Trial Outcomes & Findings for High-Dose Gemcitabine, Busulfan and Melphalan for Patients With Refactory Hodgkin's Disease (NCT NCT01200329)

NCT ID: NCT01200329

Last Updated: 2019-12-16

Results Overview

The event-free survival (EFS) of patients with poor prognosis relapse or refractory Hodgkin's disease (HD) after high-dose chemotherapy (HDC) with Gemcitabine/Busulfan/Melphalan (GemBuMel). Event is defined as relapse, tumor progression or death.Progression free survival is the length of time during and after the treatment of disease that a patient lives with the disease but it does not get worse. Toxicity is defined as the treatment related mortality (TRM) rate, which will be evaluated within 30 days post transplant, and this rate will be compared with the 5% maximum rate. For EFS analysis, patients who experience the tumor relapse, disease progression, or death will be considered to be an event.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

81 participants

Primary outcome timeframe

Enrollment up to 2 years post transplant

Results posted on

2019-12-16

Participant Flow

Patients enrolled at MD Anderson clinic starting on June 16, 2011.

Participant milestones

Participant milestones
Measure	Overall Study Group Busulfan (adjusted PK dosing) IV for 4 days+Gemcitabine 2775 mg/m2 IV for 2 days+Melphalan 60 mg/m2 IV for 2 days +/- Rituximab 375 mg/m2 IV for 1 day (for CD20 positive tumors)+ Auto Stem Cell Transplant (SCT)+Palifermin 60 mcg/kg IV for 3 days post SCT
Overall Study STARTED	81
Overall Study COMPLETED	78
Overall Study NOT COMPLETED	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Overall Study Group Busulfan (adjusted PK dosing) IV for 4 days+Gemcitabine 2775 mg/m2 IV for 2 days+Melphalan 60 mg/m2 IV for 2 days +/- Rituximab 375 mg/m2 IV for 1 day (for CD20 positive tumors)+ Auto Stem Cell Transplant (SCT)+Palifermin 60 mcg/kg IV for 3 days post SCT
Overall Study Lack of financial coverage	2
Overall Study Progressive disease	1

Baseline Characteristics

High-Dose Gemcitabine, Busulfan and Melphalan for Patients With Refactory Hodgkin's Disease

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Overall Study Group n=78 Participants Busulfan (adjusted PK dosing) IV for 4 days+Gemcitabine 2775 mg/m2 IV for 2 days+Melphalan 60 mg/m2 IV for 2 days +/- Rituximab 375 mg/m2 IV for 1 day (for CD20 positive tumors)+ Auto Stem Cell Transplant (SCT)+Palifermin 60 mcg/kg IV for 3 days post SCT
Age, Categorical <=18 years	2 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	74 Participants n=5 Participants
Age, Categorical >=65 years	2 Participants n=5 Participants
Age, Continuous	31.5 years n=5 Participants
Sex: Female, Male Female	32 Participants n=5 Participants
Sex: Female, Male Male	46 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	15 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	36 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	27 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	3 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	10 Participants n=5 Participants
Race (NIH/OMB) White	52 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	13 Participants n=5 Participants
Region of Enrollment United States	78 participants n=5 Participants
Overall Study Group Primary Refractory Hodgkin's	32 participants n=5 Participants
Overall Study Group Relapsed Hodgkin's	46 participants n=5 Participants

PRIMARY outcome

Timeframe: Enrollment up to 2 years post transplant

Population: Patients with relapsed/refractory Hodgkin's disease (ie, extranodal relapse or within 1 year of frontline therapy).

Outcome measures

Outcome measures
Measure	Overall Study Group n=78 Participants Busulfan (adjusted PK dosing) IV for 4 days+Gemcitabine 2775 mg/m2 IV for 2 days+Melphalan 60 mg/m2 IV for 2 days +/- Rituximab 375 mg/m2 IV for 1 day (for CD20 positive tumors)+ Auto Stem Cell Transplant (SCT)+Palifermin 60 mcg/kg IV for 3 days post SCT
Event-free Survival (EFS) of Patients	51 Participants

SECONDARY outcome

Timeframe: Beyond 100 days post transplant up to 84 months.

The overall survival is the length of time from the start of treatment (Auto SCT) for the cancer, that patients are diagnosed with are still alive until date of first documented progression or date of death from any cause. It is measured in months and assessed up to 84 months.

Outcome measures

Outcome measures
Measure	Overall Study Group n=78 Participants Busulfan (adjusted PK dosing) IV for 4 days+Gemcitabine 2775 mg/m2 IV for 2 days+Melphalan 60 mg/m2 IV for 2 days +/- Rituximab 375 mg/m2 IV for 1 day (for CD20 positive tumors)+ Auto Stem Cell Transplant (SCT)+Palifermin 60 mcg/kg IV for 3 days post SCT
Overall Survival (OS) of These Patients.	52 Months Interval 4.0 to 84.0

Adverse Events

Overall Study Group

Serious events: 0 serious events

Other events: 78 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Other adverse events
Measure	Overall Study Group n=78 participants at risk Busulfan (adjusted PK dosing) IV for 4 days+Gemcitabine 2775 mg/m2 IV for 2 days+Melphalan 60 mg/m2 IV for 2 days +/- Rituximab 375 mg/m2 IV for 1 day (for CD20 positive tumors)+ Auto Stem Cell Transplant (SCT)+Palifermin 60 mcg/kg IV for 3 days post SCT
Cardiac disorders Hypertension	6.4% 5/78 • Number of events 5 • Up to 100 Days post transplant
General disorders Fluid Overload	42.3% 33/78 • Number of events 33 • Up to 100 Days post transplant
General disorders Fever	10.3% 8/78 • Number of events 8 • Up to 100 Days post transplant
Gastrointestinal disorders Diarrhea	50.0% 39/78 • Number of events 40 • Up to 100 Days post transplant
Gastrointestinal disorders Mucositis	97.4% 76/78 • Number of events 76 • Up to 100 Days post transplant
Gastrointestinal disorders Nausea	87.2% 68/78 • Number of events 68 • Up to 100 Days post transplant
Hepatobiliary disorders Transaminitis	70.5% 55/78 • Number of events 63 • Up to 100 Days post transplant
Hepatobiliary disorders Elevated Bilirubin	46.2% 36/78 • Number of events 36 • Up to 100 Days post transplant
Infections and infestations Infection	25.6% 20/78 • Number of events 27 • Up to 100 Days post transplant
General disorders Neutropenic Fever	79.5% 62/78 • Number of events 62 • Up to 100 Days post transplant
Respiratory, thoracic and mediastinal disorders Pneumonitis	11.5% 9/78 • Number of events 9 • Up to 100 Days post transplant
Skin and subcutaneous tissue disorders Skin Rash	67.9% 53/78 • Number of events 54 • Up to 100 Days post transplant
Skin and subcutaneous tissue disorders Skin Hand/Foot Syndrome	7.7% 6/78 • Number of events 6 • Up to 100 Days post transplant
Skin and subcutaneous tissue disorders Pruritis	5.1% 4/78 • Number of events 4 • Up to 100 Days post transplant
Nervous system disorders Headache	23.1% 18/78 • Number of events 18 • Up to 100 Days post transplant

Additional Information

Yago Nieto/Stem Cell Transplantation and Cellular Therapy

UT MD Anderson Cancer Center

Phone: 713-792-8750

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place