NCT00894075

Brief Summary

This Clinical Trial is being conducted to study Hypophosphatasia (HPP), a bone disorder caused by gene mutations or changes. These gene mutations cause low levels of an enzyme needed to harden bone. The purpose of this study is to test the safety of the study drug called ENB-0040 and see what effects is has on human juveniles and HPP.

Trial Health

15
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Timeline
Completed

Started Jul 2009

Longer than P75 for phase_2

Status
withdrawn

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

May 4, 2009

Completed
2 days until next milestone

First Posted

Study publicly available on registry

May 6, 2009

Completed
2 months until next milestone

Study Start

First participant enrolled

July 1, 2009

Completed
5.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2014

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2014

Completed
Last Updated

August 4, 2023

Status Verified

August 1, 2023

Enrollment Period

5.4 years

First QC Date

May 4, 2009

Last Update Submit

August 3, 2023

Conditions

Hypophosphatasia

Keywords

genetic metabolic disorderalkaline phosphatasetissue non-specific alkaline phosphatasericketsosteomalacia

Outcome Measures

Primary Outcomes (1)

  • Skeletal radiographs using a qualitative Clinical Global Impression of Change (CGI-C) scoring system

    6 months

Secondary Outcomes (1)

  • PK using serum peak and trough levels and PD of plasma inorganic pyrophosphate (PPi) and plasma pyridoxal-5' phosphate (PLP) as biomarkers for HPP.

    6 months

Interventions

ENB-0040BIOLOGICAL

1mg/kg subcutaneous injection thrice weekly for 6 months

Also known as: Human Recombinant Tissue Nonspecific Alkaline Phosphatase Fusion Protein, sALP-Fc-D10

Eligibility Criteria

Age5 Years - 12 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • Written informed consent from parent or legal guardian prior to participation
  • Boys \>/= 5 and \< 12 years of age and girls \>/= 5 and \< 10 years of age with open growth plates at time of enrollment
  • Documented history of HPP, as evidenced by:
  • Presence of HPP-related rickets on skeletal radiographs
  • Serum alkaline phosphatase (ALP) below age-adjusted normal range
  • Plasma PLP at least twice the upper limit of normal (\>/=220 nM)
  • Ambulatory without the use of assistive devices
  • Ability of patient and parent/guardian to comply with study requirements

You may not qualify if:

  • Serum calcium or phosphorus below age-adjusted normal range
  • History of sensitivity to any study drug constituent
  • 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
  • Treatment with an investigational drug within 1 month before start of study drug
  • Current enrollment in any other study involving an investigational new drug, device, or treatment for HPP (eg, bone marrow transplantation)
  • Current evidence of a treatable form of rickets
  • Prior treatment with bisphosphonates
  • Bone fracture or orthopedic surgery within the past 12 months
  • Major congenital abnormality other than those associated with HPP

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Publications (2)

  • Millan JL, Narisawa S, Lemire I, Loisel TP, Boileau G, Leonard P, Gramatikova S, Terkeltaub R, Camacho NP, McKee MD, Crine P, Whyte MP. Enzyme replacement therapy for murine hypophosphatasia. J Bone Miner Res. 2008 Jun;23(6):777-87. doi: 10.1359/jbmr.071213.

    PMID: 18086009BACKGROUND

  • Drake MT, Khosla S. Bone-targeted replacement therapy for hypophosphatasia. J Bone Miner Res. 2008 Jun;23(6):775-6. doi: 10.1359/jbmr.080305. No abstract available.

    PMID: 18318644BACKGROUND

MeSH Terms

Conditions

HypophosphatasiaRicketsOsteomalacia

Interventions

asfotase alfa

Condition Hierarchy (Ancestors)

Metal Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic DiseasesBone Diseases, MetabolicBone DiseasesMusculoskeletal DiseasesCalcium Metabolism DisordersVitamin D DeficiencyAvitaminosisDeficiency DiseasesMalnutritionNutrition Disorders

Study Officials

  • Michael P. Whyte, MD

    Shriners Hospital, St. Louis. MO

    PRINCIPAL INVESTIGATOR
0

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

May 4, 2009

First Posted

May 6, 2009

Study Start

July 1, 2009

Primary Completion

December 1, 2014

Study Completion

December 1, 2014

Last Updated

August 4, 2023

Record last verified: 2023-08