Neonatal Spinal Muscular Atrophy (SMA) Screening

Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age0-2
SponsorKaohsiung Medical University Chung-Ho Memorial Hospital

About this trial

Parents or legal guardian of neonates who signed agreement will receive SMA screening test if their neonates are affected with SMA. The dried blood spots of routine newborn screening samples will be used to test if neonates have lost 2 copies of SMN1 gene. If neonates have positive SMA screening test, further confirmation with multiplex ligation-dependent probe amplification (MLPA) test and prospective motor function monitoring including physical and neurological examinations will be proved to make SMA confirmation. For any confirmed SMA patient, genetic counseling and standard of care will be proved.

Eligibility criteria

This trial accepts healthy volunteers

Qualifiers

Neonates born in Taiwan who receive regular newborn screening suggested by Ministry of Heath and Welfare.

Parents or legal guardian agree to perform SMA newborn screening.

Disqualifiers

None

Trial population

Neonates whose parents agree to be tested.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • SMA Newborn Screening

    Other intervention

Treatment groups

No treatment groups listed

Trial outcomes

Primary outcomes

1

Number of neonates with confirmed the deletion of 2 copies of SMN1 gene

Neonates with positive SMA newborn screening will be confirmed by multiplex ligation-dependent probe amplification (MLPA) test

Time frame
3 years

Secondary outcomes

1

Number of neonates with confirmed SMA

Neonates with confirmed the deletion of 2 copies of SMN1 gene by MLPA test will be followed till SMA symptoms appear.

Time frame
3 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Kaohsiung Medical University Chung-Ho Memorial Hospital

Lead sponsor

The Chinese Foundation of Health

Collaborator

Taipei Institute of Pathology

Collaborator