Recurrent Plasma Cell MyelomaPhase 1NCT05391750
Venetoclax and Tocilizumab for the Treatment of Patients With Relapsed or Refractory t(11;14) Multiple Myeloma
A Phase 1 study testing Tocilizumab and Venetoclax in people with Recurrent Plasma Cell Myeloma and Refractory Plasma Cell Myeloma, run by Emory University at 1 site in United States.
Trial summary
This phase I trial finds out the best dose and side effects of venetoclax and tocilizumab in treating patients with t(11;14) multiple myeloma that has come back (relapsed) or does not respond to treatment (refractory). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Recurrent Plasma Cell Myeloma, Refractory Plasma Cell Myeloma
- What is being tested?
- Tocilizumab and Venetoclax
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- 19 Oct 2022to 12 Feb 2028
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
None listed.
Who cannot join
- Subject exhibits evidence of other clinically significant uncontrolled condition(s), including, but not limited to:
- Acute infection within 14 days prior to first dose of study drug requiring antibiotic, antifungal, or antiviral therapy
- Diagnosis of fever and neutropenia within 1 week prior to first dose of study drug
- Subject has a cardiovascular disability status of New York Heart Association class >= 3
- Subject has a significant history of renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, cardiovascular or hepatic disease within the last 6 months that, in the opinion of the investigator, would adversely affect his/her participation in the study
- Subject has a history of other active malignancies other than multiple myeloma within the past 3 years prior to study entry, with the following exceptions:
Show all 23Show fewer
- Adequately treated in situ carcinoma of the cervix uteri,
- Basal cell carcinoma of the skin or localized squamous cell carcinoma of the skin,
- Localized prostate cancer Gleason grade 6 or lower AND with stable prostate specific antigen (PSA) levels off treatment
- Previous malignancy confined and surgically resected (or treated with other modalities) with curative intent
- Known human immunodeficiency viral (HIV) infection
- Active hepatitis B or C infection based on screening blood testing
- Subject is receiving other ongoing anti-myeloma therapy
- Subject has received any of the following within 7 days prior to the first dose of study drug:
- Strong or moderate CYP3A inhibitors, or
- Strong or moderate CYP3A inducers
- Subject has received any of the following within 14 days prior to the first dose of study drug or has not recovered to less than a grade 2 clinically significant adverse effect(s)/toxicity(s) of the previous therapy: any anti-myeloma therapy including chemotherapy, radiotherapy, or investigational therapy, including targeted small molecule agents
- Subject has received prior treatment with a BCL-2 family inhibitor
- Subject is pregnant, parturient, or breastfeeding; deprived of freedom by judicial or administrative decision; hospitalized and unable to provide consent, or otherwise unable to provide consent
- Subject has consumed grapefruit, grapefruit products, Seville oranges (including marmalade containing Seville oranges) or star fruit within 3 days prior to the first dose of study drug
- Subject has received immunization with live vaccine within 60 days of dosing
- Recent corticosteroid therapy at a cumulative dose equivalent to > 140 mg of prednisone or a single dose equivalent to >= 40 mg of dexamethasone within 2 weeks prior to the first dose of study drug
- Subject's decision to not divulge the race
Trial locations and contacts
1 site in 1 country.
United States1 site
Emory University Hospital/Winship Cancer Institute
Atlanta, Georgia, 30322
About this study
Official title: A Phase 1 Study Evaluating the Safety of Venetoclax and Tocilizumab in African American and Non-African American Subjects With Relapsed or Refractory t(11;14) Multiple Myeloma
This phase I trial finds out the best dose and side effects of venetoclax and tocilizumab in treating patients with t(11;14) multiple myeloma that has come back (relapsed) or does not respond to treatment (refractory). Venetoclax may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Tocilizumab is a monoclonal antibody that may interfere with the ability of tumor cells to grow and spread. Tocilizumab is used to treat side effects from immune therapy in patients with myeloma. Giving venetoclax and tocilizumab may kill more cancer cells.
Read the detailed description
PRIMARY OBJECTIVE:
- To determine the dose limiting toxicity (DLT), safety profile, and the recommended phase 2 dose (RPTD) of venetoclax and tocilizumab when administered in subjects with relapsed and recurrent (RR) multiple myeloma t(11;14) (MM).
SECONDARY OBJECTIVES:
I.To evaluate the preliminary efficacy data regarding the effect of venetoclax and tocilizumab by objective response rate per IMWG criteria.
II. To evaluate the effect of chronic tocilizumab administration on venetoclax exposure.
TERTIARY/EXPLORATORY OBJECTIVES:
- To evaluate the preliminary efficacy data regarding the effect of venetoclax and tocilizumab by time to response (TTR), time to disease progression (TTP), duration of response (DOR), progression free survival (PFS) and overall survival (OS).
II. To measure the effect of IL6 receptor blockade on ex vivo venetoclax sensitivity.
III. To evaluate the cell populations in the bone marrow of responders versus non-responders as well as the effect of IL6 receptor blockade on those populations.
IV. To evaluate the expression of B cell markers on venetoclax sensitive myeloma.
- To determine the expression of key BCL2 family members with and without IL6 receptor blockade.
VI. To correlate differences in somatic mutations, structural alterations, gene expression and chromatin accessibility with venetoclax response.
OUTLINE: This is a dose-escalation study of venetoclax and tocilizumab.
Patients receive tocilizumab intravenously (IV) on day -7 of cycle 1, and on day 1 of subsequent cycles. Patients also receive venetoclax orally (PO) on days 1-21. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up for 4 weeks, then every 6 months thereafter.
- Being tested
- Tocilizumab, Venetoclax
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TocilizumabBiological
Also called Actemra, Immunoglobulin G1, Anti-(Human Interleukin 6 Receptor) (Human-Mouse Monoclonal MRA Heavy Chain), Disulfide with Human-Mouse Monoclonal MRA Kappa-Chain, Dimer, MRA, R-1569
Given IV
VenetoclaxDrug
Also called ABT-0199, ABT-199, ABT199, GDC-0199
Given PO
| Study group | Role | What they receive |
|---|---|---|
| Treatment (venetoclax, tocilizumab) | Experimental | Patients receive tocilizumab IV on day -7 of cycle 1, and on day 1 of subsequent cycles. Patients also receive venetoclax PO on days 1-21. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. Receives: Biological: Tocilizumab; Drug: Venetoclax |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum tolerated dose (MTD)
The MTD for each arm is selected based on isotonic regression27, using the shiny app BOINComb (http://www.trialdesign.org). The dose is selected as the MTD for which the isotonic estimate of the toxicity rate is closest to the target toxicity rate. If there are ties, the higher dose level is selected when the isotonic estimate is lower than the target toxicity rate; the lower dose level is selected when the isotonic estimate is greater than or equal to the target toxicity rate.
Measured: Completion of cycle 1 (each cycle is 21 days)
Secondary outcomes (8)
Incidence of adverse events (AEs)
AEs, related AEs, serious (S)AEs, related SAEs, and AEs leading to interruptions and/or discontinuation of study drug will be analyzed descriptively, using frequencies and percentages.
Measured: Up to 30 days post-treatment
Overall response rate (ORR)
ORR will be calculated with an exact 95% confidence interval (CI) for each arm, using the Clopper-Pearson method.
Measured: Up to 5 years
Complete response rate
Complete response rate will be calculated with an exact 95% CI for each arm, using the Clopper-Pearson method.
Measured: Up to 5 years
Time to response (TTR)
TTR will be estimated using the Kaplan-Meier method. Median TTR will be reported, along with a 95% confidence interval estimated using the Brookmeyer-Crowley method.
Measured: From date of receipt of study treatment to date of treatment response, where those not responding are censored at date of last follow-up or death from any cause, assessed up to 5 years
Time to disease progression (TTP)
TTP will be estimated using the Kaplan-Meier method. Median TTP will be reported, along with a 95% confidence interval estimated using the Brookmeyer-Crowley method.
Measured: From date of receipt of study treatment to date of disease progression, where those not progressing or have died are censored at date of last follow-up or death from any cause, assessed up to 5 years
Duration of response (DOR)
Median DOR will be reported, along with the 25th and 75th percentiles.
Measured: From date of treatment response to date of disease progression, assessed up to 5 years
Progression-free survival (PFS)
PFS will be estimated using the Kaplan-Meier method. Median PFS will be reported, along with a 95% confidence interval estimated using the Brookmeyer-Crowley method.
Measured: From date of receipt of study treatment to date of disease progression or death from any cause, where those alive without are censored at date of last follow-up, assessed up to 5 years
Overall survival (OS)
OS will be estimated using the Kaplan-Meier method. Median OS will be reported, along with a 95% confidence interval estimated using the Brookmeyer-Crowley method.
Measured: From date of receipt of study treatment to date of death from any cause, where those alive without are censored at date of last follow-up, assessed up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 May 2022
- Study start19 Oct 2022
- Primary completion12 Feb 2027 (estimated)
- Study completion12 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Recurrent Plasma Cell MyelomaStudies of the same condition, open and under way.
- More trials run by Emory UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Lead sponsor
- Emory University
- Sponsor type
- University, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Principal investigator, Jonathan Kaufman, Emory University
- Investigators
- Jonathan L. Kaufman, MD, Principal investigator, Emory University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05391750
- Sponsor's protocol ID
- STUDY00002448
- Other IDs
- NCI-2021-02510, STUDY00002448, WINSHIP5273-21, P30CA138292
- Status last verified
- Feb 2026
- First submitted
- 13 Apr 2022
- Last updated
- 17 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Study Evaluating the Safety of Venetoclax and Tocilizumab in African American and Non-African American Subjects With Relapsed or Refractory t(11;14) Multiple Myeloma
- ClinicalTrials.gov ID
- NCT05391750
- Acronym / sponsor ID
- STUDY00002448
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 7 participants (actual)
- Lead sponsor
- Emory UniversityUniversity, hospital or other
- Collaborators
- National Cancer Institute (NCI)
- Study start
- 19 Oct 2022 (actual)
- Primary completion
- 12 Feb 2027 (estimated)
- Study completion
- 12 Feb 2028 (estimated)
- First posted
- 26 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This phase I trial finds out the best dose and side effects of venetoclax and tocilizumab in treating patients with t(11;14) multiple myeloma that has come back (relapsed) or does not respond to treatment (refractory). Venetoclax may stop the growth of cancer cells by blocking Bcl-2, a protein needed for cancer cell survival. Tocilizumab is a monoclonal antibody that may interfere with the ability of tumor cells to grow and spread. Tocilizumab is used to treat side effects from immune therapy in patients with myeloma. Giving venetoclax and tocilizumab may kill more cancer cells.
Detailed description
PRIMARY OBJECTIVE:
- To determine the dose limiting toxicity (DLT), safety profile, and the recommended phase 2 dose (RPTD) of venetoclax and tocilizumab when administered in subjects with relapsed and recurrent (RR) multiple myeloma t(11;14) (MM).
SECONDARY OBJECTIVES:
I.To evaluate the preliminary efficacy data regarding the effect of venetoclax and tocilizumab by objective response rate per IMWG criteria.
II. To evaluate the effect of chronic tocilizumab administration on venetoclax exposure.
TERTIARY/EXPLORATORY OBJECTIVES:
- To evaluate the preliminary efficacy data regarding the effect of venetoclax and tocilizumab by time to response (TTR), time to disease progression (TTP), duration of response (DOR), progression free survival (PFS) and overall survival (OS).
II. To measure the effect of IL6 receptor blockade on ex vivo venetoclax sensitivity.
III. To evaluate the cell populations in the bone marrow of responders versus non-responders as well as the effect of IL6 receptor blockade on those populations.
IV. To evaluate the expression of B cell markers on venetoclax sensitive myeloma.
- To determine the expression of key BCL2 family members with and without IL6 receptor blockade.
VI. To correlate differences in somatic mutations, structural alterations, gene expression and chromatin accessibility with venetoclax response.
OUTLINE: This is a dose-escalation study of venetoclax and tocilizumab.
Patients receive tocilizumab intravenously (IV) on day -7 of cycle 1, and on day 1 of subsequent cycles. Patients also receive venetoclax orally (PO) on days 1-21. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up for 4 weeks, then every 6 months thereafter.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment (venetoclax, tocilizumab) | Experimental | Biological: Tocilizumab, Drug: VenetoclaxPatients receive tocilizumab IV on day -7 of cycle 1, and on day 1 of subsequent cycles. Patients also receive venetoclax PO on days 1-21. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (0 listed)
Key exclusion criteria (23 listed)
- Subject exhibits evidence of other clinically significant uncontrolled condition(s), including, but not limited to:
- Acute infection within 14 days prior to first dose of study drug requiring antibiotic, antifungal, or antiviral therapy
- Diagnosis of fever and neutropenia within 1 week prior to first dose of study drug
- Subject has a cardiovascular disability status of New York Heart Association class >= 3
- Subject has a significant history of renal, neurologic, psychiatric, endocrinologic, metabolic, immunologic, cardiovascular or hepatic disease within the last 6 months that, in the opinion of the investigator, would adversely affect his/her participation in the study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TocilizumabAlso: Actemra, Immunoglobulin G1, Anti-(Human Interleukin 6 Receptor) (Human-Mouse Monoclonal MRA Heavy Chain), Disulfide with Human-Mouse Monoclonal MRA Kappa-Chain, Dimer, MRA, R-1569Given IV | Biological | Treatment (venetoclax, tocilizumab) |
| VenetoclaxAlso: ABT-0199, ABT-199, ABT199, GDC-0199Given PO | Drug | Treatment (venetoclax, tocilizumab) |
Primary outcome
Maximum tolerated dose (MTD)
Time frame: Completion of cycle 1 (each cycle is 21 days)
The MTD for each arm is selected based on isotonic regression27, using the shiny app BOINComb (http://www.trialdesign.org). The dose is selected as the MTD for which the isotonic estimate of the toxicity rate is closest to the target toxicity rate. If there are ties, the higher dose level is selected when the isotonic estimate is lower than the target toxicity rate; the lower dose level is selected when the isotonic estimate is greater than or equal to the target toxicity rate.
8 secondary outcomes
Incidence of adverse events (AEs)
Time frame: Up to 30 days post-treatment
Overall response rate (ORR)
Time frame: Up to 5 years
Complete response rate
Time frame: Up to 5 years
Time to response (TTR)
Time frame: From date of receipt of study treatment to date of treatment response, where those not responding are censored at date of last follow-up or death from any cause, assessed up to 5 years
Time to disease progression (TTP)
Time frame: From date of receipt of study treatment to date of disease progression, where those not progressing or have died are censored at date of last follow-up or death from any cause, assessed up to 5 years
Duration of response (DOR)
Time frame: From date of treatment response to date of disease progression, assessed up to 5 years
Progression-free survival (PFS)
Time frame: From date of receipt of study treatment to date of disease progression or death from any cause, where those alive without are censored at date of last follow-up, assessed up to 5 years
Overall survival (OS)
Time frame: From date of receipt of study treatment to date of death from any cause, where those alive without are censored at date of last follow-up, assessed up to 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Feb 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jonathan L. Kaufman, MD principal investigator
Emory University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Tocilizumab and Venetoclax in people with Recurrent Plasma Cell Myeloma and Refractory Plasma Cell Myeloma, run by Emory University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 0 inclusion and 23 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 17 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Maximum tolerated dose (MTD) (time frame: Completion of cycle 1 (each cycle is 21 days)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 17 Feb 2026.