Safety and Efficacy of Asfotase Alfa in Juvenile Patients 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

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-09-30

Study Completion Date

2010-07-31

Brief Summary

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

This clinical trial studied the safety and efficacy of asfotase alfa in children with HPP compared to a historical control group.

Related Clinical Trials

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

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

NCT01203826

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

Efficacy and Safety on the Use of Bisphosphonates in Paediatrics

NCT04012320

Bone Fragile Bisphosphonate-Associated Osteonecrosis Children +1 more

COMPLETED

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

NCT00195936

Hypophosphatemic Rickets, X-Linked Dominant

COMPLETED PHASE1

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 no approved disease-modifying treatments for patients with this disease. There is also limited data available on the natural course of this disease over time, particularly in patients with the juvenile-onset form.

Efficacy analyses were prospectively defined in the protocol with a comparison to historical controls. The historical control group came from patients whose characteristics matched as closely as possible the entry criteria for the trial. The control group included all patients who had x-rays within the age range defined by the inclusion criteria of this study (5 to 12 years of age, inclusive, with open growth plates).

The pre-specified plan for analysis was to combine the two asfotase alfa treated groups (asfotase alfa 2 mg/kg subcutaneous (SC) injection three times per week or 3 mg/kg subcutaneous (SC) injection three times per week) and compare them to historical controls.

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

RANDOMIZED

Intervention Model

PARALLEL

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.

2 mg/kg

2 mg/kg subcutaneous injection three times per week.

Group Type ACTIVE_COMPARATOR

asfotase alfa

Intervention Type BIOLOGICAL

2 mg/kg subcutaneous injection three times per week for 6 months.

3 mg/kg

3 mg/kg subcutaneous injection three times per week.

Group Type ACTIVE_COMPARATOR

asfotase alfa

Intervention Type BIOLOGICAL

3 mg/kg subcutaneous injection three times per week for 6 months.

Interventions

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

asfotase alfa

2 mg/kg subcutaneous injection three times per week for 6 months.

Intervention Type BIOLOGICAL

asfotase alfa

3 mg/kg subcutaneous injection three times per week for 6 months.

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 sALP-Fc-D10 Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein sALP-Fc-D10

Eligibility Criteria

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

Inclusion Criteria

1. Written informed consent from parent or legal guardian prior to participation
2. Patients \> 5 and \< 12 years of age with open growth plates at time of enrollment
3. Tanner stage of 2 or less indicating pre-pubescence
4. Documented history of HPP, as evidenced by:

* Presence of HPP-related rickets on skeletal radiographs of the wrist and knee
* Serum alkaline phosphatase (ALP) below age-adjusted normal range
* Plasma PLP at least twice the upper limit of normal
5. 25(OH) vitamin D level \> 20 ng/mL
6. Ability of patient and parent/guardian to comply with study requirements

Exclusion Criteria

1. Serum calcium or phosphorus below age-adjusted normal range
2. History of sensitivity to any study drug constituent
3. Medical condition, serious intercurrent illness, or other extenuating circumstance that, in the opinion of the Investigator, may significantly interfere with study compliance, including all prescribed evaluations and follow-up activities
4. Treatment with an investigational drug within 1 month before start of study drug
5. Current enrollment in any other study involving an investigational new drug, device, or treatment for HPP (e.g., bone marrow transplantation)
6. Current evidence of a treatable form of rickets
7. Prior treatment with bisphosphonates
8. Bone fracture or orthopedic surgery within the past 12 months that, in the opinion of the Investigator would interfere with the ability of study patient to comply with study protocol
9. Major congenital abnormality other than those associated with HPP

Minimum Eligible Age

5 Years

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No