Trial Outcomes & Findings for A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients (NCT NCT00568802)

NCT ID: NCT00568802

Last Updated: 2019-10-02

Results Overview

• Recruitment status: COMPLETED
• Study phase: PHASE1/PHASE2
• Target enrollment: 27 participants
• Primary outcome timeframe: Up to 6 years, 2 months
• Results posted on: 2019-10-02

Participant Flow

Data are presented for all participants because data relating to arm assignment are no longer accessible; the Principal Investigator (PI) has left institution and all efforts to locate the data have been exhausted.

Participant milestones

Measure	All Participants Participants received hydroxyurea, or placebo to match hydroxyurea.
Overall Study STARTED	27
Overall Study COMPLETED	24
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Pilot Therapeutic Trial Using Hydroxyurea in Type II and Type III Spinal Muscular Atrophy Patients

Baseline characteristics by cohort

Measure	All Participants n=27 Participants Participants received hydroxyurea, or placebo to match hydroxyurea.
Age, Categorical <=18 years	27 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Sex: Female, Male Female	12 Participants n=5 Participants
Sex: Female, Male Male	15 Participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 6 years, 2 months

Population: Data for this outcome measure are no longer accessible; the PI has left institution and all efforts to locate the data have been exhausted.

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Up to 6 years, 2 months

Population: Data for this outcome measure are no longer accessible; the PI has left institution and all efforts to locate the data have been exhausted.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 6 years, 2 months

Population: Data for this outcome measure are no longer accessible; the PI has left institution and all efforts to locate the data have been exhausted.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 6 years, 2 months

Population: Data for this outcome measure are no longer accessible; the PI has left institution and all efforts to locate the data have been exhausted.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Up to 6 years, 2 months

Population: Data for this outcome measure are no longer accessible; the PI has left institution and all efforts to locate the data have been exhausted.

Outcome measures

Outcome data not reported

Adverse Events

Hydroxyurea

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Placebo

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Principal Investigator

Stanford University

Email: clinicaltrials-gov@lists.stanford.edu

Results disclosure agreements

• Principal investigator is a sponsor employee
• Publication restrictions are in place