UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000023046 |
|---|---|
| Receipt number | 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 09:22:01 |
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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
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
uchiyama-t@ncchd.go.jp
Sponsor or person
Institute
Department of Human Genetics
National Center for Child Health and Development
Institute
Department
Personal name
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
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
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000022468
