CompletedPhase 2

Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP)

United States, Germany27 participantsStarted 2016-06-06

Plain-language summary

The purpose of this study was to evaluate the pharmacokinetics (PK) and pharmacodynamics (PD) of asfotase alfa in adult participants with pediatric-onset HPP.

Who can participate

Age range18 Years

SexALL

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Inclusion criteria

✓. Participants or their legal representative(s) provided written informed consent prior to undergoing any study-related procedures.
✓. Participants were ≥18 years of age at Screening.
✓. Participant had pediatric-onset hypophosphatasia (HPP), defined as onset of first sign(s)/symptom (s) of HPP prior to 18 years of age.
✓. 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:
✓. Participants had a plasma inorganic pyrophosphate (PPi) level of ≥3.9 micromolar (µM) at Screening.
✓. Female participants of childbearing potential had a negative pregnancy test at the time of enrollment.
✓. Sexually active male and female participants of childbearing potential agreed to use a highly effective method of birth control during the study.
✓. 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.

Exclusion criteria

✕. 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.
✕. Employees of Alexion Pharmaceuticals.
✕. Currently enrolled in a clinical study involving another study drug or non-approved use of a drug or device.
✕. Participated, within the last 30 days, in a clinical study involving a study drug (other than the study drug used in this study).
✕. Completed or withdrawn from this study or any other study investigating asfotase alfa in the previous 3 years.
✕. Women who were pregnant, planning to become pregnant, or breastfeeding.
✕. Serum 25-hydroxy Vitamin D levels below 20 nanogram (ng) per milliliter (mL) at Screening.
✕. Screening serum creatinine or parathyroid hormone (PTH) levels ≥1.5 times the upper limit of normal.

What they're measuring

Change In Plasma PPi From Baseline To Pre-3rd Dose At Week 9

Timeframe: Baseline to Week 9

Trial details

NCT IDNCT02797821

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2017-06-21

Results submitted2018-06-11

View on ClinicalTrials.gov More Hypophosphatasia trials