Busulfan, Melphalan, Escalating Carfilzomib Conditioning Auto Stem Cell Transplantation for Multiple Myeloma (MM) (BuMelCarAuto)
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ClinicalTrials.gov Identifier: NCT03795597 |
Recruitment Status :
Recruiting
First Posted : January 8, 2019
Last Update Posted : August 20, 2019
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Multiple Myeloma | Drug: Carfilzomib Drug: Busulfan IV Drug: Melphalan IV | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 36 participants |
Allocation: | Non-Randomized |
Intervention Model: | Sequential Assignment |
Intervention Model Description: | Busulfan IV over 3 hours every 24 hours for a total of 4 doses from D-6 to D-3. First two daily infusions will be given at a fixed dose of 3.2 mg/kg over 3 hours from D-6 to D-5. The 3rd and 4th daily doses will be adjusted to an AUC of approximately 5,000 mMol-min per dose. Melphalan 140 mg/ m2 IV over 15-30 minutes for one dose on day -3. Carfilzomib administration will follow a Phase I dose escalation design. Each cohort will start with a goal of accruing three patients to determine the dose limiting toxicities. Once the Maximum Tolerated Dose is established an expansion cohort will be treated to include a total of 10 additional patients. |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | Phase I/II Study Utilizing High Dose Busulfan and Melphalan With Escalating Carfilzomib as Conditioning in Autologous Peripheral Blood Stem Cell Transplantation for Patients With Multiple Myeloma |
Actual Study Start Date : | May 22, 2019 |
Estimated Primary Completion Date : | November 1, 2022 |
Estimated Study Completion Date : | November 1, 2023 |

Arm | Intervention/treatment |
---|---|
Experimental: Carfilzomib IV at dose: 20 mg/m2
The participants will receive Carfilzomib IV at dose: 20 mg/m2 on days -9 and -8 over 30 minutes. The participant will receive Busulfan IV over 3 hours every 24 hours for a total of 4 doses from Day -6 to Day -3 and Melphalan IV over 15-30 minutes for one dose on day -3.The participants will receive stem cell infusion on Day 0 and Granulocyte-Colony stimulating factor (G-CSF) given daily until engraftment occurs.
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Drug: Carfilzomib
Carfilzomib is an anti-cancer drug acting as a selective proteasome inhibitor that is used to treat Multiple Myeloma.
Other Name: Kyprolis Drug: Busulfan IV Busulfan is an anti-cancer drug acting as a bifunctional alkylating agent that is used to treat Multiple Myeloma.
Other Name: Busulfex Drug: Melphalan IV Melphalan is an anti-cancer drug acting as alkylating agent that is used to treat Multiple Myeloma.
Other Name: Alkeran |
Experimental: Carfilzomib IV at dose: 27 mg/m2
The participants will receive Carfilzomib IV at dose: 27 mg/m2 on days -9 and -8 over 30 minutes. The participant will receive Busulfan IV over 3 hours every 24 hours for a total of 4 doses from Day -6 to Day -3 and Melphalan IV over 15-30 minutes for one dose on day -3.The participants will receive stem cell infusion on Day 0 and G-CSF given daily until engraftment occurs.
|
Drug: Carfilzomib
Carfilzomib is an anti-cancer drug acting as a selective proteasome inhibitor that is used to treat Multiple Myeloma.
Other Name: Kyprolis Drug: Busulfan IV Busulfan is an anti-cancer drug acting as a bifunctional alkylating agent that is used to treat Multiple Myeloma.
Other Name: Busulfex Drug: Melphalan IV Melphalan is an anti-cancer drug acting as alkylating agent that is used to treat Multiple Myeloma.
Other Name: Alkeran |
Experimental: Carfilzomib IV at dose: 36 mg/m2
The participants will receive Carfilzomib IV at dose: 36 mg/m2 on days -9 and -8 over 30 minutes. The participant will receive Busulfan IV over 3 hours every 24 hours for a total of 4 doses from Day -6 to Day -3 and Melphalan IV over 15-30 minutes for one dose on day -3.The participants will receive stem cell infusion on Day 0 and G-CSF given daily until engraftment occurs.
|
Drug: Carfilzomib
Carfilzomib is an anti-cancer drug acting as a selective proteasome inhibitor that is used to treat Multiple Myeloma.
Other Name: Kyprolis Drug: Busulfan IV Busulfan is an anti-cancer drug acting as a bifunctional alkylating agent that is used to treat Multiple Myeloma.
Other Name: Busulfex Drug: Melphalan IV Melphalan is an anti-cancer drug acting as alkylating agent that is used to treat Multiple Myeloma.
Other Name: Alkeran |
Experimental: Carfilzomib IV at dose: 45 mg/m2
The participants will receive Carfilzomib IV at dose: 45 mg/m2 on days -9 and -8 over 30 minutes. The participant will receive Busulfan IV over 3 hours every 24 hours for a total of 4 doses from Day -6 to Day -3 and Melphalan IV over 15-30 minutes for one dose on day -3.The participants will receive stem cell infusion on Day 0 and G-CSF given daily until engraftment occurs.
|
Drug: Carfilzomib
Carfilzomib is an anti-cancer drug acting as a selective proteasome inhibitor that is used to treat Multiple Myeloma.
Other Name: Kyprolis Drug: Busulfan IV Busulfan is an anti-cancer drug acting as a bifunctional alkylating agent that is used to treat Multiple Myeloma.
Other Name: Busulfex Drug: Melphalan IV Melphalan is an anti-cancer drug acting as alkylating agent that is used to treat Multiple Myeloma.
Other Name: Alkeran |
Experimental: Carfilzomib IV at dose: 56 mg/m2
The participants will receive Carfilzomib IV at dose: 56 mg/m2 on days -9 and -8 over 30 minutes. The participant will receive Busulfan IV over 3 hours every 24 hours for a total of 4 doses from Day -6 to Day -3 and Melphalan IV over 15-30 minutes for one dose on day -3.The participants will receive stem cell infusion on Day 0 and G-CSF given daily until engraftment occurs.
|
Drug: Carfilzomib
Carfilzomib is an anti-cancer drug acting as a selective proteasome inhibitor that is used to treat Multiple Myeloma.
Other Name: Kyprolis Drug: Busulfan IV Busulfan is an anti-cancer drug acting as a bifunctional alkylating agent that is used to treat Multiple Myeloma.
Other Name: Busulfex Drug: Melphalan IV Melphalan is an anti-cancer drug acting as alkylating agent that is used to treat Multiple Myeloma.
Other Name: Alkeran |
- 36 participants evaluated for safety with treatment-related adverse events and grading by using CTCAE v4.0. [ Time Frame: 3 years ]To determine the maximal tolerated dose of carfilzomib when added to busulfan and melphalan as a preparative regimen for high dose therapy with autologous hematopoietic transplantation for patients with multiply myeloma.
- 36 participants evaluated for tolerability with treatment-related adverse events and grading by using CTCAE v4.0. [ Time Frame: 3 years ]To determine the maximal tolerated dose of carfilzomib when added to busulfan and melphalan as a preparative regimen for high dose therapy with autologous hematopoietic transplantation for patients with multiply myeloma.
- 36 participants evaluated for response to treatment by testing blood for multiple myeloma levels. [ Time Frame: 100 days ]To evaluate complete, very good partial, partial and stable disease response rate for both clinical and biologic endpoints.
- 36 participants evaluated for progression by testing blood for multiple myeloma levels. [ Time Frame: 3 years ]Progression Free Survival
- 36 participants evaluated for overall survival by clinical visit or contact by phone. [ Time Frame: 3 years ]Overall Survival
- 36 participants evaluated for absolute neutrophil count by testing white blood cells levels. [ Time Frame: 100 days ]Days to neutrophil engraftment: Absolute neutrophil count > 500/microliter
- 36 participants evaluated for platelet engraftment by testing platelet count in blood cells. [ Time Frame: 100 days ]Days to platelet engraftment: platelet count > 20,000/microliter untransfused
- 36 participants evaluated by oral exam to assess mucositis events and grading levels by using CTCAE v4.0. [ Time Frame: 100 days ]Mucositis: CTCAE v 4.0 grade and severity
- 36 participants evaluated by the liver to assess Veno-occlusive disease and grading levels by using CTCAE v4.0. [ Time Frame: 100 days ]Veno-occlusive disease
- 36 participants evaluated by a physical exam to assess peripheral neuropathy and grading by using CTCAE v4.0. [ Time Frame: 3 years ]Peripheral neuropathy greater than or equal to CTCAE V 4.0 Grade 3
- 36 participants evaluated for response to treatment by testing urine for multiple myeloma levels. [ Time Frame: 100 days ]To evaluate complete, very good partial, partial no stable disease response rate for both clinical and biologic endpoints
- 36 participants evaluated for progression by testing urine for multiple myeloma levels. [ Time Frame: 3 years ]Progression Free Survival

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Participants must be greater than or equal to 18 years of age.
- Participants must have been diagnosed with multiple myeloma in a first or subsequent remission and have undergone a successful pre-transplant work up and are otherwise eligible for an autotransplant with a busulfan/melphalan preparative regimen at Loyola University Medical Center.
- Participants may receive this preparative regimen if in first or subsequent remission. Participants may enter if they have received a prior autologous stem cell transplant and this therapy produced a remission that lasted greater than 18 months before progression of disease. Participants who have undergone prior allogeneic transplantation are excluded.
- All participants must have responsive disease as defined by a Partial Response or greater to most recent conventional regimen.
- Participants receiving prior carfilzomib will be eligible for inclusion provided they demonstrated responsive disease to this agent and either had a remission that lasted greater than 6 months after its discontinuance, or if in remission after a carfilzomib containing regimen administered to qualify for transplant.
- Participants must have an Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) less than or equal to 2.
- Acceptable heart function test.
Exclusion Criteria:
- Participants must not have below normal kidney function.
- Participants must not have below normal liver function.
- Participants must not have active bacterial, fungal, or viral infection.
- Participants must not have severe lung function.
- Participants must not have Grade 2 or greater peripheral neuropathy.
- Participants must not have uncontrolled hypertension.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03795597
Contact: Patrick Stiff, MD | 708-327-3148 | mailto:pstiff@lumc.edu | |
Contact: Mary Lee, RN | 708-327-2241 | mailto:mlee@luc.edu |
United States, Illinois | |
Loyola University Medical Center | Recruiting |
Maywood, Illinois, United States, 60153 | |
Contact: Patrick Stiff, MD 708-327-3148 pstiff@lumc.edu | |
Contact: Mary Lee, RN 708-327-2241 mlee@luc.edu |
Principal Investigator: | Patrick Stiff, MD | Loyola University |
Responsible Party: | Patrick Stiff, Principal Investigator, Loyola University |
ClinicalTrials.gov Identifier: | NCT03795597 |
Other Study ID Numbers: |
209274 |
First Posted: | January 8, 2019 Key Record Dates |
Last Update Posted: | August 20, 2019 |
Last Verified: | August 2019 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Multiple Myeloma Autologous Stem Cell Transplantation Bone Marrow Transplant Carfilzomib |
Multiple Myeloma Neoplasms, Plasma Cell Neoplasms by Histologic Type Neoplasms Hemostatic Disorders Vascular Diseases Cardiovascular Diseases Paraproteinemias Blood Protein Disorders Hematologic Diseases Hemorrhagic Disorders Lymphoproliferative Disorders |
Immunoproliferative Disorders Immune System Diseases Melphalan Busulfan Antineoplastic Agents, Alkylating Alkylating Agents Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Myeloablative Agonists Immunosuppressive Agents Immunologic Factors Physiological Effects of Drugs |