Condition category
Nutritional, Metabolic, Endocrine
Date applied
21/11/2007
Date assigned
04/12/2007
Last edited
04/12/2007
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

Not provided at time of registration

Trial website

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 measures

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

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

http://www.uib.no/info/english/

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

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes