Post-approval Clinical Study of Asfotase Alfa Treatment for Patients With Hypophosphatasia (HPP) in Japan

NCT02531867 | Completed Phase 4 Results

• 1 other identifier: AA-HPP-409
• Study Type: interventional
• Target: 13
• Locations: 0 countries
• Sites: N/A

Brief Summary

This is a multicenter study in Japan. Eleven sites which have already participated in the investigator-initiated clinical study (Early Access Program) will participate in this study.The objective of this study is to gain further information on the safety and efficacy of treatment with asfotase alfa.

Conditions

Hypophosphatasia

Keywords

HPP; Bone Disease; Soft Bones; Low Alkaline Phosphatase; Genetic Metabolic Disorder; Alkaline Phosphatase; Rickets; Osteomalacia

Outcome Measures

Primary Outcomes (1)

• Number of Participants With Adverse Events (AEs) Including Injection Site Reactions (ISRs) and Injection Associated Reactions (IARs)

  Adverse events are any unwanted adverse medical occurrence in patients who are treated with a medicinal drug, whether or not considered drug-related. This includes events observed in patients administered with asfotase alfa between the first dose of asfotase alfa and the completion of patient's last visit for the clinical study.

  Events that occurred between the first dose of asfotase alfa and the completion of the patient's last visit, which was up to 5 months.

Study Arms (1)

Asfotase Alfa

EXPERIMENTAL

Patients will receive asfotase alfa by subcutaneous injection. Asfotase alfa will be administered at either 2 mg/kg 3 times per week or 1 mg/kg 6 times per week depending on investigator's discretion.

Biological: Asfotase Alfa

Interventions

Asfotase Alfa BIOLOGICAL

Also known as: Strensiq

Asfotase Alfa

Eligibility Criteria

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

You may qualify if:

• Patient or parent (or legal guardian) must provide written informed consent prior to the performance of any study-related procedures and must be willing to comply with study procedures. Where appropriate and required by local regulations, patient assent for participation must also be obtained.
• Patient has completed the investigator-initiated clinical study (HPPJEAP-01) protocol for asfotase alfa

You may not qualify if:

• Patient has a documented form of rickets caused by a condition other than HPP, including, but not limited to, rickets caused by 25(OH) vitamin D deficiency
• Patient has serum calcium and/or phosphorus levels below the normal range
• Patient is pregnant or lactating
• Patient received treatment with bisphosphonates within 2 years prior to the Screening visit
• Patient has a documented sensitivity to any of the components of asfotase alfa
• Patient is currently enrolled in any other program or clinical study involving an investigational new drug, device, or treatment for HPP (eg, bone marrow transplantation)
• Patient has clinically significant other disease in the opinion of the Investigator, defined as any other non HPP-related condition for which the patient is considered medically unstable.

Sponsors & Collaborators

• Alexion Pharmaceuticals, Inc.: lead

MeSH Terms

Conditions

Hypophosphatasia; Bone Diseases; Rickets; Osteomalacia

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; Musculoskeletal Diseases; Bone Diseases, Metabolic; Calcium Metabolism Disorders; Vitamin D Deficiency; Avitaminosis; Deficiency Diseases; Malnutrition; Nutrition Disorders

Results Point of Contact

• Title: Medical Director
• Organization: Alexion

clinicaltrials@alexion.com

(781) 357-9900

Study Officials

• Kenji Fujita, MD

  Medical Monitor

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 11, 2015
• First Posted: August 25, 2015
• Study Start: June 1, 2015
• Primary Completion: November 1, 2015
• Study Completion: November 1, 2015
• Last Updated: May 8, 2017
• Results First Posted: May 8, 2017

Record last verified: 2017-03