CompletedPhase 1/2

Safety and Efficacy Study of Asfotase Alfa in Severely Affected Infants With Hypophosphatasia (HPP)

United States, Canada, United Arab Emirates11 participantsStarted 2008-09

Plain-language summary

This clinical trial studies the safety and efficacy of asfotase alfa in infants and young children with infantile onset HPP.

Who can participate

Age range

36 Months

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Legal guardian(s) must provide informed consent prior to any study procedures * Documented diagnosis of severe HPP as indicated by: * Total serum alkaline phosphatase at least 3 standard deviations (SD) below the mean for age * Plasma pyridoxal 5'-phosphate (PLP) at least 4 times the upper limit of normal * Radiographic evidence of HPP (hypophosphatasia), characterized by: * Flared and frayed metaphyses * Severe, generalized osteopenia * Widened growth plates * One or more HPP-related findings: * History or presence of: * Non-traumatic post-natal fracture * Delayed fracture healing * History of elevated serum calcium * Functional craniosynostosis with decreased head circumference growth * Nephrocalcinosis * Respiratory compromise * Rachitic chest deformity and/or vitamin B6 dependent seizures * Failure to thrive * Onset of symptoms prior to 6 months of age * Age ≤ 36 months * Otherwise medically stable (patient may be on ventilatory support) * Legal guardian(s) must be willing to comply with the study Exclusion Criteria: * History of sensitivity to any of the constituents of the study drug * Current or prior clinically significant cardiovascular, endocrinologic, hematologic, hepatic, immunologic, metabolic, infectious, urologic, pulmonary, neurologic, dermatologic, renal condition and/or other major disease which, in the opinion of the investigator, precludes study participation * Treatment w…

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Change in Rickets Severity From Baseline to Week 24, Based on Assessment of Skeletal Radiographs Using Radiologic Global Impression of Change (RGI-C)

Timeframe: 24 weeks

Trial details

NCT IDNCT00744042

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2010-05

Results submitted2011-05-15

View on ClinicalTrials.gov More Hypophosphatasia (HPP) trials

Who can participate

Age range

36 Months

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.