UMIN-CTR Clinical Trial

BACK TOP
UMIN-CTR English Home Glossary (Simple) FAQ Search clinical trials

Name:
UMIN ID:

Recruitment status Terminated
Unique ID issued by UMIN UMIN000023046
Receipt No. R000022468
Scientific Title Study of Efficacy of Sirolimus in Patients with Activated PI3Kdelta Syndrome
Date of disclosure of the study information 2016/07/08
Last modified on 2020/07/09

* This page includes information on clinical trials registered in UMIN clinical trial registed system.
* We don't aim to advertise certain products or treatments


Basic information
Public title Study of Efficacy of Sirolimus in Patients with Activated PI3Kdelta Syndrome
Acronym Treatment of APDS with Sirolimus
Scientific Title Study of Efficacy of Sirolimus in Patients with Activated PI3Kdelta Syndrome
Scientific Title:Acronym Treatment of APDS with Sirolimus
Region
Japan

Condition
Condition Activated PI3Kdelta Syndrome
Classification by specialty
Hematology and clinical oncology Pediatrics
Classification by malignancy Others
Genomic information YES

Objectives
Narrative objectives1 To assess the efficacy of Sirolimus in patients with Activated PI3Kdelta syndrome (APDS).
Basic objectives2 Efficacy
Basic objectives -Others
Trial characteristics_1 Confirmatory
Trial characteristics_2
Developmental phase

Assessment
Primary outcomes Reduction of lymphadenopathy, hepatosplenomegaly and lymphoid follicles in colon, due to abnormal activation and proliferation of lymphocytes.
Key secondary outcomes To assess the improvement in increased susceptibility to infection.
1) immunological test
2) lymphocyte function
3) test for fungal infection
4) test for viral infection (CMV, EBV, HHV, HSV)

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 An initial dose of 0.3 mg per surface area of sirolimus is administered once a day.
Dose is adjusted to maintain the blood level at 5-10 ng/ml.
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

Not applicable
Gender Male and Female
Key inclusion criteria 1) Patients with a diagnosis of APDS
2) Patients with clinical signs of lymphoproliferation as follows,
lymphadenopathy
hepatosplenomegaly and/or ascites
lymphoid follicles in colon with diarrhea and bloody stool
Key exclusion criteria 1) Patients with severe infections
2) Patients with severe thrombocytopenia
Target sample size 5

Research contact person
Name of lead principal investigator
1st name
Middle name
Last name Toru Uchiyama
Organization National Center for Child Health and Development
Division name Department of Human Genetics
Zip code
Address 2-10-1 Okura, Setagaya-ku, Tokyo, Japan
TEL 03-3416-0181
Email uchiyama-t@ncchd.go.jp

Public contact
Name of contact person
1st name
Middle name
Last name Toru Uchiyama
Organization National Center for Child Health and Development
Division name Department of Human Genetics
Zip code
Address 2-10-1 Okura, Setagaya-ku, Tokyo, Japan
TEL 03-3416-0181
Homepage URL
Email uchiyama-t@ncchd.go.jp

Sponsor
Institute Department of Human Genetics
National Center for Child Health and Development
Institute
Department

Funding Source
Organization National Center for Child Health and Development
Organization
Division
Category of Funding Organization Other
Nationality of Funding Organization

Other related organizations
Co-sponsor
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

Other administrative information
Date of disclosure of the study information
2016 Year 07 Month 08 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 Terminated
Date of protocol fixation
2015 Year 09 Month 25 Day
Date of IRB
2015 Year 05 Month 08 Day
Anticipated trial start date
2015 Year 09 Month 25 Day
Last follow-up date
2020 Year 07 Month 31 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date
2016 Year 07 Month 06 Day
Last modified on
2020 Year 07 Month 09 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000022468

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


Contact us.