UMIN-CTR Clinical Trial

Basic information
Public title	Continuous subcutaneous injection of octreotide for congtenital hyperinsulinism: efficacy and safety.
Acronym	Subcutaneous continuous octreotide for congenital hyperinsulinism (SCORCH study)
Scientific Title	Continuous subcutaneous injection of octreotide for congtenital hyperinsulinism: efficacy and safety.
Scientific Title:Acronym	Subcutaneous continuous octreotide for congenital hyperinsulinism (SCORCH study)
Region	Japan

Condition
Condition	congenital hyperinsulinism
Classification by specialty	Pediatrics
Classification by malignancy	Others
Genomic information	YES

Objectives
Narrative objectives1	To examine the efficacy and safety of continuous subcutaneous octreotide injection for congenital hyperinsulinism.
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes	Short term increase in blood glucose comparing the mean blood glucose before 24 hours and after 48 hours of the initiation of the treatment.
Key secondary outcomes	1) Long-term reduction in the amount of glucose infusion to maintain blood glucose at 1 week, 2 weeks, 4 weeks, and at every 3 month after 5 weeks. 2) Neurological outcome after > 1 year of the initiation of the treatment. 3) Number of ypoglycemic episodes < 45 mg/dL during the treatment, categorized as asymptomatic, symptomatic, or severe.

Base
Study type	Interventional

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

Intervention
No. of arms	1
Purpose of intervention	Treatment
Type of intervention	Medicine
Interventions/Control_1	After stabilizing the blood glucse by continuous high concentration glucose infusion, continuous subcutaneous octreotide injection is initiated at a dose of up to 25 micrograms/kg/day with continuous monitoring of blood glucose for the first 48 hours
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 weeks-old <=
Age-upper limit	52 weeks-old >
Gender	Male and Female
Key inclusion criteria	Patients who meet all criteria below. 1 Age between 2 weeks to 1 year and with congenital hyperinsulinemic hypoglycemia (insulin > 3 microunits/mL at blood glucose <45 mg/dL) 2 Requires continuous glucose infusion at > 6 mg/kg/min to maintain blood glucose > 60 mg/dL. 3 Inability to maintain blood glucose > 60 mg/dL with diazoxide at15 mg/kg/d. 4 Consent to participate in the study from the guardians. 5 Consent to undergo molecular testing for congenital hyperinsulinism.
Key exclusion criteria	1) Hypoglycemic patients with causes other than hyperinsulinemia. 2) Patients with severe extrapancreatic comorbidities. a. intestinal dysfunction b. respiratory failure c. severe liver dysfunction with transaminases or bilirubin > 3x ULN for age. d. renal failure with urea nitrogen or creatinine > 3x ULN for age. e. patient with other comorbidities who would not be appropriate for enrollment.
Target sample size	7

Research contact person
Name of lead principal investigator	1st name Middle name Last name Tohru Yorifuji
Organization	Osaka City General Hospital
Division name	Pediatric Endocrinology and Metabolism
Zip code
Address	2-13-22 Miyakojima-Hondori, Miyakojima, Osaka 534-0021
TEL	06-6929-1221
Email	t-yorifuji@med.osakacity-hp.or.jp

Public contact
Name of contact person	1st name Middle name Last name Tohru Yorifuji
Organization	Osaka City General Hospital
Division name	Pediatric Endocrinology and Metabolism
Zip code
Address	2-13-22 Miyakojima-Hondori, Miyakojima, Osaka 534-0021
TEL	06-6929-1221
Homepage URL
Email	scorch-study@med.osakacity-hp.or.jp

Sponsor
Institute	Japan Agency for Medical Research and Development. Research group for the study on "Efficacy and safety of subcutaneous continuous octreotide infusion for congenital hyperinsulinism."
Institute
Department

Funding Source
Organization	Japan Agency for Medical Research and Development.
Organization
Division
Category of Funding Organization	Japanese Governmental office
Nationality of Funding Organization

Other related organizations
Co-sponsor	Japanese Society for Pediatric Endocrinology
Name of secondary funder(s)

IRB Contact (For public release)
Organization
Address
Tel
Email

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	大阪市立総合医療センター（大阪府）、聖マリアンナ医科大学病院（神奈川県）、北海道大学病院（北海道）

Other administrative information
Date of disclosure of the study information	2013 Year 12 Month 19 Day

Related information
URL releasing protocol
Publication of results	Partially published

Result
URL related to results and publications	https://www.jstage.jst.go.jp/article/endocrj/64/9/64_EJ17-0024/_article/-char/en
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status	Main results already published
Date of protocol fixation	2013 Year 10 Month 06 Day
Date of IRB	2013 Year 03 Month 12 Day
Anticipated trial start date	2014 Year 01 Month 01 Day
Last follow-up date	2019 Year 03 Month 31 Day
Date of closure to data entry	2019 Year 04 Month 30 Day
Date trial data considered complete	2019 Year 10 Month 30 Day
Date analysis concluded	2019 Year 11 Month 30 Day

Other
Other related information

Management information
Registered date	2013 Year 12 Month 19 Day
Last modified on	2019 Year 07 Month 21 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000014652