UMIN-CTR Clinical Trial

Basic information
Official scientific title of the study	Evaluation of the correlation between serum carnitine and constipation
Title of the study (Brief title)	Evaluation of the correlation between serum carnitine and constipation
Region	Japan

Condition
Condition	carnitine defeciensy, constipatin\on
Classification by specialty	Medicine in general Gastroenterology
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	Carnitine, which is acquired mostly through the diet, is an essential nutrient related to the mitochondrial transport of fatty acids. Carnitine deficiency causes a variety of symptoms in multiple organs due to mitochondrial dysfunction. Most patients with severe motor and intellectual disabilities have epilepsy and are prescribed anti-epileptic drugs such as valproate sodium, which promotes the excretion of carnitine. Furthermore, as most of these patients cannot feed themselves, they receive enteral nutrition, which has less carnitine content than a normal diet. Use of valproate sodium and most enteral nutrition formulas for an extended time has been reported as a risk factor for carnitine deficiency. Therefore, patients with severe motor and intellectual disabilities are known to be at risk of deficiency of carnitine. In our clinical practice we have treated many patients with severe motor and intellectual disabilities who have experienced relief from constipation after supplementation of carnitine. However, to our knowledge, there are few reports of studies designed to examine correlations between gastrointestinal dysmotility and deficiency of carnitine. Therefore, this study aimed to retrospectively review clinical data of patients with severe motor and intellectual disabilities in order to assess our suspicion of a correlation between constipation and carnitine deficiency.
Basic objectives2	Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	First, we examined the correlation between level of free carnitine and severity of constipation. Second, we compared the constipation group with the non-constipation group in terms of age, sex, BMI, bed rest period (years), use of anti-epileptic drugs, use of valproate sodium, use of enteral nutrition, recurrent pulmonary infection (more than twice a year), presence of pressure ulcer, and results of blood tests for nutritional assessment (including serum levels of albumin, pre-albumin, total cholesterol, free carnitine, folic acid, and the trace elements Cu, Fe, Se, and Zn). Blood samples were collected in all cases during times when there were no observed inflammation reactions and no prescribed antibiotics. Carnitine levels also were obtained from patients without motor and intellectual disabilities who had severe chronic constipation requiring disimpaction with an enema on a daily basis. Third, we compared the frequency of defecation and the form of feces before and after therapeutic supplementation with carnitine.
Key secondary outcomes

Base
Study type	Interventional

Study design
Basic design	Single arm
Randomization	Non-randomized
Randomization unit
Blinding	Open -no one is blinded
Control	Self control
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	Supplementation with 10-50 mg/kg/day carnitine (P<0.001). No other changes of medication were made during the supplementation period.
Interventions/Control_2
Interventions/Control_3
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit	2 years-old <=
Age-upper limit	45 years-old >=
Gender	Male and Female
Key inclusion criteria	Patients with severe motor and intellectual disabilities were defined as having an inability to move by themselves and IQ <35
Key exclusion criteria	The patients with infection or prescribed with antibiotics
Target sample size	27

Research contact person
Name of lead principal investigator	Shinya Murata
Organization	Hirakata City Hospital,
Division name	Department of Pediatrics,
Address	2-14-1 Kinya-honmachi, Hirakata, Osaka 573-1013, Japan
TEL	072-847-2821
Email	wildwind1980@live.jp

Public contact
Name of contact person	Shinya Murata
Organization	Hirakata City Hospital,
Division name	Department of Pediatrics,
Address	2-14-1 Kinya-honmachi, Hirakata, Osaka 573-1013, Japan
TEL	072-847-2821
Homepage URL
Email	wildwind1980@live.jp

Sponsor
Institute	Department of Pediatrics, Hirakata City Hospital
Institute
Department

Funding Source
Organization	Department of Pediatrics, Hirakata City Hospital
Organization
Division
Category of Funding Organization	Local Government
Nationality of Funding Organization

Other related organizations
Co-sponsor
Name of secondary funｄer(s)

Secondary IDs
Secondary IDs	NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information	2016 Year 06 Month 25 Day

Progress
Recruitment status	Completed
Date of protocol fixation	2015 Year 01 Month 01 Day
Anticipated trial start date	2015 Year 01 Month 01 Day
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Related information
URL releasing protocol
Publication of results	Published
URL releasing results
Results
Other related information

Management information
Registered date	2016 Year 06 Month 23 Day
Last modified on	2017 Year 07 Month 18 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000026340