Valproate in spinal muscular atrophy

Randomized placebo controlled trial of valproate in children with spinal muscular atrophy

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

CTRI

Registry ID

CTRI/2008/091/000228

Enrollment

Registered

2008-12-11

Start date

Unknown

Completion date

Unknown

Last updated

2021-11-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

None listed

Interventions

Intervention1: valproate: 0-10 kg- syr 2.5 ml 10-20 kg- 5 ml 20-30 kg- 300 mg 30-40 kg- 400 mg all 8 hrly for 52 wks Control Intervention1: placebo:

Eligibility

Inclusion criteria

Inclusion criteria: all children havintg motor weakness, hypotonia, hyporeflexia with onset noticed after 6 mo of age presence of exon 7 of SMNT gene OR normal/ mildly elevated CPK with electrodiagnostic characterstics suggestive of neurogenic weakness, normal motor & sensory NCVs and muscle biopsy showing neurogenic atrophy and/ or evidence of re-innervation

Exclusion criteria

Exclusion criteria: SMA type 1, onset before 6 mo of age severely ill patients on life support other causes of hypotonia e.g. CP, down syndrome, metabolic pre-existing liver damage, bone marrow depression and coagulation disorders current use of valproate BMI > 90th centile for age

Design outcomes

Primary

Measure	Time frame
change in muscle powerTimepoint: 52 weeks	—

Secondary

Measure	Time frame
improvement on functional measureTimepoint: 52 weeks	—

Countries

India

Contacts

Public Contactsheffali gulati

sheffaligulati@gmail.com09868397532

Outcome results