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Recruitment status Completed
Unique ID issued by UMIN UMIN000014816
Receipt No. 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

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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

In outcomes field, the entry of just a few words such as "safety" or "efficiency" will not be accepted. Specify the name of outcome measures, including the time when you plan to measure. Usually, only one primary outcome is accepted. Write the other outcomes in "secondary outcomes" field.

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

In interventions field, include the details of interventions, such as duration, amount, and frequency. If the intervention includes prescription or use of medical devices, duration is required.

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
Email 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
Email hpp-studygp@ped.med.osaka-u.ac.jp

Sponsor
Institute HPP study group
Institute
Department

Sponsor means an organization that is responsible for plan, deployment and
report of the research including funding management. It doesn't mean
funding agency". Therefore, all clinical trial should have the one.

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
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
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
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000017019


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