 1 year after transplant ]by continuous glycemic measurement Holter (CGMS) & glycemic reader memory analysis
- duration of hypoglycemia [ Time Frame: Every 6 months during 1 year after transplant ]by continuous glycemic measurement Holter (CGMS) & glycemic reader memory analysis
- Forced Expiratory Volume (FEV1) [ Time Frame: Every month during 1 year after transplant ]
- Forced Vital Capacity (FVC) [ Time Frame: Every month during 1 year after transplant ]
- Tiffeneau-Pinelli index [ Time Frame: Every month during 1 year after transplant ]FEV1/FVC
- Nature of pulmonary infection episodes and nature of acute lung rejection if need be [ Time Frame: Every month during 1 year after transplant ]
- Dyspnea score according to the mMRC scale [ Time Frame: Every month during 1 year after transplant ]
- Oxygen saturation SaO2 room air [ Time Frame: Every month during 1 year after transplant ]
- Median maximum expiration flow [ Time Frame: Every month during 1 year after transplant ]
- Number of episodes of pulmonary rejection [ Time Frame: Every visit during 1 year after transplant ]requiring a corticosteroid bolus
- Number of days of post-transplant hospitalization and during the follow-up [ Time Frame: Every visit during 1 year after transplant ]
- Mortality [ Time Frame: Every visit during 1 year after transplant ]
- Adverse events [ Time Frame: Every visit during 1 year after transplant ]
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| Ages Eligible for Study: | 14 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion criteria:
- Patient with cystic fibrosis
- Patient able to respect the protocol procedures
- Patient with end-stage respiratory insufficiency indicating a lung transplant
- Clinical history of cystic fibrosis related diabetes and/or insulin-dependent diabetes, with no residual insulin secretion (C-peptide < 0,5 ng/mL) and/or no response to IV glucagon stimulation: [peak stimulated C-peptide (T6min)/basal plasma C-peptide(T0)] < 2. The absence of insulin secretion will be verified 2 times before inclusion
- Evolution of diabetes for over 3 years
- Patient whose glycaemic control obtained with insulin therapy is not satisfactory and could significantly alter quality of life (HbA1c > 7% and/or MAGE index > 1,25). This situation is assessed by a diabetologist.
- Social Security membership or benefit from Social Welfare
- Patient who received the results of the medical evaluation required
Non-inclusion criteria:
- Patient with contra-indication for undergo a lung transplant
- Patient with an indication of heart, liver or kidney transplantation
- Patient for which poor therapeutic compliance is expected
- Patient under oral antidiabetic drug
- In women of childbearing potential: pregnancy test (urine and / or blood) positive, lactation, or unwilling to use effective contraception for the duration of the study until 3 months after the end . Men: procreation project during the study period up to 3 months after its end, or unwilling to use effective contraception patient.
- Active infection, including hepatitis B, hepatitis C, HIV
- Any history of malignancy within the past 5 years, with the exception of cutaneous basal cell carcinomas completely resected. The history of breast or melanoma cancers are an absolute contraindication
- Alcoholic intoxication or drug addiction
- Anemia (hemoglobin Hb <10g / dL in women and Hb <11 g / dL in men), lymphopenia (<1000 / uL), neutropenia (<1500 / uL) or thrombocytopenia (<100,000 / uL).
- Persistent elevated liver enzymes at baseline
- Portal hypertension identified by oesophageal varice and/or hypersplenism (platelets < 120 000 /mm3) or Child-Pugh score > 6.
- Use of a medical treatment under investigation within 4 weeks before inclusion
- All medical situation assessed by an investigator which could interfere with the good management of the project
- Patient restricted of freedom or unable to give his consent
- Patient has been included in another study that could interfere with the results of the study
- Contraindications to the use of experimental drugs (Cellcept®, prednisone, Prograf, prednisolone, methylprednisolone, and pancreatic islets)
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01548729
Locations
Show 28 study locations
Sponsors and Collaborators
University Hospital, Strasbourg, France
Investigators
| Principal Investigator: | Laurence KESSLER, MD, PhD | Hôpitaux Universitaires de Strasbourg | |
| Principal Investigator: | Pierre Yves BENHAMOU, MD, phD | University Hospital, Grenoble | |
| Principal Investigator: | Dominique Grenet, MD | HOPITAL FOCH DE SURESNES | |
| Principal Investigator: | Charles THIVOLET, MD | Hospices Civils de Lyon | |
| Principal Investigator: | Thierry BERNEY, MD PhD | CENTRE ROMAND DE TRANSPLANTATION - SUISSE |
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | University Hospital, Strasbourg, France |
| ClinicalTrials.gov Identifier: | NCT01548729 |
| Other Study ID Numbers: |
4790 |
| First Posted: | March 8, 2012 Key Record Dates |
| Last Update Posted: | March 30, 2021 |
| Last Verified: | March 2021 |
Keywords provided by University Hospital, Strasbourg, France:
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Cystic Fibrosis Diabetes Related Cystic Fibrosis Lung transplantation Pancreatic islets Insulin-dependent diabetes |
Additional relevant MeSH terms:
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Cystic Fibrosis Fibrosis Diabetes Mellitus Diabetes Mellitus, Type 1 Glucose Metabolism Disorders Metabolic Diseases Endocrine System Diseases Pathologic Processes |
Pancreatic Diseases Digestive System Diseases Lung Diseases Respiratory Tract Diseases Genetic Diseases, Inborn Infant, Newborn, Diseases Autoimmune Diseases Immune System Diseases |