Trial Outcomes & Findings for A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease (NCT NCT01279616)

Results Overview

Event-free survival

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

8 participants

Primary outcome timeframe

1 year

Results posted on

2019-04-03

Participant Flow

Participant milestones

Participant milestones
Measure	Hematopoietic Stem Cell Transplant Drug: Fludarabine monophosphate 180 mg/m2 over 6 days. Drug: Rituximab 375 mg/m2 on day -13 and day -3 Other Names: •Rituxan Drug: Busulfan AUC 1000-1200 microM.mt Other Names: •busulfex Drug: ATG 2.5 mg/kg for 3 days Other Names: •Thymoglobulin Drug: Cyclophosphamide 50 mg/kg on day +3 Other Names: •Cytoxan Drug: Mycophenolate mofetil 15 mg/kg q 8 hours Other Names: •MMF, Cell-cept. Drug: Tacrolimus 0.03 mg/kg /d Other Names: •FK-506
Overall Study STARTED	8
Overall Study COMPLETED	1
Overall Study NOT COMPLETED	7

Reasons for withdrawal

Reasons for withdrawal
Measure	Hematopoietic Stem Cell Transplant Drug: Fludarabine monophosphate 180 mg/m2 over 6 days. Drug: Rituximab 375 mg/m2 on day -13 and day -3 Other Names: •Rituxan Drug: Busulfan AUC 1000-1200 microM.mt Other Names: •busulfex Drug: ATG 2.5 mg/kg for 3 days Other Names: •Thymoglobulin Drug: Cyclophosphamide 50 mg/kg on day +3 Other Names: •Cytoxan Drug: Mycophenolate mofetil 15 mg/kg q 8 hours Other Names: •MMF, Cell-cept. Drug: Tacrolimus 0.03 mg/kg /d Other Names: •FK-506
Overall Study donor could not be located	5
Overall Study no signs of sickle cell disease	2

Baseline Characteristics

A Reduced Toxicity Allogeneic Unrelated Donor Stem Cell Transplantation (SCT) for Severe Sickle Cell Disease

Baseline characteristics by cohort

Baseline data not reported

PRIMARY outcome

Timeframe: 1 year

Population: The study was terminated and the PI has left the institution. Efforts were made to contact the PI but unsuccessful. No outcome measure data is available for the study.

Event-free survival

Outcome measures

Outcome data not reported

Adverse Events

No Arm Analyzed: N/A

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

Kristy Ott

Nationwide Children's Hospital

Phone: 614-722-6313

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee No Outcome data are available for this study as the study was terminated. The PI has left the institution and cannot be contacted.
Publication restrictions are in place

Restriction type: OTHER