Condition category
Nutritional, Metabolic, Endocrine
Date applied
23/01/2014
Date assigned
14/04/2014
Last edited
14/04/2014
Prospective/Retrospective
Retrospectively registered
Overall trial status
Completed
Recruitment status
No longer recruiting

Plain English Summary

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, we do not know if exocrine pancreatic dysfunction in childhood severe malnutrition can be corrected by PERT and will lead to additional weight gain. Our objective 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 will be randomly divided in 2 groups: the group getting the enzymes (PERT) and the control group. Both groups will be treated according to the current departmental refeeding guidelines that are based on what the World Health Organization recommends. The group getting PERT will 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 will take 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?
The study will start mid February 2014. We are aiming to recruit all patients within 6 months.

Who is funding the study?
Stichting Steun Emma Kinderziekenhuis” (Foundation Support at the Emma Children’s Hospital in Amsterdam). Netherlanfds. See also: http://steunemma.nl/site/stichting/
Who is the main contact?
Dr. Wieger P. Voskuijl

Trial website

Contact information

Type

Scientific

Primary contact

Dr Wieger Voskuijl

ORCID ID

Contact details

College of Medicine
Department of Paediatrics
Private Bag 360
Chichiri
Blantyre
N/A
Malawi

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

P.11/12/1306

Study information

Scientific title

The OPTIMISM trial: pancreatic exocrine replacement therapy in severely malnourished children: a pilot study

Acronym

OPTIMISM

Study hypothesis

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

College of Medicine Research Ethics Committee, University of Malawi, Blantyre, Malawi, 02/10/2013, P.11/12/1306

Study design

Prospective randomised single blinded study

Primary study design

Interventional

Secondary study design

Randomised controlled trial

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Not available in web format, please use the contact details below to request a patient information sheet

Condition

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

Phase

Not Applicable

Drug names

Primary outcome measures

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

We will compare 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 trial start date

10/02/2014

Overall trial end date

01/08/2014

Reason abandoned

Eligibility

Participant 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

Participant type

Patient

Age group

Child

Gender

Both

Target number of participants

100

Participant 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

Recruitment start date

10/02/2014

Recruitment end date

01/08/2014

Locations

Countries of recruitment

Malawi

Trial participating centre

College of Medicine
Blantyre
N/A
Malawi

Sponsor information

Organisation

Emma Children's Hospital Foundation Support (Stichting Steun Emma Kinderziekenhuis AMC) (Netherlands)

Sponsor details

Meibergdreef 9 (TKs0-253)
Amsterdam
1105 AZ
Netherlands

Sponsor type

Research organisation

Website

http://www.steunemma.nl/site/de-stichting/

Funders

Funder type

Hospital/treatment centre

Funder name

Emma Children's Hospital Foundation Support (Stichting Steun Emma Kinderziekenhuis) (Netherlands)

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