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

NCT02456038 | Completed Phase 2

• 2 other identifiers: HPPJEAP-01UMIN000014816
• Study Type: interventional
• Target: 13
• Locations: 1 country
• Sites: 12

Brief Summary

The aim of this study is to assess safety and efficacy of Asfotase Alfa (ALXN1215) in patients with hypophosphatasia

Conditions

Hypophosphatasia

Outcome Measures

Primary Outcomes (1)

• Number of subjects with Adverse Events as an assessment of the Safety of repeated subcutaneous (SC) injections of asfotase alfa

  Safety of repeated subcutaneous (SC) injections of asfotase alfa for all treated patients

  Up to 50 months or until regulatory approval

Secondary Outcomes (6)

• Overall survival

  Up to 50 months or until regulatory approval

• Effect of asfotase alfa treatment on skeletal manifestations of HPP as measured by radiographs using a qualitative Radiographic Global Impression of Change (RGI-C) scale

  Up to 50 months or until regulatory approval

• Effect of asfotase alfa treatment on ventilator-free survival: (percentage of patients who are alive and ventilator-free after receiving asfotase alfa)

  Up to 50 months or until regulatory approval

• Profile of asfotase alfa treatment on respiratory function

  Up to 50 months or until regulatory approval

• Profile of asfotase alfa treatment on physical growth

  Up to 50 months or until regulatory approval

Study Arms (1)

Asfotase Alfa (ALXN1215)

EXPERIMENTAL

Asfotase Alfa (ALXN1215) is administered 6mg/kg in total per week, divided into 3 times.

Drug: Asfotase Alfa (ALXN1215)

Interventions

Asfotase Alfa (ALXN1215) DRUG

Also known as: ALXN1215

Asfotase Alfa (ALXN1215)

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients must meet one selection criteria of following "1", "2", and "3", and must meet the selection criteria of "4."
• Patient who has been already treated with Asfotae Alfa (ALXN1215) out of this clinical trial
• Patient who has been diagnosed as HPP
• Documented diagnosis of HPP as indicated by:
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• Total serum alkaline phosphatase below the lower limit of normal for age
• Ultrasonographic features of prenatal, characterized by:
• \) severe short extremities (femur length \<-4SD in second and third trimesters) 2) extending into the metaphysis (femur metaphysis length or femur length \>0.33) 3) craniotabes 4) Hypoplastic thorax (Thoracic or abdominal circumference \<0.6) (3) Computed tomographic findings of prenatal, characterized by:
• Generalized decreased ossification
• Extreme shortening of tubular bones
• Hypoplastic thorax (4) Radiographic evidence of HPP, characterized by:
• \) Flared and frayed metaphyses 2) Severe, generalized osteopenia 3) Widened growth plates 4) Areas of radiolucency or sclerosis (5) Two or more of the following HPP-related findings:
• History or presence of:
• \- Nontraumatic post-natal fracture
• \- Delayed fracture healing

You may not qualify if:

• Current evidence of treatable form of rickets
• Serum calcium or phosphate levels below the normal range
• Pregnant women and nursing mothers
• Patient who cannot enforce suitable contraceptive measures during the clinical trial
• Prior treatment with bisphosphonates
• Treatment with an investigational drug within 1 month prior to the start of asfotase alfa treatment
• Current enrollment in any other study involving an investigational new drug, device or treatment for HPP (e.g., bone marrow transplantation)
• Clinically significant disease that precludes study participation, in the opinion of the Investigator

Sponsors & Collaborators

• Translational Research Center for Medical Innovation, Kobe, Hyogo, Japan: lead
• Osaka University Graduate School of Medicine: collaborator

Study Sites (12)

Kurume University Hospital

Kurume, Fukuoka, 830-0011, Japan

Location

National Hospital Organization Nagara Medical Center

Gifu, Gifu, 502-8558, Japan

Location

Hiroshima University Hospital

Hiroshima, Hiroshima, 734-8551, Japan

Location

Hokkaido University Hospital

Sapporo, Hokkaido-prefecture, 060-8648, Japan

Location

Tohoku University Hospital

Sendai, Miyagi, 980-8574, Japan

Location

Niigata University Medical & Dental Hospital

Niigata, Niigata, 951-8510, Japan

Location

Osaka Medical Center and Research Institute for Maternal and Child Health

Izumi, Osaka, 594-1101, Japan

Location

Osaka University Hospital

Suita, Osaka, 565-0871, Japan

Location

Saitama Children's Medical Center

Saitama, Saitama, 339-0077, Japan

Location

Hamamatsu University Hospital

Hamamatsu, Shizuoka, 431-3192, Japan

Location

Nippon Medical School Hospital

Bunkyo-ku, Tokyo, 113-8603, Japan

Location

Showa General Hospital

Kodaira, Tokyo, 187-8510, Japan

Location

MeSH Terms

Conditions

Hypophosphatasia

Interventions

asfotase alfa

Condition Hierarchy (Ancestors)

Metal Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Keiichi Ozono

  Osaka University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: March 26, 2015
• First Posted: May 28, 2015
• Study Start: August 1, 2014
• Primary Completion: July 1, 2015
• Study Completion: July 1, 2015
• Last Updated: March 31, 2016

Record last verified: 2016-03

Locations

JP (12)