A Prospective Sub-Study of the Global Hypophosphatasia Registry

Hypophosphatasia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05234567
• Other study ID #: ALX-HPP-501s
• Status: Recruiting
• Start date: March 17, 2022
• Est. completion date: June 30, 2028

Study information

• Verified date: July 2023
• Source: Alexion
• Contact: Alexion Pharmaceuticals, Inc.
• Phone: 1.855.752.2356
• Email: clinicaltrials[@]alexion.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

In this prospective observational sub-study, participants with pediatric-onset hypophosphatasia (HPP) (perinatal/infantile- or juvenile-onset) of any age will be followed for a minimum of 5 years at sites in the United States and potentially 1 or 2 other countries.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 40
• Est. completion date: June 30, 2028
• Est. primary completion date: June 30, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Hypophosphatasia

Intervention

Biological:
• Asfotase Alfa
All participants will receive asfotase alfa subcutaneously per standard of care. Unless otherwise specified per the Physician's standard of care, participants aged < 2 years are recommended for a clinic visit approximately every 3 months after Enrollment until 2 years of age, after which they should have a clinic visit approximately every 6 months. Participants should be followed for 5 years, as possible.

Locations

Country	Name	City	State
United States	Clinical Trial Site	Boston	Massachusetts
United States	Clinical Trial Site	Charlottesville	Virginia
United States	Clinical Trial Site	Chicago	Illinois
United States	Clinical Trial Site	Cincinnati	Ohio
United States	Clinical Trial Site	Columbus	Ohio
United States	Clinical Trial Site	Hartford	Connecticut
United States	Clinical Trial Site	Kansas City	Missouri
United States	Clinical Trial Site	Las Vegas	Nevada
United States	Clinical Trial Site	Mineola	New York
United States	Clinical Trial Site	Nashville	Tennessee
United States	Clinical Trial Site	Philadelphia	Pennsylvania
United States	Clinical Trial Site	Pittsburgh	Pennsylvania
United States	Clinical Trial Site	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Alexion

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Occurrence Of Immune-mediated Loss Of Effectiveness According To The Treating Physician	This will be based on clinical and biochemical assessments as well as positive anti-drug antibodies and positive neutralizing antibodies.	Up to 5 years
Primary	Occurrence Of Immune-mediated Serious Adverse Events	These serious adverse events will include serious hypersensitivity reactions and anaphylaxis.	Up to 5 years