Observational study of pediatric hypophosphatasia patients receiving treatment

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This observational sub-study aims to follow participants with pediatric-onset hypophosphatasia (HPP) for a minimum of 5 years. It includes individuals of any age who have been diagnosed with HPP and are currently receiving or will start treatment with asfotase alfa. The study will track the effects of the treatment and the progression of the condition across multiple sites in the United States and potentially in one or two other countries.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Any age or sex with a confirmed diagnosis of pediatric-onset HPP (that is, first HPP sign or symptom presented at \< 18 years of age).
* Currently receiving asfotase alfa treatment at Enrollment (not treatment-naïve) or the Physician has decided to resume (not treatment-naïve) or start (treatment-naïve) the participant's asfotase alfa treatment within 6 months after Enrollment.
* Participant must have documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, and a documented ALPL gene mutation (Note: An exception is made for infants with clinical features of HPP plus low ALP who need to start asfotase alfa treatment right away, at the Physician's discretion, but do not yet have a genetic result. In this case, ALPL gene documentation is not required at the time of sub-study enrollment but should be documented within 6 months after Enrollment).
* Participant or participant's parent/legally authorized representative is able to read and/or understand the informed consent and study questionnaires in the local language.
* Participant or participant's parent/legally authorized representative must be willing and able to give signed informed consent for this sub-study, and the participant must be willing to give written informed assent, if appropriate and required by local regulations.

Exclusion Criteria:

* Currently participating in an Alexion-sponsored interventional clinical study. Participants who have concluded participation in an Alexion-sponsored asfotase alfa clinical study are eligible to enroll in this sub-study.

Where it's running

• Clinical Trial Site
  Hartford, Connecticut, United States Recruiting
• Clinical Trial Site
  Chicago, Illinois, United States Recruiting
• Clinical Trial Site
  Boston, Massachusetts, United States Recruiting
• Clinical Trial Site
  Kansas City, Missouri, United States Recruiting
• Clinical Trial Site
  Mineola, New York, United States Recruiting
• Clinical Trial Site
  Columbus, Ohio, United States Recruiting

Study contacts

• Study coordinator: Alexion Pharmaceuticals, Inc. (Sponsor)
• Email: clinicaltrials@alexion.com
• Phone: 1-855-752-2356

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• How a single ALPL gene change affects hypophosphatasia over time in Russia

  This observational study will follow children and adults with hypophosphatasia who carry one ALPL gene variant to see how their symptoms and health change during routine care in Russia.

  Recruiting at 3 sites AstraZeneca · Moscow and 3 other locations · NCT07390240

• Understanding Hypophosphatasia in Adults with Rheumatology Issues

  This study is trying to learn more about adults with hypophosphatasia who have joint and bone issues to help improve how they are diagnosed and treated.

  Recruiting University of Bonn · Bonn, North Rhine-Westphali · NCT06574282

• Registry of adults with hypophosphatasia

  This study is tracking adults with hypophosphatasia to see how the condition affects their bone health and symptoms over time.

  Recruiting at 11 sites Assistance Publique - Hôpitaux de Paris · Bordeaux, France and 10 other locations · NCT05596539

• Observing the natural history of hypophosphatasia

  This study is tracking patients with hypophosphatasia to better understand how the condition affects them and how it runs in families.

  Recruiting Duke University · Durham, North Carolina · NCT02237625

• Registry for patients with rare diseases

  This study is creating a central registry for people with rare diseases to help connect them with researchers and improve treatments.

  Recruiting Sanford Health · Sioux Falls, South Dakota · NCT01793168

• Understanding the disease burden of hypophosphatasia in Chinese patients

  This study tests how hypophosphatasia affects people in China by collecting real-world data on symptoms, quality of life, and costs.

  Recruiting AstraZeneca · Beijing and 21 other locations · NCT07905287

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.