Pancreatic exocrine replacement therapy in severely malnourished children
| ISRCTN | ISRCTN57423639 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN57423639 |
| Secondary identifying numbers | P.11/12/1306 |
- Submission date
- 23/01/2014
- Registration date
- 14/04/2014
- Last edited
- 18/09/2017
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Nutritional, Metabolic, Endocrine
Plain English summary of protocol
Background and study aims
Every day, 24000 children under five die of which more than one third of deaths are associated with malnutrition. Malnourished children have an inability to properly digest food because they lack digestive enzymes made by the pancreas, an abdominal digestive organ. These enzymes are used to break down fats, proteins and carbohydrates into units that can be digested. Loss of digestive enzymes leads to incomplete digestion and insufficient absorption of nutrients. In other diseases with these characteristics, like Cystic Fibrosis, improving this maldigestion with pancreatic enzyme replacement therapy (PERT) is very common. The goal of PERT is to restore normal digestion, achieve adequate nutritional status and to reach a normal pattern of growth. However, it is not known whether exocrine pancreatic dysfunction in childhood severe malnutrition can be corrected by PERT and will lead to additional weight gain. The aim of this study is to test whether PERT results in additional weight gain in severely malnourished children.
Who can participate?
Severely malnourished children, aged between 6 and 60 months, admitted to the ward dedicated to malnourished children: MOYO House or Moyo Nutritional Rehabilitation Unit. Both HIV positive and negative children can participate.
What does the study involve?
100 children are randomly allocated into two groups: the group getting the enzymes (PERT) and the control group. Both groups are treated according to the current departmental refeeding guidelines that are based on what the World Health Organization recommends. The group getting PERT receive this during a four-week period.
What are the possible benefits and risks of participating?
Several benefits are present for participants. A special study team will follow and take care of the children during the entire admission to MOYO. The study information provided to the parents creates an opportunity to learn more about the nutritional problem and the health of their child. No more than the maximum recommended daily dose per kilogram bodyweight, as is currently practiced in children with Cystic Fibrosis, will be prescribed. Mild discomfort and minimal bleeding is related to the venous blood drawing.
Where is the study run from?
The study takes place in MOYO Nutritional Rehabilitation Unit, Paediatric Department, Queen Elizabeth Central Hospital, Blantyre, Malawi.
When is the study starting and how long is it expected to run for?
February 2014 to August 2014
Who is funding the study?
Stichting Steun Emma Kinderziekenhuis (Foundation Support at the Emma Childrens Hospital in Amsterdam) (Netherlands)
Who is the main contact?
Dr Wieger P. Voskuijl
Contact information
Scientific
College of Medicine
Department of Paediatrics
Private Bag 360
Chichiri
Blantyre
N/A
Malawi
Study information
| Study design | Prospective randomised single-blinded study |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Hospital |
| Study type | Treatment |
| Participant information sheet | Not available in web format, please use the contact details to request a patient information sheet |
| Scientific title | The OPTIMISM trial: pancreatic exocrine replacement therapy in severely malnourished children: a pilot study |
| Study acronym | OPTIMISM |
| Study objectives | Exocrine pancreatic dysfunction in childhood severe malnutrition can be corrected by pancreatic enzymatic replacement therapy and will lead to enhanced weight gain and ultimately to a reduced morbidity and mortality. |
| Ethics approval(s) | College of Medicine Research Ethics Committee, University of Malawi, Blantyre, Malawi, 02/10/2013, P.11/12/1306 |
| Health condition(s) or problem(s) studied | Severe acute malnutrition in children |
| Intervention | Children are randomised to two groups: receiving pancreatic enzyme replacement therapy (PERT) or standard treatment. All children admitted to MOYO have a thick blood film examined for parasitaemia and a haematocrit measured. They are all offered an HIV antibody test with appropriate pre- and post counselling. After admission children will be treated according to the current departmental refeeding guidelines that are WHO based. The intervention will be given during a four-week period. PERT is prescribed as 3000 Units Lipase/kilogram bodyweight 3 times per day with an upper limit dose of 10,000 Units Lipase/kg bodyweight per day 35. If the child is discharged before this period, parents will be given transport money to return to the hospital after the four-week period has ended. |
| Intervention type | Other |
| Primary outcome measure | 1. To compare the percentage of weight gain after 28 days of PERT with the current standard of care (calculated using the final weight at 28 days and the minimum weight during admission) 2. An additional 10% increase in weight will be considered clinically relevant |
| Secondary outcome measures | The differences after 28 days of PERT compared to the current standard of care with respect to: 1. Improvement in exocrine pancreatic function assessed by increased serum cationic and faecal elastase-1 levels 2. Decreased time on the ward in the PERT group 3. Overall mortality by 4 weeks in both groups |
| Overall study start date | 10/02/2014 |
| Completion date | 01/08/2014 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Child |
| Lower age limit | 6 Months |
| Upper age limit | 60 Months |
| Sex | Both |
| Target number of participants | 100 |
| Key inclusion criteria | 1. Severely malnourished children 2. Aged between 6 and 60 months 3. Admitted to MOYO House 4. Diagnosed with kwashiorkor, and/or marasmus (WHO criteria) 5. Both HIV positive and negative children |
| Key exclusion criteria | 1. Malaria (as proven by a positive blood smear) 2. Sepsis (i.e. circulatory failure) 3. Severe pneumonia, vomiting or severe diarrhoea 4. Those whose parent/guardian has not given consent |
| Date of first enrolment | 10/02/2014 |
| Date of final enrolment | 01/08/2014 |
Locations
Countries of recruitment
- Malawi
Study participating centre
N/A
Malawi
Sponsor information
Research organisation
Meibergdreef 9 (TKs0-253)
Amsterdam
1105 AZ
Netherlands
| Website | http://www.steunemma.nl/site/de-stichting/ |
|---|---|
"ROR" |
https://ror.org/00bmv4102 |
Funders
Funder type
Hospital/treatment centre
No information available
Results and Publications
| Intention to publish date | |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Not provided at time of registration |
| Publication and dissemination plan | Not provided at time of registration |
| IPD sharing plan |
Study outputs
| Output type | Details | Date created | Date added | Peer reviewed? | Patient-facing? |
|---|---|---|---|---|---|
| Results article | results | 01/11/2017 | Yes | No |
Editorial Notes
18/09/2017: Publication reference added.
