A preliminary safety and efficacy evaluation of direct delivery of autologous bone marrow-derived cells in Egyptian patients with type 1 diabetes mellitus

• Register: ISRCTN
• Last refreshed on: 5 June 2017
• Main ID: ISRCTN15591075
• Date of registration: 02/05/2017
• Prospective Registration: No
• Primary sponsor: Wadi EL-Neel hospital (JCI)
• Public title: A preliminary safety and efficacy evaluation of direct delivery of autologous bone marrow-derived cells in Egyptian patients with type 1 diabetes mellitus
• Scientific title: A preliminary safety and efficacy evaluation of direct intrapancreatic tail delivery of autologous bone marrow-derived cells in Egyptian patients with type 1 diabetes mellitus
• Date of first enrolment: 13/02/2014
• Target sample size: 3
• Recruitment status: Completed
• URL: http://isrctn.com/ISRCTN15591075
• Study type: Interventional
• Study design: Prospective single centre interventional non randomised study (Treatment)
• Phase: Not Applicable

Countries of recruitment

Egypt

Contacts

• Name: Hazem Khamis

Key inclusion & exclusion criteria

Inclusion criteria: 1. Males and females aged between 2 to 30 years of age
2. Confirmed T1D based on the American Diabetes association (ADA) 2015 criteria for diagnosis
3. Body mass index (BMI) of = 29.9
Exclusion criteria: 1. Active infections
2. Any chronic or acute illness
3. Antibodies to hepatitis B surface antigen
4. Hepatitis C
5. Human immunodeficiency virus or evidence of diabetic complications at baseline
6. Pregnant, are breast-feeding or intend to become pregnant during the study
7. Clinically relevant uncontrolled medical conditions not associated with diabetes (such as hematologic, renal, hepatic, neurologic, cardiac and respiratory conditions)
8. Evidence of active malignancy or a prior history of active malignancy

Age minimum:
Age maximum:
Gender: Both

Health Condition(s) or Problem(s) studied

Type 1 diabetes mellitus
Digestive System
Type 1 diabetes mellitus

Intervention(s)

At baseline, participants undergo a clinical examinations and provide blood samples to assess for HbA1c, fasting C-peptide (FCP) and 2hour-C-peptide (2h-CP) levels (as indirect measures of endogenous insulin secretion), levels of islet cells and insulin antibodies. Insulin doses for each participant are determined.

Participants then undergo an autologus bone marrow transplant derived mononuclear cells. This is done using a new strategy (designed to enhance localisation of the injected cells to the diseased pancreas along with the avoidance of nonspecific organ entrapment) by a single administration of bone marrow-derived mononuclear cells (BM-MNCs) via a computed tomography (CT) scan-guided direct transgastricintrapancreatic (DTI) delivery route.

During the procedure, participants are assessed for bleeding from the bone marrow aspiration and transgastric puncture sites, abdominal pain and other clinical complaints (i.e. nausea, vomiting, pain and fever). Blood amylase levels are measured to exclude acute pancreatitis complications. Haematocrit values are obtained only in suspected bleed cases.

Participants are followed up between month one-two, three-five, six-eight, and after eight months (around 12 months) for any clinical complaints. After six months, if islet cells and insulin antibodies are abnormal and serologically positive before the transplantation, there are re-measured to record any improvement or decline in these antibodies. If the patients are negative for islet cell or insulin antibodies before the transplantation, the test is not repeated after six months (as it is not recommended as routine clinical practice). Participants are also followed up to measure their HbA1c, FCP, 2h-CP, insulin doses levels as well as their frequency of hospitalisation for hypo- and hyper-glycemic attacks (common acute short-term complications of uncontrolled diabetes that can lead to hospitalization and account for the majority of costs associated with diabetes care).

Primary Outcome(s)

Safety is measured using the morbidity, mortality, and unwanted side effects (through patient records) during the procedure and at 12 month follow up.

Secondary Outcome(s)

1. Serological changes in positive islet cell and insulin antibody levels are measured using blood samples at baseline and six months
2. ß-cell function is measured using blood samples( HbA1c and in FCP and 2h-CP levels) at baseline, month one-two, month three-five, month six-eight and months eight to 12
3. Abdominal pain and other clinical parameters are measured using clinical examination during the procedure and at month one-two, month three-five, month six-eight and month 12

Secondary ID(s)

0000006555

Source(s) of Monetary Support

Wadi EL-Neel Hospital (JCI)