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Cystic FibrosisPhase 1/2PIMNCT01548729

Metabolic Efficiency of Combined Pancreatic Islet and Lung Transplant for the Treatment of End-Stage Cystic Fibrosis

A Phase 1/2 study testing Combined pancreatic islet and lung transplantation in people with Cystic Fibrosis and Diabetes Related Cystic Fibrosis, run by University Hospital, Strasbourg, France at 28 sites in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 14 years and older
What is being tested?
Combined pancreatic islet and lung transplantation
Type of treatment
Surgery or procedure
Could I receive a placebo?
No, everyone receives an active treatment
How many people
14 participants
Where
28 sitesFrance
Study dates
25 Feb 2012to 20 Dec 2019
Phase
Phase 1/2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 14 years and older
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • 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.
Show all 25Show fewer
  • 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)

Who cannot join

None listed.

Trial locations and contacts

28 sites in 1 country.

28 sites in 1 country

France28 sites

  • CRCM AdulteCHU de Grenoble, Hôpital A. Michallon

    Grenoble, 38 043

  • Nephrologie, CHU Grenoble

    Grenoble, 38043

  • Réanimation Cardiovasculaire et Thoracique, Hôpital Michallon

    Grenoble, 38043

  • Service d'Endocrinologie, CHU de Grenoble

    Grenoble, 38043

  • Service de Chirurgie Cardiaque, CHU Grenoble

    Grenoble, 38043

  • Service de Radiologie Interventionnelle, CHU de Grenoble

    Grenoble, 38043

  • Service de Pneumologie, Hôpital A. MICHALLON , CHU de Grenoble

    Grenoble, 38700

  • Service d'Urologie et chirurgie de la Transplantation, Groupement Hospitalier Edouard Herriot

    Lyon, 69 437

  • Service de médecine de la transplantation et immunologie clinique, Hôpital Edouard Herriot

    Lyon, 69437

  • CRCM adulte, Centre Hospitalier Lyon-Sud

    Lyon, 69495

  • Service d'Endocrinologie, Pavillon médical

    Lyon, 69495

  • Service d'imagerie radiologique et de médecine nucléaire, Centre Hospitalier Lyon-Sud

    Lyon, 69495

  • Service de Chirurgie Thoracique, HOPITAL LOUIS PRADEL

    Lyon, 69677

  • Service de pneumologie, Hôpital Louis Pradel

    Lyon, 69677

  • Service de la Clinique d'Endocrinologie, maladies métaboliques et Nutrition

    Nantes, 44093

  • Service de Néphrologie et Immuno-transplantation

    Nantes, 44093

  • Service de Pneumologie

    Nantes, 44093

  • Service de Radiologie

    Nantes, 44093

  • Service d'endocrinologie, diabète et maladies métaboliques

    Strasbourg, 67 091

  • Service d'Anesthésie-Réanimations chirurgicales, Nouvel Hôpital Civil

    Strasbourg, 67091

  • Service d'Anesthésie-Réanimations Chirurgicales

    Strasbourg, 67091

  • Service de chirurgie, Nouvel Hôpital Civil

    Strasbourg, 67091

  • Service de pneumologie, Nouvel Hôpital Civil

    Strasbourg, 67091

  • Service de Radiologie, Hôpital de Hautepierre

    Strasbourg, 67098

  • Unité d'Endocrinologie, Diabétologie et Médecine Métabolique - Hôpital Foch

    Suresnes, 92150

  • Service d'Imagerie - Hôpital Foch

    Suresnes, 92151

  • Service de Chirurgie Thoracique et Transplantation Pulmonaire - Hôpital Foch

    Suresnes, 92151

  • Service de Pneumologie - Hôpital Foch

    Suresnes, 92151

Other trials in France

About this study

Official title: Metabolic Efficiency of Combined Pancreatic Islet and Lung Transplant for the Treatment of End-Stage Cystic Fibrosis : a Pilot Study

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.

Being tested
Combined pancreatic islet and lung transplantation
Keywords
Cystic Fibrosis, Diabetes Related Cystic Fibrosis, Lung transplantation, Pancreatic islets, Insulin-dependent diabetes
Medical subject headings
Cystic Fibrosis, Diabetes Mellitus, Type 1

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Combined pancreatic islet and lung transplantationProcedure or surgery

    Combined pancreatic islet and lung transplant from the same donor for the treatment of patients with end-stage cystic fibrosis

Study groups in NCT01548729
Study groupRoleWhat they receive
Patient with cystic fibrosisExperimentalPatients with end-stage cystic fibrosis
Receives: Procedure: Combined pancreatic islet and lung transplantation

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Metabolic efficiency at 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

    Measured: 1 year

Secondary outcomes (22)

  1. Ratio [C-peptide stimulated T6min/ C-peptide basal T0]

    Δ C peptide = [ C-peptide stimulated T6min/ C-peptide basal T0] Success if Δ C peptide > 2

    Measured: 1 year after transplant

  2. Ratio [C-peptide/Glucose-Creatinine] & ratio [C-peptide/Glucose]

    Measured: Every week during the first month, and every month during 1 year

  3. HbA1c

    Measured: Every 3 months during 1 year after transplant

  4. C-peptide stimulated by glucagon

    Measured: Every 3 months during 1 year after transplant

  5. Ratio [C-peptide stimulated T6min/ C-peptide basal T0]

    Measured: Every 3 months during 1 year after transplant

  6. Microalbuminuria & proteinuria

    Measured: Every 3 months during 1 year after transplant

  7. Insulin requirements

    Unit/day

    Measured: Every month during 1 year after transplant

  8. Number of minor hypoglycemia

    defined by a blood glucose level < 0.6g/L at which the patient is capable of self-sugaring

    Measured: Every month during 1 year after transplant

  9. Number of major hypoglycemia

    Measured: Every month during 1 year after transplant

  10. Glycemic variability (MAGE)

    by continuous glycemic measurement Holter (CGMS) \& glycemic reader memory analysis

    Measured: Every 6 months during 1 year after transplant

  11. duration of hypoglycemia

    by continuous glycemic measurement Holter (CGMS) \& glycemic reader memory analysis

    Measured: Every 6 months during 1 year after transplant

  12. Forced Expiratory Volume (FEV1)

    Measured: Every month during 1 year after transplant

  13. Forced Vital Capacity (FVC)

    Measured: Every month during 1 year after transplant

  14. Tiffeneau-Pinelli index

    FEV1/FVC

    Measured: Every month during 1 year after transplant

  15. Nature of pulmonary infection episodes and nature of acute lung rejection if need be

    Measured: Every month during 1 year after transplant

  16. Dyspnea score according to the mMRC scale

    Measured: Every month during 1 year after transplant

  17. Oxygen saturation SaO2 room air

    Measured: Every month during 1 year after transplant

  18. Median maximum expiration flow

    Measured: Every month during 1 year after transplant

  19. Number of episodes of pulmonary rejection

    requiring a corticosteroid bolus

    Measured: Every visit during 1 year after transplant

  20. Number of days of post-transplant hospitalization and during the follow-up

    Measured: Every visit during 1 year after transplant

  21. Mortality

    Measured: Every visit during 1 year after transplant

  22. Adverse events

    Measured: Every visit during 1 year after transplant

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Mar 2012 (estimated)
  2. Study start25 Feb 2012
  3. Primary completion20 Dec 2019
  4. Study completion20 Dec 2019

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
14 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01548729
Sponsor's protocol ID
4790
Acronym
PIM
Status last verified
Mar 2021
First submitted
10 Feb 2012
Last updated
30 Mar 2021
Results posted
No
Data monitoring committee
Yes

Publications

  • Rakotoarisoa L, Wagner C, Munch M, Renaud Picard B, Grenet D, Olland A, Greget M, Enescu I, Bouilloud F, Bonnette P, Guth A, Bosco D, Mercier C, Rabilloud M, Berney T, Yves Benhamou P, Massard G, Camilo C, Colin C, Arnold C, Kessler R, Kessler L; GRAGIL-TREPID Group. Feasibility and efficacy of combined pancreatic islet-lung transplantation in cystic fibrosis-related diabetes-PIM study: A multicenter phase 1-2 trial. Am J Transplant. 2022 Jul;22(7):1861-1872. doi: 10.1111/ajt.17058. Epub 2022 Apr 26.

    PubMed 35403818

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Combined pancreatic islet and lung transplantation in people with Cystic Fibrosis and Diabetes Related Cystic Fibrosis, run by University Hospital, Strasbourg, France at 28 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 25 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 30 Mar 2021. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 28 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Metabolic efficiency at 1 year (time frame: 1 year). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01548729, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Mar 2021.

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