Extension Study of Protocol ENB-006-09 - Study of Asfotase Alfa in Children With Hypophosphatasia (HPP)

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

View full results

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE2

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2010-04-30

Study Completion Date

2016-06-30

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

This clinical trial studies the long term safety and efficacy of asfotase alfa in children with HPP who completed Study ENB-006-09 (NCT00952484).

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Safety and Efficacy of Asfotase Alfa in Juvenile Patients With Hypophosphatasia (HPP)

NCT00952484

Hypophosphatasia (HPP)

COMPLETED PHASE2

A Prospective Sub-Study of the Global Hypophosphatasia Registry

NCT05234567

Hypophosphatasia

RECRUITING

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

NCT04195763

Hypophosphatasia

COMPLETED

Effect of Cinacalcet on Parathyroid Hormone Secretion in Children and Adolescents With Hypophosphatemic Rickets

NCT00195936

Hypophosphatemic Rickets, X-Linked Dominant

COMPLETED PHASE1

Study of Weekly ALTU-238 Compared With Daily Nutropin AQ in Prepubertal Children With Growth Hormone Deficiency

NCT00837863

Growth Hormone Deficiency

UNKNOWN PHASE2

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Asfotase alfa was formerly referred to as ENB-0040

Hypophosphatasia (HPP) is a life-threatening, genetic, and ultra-rare metabolic disease characterized by defective bone mineralization and impaired phosphate and calcium regulation that can lead to progressive damage to multiple vital organs, including destruction and deformity of bones, profound muscle weakness, seizures, impaired renal function, and respiratory failure. There are limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Hypophosphatasia (HPP)

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

asfotase alfa

asfotase alfa starting dose 3 mg/kg/week SC injection, increased to 6 mg/kg/week SC injection

Group Type EXPERIMENTAL

Asfotase Alfa

Intervention Type BIOLOGICAL

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Asfotase Alfa

Intervention Type BIOLOGICAL

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

human recombinant tissue nonspecific alkaline phosphatase fusion protein

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* Compliant and satisfactory completion of Enobia-sponsored clinical trial ENB-006-09
* Written informed consent by parent or other legal guardian prior to any study procedures being performed
* Parent or other legal guardian willing to comply with study requirements

Exclusion Criteria

* Clinically significant disease that precludes study participation, in the Investigator's opinion
* Treatment with an investigational drug other than asfotase alfa
* Enrollment in any study (other than ENB-006-09) involving an investigational drug, device, or treatment for HPP
* Prior treatment with bisphosphonates

Minimum Eligible Age

5 Years

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No