Trial Outcomes & Findings for A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients (NCT NCT00568698)

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

29 participants

Primary outcome timeframe

Up to 8 years, 1 month

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

Participant milestones
Measure	All Participants Participants received hydroxyurea, or placebo to match hydroxyurea.
Overall Study STARTED	29
Overall Study COMPLETED	18
Overall Study NOT COMPLETED	11

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Pilot Therapeutic Trial Using Hydroxyurea in Type I Spinal Muscular Atrophy Patients

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	All Participants n=29 Participants Participants received hydroxyurea, or placebo to match hydroxyurea.
Age, Categorical <=18 years	29 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=99 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants
Sex: Female, Male Female	13 Participants n=99 Participants
Sex: Female, Male Male	16 Participants n=99 Participants

PRIMARY outcome

Timeframe: Up to 8 years, 1 month

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 8 years, 1 month

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 8 years, 1 month

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 8 years, 1 month

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: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place