Trial Outcomes & Findings for Allogeneic Hematopoietic Stem Cell Transplantation for Multiple Myeloma (NCT NCT01453101)

Last Updated: 2022-05-25

Results Overview

The main primary endpoint of this study is two-year progression free survival. Patients are considered a failure with respect to PFS if they die or experience disease progression or relapse. The time to this event is the time from transplantation to relapse/progression, initiation of non-protocol anti-myeloma therapy, or death from any cause. Subjects alive without confirmed disease progression will be censored at the time of last disease evaluation. Deaths without progression are treated as failures no matter when they occur.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

54 participants

Primary outcome timeframe

Subjects will be followed for progression-free survival for at least 36 months

Results posted on

2022-05-25

Participant Flow

Participant milestones

Participant milestones
Measure	Fludarabine, Melphalan, Bortezomib Fludarabine monophosphate, melphalan, Bortezomib: •Fludarabine will be administered at a dose of 30/mg/m2 IV daily for 4 days starting on transplant day -5. * Melphalan will be administered at a dose of 140 mg/m2 on transplant day-2 * Bortezomib will be administered by rapid IV push at a dose of 1.6mg/m2 on days-4 and -1. The bortezomib should be given at least 20 hours after the melphalan.
Overall Study STARTED	54
Overall Study COMPLETED	54
Overall Study NOT COMPLETED	0

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Race and Ethnicity were not collected from any participant.

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Fludarabine, Melphalan, Bortezomib n=54 Participants Fludarabine monophosphate, melphalan, Bortezomib: •Fludarabine will be administered at a dose of 30/mg/m2 IV daily for 4 days starting on transplant day -5. * Melphalan will be administered at a dose of 140 mg/m2 on transplant day-2 * Bortezomib will be administered by rapid IV push at a dose of 1.6mg/m2 on days-4 and -1. The bortezomib should be given at least 20 hours after the melphalan.
Age, Continuous	56 years n=54 Participants
Sex: Female, Male Female	19 Participants n=54 Participants
Sex: Female, Male Male	35 Participants n=54 Participants
Region of Enrollment United States	54 participants n=54 Participants

PRIMARY outcome

Timeframe: Subjects will be followed for progression-free survival for at least 36 months

Outcome measures

Outcome measures
Measure	Fludarabine, Melphalan, Bortezomib n=54 Participants Fludarabine monophosphate, melphalan, Bortezomib: •Fludarabine will be administered at a dose of 30/mg/m2 IV daily for 4 days starting on transplant day -5. * Melphalan will be administered at a dose of 140 mg/m2 on transplant day-2 * Bortezomib will be administered by rapid IV push at a dose of 1.6mg/m2 on days-4 and -1. The bortezomib should be given at least 20 hours after the melphalan.
Progression Free Survival	16.7 Months Interval 0.3 to 61.1

SECONDARY outcome

Timeframe: Up to 3 years

Overall survival (OS): Defined as time from the first dose of administration to death from any cause

Outcome measures

Outcome measures
Measure	Fludarabine, Melphalan, Bortezomib n=54 Participants Fludarabine monophosphate, melphalan, Bortezomib: •Fludarabine will be administered at a dose of 30/mg/m2 IV daily for 4 days starting on transplant day -5. * Melphalan will be administered at a dose of 140 mg/m2 on transplant day-2 * Bortezomib will be administered by rapid IV push at a dose of 1.6mg/m2 on days-4 and -1. The bortezomib should be given at least 20 hours after the melphalan.
Overall Survival (OS)	42 percentage

SECONDARY outcome

Timeframe: Up to 3 years

Overall response rate: Defined as the composite endpoint of response to treatment which includes Complete Response (CR), Partial Response (PR), stable disease (SD) as defined in International Response Criteria. International Myeloma Working Group Response Criteria for Multiple Myeloma: CR: Negative immunofixation on the serum and urine and disappearance of any soft tissue plasmacytomas and \< 5% plasma cells in bone marrow PR: \> 50% reduction of serum M-protein and reduction in 24 hours urinary M-protein by \>90% or to \< 200 mg/24 h SD: Not meeting criteria for CR, VGPR, PR, or progressive disease

Outcome measures

Outcome measures
Measure	Fludarabine, Melphalan, Bortezomib n=54 Participants Fludarabine monophosphate, melphalan, Bortezomib: •Fludarabine will be administered at a dose of 30/mg/m2 IV daily for 4 days starting on transplant day -5. * Melphalan will be administered at a dose of 140 mg/m2 on transplant day-2 * Bortezomib will be administered by rapid IV push at a dose of 1.6mg/m2 on days-4 and -1. The bortezomib should be given at least 20 hours after the melphalan.
Overall Response Rate	45 Participants

Adverse Events

Fludarabine, Melphalan, Bortezomib

Serious events: 29 serious events

Other events: 0 other events

Deaths: 27 deaths

Serious adverse events

Serious adverse events
Measure	Fludarabine, Melphalan, Bortezomib n=54 participants at risk Fludarabine monophosphate, melphalan, Bortezomib: •Fludarabine will be administered at a dose of 30/mg/m2 IV daily for 4 days starting on transplant day -5. * Melphalan will be administered at a dose of 140 mg/m2 on transplant day-2 * Bortezomib will be administered by rapid IV push at a dose of 1.6mg/m2 on days-4 and -1. The bortezomib should be given at least 20 hours after the melphalan.
Gastrointestinal disorders Rectal Hemmorhage	1.9% 1/54 • Collected for 3 years post transplant per patient
Gastrointestinal disorders hemorrhoidal rectal bleed	1.9% 1/54 • Collected for 3 years post transplant per patient
General disorders fever	18.5% 10/54 • Collected for 3 years post transplant per patient
Infections and infestations infection/sepsis/URI	53.7% 29/54 • Collected for 3 years post transplant per patient
Renal and urinary disorders Renal Failure	3.7% 2/54 • Collected for 3 years post transplant per patient
Respiratory, thoracic and mediastinal disorders Dyspnea	3.7% 2/54 • Collected for 3 years post transplant per patient
Immune system disorders Graft versus Host Disease	16.7% 9/54 • Collected for 3 years post transplant per patient
Gastrointestinal disorders Diarrhea	5.6% 3/54 • Collected for 3 years post transplant per patient
General disorders Intractable Migraine	1.9% 1/54 • Collected for 3 years post transplant per patient
Respiratory, thoracic and mediastinal disorders Pneumonia	7.4% 4/54 • Collected for 3 years post transplant per patient
Vascular disorders Hypertension	1.9% 1/54 • Collected for 3 years post transplant per patient
Vascular disorders Hypotension	1.9% 1/54 • Collected for 3 years post transplant per patient
Respiratory, thoracic and mediastinal disorders Respiratory Distress/Fluid Overload	1.9% 1/54 • Collected for 3 years post transplant per patient
Eye disorders Decreased Visual Acuity	1.9% 1/54 • Collected for 3 years post transplant per patient
General disorders Multisystem Failure	5.6% 3/54 • Collected for 3 years post transplant per patient
Gastrointestinal disorders Nausea and Vomiting	1.9% 1/54 • Collected for 3 years post transplant per patient
Respiratory, thoracic and mediastinal disorders Metapneumovirus Infection	5.6% 3/54 • Collected for 3 years post transplant per patient
Vascular disorders Veno Occlusive Disease	1.9% 1/54 • Collected for 3 years post transplant per patient
Respiratory, thoracic and mediastinal disorders Shortness of Breath	3.7% 2/54 • Collected for 3 years post transplant per patient
Cardiac disorders Atrial Fibrillation	3.7% 2/54 • Collected for 3 years post transplant per patient
Vascular disorders Hemolytic Anemia	1.9% 1/54 • Collected for 3 years post transplant per patient
Nervous system disorders Epidural Tumor	1.9% 1/54 • Collected for 3 years post transplant per patient
General disorders Progressive Disease	13.0% 7/54 • Collected for 3 years post transplant per patient
Gastrointestinal disorders GI Disorder	3.7% 2/54 • Collected for 3 years post transplant per patient
General disorders Confusion	3.7% 2/54 • Collected for 3 years post transplant per patient
Blood and lymphatic system disorders Hyponatremia	1.9% 1/54 • Collected for 3 years post transplant per patient
Blood and lymphatic system disorders Thrombocytopenia	1.9% 1/54 • Collected for 3 years post transplant per patient
Gastrointestinal disorders GI Pain	1.9% 1/54 • Collected for 3 years post transplant per patient
Endocrine disorders Diabetic Ketoacidosis	1.9% 1/54 • Collected for 3 years post transplant per patient
Nervous system disorders Cerebral Edema	1.9% 1/54 • Collected for 3 years post transplant per patient
Respiratory, thoracic and mediastinal disorders Respiratory Syncytial Virus	1.9% 1/54 • Collected for 3 years post transplant per patient
Gastrointestinal disorders Malnutrition	1.9% 1/54 • Collected for 3 years post transplant per patient
General disorders Fall	1.9% 1/54 • Collected for 3 years post transplant per patient
Infections and infestations Abdominal Wall Cellulitis	1.9% 1/54 • Collected for 3 years post transplant per patient

Other adverse events

Adverse event data not reported

Additional Information

Joshua Zenreich

Hackensack Meridian Health

Phone: 15519964248

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place