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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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21 August 2023 |
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Main ID: |
NCT00894075 |
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Date of registration:
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04/05/2009 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)
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Scientific title:
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Single-Center, Case-Control Study of Safety, Efficacy and Pharmacokinetics of ENB-0040 (Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein) for Treatment of Hypophosphatasia in Children |
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Date of first enrolment:
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July 2009 |
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Target sample size:
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0 |
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Recruitment status: |
Withdrawn |
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URL:
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https://clinicaltrials.gov/ct2/show/NCT00894075 |
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Study type:
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Interventional |
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Study design:
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Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
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Phase:
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Phase 2
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Countries of recruitment
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United States
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Contacts
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Name:
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Michael P. Whyte, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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Shriners Hospital, St. Louis. MO |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Written informed consent from parent or legal guardian prior to participation
2. Boys >/= 5 and < 12 years of age and girls >/= 5 and < 10 years of age with open
growth plates at time of enrollment
3. Documented history of HPP, as evidenced by:
1. Presence of HPP-related rickets on skeletal radiographs
2. Serum alkaline phosphatase (ALP) below age-adjusted normal range
3. Plasma PLP at least twice the upper limit of normal (>/=220 nM)
4. Ambulatory without the use of assistive devices
5. Ability of patient and parent/guardian to comply with study requirements
Exclusion Criteria:
1. Serum calcium or phosphorus below age-adjusted normal range
2. History of sensitivity to any study drug constituent
3. Medical condition, serious intercurrent illness, or other extenuating circumstance
that, in the opinion of the investigator, may significantly interfere with study
compliance, including all prescribed evaluations and follow-up activities
4. Treatment with an investigational drug within 1 month before start of study drug
5. Current enrollment in any other study involving an investigational new drug, device,
or treatment for HPP (eg, bone marrow transplantation)
6. Current evidence of a treatable form of rickets
7. Prior treatment with bisphosphonates
8. Bone fracture or orthopedic surgery within the past 12 months
9. Major congenital abnormality other than those associated with HPP
Age minimum:
5 Years
Age maximum:
12 Years
Gender:
All
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Health Condition(s) or Problem(s) studied
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Hypophosphatasia
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Intervention(s)
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Biological: ENB-0040
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Primary Outcome(s)
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Skeletal radiographs using a qualitative Clinical Global Impression of Change (CGI-C) scoring system
[Time Frame: 6 months]
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Secondary Outcome(s)
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PK using serum peak and trough levels and PD of plasma inorganic pyrophosphate (PPi) and plasma pyridoxal-5' phosphate (PLP) as biomarkers for HPP.
[Time Frame: 6 months]
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Secondary ID(s)
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ENB-004-09
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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