NCT00568698

Brief Summary

The objectives of this trial are: to establish a safety profile for use of Hydroxyurea in children with Type I Spinal Muscular Atrophy; to identify reliable outcome measures for HU treatment in Type I SMA; and to detect the clinical efficacy of HU treatment in children with Type I SMA.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
29

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Jan 2004

Longer than P75 for phase_1

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

January 1, 2004

Completed
3.9 years until next milestone

First Submitted

Initial submission to the registry

December 4, 2007

Completed
2 days until next milestone

First Posted

Study publicly available on registry

December 6, 2007

Completed
4.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 1, 2012

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 1, 2012

Completed
7.7 years until next milestone

Results Posted

Study results publicly available

October 2, 2019

Completed
Last Updated

October 2, 2019

Status Verified

September 1, 2019

Enrollment Period

8.1 years

First QC Date

December 4, 2007

Results QC Date

September 6, 2019

Last Update Submit

September 11, 2019

Conditions

Muscular Atrophy, Spinal

Outcome Measures

Primary Outcomes (2)

  • Safety: Frequency of Adverse Events/Lab Abnormalities

    Up to 8 years, 1 month

  • Efficacy: Length of Survival (LOS) and Age of Ventilator Dependence (AVD)

    Up to 8 years, 1 month

Secondary Outcomes (2)

  • Motor Unit Number Estimation (MUNE)

    Up to 8 years, 1 month

  • Biomarker Assays: SMN Protein and SMN mRNA

    Up to 8 years, 1 month

Study Arms (2)

Hydroxyurea

EXPERIMENTAL
Drug: Hydroxyurea

Placebo

PLACEBO COMPARATOR
Drug: Placebo to match hydroxyurea

Interventions

HydroxyureaDRUG
Hydroxyurea
Placebo to match hydroxyureaDRUG
Placebo

Eligibility Criteria

AgeUp to 2 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Stanford University School of Medicine

Stanford, California, 94305, United States

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Interventions

Hydroxyurea

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Intervention Hierarchy (Ancestors)

UreaAmidesOrganic Chemicals

Results Point of Contact

Title
Principal Investigator
Organization
Stanford University
clinicaltrials-gov@lists.stanford.edu

Study Officials

  • Dr Ching H. Wang

    Stanford University

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
DOUBLE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 4, 2007

First Posted

December 6, 2007

Study Start

January 1, 2004

Primary Completion

February 1, 2012

Study Completion

February 1, 2012

Last Updated

October 2, 2019

Results First Posted

October 2, 2019

Record last verified: 2019-09

Locations

US(1)