Trial Outcomes & Findings for Stem Cell Transplantation for Sickle Cell Anemia (NCT NCT01877837)

NCT ID: NCT01877837

Last Updated: 2022-11-07

Results Overview

Primary endpoint: In each group, the Number of participants with Graft Failure at the 2 years endpoint will be estimated using the Kaplan Meier product limit estimator.

• Recruitment status: COMPLETED
• Study phase: PHASE3
• Target enrollment: 30 participants
• Primary outcome timeframe: 2 years
• Results posted on: 2022-11-07

Participant Flow

Participant milestones

Measure	Patients With Sickle Cell Anemia Patients with sickle cell anemia, age 2-30, that received transplant after after conditioning regimen Alemtuzumab, Fludarabine, and Melphalan.
Overall Study STARTED	30
Overall Study COMPLETED	25
Overall Study NOT COMPLETED	5

Reasons for withdrawal

Measure	Patients With Sickle Cell Anemia Patients with sickle cell anemia, age 2-30, that received transplant after after conditioning regimen Alemtuzumab, Fludarabine, and Melphalan.
Overall Study Lost to Follow-up	1
Overall Study Ineligible	4

Baseline Characteristics

Race/Ethnicity Information was not collected.

Baseline characteristics by cohort

Measure	Patients With Sickle Cell Anemia n=26 Participants Patients with sickle cell anemia, age 2-30, that received transplant after after conditioning regimen Alemtuzumab, Fludarabine, and Melphalan.
Age, Categorical <=18 years	22 Participants n=26 Participants
Age, Categorical Between 18 and 65 years	4 Participants n=26 Participants
Age, Categorical >=65 years	0 Participants n=26 Participants
Age, Continuous	11.68 years n=26 Participants
Sex: Female, Male Female	11 Participants n=26 Participants
Sex: Female, Male Male	15 Participants n=26 Participants
Race/Ethnicity, Customized Race/Ethnicity	0 Participants Race/Ethnicity Information was not collected.
Region of Enrollment United States	26 participants n=26 Participants

PRIMARY outcome

Timeframe: 2 years

Population: Patients that were followed at 2 years post transplant for graft failure

Primary endpoint:

In each group, the Number of participants with Graft Failure at the 2 years endpoint will be estimated using the Kaplan Meier product limit estimator.

Outcome measures

Measure	Patients With Sickle Cell Anemia n=23 Participants Patients with sickle cell anemia, age 2-30, that received transplant after after conditioning regimen Alemtuzumab, Fludarabine, and Melphalan.
Number of Participants With Graft Failure	3 Participants

SECONDARY outcome

Timeframe: 2 years

Population: Patients surviving at 2 years post transplant

Secondary endpoints:

Overall survival: The distribution of time to death from any cause will be estimated by Kaplan- Meier product limit function and plotted. The overall survival will be measured from the time of transplant to any death and patients will be followed for 2 years.

Outcome measures

Measure	Patients With Sickle Cell Anemia n=25 Participants Patients with sickle cell anemia, age 2-30, that received transplant after after conditioning regimen Alemtuzumab, Fludarabine, and Melphalan.
Overall Survival	23 Participants

Adverse Events

Patients With Sickle Cell Anemia

Serious events: 13 serious events

Other events: 0 other events

Deaths: 2 deaths

Serious adverse events

Measure	Patients With Sickle Cell Anemia n=25 participants at risk Patients with sickle cell anemia, age 2-30, that received transplant after after conditioning regimen Alemtuzumab, Fludarabine, and Melphalan.
Infections and infestations Infection (grade 3 and above)	36.0% 9/25 • Number of events 9 • 2 year post transplant Adverse events grade 3 and above (including deaths) were considered as serious adverse events and are reported
Renal and urinary disorders Renal insufficiency	8.0% 2/25 • Number of events 2 • 2 year post transplant Adverse events grade 3 and above (including deaths) were considered as serious adverse events and are reported
Cardiac disorders SupraVentricular Tachycardia	4.0% 1/25 • Number of events 1 • 2 year post transplant Adverse events grade 3 and above (including deaths) were considered as serious adverse events and are reported
Nervous system disorders Alerted mental status	4.0% 1/25 • Number of events 1 • 2 year post transplant Adverse events grade 3 and above (including deaths) were considered as serious adverse events and are reported
Nervous system disorders Posterior Reversible Encephalopathy Syndrome	4.0% 1/25 • Number of events 1 • 2 year post transplant Adverse events grade 3 and above (including deaths) were considered as serious adverse events and are reported
Gastrointestinal disorders Gastrointestinal bleed	4.0% 1/25 • Number of events 1 • 2 year post transplant Adverse events grade 3 and above (including deaths) were considered as serious adverse events and are reported
Respiratory, thoracic and mediastinal disorders Respiratory failure	4.0% 1/25 • Number of events 1 • 2 year post transplant Adverse events grade 3 and above (including deaths) were considered as serious adverse events and are reported
Metabolism and nutrition disorders Hypokalemia	4.0% 1/25 • Number of events 1 • 2 year post transplant Adverse events grade 3 and above (including deaths) were considered as serious adverse events and are reported
Immune system disorders Graft versus host disease	24.0% 6/25 • Number of events 6 • 2 year post transplant Adverse events grade 3 and above (including deaths) were considered as serious adverse events and are reported

Other adverse events

Adverse event data not reported

Additional Information

Elana Smilow

Hackensack Meridian Health

Phone: 5519965673

Email: Elana.Smilow@hmhn.org

Results disclosure agreements

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