UMIN-CTR Clinical Trial
| Unique ID issued by UMIN | UMIN000014816 |
|---|---|
| Receipt number | R000017019 |
| Scientific Title | A Multicenter study of the safety and efficacy of Asfotase Alfa (ALXN1215) (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in patients with hypophosphatasia (HPP) |
| Date of disclosure of the study information | 2014/08/11 |
| Last modified on | 2015/09/02 18:24:49 |
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Basic information
Public title
A Multicenter study of the safety and efficacy of Asfotase Alfa (ALXN1215) (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in patients with hypophosphatasia (HPP)
Acronym
Safety and efficacy of Asfotase Alfa in patients with hypophosphatasia (HPP)
Scientific Title
A Multicenter study of the safety and efficacy of Asfotase Alfa (ALXN1215) (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) in patients with hypophosphatasia (HPP)
Scientific Title:Acronym
Safety and efficacy of Asfotase Alfa in patients with hypophosphatasia (HPP)
Region
| Japan |
Condition
Condition
hypophosphatasia
Classification by specialty
| Pediatrics |
Classification by malignancy
Others
Genomic information
NO
Objectives
Narrative objectives1
Safety and efficacy of Asfotase Alfa (ALXN1215) in patients with hypophosphatasia
Basic objectives2
Safety,Efficacy
Basic objectives -Others
Trial characteristics_1
Trial characteristics_2
Developmental phase
Assessment
Primary outcomes
Safety of repeated subcutaneous injections of Asfotase Alfa (ALXN1215)
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
Uncontrolled
Stratification
Dynamic allocation
Institution consideration
Blocking
Concealment
Intervention
No. of arms
1
Purpose of intervention
Treatment
Type of intervention
| Medicine |
Interventions/Control_1
Drug: Asfotase Alfa (ALXN1215)
Dose: 6mg/kg/week (divided 3 times a week)
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
| Not applicable |
Gender
Male and Female
Key inclusion criteria
Patients must meet one selection criteria of following "1", "2", and "3", and must meet the selection criteria of "4."
1. Patient who have been already treated with Asfotae Alfa (ALXN1215) out of this clinical trial
2. Patient who have diagnosed as HPP
3. Documented diagnosis of HPP as indicated by:
(1) Total serum alkaline phosphatase below the lower limit of normal for age
(2) Ultrasonographic features of prenatal, characterized by:
1) severe short extremities (femur length <-4SD in second and third trimesters)
2) extending into the metaphysis (femur metaphysis length or femur length >0.33)
3) craniotabes
4) Hypoplastic thorax (Thoracic or abdominal circumference <0.6)
(3) Computed tomographic findings of prenatal, characterized by:
1) Generalized decreased ossification
2) Extreme shortening of tubular bones
3) Hypoplastic thorax
(4) Radiographic evidence of HPP, characterized by:
1) Flared and frayed metaphyses
2) Severe, generalized osteopenia
3) Widened growth plates
4) Areas of radiolucency or sclerosis
(5) Two or more of the following HPP-related findings:
1) History or presence of:
- Nontraumatic post-natal fracture
- Delayed fracture healing
2) Nephrocalcinosis or history of elevated serum calcium
3) Functional craniosynostosis
4) Respiratory compromise or rachitic chest deformity
5) Vitamin B6 dependent seizures
6) Failure to thrive
7) Premature tooth loss
(6) Patient who have the mutation of tissue non-specific ALP gene
4. Parent or legal guardian(s) must provide written informed consent prior to any study procedures being performed and must be willing to comply with all study-required procedures
Key exclusion criteria
Patients will be excluded from enrollment in this study if they meet any of the following exclusion criteria:
1. Current evidence of treatable form of rickets
2. Serum calcium or phosphate levels below the normal range
3. Pregnant women and nursing mothers
4. Patient who cannot enforce suitable contraceptive measures during the clinical trial
5. Prior treatment with bisphosphonates
Treatment with an investigational drug within 1 month prior to the start of asfotase alfa treatment
7. Current enrollment in any other study involving an investigational new drug, device or treatment for HPP (e.g., bone marrow transplantation)
8. Clinically significant disease that precludes study participation, in the opinion of the Investigator
Target sample size
20
Research contact person
Name of lead principal investigator
| 1st name | |
| Middle name | |
| Last name | Keiichi Ozono |
Organization
Osaka University Graduate School of Medicine
Division name
Department of Pediatrics
Zip code
Address
2-2 Yamada-oka, Suita, Osaka 565-0871, Japan
TEL
06-6879-3932
keioz@ped.med.osaka-u.ac.jp
Public contact
Name of contact person
| 1st name | |
| Middle name | |
| Last name | Taichi Kitaoka |
Organization
Osaka University Graduate School of Medicine
Division name
Department of Pediatrics
Zip code
Address
2-2 Yamada-oka, Suita, Osaka 565-0871, Japan
TEL
06-6879-3932
Homepage URL
hpp-studygp@ped.med.osaka-u.ac.jp
Sponsor or person
Institute
HPP study group
Institute
Department
Personal name
Funding Source
Organization
ALEXION Phalmaceuticals
Organization
Division
Category of Funding Organization
Profit organization
Nationality of Funding Organization
Other related organizations
Co-sponsor
Translational Research Informatics Center
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
| 2014 | Year | 08 | Month | 11 | 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
Completed
Date of protocol fixation
| 2014 | Year | 07 | Month | 28 | Day |
Date of IRB
Anticipated trial start date
| 2014 | Year | 08 | Month | 11 | Day |
Last follow-up date
Date of closure to data entry
Date trial data considered complete
Date analysis concluded
Other
Other related information
Management information
Registered date
| 2014 | Year | 08 | Month | 10 | Day |
Last modified on
| 2015 | Year | 09 | Month | 02 | Day |
Link to view the page
Value
https://center6.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000017019
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