UMIN-CTR Clinical Trial

Basic information
Public title	Clinical study on efficacy of alendronate infusion for pediatric osteogenesis imperfecta (including severe form)
Acronym	Alendronate infusion for osteogenesis imperfecta
Scientific Title	Clinical study on efficacy of alendronate infusion for pediatric osteogenesis imperfecta (including severe form)
Scientific Title:Acronym	Alendronate infusion for osteogenesis imperfecta
Region	Japan

Condition
Condition	osteogenesis imperfecta
Classification by specialty	Pediatrics Orthopedics
Classification by malignancy	Others
Genomic information	NO

Objectives
Narrative objectives1	To evaluate efficacy and safety of alendronate infusion for pediatric osteogenesis imperfecta
Basic objectives2	Safety,Efficacy
Basic objectives -Others
Trial characteristics_1	Confirmatory
Trial characteristics_2	Pragmatic
Developmental phase	Not applicable

Assessment
Primary outcomes	Bone mineral density (BMD)
Key secondary outcomes	Frequency of fracture

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	Eighteen micrograms (2 ml)/kg of alendronate will be infused in 1 hour every 4 weeks. The study duration is 6 months. When the patient's BMD reaches to the normal range for age, treatment with alendronate will be finished. When the patient's BMD does not reach to the normal range for age, alendronate will be continued for another 6 months.
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	Not applicable
Age-upper limit	15 years-old >=
Gender	Male and Female
Key inclusion criteria	-Informed consent obtained from each subject's parent or the subject's legally acceptable representative before any study-related activities. -Patients clinically diagnosed as osteogenesis imperfecta with all the types according to Sillence phenotypes (I - IV).
Key exclusion criteria	-Patients with the following diseases which show fracture easily: Juvenile osteoporosis, hypophosphatasia, Ehlers-Danlos syndrome, Osteoporosis-pseudoglioma syndrome, Bruck syndrome. -Patients with osteogenesis imperfecta who falls under any of the following conditions shall be excluded: 1) Patients previously treated with any medical treatment including bisphosphonates 2) Patients with severe renal dysfunction 3) Patients with severe brain complication such as intracranial hemorrhage 4) Patients with congenital heart disease
Target sample size	10

Research contact person
Name of lead principal investigator	1st name Ikuma Middle name Last name Fujiwara
Organization	Tohoku University School of Medicine
Division name	Department of Pediatrics
Zip code	022-0872
Address	1-1 Seiryo-machi, Aoba-ku, Sendai
TEL	022-717-7287
Email	ifujiwara-endo@umin.ac.jp

Public contact
Name of contact person	1st name Ikuma Middle name Last name Fujiwara
Organization	Tohoku University School of Medicine
Division name	Department of Pediatrics
Zip code	022-0872
Address	1-1 Seiryo-machi, Aoba-ku, Sendai
TEL	022-717-7287
Homepage URL
Email	ifujiwara-endo@umin.ac.jp

Sponsor
Institute	Tohoku University School of Medicine, Department of Pediatrics
Institute
Department

Funding Source
Organization	None
Organization
Division
Category of Funding Organization	Self funding
Nationality of Funding Organization

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

IRB Contact (For public release)
Organization	Tohoku University Hospital IRB
Address	1-1 Seiryo-machi, Aoba-ku, Sendai
Tel	022-717-7146
Email	rinri-2@proj.med.tohoku.ac.jp

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	東北大学病院（宮城県）

Other administrative information
Date of disclosure of the study information	2013 Year 04 Month 30 Day

Related information
URL releasing protocol
Publication of results	Unpublished

Result
URL related to results and publications
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	No longer recruiting
Date of protocol fixation	2013 Year 04 Month 30 Day
Date of IRB	2013 Year 03 Month 26 Day
Anticipated trial start date	2013 Year 04 Month 30 Day
Last follow-up date	2018 Year 02 Month 28 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date	2013 Year 04 Month 30 Day
Last modified on	2019 Year 11 Month 04 Day

Link to view the page
URL(English)	https://upload.umin.ac.jp/cgi-open-bin/icdr_e/ctr_view.cgi?recptno=R000012407