NCT01556516

Brief Summary

This study explores the outcome and effect of pregnancy on Pompe Disease. The results are expected to guide clinicians in counseling and care of women with Pompe disease, who are planning to become pregnant, and during the pregnancy.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
20

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Feb 2012

Longer than P75 for all trials

Geographic Reach
1 country

1 active site

Status
completed

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

Study Start

First participant enrolled

February 1, 2012

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

March 9, 2012

Completed
7 days until next milestone

First Posted

Study publicly available on registry

March 16, 2012

Completed
2.5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 1, 2014

Completed
1.8 years until next milestone

Study Completion

Last participant's last visit for all outcomes

July 1, 2016

Completed
Last Updated

September 28, 2016

Status Verified

September 1, 2016

Enrollment Period

2.7 years

First QC Date

March 9, 2012

Last Update Submit

September 27, 2016

Conditions

Pompe Disease

Outcome Measures

Primary Outcomes (1)

  • Effect of pregnancy on Pompe disease severity and progression.

    The effect of pregnancy on Pompe disease severity and progression as measured by increase in muscle weakness, use of assistive devices for mobility or breathing based on subject's reporting.

    5years

Secondary Outcomes (1)

  • Effect of Pompe disease on pregnancy and pregnancy outcomes.

    5 years

Study Arms (1)

Women with Pompe Disease

Eligibility Criteria

Age18 Years+
Sexfemale
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

20 female subjects (18 and above) who meet eligibility criteria.

You may qualify if:

  • Confirmed diagnosis of Pompe disease
  • Females age 18 and over
  • Have been pregnant or anticipating pregnancy in near future.

You may not qualify if:

  • If the diagnosis of Pompe disease is not confirmed
  • If the subject is not able to consent

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

O&O Alpan

Fairfax, Virginia, 22031, United States

Location

MeSH Terms

Conditions

Glycogen Storage Disease Type II

Condition Hierarchy (Ancestors)

Lysosomal Storage Diseases, Nervous SystemBrain Diseases, Metabolic, InbornBrain Diseases, MetabolicBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGlycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsLysosomal Storage DiseasesMetabolic DiseasesNutritional and Metabolic Diseases

Study Officials

  • Ozlem Goker-Alpan, MD

    O&O Alpan

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 9, 2012

First Posted

March 16, 2012

Study Start

February 1, 2012

Primary Completion

October 1, 2014

Study Completion

July 1, 2016

Last Updated

September 28, 2016

Record last verified: 2016-09

Locations

US(1)