Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onse… (NCT02496689) | Clinical Trial Compass
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Expanded Access Program for Asfotase Alfa Treatment for Patients With Infantile- or Juvenile-onset Hypophosphatasia (HPP)
United States, France
Plain-language summary
This clinical trial is being conducted in Hypophosphatasia, a bone disorder caused by gene mutation(s) resulting in bone defects. These gene mutations cause low levels of an enzyme needed to harden bone. The purpose of this study is to provide access to treatment in a disease where no approved treatment exists. This is an experimental treatment provided under specific treatment guidelines in which safety endpoints will be collected.
Who can participate
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
. Patient or parent (or legal guardian) must provide written informed consent prior to the performance of any program-related procedures and must be willing to comply with program procedures. Where appropriate and required by local regulations, patient assent for participation must also be obtained.
. Patient must have a documented diagnosis of HPP as indicated by a documented history of HPP-related skeletal abnormalities and one or more of the following:
. Patient must have infantile- or juvenile-onset HPP, defined as documented onset of signs/symptoms of HPP prior to 18 years of age.
. Male patient is:
. Female patient is:
. Male patients who are pubertal or post-pubertal and not surgically sterile (surgically sterile defined as vasectomized for ≥6 months) must agree to not donate sperm during program participation and for 3 months after program completion.
. Female patients of childbearing potential (ie, those who are not prepubertal or postmenopausal or surgically sterile as defined in inclusion criteria 5 above) must have a negative urine or serum pregnancy test at Screening.
Exclusion criteria
. Patient has geographic access to and qualifies for enrollment (as determined by the Sponsor and Investigator) in any other Alexion-sponsored clinical trials of asfotase alfa that are open to enrollment (eg, ENB-010-10).
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.
. Patient is pregnant, trying to become pregnant, or is lactating.
. Patient has a documented sensitivity to any of the components of asfotase alfa.
. Patient has serum calcium and/or phosphorus levels below the normal range at Screening.
. Patient is currently enrolled in any other program or study involving an investigational new drug, device, or treatment for HPP (eg, bone marrow transplantation).
. Patient has any other medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with program compliance, including all evaluations and follow-up activities. Patients who previously received treatment with asfotase alfa may be considered for enrollment.