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NCT01409486 Clinical Trial

Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry

Pompe Disease Clinical Trial

— LC-MS-MS  

Official title:
Screening for Early Detection and Prevention of Pompe Disease in Israel Using Tandem Mass Spectrometry

Clinical Trial Details — Status: Not yet recruiting

Administrative data
NCT number NCT01409486
Other study ID # 0290-09-RMB-CTIL
Secondary ID
Status Not yet recruiting
Phase N/A
First received July 7, 2011
Last updated August 3, 2011
Start date September 2011
Est. completion date September 2013

Study information
Verified date August 2011
Source Rambam Health Care Campus
Contact Hanna Mandel, Prof.
Phone 972-50-2062637
Email h_mandel@rambam.health.gov.il
Is FDA regulated No
Health authority Israel: Ministry of Health
Study type Observational

Clinical Trial Summary

The aim of the study is:

to develop a comprehensive biochemical assay for detection of Pompe disease (glycogen storage disease type II), to be implemented in the Newborn screening program among the Israeli population.

Recruitment information / eligibility
Status Not yet recruiting
Enrollment 10000
Est. completion date September 2013
Est. primary completion date September 2013
Accepts healthy volunteers Accepts Healthy Volunteers
Gender Both
Age group N/A to 7 Days
Eligibility Inclusion Criteria:

- New born babies born during the study period

Exclusion Criteria:

- Premature babies

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

Intervention

Other:
• Drawing blood spots from Newborns
Dry blood spots would be taken for determination of Alpha Glucosidase activity using LC-MS-MS

Locations
Country Name City State
Israel Rambam Health Care Campus Haifa

Sponsors (1)
Lead Sponsor Collaborator
Rambam Health Care Campus

Country where clinical trial is conducted

Israel, 

Outcome
Type Measure Description Time frame Safety issue
Primary Identification of the normal mean control value of Alpha glucosidase activity in Dry blood spots among Newborns in Israel Two years No
See also
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Recruiting NCT01665326 - Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease
Completed NCT01758354 - Newborn Screening Assay of Pompe's Disease N/A
Recruiting NCT00231400 - Pompe Disease Registry Protocol
Recruiting NCT04476550 - Clinical Specimen Collection From Pompe Disease Patients
Recruiting NCT05687474 - Baby Detect : Genomic Newborn Screening
Completed NCT01380743 - Drug-drug Interaction Study Phase 2
Recruiting NCT05734521 - Avalglucosidase Alfa Pregnancy Study
Completed NCT02742298 - Pompe Disease QMUS and EIM N/A
Terminated NCT02185651 - A Pilot Study of Zavesca® in Patients With Pompe Disease and Infusion Associated Reaction Phase 1
Completed NCT02654886 - Safety and Effectiveness of Resistance Exercise Training in Patients With Pompe Disease. N/A
Completed NCT02405598 - Evaluation of Salbutamol as an Adjuvant Therapy for Pompe Disease Phase 4
Completed NCT00701129 - An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease Phase 4
Completed NCT01451879 - Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies N/A
Completed NCT02240407 - Re-administration of Intramuscular AAV9 in Patients With Late-Onset Pompe Disease Phase 1
Completed NCT05073783 - A Study to Assess the Safety of Myozyme® and of Aldurazyme® in Male and Female Participants of Any Age Group With Pompe Disease or With Mucopolysaccharidosis Type I (MPS I) in a Home-care Setting
Active, not recruiting NCT04093349 - A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) Phase 1/Phase 2
Completed NCT02363153 - Diet and Exercise in Pompe Disease N/A
Completed NCT01410890 - Pharmacokinetics of Alglucosidase Alfa in Patients With Pompe Disease Phase 4