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NCT04195763 Clinical Trial

Patient Reported Outcomes in Adults With Pediatric-onset Hypophosphatasia Treated With Strensiq® (Asfotase Alfa)

Hypophosphatasia Clinical Trial

Official title:
A Prospective Study to Evaluate the Patient Reported Quality of Life Prior to and After Strensiq® Treatment in Adults With Pediatric Onset Hypophosphatasia

Clinical Trial Details — Status: Active, not recruiting

Administrative data
NCT number NCT04195763
Other study ID # ALX-HPP-503
Secondary ID
Status Active, not recruiting
Phase
First received
Last updated
Start date November 6, 2019
Est. completion date May 3, 2024

Study information
Verified date March 2024
Source Alexion Pharmaceuticals, Inc.
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

Clinical Trial Summary

This observational study will evaluate the treatment effect of Strensiq (asfotase alfa) on Patient Reported Outcomes (PROs) in participants diagnosed with pediatric-onset hypophosphatasia (HPP) registered in the patient support program managed by OneSource™.

Description:

Participants consenting to participate in this study will be asked to complete study questionnaires by phone interviews upon consenting (Baseline), and up to 12 months following treatment initiation with asfotase alfa. Demographic and clinical characteristics of participants in the study will be characterized. This is an observational study and no intervention will be administered. Participants will be treated in accordance with standard of care.

Recruitment information / eligibility
Status Active, not recruiting
Enrollment 100
Est. completion date May 3, 2024
Est. primary completion date May 3, 2024
Accepts healthy volunteers No
Gender All
Age group 18 Years to 99 Years
Eligibility Inclusion Criteria: - = 18 years old - Clinical diagnosis of pediatric-onset HPP - Naïve to asfotase alfa - Expected to begin treatment with asfotase alfa for HPP - Registered in OneSource - Willing and able to provide voluntary, verbal informed consent to participate in this study Exclusion Criteria: - Pregnant or breastfeeding - Unable to speak and understand English - Unable or unwilling to complete the study surveys via telephone interview at the protocol-required time points

Study Design

Related Conditions & MeSH terms

Intervention

Drug:
• asfotase alfa
This is an observational study and no intervention will be administered. All participants will be treated by their physician in accordance with standard of care. All medications are commercially available and will be used as directed by the treating physician.

Locations
Country Name City State
United States Xcenda, LLC Palm Harbor Florida

Sponsors (2)
Lead Sponsor Collaborator
Alexion Pharmaceuticals, Inc. Xcenda, LLC

Country where clinical trial is conducted

United States, 

Outcome
Type Measure Description Time frame Safety issue
Primary Change From Baseline In Patient Reported Outcomes (PROs) Questionnaire Scores Baseline, up to 12 months
See also
  Status Clinical Trial Phase
Completed NCT03418389 - Evaluate and Monitor Physical Performance of Adults Treated With Asfotase Alfa for Hypophosphatasia
Recruiting NCT02237625 - Natural History Study of Patients With Hypophosphatasia (HPP)
Completed NCT02291497 - Burden of Disease in Hypophosphatasia (HPP) N/A
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Not yet recruiting NCT05596539 - Prospective, Longitudinal, Observational Registry of Adult Patients With Hypophosphatasia (REG-HYPO)
Completed NCT02751801 - Health Burden of Hypophosphatasia
Completed NCT02796885 - Characterisation of Adult-Onset Hypophosphatasia
Completed NCT05890794 - Pilot Trial of Single Dose Ilofotase Alfa in Hypophosphatasia Phase 1/Phase 2
Recruiting NCT06079359 - Phase 3 Study of ALXN1850 in Treatment-Naïve Pediatric Participants With HPP Phase 3
Recruiting NCT05234567 - A Prospective Sub-Study of the Global Hypophosphatasia Registry
Completed NCT02797821 - Pharmacokinetic and Dose Response Study of Asfotase Alfa in Adult Patients With Pediatric-Onset Hypophosphatasia (HPP) Phase 2
Completed NCT01163149 - Safety and Efficacy Study of Asfotase Alfa in Adolescents and Adults With Hypophosphatasia (HPP) Phase 2
Completed NCT04925804 - Unraveling Genetics of HypoPhosPhatasia (HPP Genetics)
Completed NCT02531867 - Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan Phase 4
Completed NCT01406977 - Dose Escalation Study to Evaluate the Safety and Tolerability of Multiple Infusions of BPS804 in Adults With Hypophosphatasia (HPP) Phase 2
Recruiting NCT01793168 - Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
Completed NCT01176266 - Open-Label Study of Asfotase Alfa in Infants and Children ≤ 5 Years of Age With Hypophosphatasia (HPP) Phase 2/Phase 3
Withdrawn NCT00894075 - Safety and Efficacy Study of ENB-0040 in Juvenile Patients With Hypophosphatasia (HPP) Phase 2
Active, not recruiting NCT04222452 - The PORTRAIT Study
Recruiting NCT06079281 - Phase 3 Study of ALXN1850 Versus Placebo in Adolescent and Adult Participants With HPP Who Have Not Previously Been Treated With Asfotase Alfa Phase 3