Treatment of Adult Patients With Hemoglobin SC Disease (SCYTHE)

• ClinicalTrials.gov Identifier: NCT02640573
• Recruitment Status: Terminated
• First Posted: December 29, 2015
• Results First Posted: January 27, 2020
• Last Update Posted: January 27, 2020
• Sponsor: Baylor College of Medicine
• Collaborator: The University of Texas Health Science Center, Houston
• Information provided by (Responsible Party): Vivien Sheehan, Baylor College of Medicine

• Study Type: Interventional
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Hemoglobin SC Disease
• Intervention: Drug: Hydroxyurea
• Enrollment: 1

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Hydroxurea
Arm/Group Description	Initiate hydroxyurea at 10 mg/kg daily and escalate hydroxyurea dose by 5 mg/kg/day every 8 weeks up to a maximum dose of 35 mg/kg/day if blood counts meet escalation criteria. Hydroxyurea: Treat symptomatic HbSC patients to MTD on hydroxyurea, and assess for clinical improvement using the AdultsQLTM 3.0 Sickle Cell Disease Module after 6 months at MTD, compared to entrance scores
Period Title: Overall Study
Started	1
Completed	0
Not Completed	1

Baseline Characteristics

Arm/Group Title		Hydroxurea
Arm/Group Description		Initiate hydroxyurea at 10 mg/kg daily and escalate hydroxyurea dose by 5 mg/kg/day every 8 weeks up to a maximum dose of 35 mg/kg/day if blood counts meet escalation criteria. Hydroxyurea: Treat symptomatic HbSC patients to MTD on hydroxyurea, and assess for clinical improvement using the AdultsQLTM 3.0 Sickle Cell Disease Module after 6 months at MTD, compared to entrance scores
Overall Number of Baseline Participants		1
Baseline Analysis Population Description		[Not Specified]
Age, Categorical [1]; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	1 participants
	<=18 years	0 0.0%
	Between 18 and 65 years	1 100.0%
	>=65 years	0 0.0%
		[1] Measure Description: chart review
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	1 participants
		35 (0)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	1 participants
	Female	1
	Male	0
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	1 participants
	American Indian or Alaska Native	0 0.0%
	Asian	0 0.0%
	Native Hawaiian or Other Pacific Islander	0 0.0%
	Black or African American	1 100.0%
	White	0 0.0%
	More than one race	0 0.0%
	Unknown or Not Reported	0 0.0%
Region of Enrollment; Measure Type: Count of Participants; Unit of measure: Participants
United States	Number Analyzed	1 participants
		1 100.0%
Fetal hemoglobin level at baseline; Mean (Full Range); Unit of measure: %
	Number Analyzed	1 participants
		2; (2 to 2)

Outcome Measures

1. Primary Outcome

Title	Fetal Hemoglobin Response to HU
Description	Change in HbF on hydroxyurea from baseline
Time Frame	Baseline to 6 months

Outcome Measure Data

Analysis Population Description

Baseline QoL only; no subsequent values obtained.

Arm/Group Title	Single Arm Study
Arm/Group Description:	adult patient on HU, measure of QoL
Overall Number of Participants Analyzed	0

No data displayed because Outcome Measure has zero total analyzed.

Adverse Events

Time Frame	1 month
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Hydroxurea
Arm/Group Description	Initiate hydroxyurea at 10 mg/kg daily and escalate hydroxyurea dose by 5 mg/kg/day every 8 weeks up to a maximum dose of 35 mg/kg/day if blood counts meet escalation criteria. Hydroxyurea: Treat symptomatic HbSC patients to MTD on hydroxyurea, and assess for clinical improvement using the AdultsQLTM 3.0 Sickle Cell Disease Module after 6 months at MTD, compared to entrance scores
All-Cause Mortality
	Hydroxurea
	Affected / at Risk (%)
Total	0/1 (0.00%)
Serious Adverse Events
	Hydroxurea
	Affected / at Risk (%)
Total	0/1 (0.00%)
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	0%
	Hydroxurea
	Affected / at Risk (%)
Total	0/1 (0.00%)

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

All Principal Investigators ARE employed by the organization sponsoring the study.

Results Point of Contact

• Name/Title: Vivien Sheehan
• Organization: Baylor College of Medicine
• Phone: 832 824 4459
• EMail: vsheehan@bcm.edu

• Responsible Party: Vivien Sheehan, Baylor College of Medicine
• ClinicalTrials.gov Identifier: NCT02640573
• Other Study ID Numbers: 37290 IM-SCYTHE
• First Submitted: December 17, 2015
• First Posted: December 29, 2015
• Results First Submitted: August 29, 2019
• Results First Posted: January 27, 2020
• Last Update Posted: January 27, 2020