Trial Outcomes & Findings for Hydroxyurea for Children and Young Adults With Sickle Cell Disease and Pulmonary Hypertension (NCT NCT00350844)

Results Overview

Primary outcome measure was tricuspid regurgitant jet velocity (TRJV) by echocardiogram after 6 and 12 months of hydroxyurea therapy.

Recruitment status

TERMINATED

Study phase

PHASE1/PHASE2

Target enrollment

6 participants

Primary outcome timeframe

6 and 12 months after HU therapy begins

Results posted on

2019-08-06

Participant Flow

This study is currently closed to patient accrual at all sites due to poor enrollment, including Children's Memorial Hospital, Washington University, Johns Hopkins and the Medical College of Wisconsin. The FDA was notified in 2010, and the IND was removed for this study.

Participant milestones

Participant milestones
Measure	Hydroxyurea Hydroxyurea : 20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose.
Overall Study STARTED	6
Overall Study COMPLETED	5
Overall Study NOT COMPLETED	1

Reasons for withdrawal

Reasons for withdrawal
Measure	Hydroxyurea Hydroxyurea : 20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose.
Overall Study Lost to Follow-up	1

Baseline Characteristics

Hydroxyurea for Children and Young Adults With Sickle Cell Disease and Pulmonary Hypertension

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Hydroxyurea n=6 Participants Hydroxyurea : 20 mg/kg/day and dose escalating every 2 months until maximum tolerated dose.
Age, Categorical <=18 years	6 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	2 Participants n=5 Participants
Sex: Female, Male Male	4 Participants n=5 Participants
Region of Enrollment United States	6 participants n=5 Participants

PRIMARY outcome

Timeframe: 6 and 12 months after HU therapy begins

Population: Study was terminated and 0 participants were analyzed.

Primary outcome measure was tricuspid regurgitant jet velocity (TRJV) by echocardiogram after 6 and 12 months of hydroxyurea therapy.

Outcome measures

Outcome data not reported

SECONDARY outcome

Timeframe: Throughout study

Secondary outcome measures included compliance; laboratory measures of therapy-related toxicity; laboratory biomarkers for hemolysis, oxidative stress and endothelial injury; and quality of life measures by Child Health Questionnaire (CHQ).

Outcome measures

Outcome data not reported

Adverse Events

Hydroxyurea

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

Dr. Robert I. Liem

Ann and Robert H. Lurie Children's Hospital of Chicago

Phone: 312-227-4842

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place