Registry of Patients With Hypophosphatasia

Sponsor

Alexion Pharmaceuticals (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT02306720

Collaborator

(none)

900

Enrollment

Locations

187.9

Anticipated Duration (Months)

13.2

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

In this prospective, observational, long term registry patients of all ages with a diagnosis of hypophosphatasia (HPP) are followed at participating sites in multiple countries.

Condition or Disease	Intervention/Treatment	Phase
Hypophosphatasia (HPP)

Detailed Description

The HPP Registry is an observational, prospective, long-term registry designed to collect data on HPP epidemiology, disease history, clinical course, symptoms and burden of disease from patients of all ages who have a diagnosis of HPP.

Evaluation of safety and effectiveness data in patients with HPP who have/are receiving treatment with Asfotase alfa

Study Design

Study Type:

Observational [Patient Registry]

Anticipated Enrollment :

900 participants

Observational Model:

Case-Control

Time Perspective:

Prospective

Official Title:

An Observational, Longitudinal, Prospective, Long-Term Registry Of Patients With Hypophosphatasia (HPP)

Actual Study Start Date :

Dec 31, 2014

Anticipated Primary Completion Date :

Dec 31, 2024

Anticipated Study Completion Date :

Aug 28, 2030

Outcome Measures

Primary Outcome Measures

Natural History Information [1 Year]
To collect information on the natural history of HPP from patients of all ages, including pediatric patients and adults with HPP, regardless of age at onset.
Burden of Disease/Patient-reported Outcomes [1 year]
Age-appropriate PRO data will be collected using instruments to asses below: Pain, Motor Capacity, Functional Status/Disability, including Activities of Daily Living (ADL), Quality of Life (QoL)
Characterize the epidemiology of the HPP population. [1 year]
To characterize the epidemiology of the HPP population. Inclusion of all classifications of HPP is planned: pediatric-onset (perinatal-, infantile-, and juvenile-onset), adult-onset, benign perinatal, and odontohypophosphatasia.
Long-Term Safety and Effectiveness of Asfotase Alfa [1 year]
To collect and evaluate long-term safety and effectiveness data in HPP patients who have/are receiving treatment with asfotase alfa.

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Male and female patients, of any age, with a confirmed diagnosis of HPP. Patient or legal representative is able to read and/or understand the informed consent and study questionnaires in the local language.

Signed informed consent and medical records release by the patient or legal representative. Patient or patient's parent/legal representative must be willing and able to give written informed consent, and the patient must be willing to give written informed assent, if appropriate and required by local regulations. Patient must have documented alkaline phosphatase (ALP) activity below the lower limit of normal for age and sex, or a documented ALPL gene mutation.

Exclusion Criteria:

Currently participating in an Alexion-sponsored clinical trial . Enrollment in the Registry will not exclude a patient from enrolling in a future clinical trial.

Contacts and Locations

Locations

	City	State	Country	Postal Code
1	Little Rock	Arkansas	United States	72202
2	Centennial	Colorado	United States	80112
3	Golden	Colorado	United States	80401
4	Hartford	Connecticut	United States	06106
5	Tampa	Florida	United States	33606
6	Decatur	Georgia	United States	30033
7	Chicago	Illinois	United States	60611
8	Baltimore	Maryland	United States	21287
9	Boston	Massachusetts	United States	02111
10	Kansas City	Missouri	United States	64108
11	Saint Louis	Missouri	United States	63110
12	Mineola	New York	United States	11501
13	Rochester	New York	United States	14642
14	Syracuse	New York	United States	13214
15	Durham	North Carolina	United States	27710
16	Cincinnati	Ohio	United States	45229
17	Columbus	Ohio	United States	43205
18	Pittsburgh	Pennsylvania	United States	15224
19	Nashville	Tennessee	United States	37212
20	Salt Lake City	Utah	United States	84112
21	Madison	Wisconsin	United States	53792
22	Westmead	New South Wales	Australia
23	Clayton	Victoria	Australia
24	Parkville	Victoria	Australia
25	Herston		Australia
26	South Brisbane		Australia
27	Winnipeg	Manitoba	Canada
28	Toulouse	Haute Garonne	France
29	Paris cedex 14	Paris	France
30	Lyon		France
31	Le Kremlin-Bicêtre	Île-de-France	France
32	Bad Reichenhall	Bayern	Germany
33	Würzburg	Bayern	Germany
34	Bochum	Nordrhein Westfalen	Germany
35	Düsseldorf	Nordrhein Westfalen	Germany
36	Dresden	Sachsen	Germany
37	Berlin		Germany
38	Giessen		Germany
39	Hamburg		Germany
40	Homburg		Germany
41	Koeln		Germany
42	Mainz		Germany
43	Muenchen		Germany
44	Müllheim		Germany
45	Rostock		Germany
46	Firenze		Italy
47	Pisa		Italy
48	Rome		Italy
49	San Giovanni Rotondo		Italy
50	Verona		Italy
51	Warsaw		Poland
52	Moscow		Russian Federation
53	Saint Petersburg		Russian Federation
54	Riyadh		Saudi Arabia
55	Barcelona		Spain
56	Madrid		Spain
57	Bristol	Avon	United Kingdom
58	Belfast		United Kingdom
59	Birmingham		United Kingdom
60	Dundee		United Kingdom
61	Leicester		United Kingdom
62	London		United Kingdom
63	Newcastle upon Tyne		United Kingdom
64	Newport		United Kingdom
65	Norwich		United Kingdom
66	Oxford		United Kingdom
67	Sheffield		United Kingdom
68	Stanmore		United Kingdom

Sponsors and Collaborators

Alexion Pharmaceuticals

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Alexion Pharmaceuticals

ClinicalTrials.gov Identifier:

NCT02306720

Other Study ID Numbers:

ALX-HPP-501

First Posted:

Dec 3, 2014

Last Update Posted:

Apr 22, 2022

Last Verified:

Apr 1, 2022

Additional relevant MeSH terms:

Hypophosphatasia

Study Results

No Results Posted as of Apr 22, 2022