Trial Outcomes & Findings for Thymoglobulin, Sirolimus and Mycophenolate Mofetil for Prevention of Acute Graft-Versus-Host Disease (GVHD) (NCT NCT00506948)

NCT ID: NCT00506948

Last Updated: 2020-09-24

Results Overview

Efficacy failure defined as a participants who had either grade 3-4 acute graft-versus-host disease (aGVHD) or treatment related mortality (TRM) within 100 days post transplant. Failure Rate calculated as (# of failures) / (# participants evaluated). Physical exam and bloodwork every week (for the first 90-100 days after the transplant).

• Recruitment status: TERMINATED
• Study phase: PHASE2
• Target enrollment: 13 participants
• Primary outcome timeframe: Baseline to 100 days post transplant
• Results posted on: 2020-09-24

Participant Flow

Recruitment Period: September 08, 2006 to October 27, 2009. All recruitment done in a medical clinic setting.

Of the 13 participants enrolled, three participants were ineligible and excluded from the study before treatment.

Participant milestones

Measure	Thymoglobulin + Sirolimus + MMF Thymoglobulin 1.5 mg/kg intravenous (IV) days -4, -3, -2, -1 before Stem Cell Transplant (Day 0); Sirolimus 6 mg IV on day -2 followed by 2 mg daily to maintain therapeutic levels and Mycophenolate Mofetil (MMF) 15 mg/kg IV or orally every 12 hours starting on day 0 until day+27.
Overall Study STARTED	10
Overall Study COMPLETED	0
Overall Study NOT COMPLETED	10

Reasons for withdrawal

Measure	Thymoglobulin + Sirolimus + MMF Thymoglobulin 1.5 mg/kg intravenous (IV) days -4, -3, -2, -1 before Stem Cell Transplant (Day 0); Sirolimus 6 mg IV on day -2 followed by 2 mg daily to maintain therapeutic levels and Mycophenolate Mofetil (MMF) 15 mg/kg IV or orally every 12 hours starting on day 0 until day+27.
Overall Study Death	4
Overall Study Adverse Event	5
Overall Study Physician Decision	1

Baseline Characteristics

Thymoglobulin, Sirolimus and Mycophenolate Mofetil for Prevention of Acute Graft-Versus-Host Disease (GVHD)

Baseline characteristics by cohort

Measure	Thymoglobulin + Sirolimus + MMF n=10 Participants Thymoglobulin 1.5 mg/kg intravenous (IV) days -4, -3, -2, -1 before Stem Cell Transplant (Day 0); Sirolimus 6 mg IV on day -2 followed by 2 mg daily to maintain therapeutic levels and Mycophenolate Mofetil (MMF) 15 mg/kg IV or orally every 12 hours starting on day 0 until day+27.
Age, Continuous	55 years n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants
Sex: Female, Male Male	3 Participants n=5 Participants
Region of Enrollment United States	10 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline to 100 days post transplant

Efficacy failure defined as a participants who had either grade 3-4 acute graft-versus-host disease (aGVHD) or treatment related mortality (TRM) within 100 days post transplant. Failure Rate calculated as (# of failures) / (# participants evaluated). Physical exam and bloodwork every week (for the first 90-100 days after the transplant).

Outcome measures

Outcome data not reported

PRIMARY outcome

Timeframe: Baseline to 100 days post transplant

Participants who had acute graft-versus-host disease (aGVHD) within 100 days post transplant. Physical exam and bloodwork every week (for the first 90-100 days after the transplant).

Outcome measures

Measure	Thymoglobulin + Sirolimus + MMF n=10 Participants Thymoglobulin 1.5 mg/kg intravenous (IV) days -4, -3, -2, -1 before Stem Cell Transplant (Day 0); Sirolimus 6 mg IV on day -2 followed by 2 mg daily to maintain therapeutic levels and Mycophenolate Mofetil (MMF) 15 mg/kg IV or orally every 12 hours starting on day 0 until day+27.
Number of Participants With Acute Graft-versus-host Disease (aGVHD)	6 participants

Adverse Events

Thymoglobulin + Sirolimus + MMF

Serious events: 6 serious events

Other events: 4 other events

Deaths: 0 deaths

Serious adverse events

Measure	Thymoglobulin + Sirolimus + MMF n=10 participants at risk Thymoglobulin 1.5 mg/kg intravenous (IV) days -4, -3, -2, -1 before Stem Cell Transplant (Day 0); Sirolimus 6 mg IV on day -2 followed by 2 mg daily to maintain therapeutic levels and Mycophenolate Mofetil (MMF) 15 mg/kg IV or orally every 12 hours starting on day 0 until day+27.
General disorders Death	40.0% 4/10 • Adverse event reporting from active treatment completed on day 90 (last day of sirolimus) through post-study surveillance to Day +100. Adverse events were collected for a total study period of 2 years and 11 months.
Renal and urinary disorders Veno-occlusive disease of the liver	40.0% 4/10 • Adverse event reporting from active treatment completed on day 90 (last day of sirolimus) through post-study surveillance to Day +100. Adverse events were collected for a total study period of 2 years and 11 months.
Infections and infestations Pneumonia	10.0% 1/10 • Adverse event reporting from active treatment completed on day 90 (last day of sirolimus) through post-study surveillance to Day +100. Adverse events were collected for a total study period of 2 years and 11 months.

Other adverse events

Measure	Thymoglobulin + Sirolimus + MMF n=10 participants at risk Thymoglobulin 1.5 mg/kg intravenous (IV) days -4, -3, -2, -1 before Stem Cell Transplant (Day 0); Sirolimus 6 mg IV on day -2 followed by 2 mg daily to maintain therapeutic levels and Mycophenolate Mofetil (MMF) 15 mg/kg IV or orally every 12 hours starting on day 0 until day+27.
Gastrointestinal disorders Nausea	20.0% 2/10 • Adverse event reporting from active treatment completed on day 90 (last day of sirolimus) through post-study surveillance to Day +100. Adverse events were collected for a total study period of 2 years and 11 months.
Renal and urinary disorders Hemorrhagic Cystitis	10.0% 1/10 • Adverse event reporting from active treatment completed on day 90 (last day of sirolimus) through post-study surveillance to Day +100. Adverse events were collected for a total study period of 2 years and 11 months.
Renal and urinary disorders Veno-occlusive disease of the liver (Moderate)	20.0% 2/10 • Adverse event reporting from active treatment completed on day 90 (last day of sirolimus) through post-study surveillance to Day +100. Adverse events were collected for a total study period of 2 years and 11 months.
Skin and subcutaneous tissue disorders Skin GVHD	20.0% 2/10 • Adverse event reporting from active treatment completed on day 90 (last day of sirolimus) through post-study surveillance to Day +100. Adverse events were collected for a total study period of 2 years and 11 months.
Gastrointestinal disorders Gastrointestinal GVHD	20.0% 2/10 • Adverse event reporting from active treatment completed on day 90 (last day of sirolimus) through post-study surveillance to Day +100. Adverse events were collected for a total study period of 2 years and 11 months.
General disorders Acute GVHD	20.0% 2/10 • Adverse event reporting from active treatment completed on day 90 (last day of sirolimus) through post-study surveillance to Day +100. Adverse events were collected for a total study period of 2 years and 11 months.

Additional Information

Amin Alousi, MD /Professor, Stem Cell Transplantation

University of Texas (UT) MD Anderson Cancer Center

Phone: 713-745-8613

Email: aalousi@mdanderson.org

Results disclosure agreements

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