Contact information
Type
Scientific
Primary contact
Prof Pal Njolstad
ORCID ID
Contact details
Section for Paediatrics
Department of Clinical Medicine
Haukeland University Hospital
Bergen
5021
Norway
+47 55 97 51 53
pal.njolstad@pedi.uib.no
Additional identifiers
EudraCT number
ClinicalTrials.gov number
Protocol/serial number
N/A
Study information
Scientific title
Acronym
Study hypothesis
The MODY8 syndrome is a monogenically inherited syndrome of diabetes and pancreatic exocrine dysfunction due to single-base deletion mutations in the Carboxyl-Ester Lipase (CEL) gene Variable Number of Tandem Repeats (VNTR), registered in OMIM as MODY8 or DPED.
Hypotheses:
That pancreatic enzyme substitution therapy will:
1. Ameliorate exocrine function as reflected by fecal fat excretion and fat-soluble vitamin status
2. Improve glycemic control as measured by HbA1c
3. Improve neuropathology in patients with the MODY8 syndrome
Ethics approval
The Regional Committee for Research Ethics of Western Norway (REK Vest), approved on 11 November 2004, (ref: REK Vest 209.04)
Study design
Open, non-randomized, single-center, interventional study.
Primary study design
Interventional
Secondary study design
Non randomised controlled trial
Trial setting
Not specified
Trial type
Treatment
Patient information sheet
Condition
MODY8 syndrome
Intervention
All participants initially received one Creon enterocapsule (Solvay Pharmaceuticals, Germany) containing 10,000 units lipase, 18,000 units amylase, 600 units protease three times daily, orally at meals. If the clinical effect was unsatisfactory based on patient symptoms, the dose was first increased to 1-2 capsules 3-4 times daily, and if the clinical effect was still unsatisfactory, the medication was changed to Creon Forte, taken orally with meals, 1-2 capsules per meal.
Intervention type
Drug
Phase
Not Specified
Drug names
Creon enterocapsule and Creon Forte,Creon enterocapsule and Creon Forte
Primary outcome measure
1. Fecal elastase-1, assessed at 0, 6, 12 and 30 months
2. Fecal fat excretion, assessed at 0, 12 and 30 months
3. HbA1c (blood test), assessed at 0, 6, 12 and 30 months
4. Vitamins A, D and E (blood test), assessed at 0, 6, 12 and 30 months
5. Creatinine (blood test), assessed at 0, 6, 12 and 30 months
6. Total calcium (blood test), assessed at 0, 6, 12 and 30 months
7. Total High Density Lipoprotein (HDL) and Low Density Lipoprotein (LDL) cholesterols (blood test), assessed at 0, 6, 12 and 30 months
8. Triglycerides (blood test), assessed at 0, 6, 12 and 30 months
9. C-peptide (blood test), assessed at 0, 6, 12 and 30 months
10. Bone mass density (age-matched Z-scores), assessed at 0, 12 and 30 months
11. Visual evoked potential, assessed at 0 and 18 months
12. Sensory evoked potential, assessed at 0 and 18 months
13. Nerve conduction velocity, assessed at 0 and 18 months
Secondary outcome measures
No secondary outcome measures
Overall trial start date
01/09/2004
Overall trial end date
30/06/2007
Reason abandoned (if study stopped)
Eligibility
Participant inclusion criteria
Participants should:
1. Be a carrier of a single-nucleotide deletion mutation in the CEL VNTR
2. Have diabetes by the World Health Organization criteria
3. Have exocrine dysfunction defined by fecal elastase <200 micrograms/ml in two consecutive tests
4. Patients of both sexes and all ages should be included
Participant type
Patient
Age group
Not Specified
Gender
Both
Target number of participants
16
Participant exclusion criteria
1. Ongoing treatment with pancreatic enzyme supplements
2. Inability to attend clinical examinations and other necessary investigations for geographical reasons
3. Side effects of medication (strong stomach ache)
Recruitment start date
01/09/2004
Recruitment end date
30/06/2007
Locations
Countries of recruitment
Norway
Trial participating centre
Section for Paediatrics
Bergen
5021
Norway
Sponsor information
Organisation
University of Bergen (Norway)
Sponsor details
Faculty of Medicine
Post Box 7804
Bergen
5020
Norway
+47 55 58 20 86
post@medfa.uib.no
Sponsor type
University/education
Website
Funders
Funder type
University/education
Funder name
Haukeland University Hospital, Innovest, University of Bergen (Norway)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Funder name
The Norwegian Research Council (FUGE Program)
Alternative name(s)
Funding Body Type
Funding Body Subtype
Location
Results and Publications
Publication and dissemination plan
Not provided at time of registration
Intention to publish date
Participant level data
Not provided at time of registration
Basic results (scientific)
Publication list