NCT01722682

Brief Summary

The goal of this study is to evaluate safety and efficacy of bone marrow (BM) as site for pancreatic islet transplantationin humans. Our hypothesis is that BM represents a better site than liver (currently the location of choice for this procedure) thanks to its potential capacity to favor islet engraftment. To address our hypothesis we propose herein a randomized phase II trial to compare BM and liver as sites for islet transplantation in T1D patients.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
9

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jun 2012

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

June 1, 2012

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

October 31, 2012

Completed
7 days until next milestone

First Posted

Study publicly available on registry

November 7, 2012

Completed
4.8 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2017

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2017

Completed
Last Updated

November 4, 2020

Status Verified

November 1, 2020

Enrollment Period

5.3 years

First QC Date

October 31, 2012

Last Update Submit

November 2, 2020

Conditions

Type 1 Diabetes

Keywords

Diabetes

Outcome Measures

Primary Outcomes (1)

  • Insulin secretion under stimulation

    basal and -10 to 120 min time course of glucose, C-pep levels and insulin derived from the mixed meal tolerance test

    month 12 post-Tx

Secondary Outcomes (3)

  • Incidence and severity of Adverse Events (AE) and Serious Adverse Events (SAE)

    throughout the study up to 1 year after first transplant

  • Insulin requirement

    month 1, 3, 6, 9, 12 post- transplant

  • Islet function

    month 1, 3, 6, 9, 12 post-Tx

Study Arms (2)

A: intraportal islet infusion

ACTIVE COMPARATOR

Patients will received islet into the liver through the portal venous circulation (standard procedure)

Biological: Human pancreatic islet transplantation

B: intra BM islet infusion

EXPERIMENTAL

Patients will received an intra BM islet infusion at the level of the iliac crest. The direct intra BM administration will be performed following the same procedures that our institution utilizes for administration of cord-blood cells in patients with acute leukemia (Lancet Oncol. 2008;9:831). The procedure is easy and reproducible: a standard needle for BM aspiration is inserted in the iliac crest and cells are gently infused.

Biological: Human pancreatic islet transplantation

Interventions

Human pancreatic islet transplantationBIOLOGICAL
A: intraportal islet infusionB: intra BM islet infusion

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • diabetic patients eligible for pancreatic islet transplantation based on local accepted practice and guidelines. This includes at least: a)clinical history compatible with T1D with insulin-dependence for \>5 years; b) undetectable stimulated (arginine or MMTT) C-peptide levels (\<0.3 ng/mL) in the 12 months before transplant c)presence of severe hypoglycaemic events

You may not qualify if:

  • presence of hematologic disease

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Ospedale San Raffaele

Milan, 20132, Italy

Location

Related Publications (1)

  • Maffi P, Nano R, Monti P, Melzi R, Sordi V, Mercalli A, Pellegrini S, Ponzoni M, Peccatori J, Messina C, Nocco A, Cardillo M, Scavini M, Magistretti P, Doglioni C, Ciceri F, Bloem SJ, Roep BO, Secchi A, Piemonti L. Islet Allotransplantation in the Bone Marrow of Patients With Type 1 Diabetes: A Pilot Randomized Trial. Transplantation. 2019 Apr;103(4):839-851. doi: 10.1097/TP.0000000000002416.

    PMID: 30130323RESULT

Related Links

MeSH Terms

Conditions

Diabetes Mellitus, Type 1Diabetes Mellitus

Condition Hierarchy (Ancestors)

Glucose Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesEndocrine System DiseasesAutoimmune DiseasesImmune System Diseases

Study Officials

  • Lorenzo Piemonti, MD

    Ospedale San Raffaele

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Head of Beta Cell Biology Unit, CO-Director Human Islet Transplantation Program (ITP)

Study Record Dates

First Submitted

October 31, 2012

First Posted

November 7, 2012

Study Start

June 1, 2012

Primary Completion

September 1, 2017

Study Completion

December 1, 2017

Last Updated

November 4, 2020

Record last verified: 2020-11

Locations

IT(1)