Trial Outcomes & Findings for A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen (NCT NCT00427661)

NCT ID: NCT00427661

Last Updated: 2016-08-18

Results Overview

GVHD is assessed by physical exam, bloodwork and biopsy.

• Recruitment status: COMPLETED
• Study phase: NA
• Target enrollment: 8 participants
• Primary outcome timeframe: 1 year
• Results posted on: 2016-08-18

Participant Flow

Participant milestones

Measure	AHSC in Severe SCD The patient population for this study included severe SCD patients, both pediatric and adult, who did not have end-organ failure, and met all of the eligibility criteria.
Overall Study STARTED	8
Overall Study COMPLETED	8
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

A Pilot Study of HSCT for Patients With High-risk Hemoglobinopathy Using a Nonmyeloablative Preparative Regimen

Baseline characteristics by cohort

Measure	AHSC in Severe SCD n=8 Participants The patient population for this study included severe SCD patients, both pediatric and adult, who did not have end-organ failure, and met all of the eligibility criteria.
Age, Categorical <=18 years	6 Participants n=93 Participants
Age, Categorical Between 18 and 65 years	2 Participants n=93 Participants
Age, Categorical >=65 years	0 Participants n=93 Participants
Sex: Female, Male Female	4 Participants n=93 Participants
Sex: Female, Male Male	4 Participants n=93 Participants
Region of Enrollment United States	8 participants n=93 Participants

PRIMARY outcome

Timeframe: 1 year

GVHD is assessed by physical exam, bloodwork and biopsy.

Outcome measures

Measure	AHSC in Severe SCD n=7 Participants The patient population for this study included severe SCD patients, both pediatric and adult, who did not have end-organ failure, and met all of the eligibility criteria.
Development of GVHD Within 1 Year of BMT	2 participants

PRIMARY outcome

Timeframe: 1 year

Measurement of total PBMC chimerism

Outcome measures

Measure	AHSC in Severe SCD n=7 Participants The patient population for this study included severe SCD patients, both pediatric and adult, who did not have end-organ failure, and met all of the eligibility criteria.
Engraftment at 1 Year Post BMT.	6 participants

SECONDARY outcome

Timeframe: 100 days

Outcome measures

Measure	AHSC in Severe SCD n=8 Participants The patient population for this study included severe SCD patients, both pediatric and adult, who did not have end-organ failure, and met all of the eligibility criteria.
Incidence of Grade 2-4 Acute GVHD.	1 participants with grade 2-4 AGVHD

Adverse Events

AHSC in Severe SCD

Serious events: 1 serious events

Other events: 8 other events

Deaths: 0 deaths

Serious adverse events

Measure	AHSC in Severe SCD n=8 participants at risk The patient population for this study included severe SCD patients, both pediatric and adult, who did not have end-organ failure, and met all of the eligibility criteria.
Respiratory, thoracic and mediastinal disorders Pulmonary Arterial Hypertension	12.5% 1/8

Other adverse events

Measure	AHSC in Severe SCD n=8 participants at risk The patient population for this study included severe SCD patients, both pediatric and adult, who did not have end-organ failure, and met all of the eligibility criteria.
Nervous system disorders Seizure	14.3% 1/7 • Number of events 1
Nervous system disorders Headache	12.5% 1/8 • Number of events 1
Infections and infestations Line related bacteremia	12.5% 1/8 • Number of events 1
Gastrointestinal disorders Pancreatitis	12.5% 1/8 • Number of events 1
Infections and infestations Infection	12.5% 1/8 • Number of events 1
Gastrointestinal disorders Poor oral intake	25.0% 2/8 • Number of events 2
Renal and urinary disorders Hemoglobinuria	12.5% 1/8 • Number of events 1

Additional Information

Nancy Harter-Administrative Director of Research

University of Pittsburgh-Department of Pediatrics

Phone: 412-692-7487

Email: harterne@upmc.edu

Results disclosure agreements

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