Observational study of pediatric hypophosphatasia patients receiving treatment
A Prospective Observational Sub-Study of the Global Hypophosphatasia Registry to Describe the Potential Risk of Immune-Mediated Loss of Pharmacological Effect of Asfotase Alfa in Participants With Hypophosphatasia
This study is tracking kids with hypophosphatasia who are being treated with asfotase alfa to see how the treatment affects them over at least five years.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 30 (estimated) |
| Sex | All |
| Sponsor | Alexion Pharmaceuticals, Inc. Industry-sponsored |
| Locations | 12 sites (Hartford, Connecticut and 11 other locations) |
| Trial ID | NCT05234567 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates include individuals of any age with a confirmed diagnosis of pediatric-onset hypophosphatasia who are currently receiving or will start asfotase alfa treatment.
Not a fit: Patients who do not have a confirmed diagnosis of pediatric-onset hypophosphatasia or are treatment-naïve without plans to start treatment may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the long-term effects of asfotase alfa treatment in pediatric patients with hypophosphatasia.
How similar studies have performed: Other studies involving asfotase alfa have shown promising results in treating hypophosphatasia, indicating that this approach has been successful in similar contexts.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Hartford, Connecticut and 11 other locations
- 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 — Cincinnati, Ohio, United States (Not_yet_recruiting)
- Clinical Trial Site — Columbus, Ohio, United States (Recruiting)
- Clinical Trial Site — Pittsburgh, Pennsylvania, United States (Not_yet_recruiting)
- Clinical Trial Site — Nashville, Tennessee, United States (Recruiting)
- Clinical Trial Site — Salt Lake City, Utah, United States (Recruiting)
- Clinical Trial Site — Charlottesville, Virginia, United States (Recruiting)
- Clinical Trial Site — Madison, Wisconsin, United States (Recruiting)
Study contacts
- Study coordinator: Alexion Pharmaceuticals, Inc. (Sponsor)
- Email: clinicaltrials@alexion.com
- Phone: 1-855-752-2356
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.