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. |
Register:
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ClinicalTrials.gov |
Last refreshed on:
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12 December 2020 |
Main ID: |
NCT02797821 |
Date of registration:
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23/05/2016 |
Prospective Registration:
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Yes |
Primary sponsor: |
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Public title:
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Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)
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Scientific title:
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A Phase 2a, Randomized, Multicenter, Open-Label, Pharmacokinetic, and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia |
Date of first enrolment:
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June 6, 2016 |
Target sample size:
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27 |
Recruitment status: |
Completed |
URL:
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https://clinicaltrials.gov/show/NCT02797821 |
Study type:
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Interventional |
Study design:
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Allocation: Randomized. Intervention model: Parallel 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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Germany
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United States
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Key inclusion & exclusion criteria
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Inclusion Criteria:
1. Participants or their legal representative(s) provided written informed consent prior
to undergoing any study-related procedures.
2. Participants were =18 years of age at Screening.
3. Participant had pediatric-onset hypophosphatasia (HPP), defined as onset of first
sign(s)/symptom (s) of HPP prior to 18 years of age.
4. Participants had a documented diagnosis of HPP as indicated by a documented history of
HPP-related skeletal abnormalities and 1 or more of the following:
- Documented tissue-nonspecific alkaline phosphatase (TNSALP) gene mutation(s) from
a certified laboratory.
- Serum alkaline phosphatase (ALP) level below the age-adjusted normal range AND
plasma pyridoxal-5'-phosphate (PLP) above the upper limit of normal at Screening.
5. Participants had a plasma inorganic pyrophosphate (PPi) level of =3.9 micromolar (µM)
at Screening.
6. Female participants of childbearing potential had a negative pregnancy test at the
time of enrollment.
7. Sexually active male and female participants of childbearing potential agreed to use a
highly effective method of birth control during the study.
8. Female participants not of child-bearing potential due to sterilization (at least 6
weeks after surgical bilateral oophorectomy with or without hysterectomy or at least 6
weeks after tubal ligation) confirmed by medical history, or menopause.
9. Participants were willing to comply with study procedures and the visit schedule.
Exclusion Criteria:
1. Investigational site personnel directly affiliated with this study and/or their
immediate families. Immediate family was defined as a spouse, parent, child, or
sibling, whether biological or legally adopted.
2. Employees of Alexion Pharmaceuticals.
3. Currently enrolled in a clinical study involving another study drug or non-approved
use of a drug or device.
4. Participated, within the last 30 days, in a clinical study involving a study drug
(other than the study drug used in this study).
5. Completed or withdrawn from this study or any other study investigating asfotase alfa
in the previous 3 years.
6. Women who were pregnant, planning to become pregnant, or breastfeeding.
7. Serum 25-hydroxy Vitamin D levels below 20 nanogram (ng) per milliliter (mL) at
Screening.
8. Screening serum creatinine or parathyroid hormone (PTH) levels =1.5 times the upper
limit of normal.
9. Any medical condition, serious concurrent illness and/or injury, recent orthopedic
surgery, or other extenuating circumstance that, in the opinion of the Investigator,
may have significantly interfered with study compliance or study endpoints.
10. Prior treatment with bisphosphonates within 2 years of study entry for any length of
time or for more than 2 consecutive years at any prior timepoint.
11. Treatment with PTH, strontium, or sclerostin inhibitors within 6 months prior to the
first dose of study drug.
12. Unwilling or unable to comply with the use of a data collection device on which study
participants directly recorded data.
Age minimum:
18 Years
Age maximum:
N/A
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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Drug: Asfotase alfa
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Primary Outcome(s)
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Change In Plasma PPi From Baseline To Pre-3rd Dose At Week 9
[Time Frame: Baseline to Week 9]
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Secondary Outcome(s)
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Change In Plasma PLP From Baseline To Pre-3rd Dose At Week 9
[Time Frame: Baseline to Week 9]
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Secondary ID(s)
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AA-HPP-208
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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