WithdrawnPhase 2

Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP)

Stopped: Withdrawn pending further review of clinical design.

0Started 2009-07

Plain-language summary

This Clinical Trial is being conducted to study Hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone. The purpose of this study is to test the safety of the study drug called ENB-0040 and see what effects is has on human juveniles and HPP.

Who can participate

Age range

5 Years – 12 Years

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

. Written informed consent from parent or legal guardian prior to participation
. Boys \>/= 5 and \< 12 years of age and girls \>/= 5 and \< 10 years of age with open growth plates at time of enrollment
. Documented history of HPP, as evidenced by:
. Presence of HPP-related rickets on skeletal radiographs
. Serum alkaline phosphatase (ALP) below age-adjusted normal range
. Plasma PLP at least twice the upper limit of normal (\>/=220 nM)
. Ambulatory without the use of assistive devices
. Ability of patient and parent/guardian to comply with study requirements

Exclusion criteria

. Serum calcium or phosphorus below age-adjusted normal range
. History of sensitivity to any study drug constituent
. 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

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

Skeletal radiographs using a qualitative Clinical Global Impression of Change (CGI-C) scoring system

Timeframe: 6 months

Trial details

NCT IDNCT00894075

SponsorAlexion Pharmaceuticals, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2014-12

View on ClinicalTrials.gov More Hypophosphatasia trials