Monoclonal GammopathyPhase 2NCT03236428
Phase II Study of the CD38 Antibody Daratumumab in Patients With High-Risk MGUS and Low-Risk Smoldering Multiple Myeloma
A Phase 2 study testing Daratumumab in people with Monoclonal Gammopathy and Smoldering Multiple Myeloma, run by Dana-Farber Cancer Institute at 6 sites in United States.
Trial summary
This research study is studying a drug as a possible treatment for Monoclonal Gammopathy of Unknown Significance (MGUS) or Smoldering Multiple Myeloma (SMM). The drug involved in this study is: -Daratumumab Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Monoclonal Gammopathy, Smoldering Multiple Myeloma
- What is being tested?
- Daratumumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 6 sitesUnited States
- Study dates
- 24 Nov 2017to 4 Oct 2026
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Age ≥ 18 years
- Must meet criteria for high-risk MGUS or low-risk smoldering myeloma as described below:
- High-Risk MGUS
- Must have <10% plasma cells and <3.0g/dL M-spike and at least 2 of the following 3 criteria:
- Abnormal free light-chain (FLC) ratio (<0.26 or >1.65)
- M-protein concentration (≥1.5 g/dL)
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- Non-IgG M protein (including IgA)
- Low-Risk Smoldering Multiple Myeloma
- Must only present with 1 of the following criterion:
- Monoclonal Protein ≥ 3 g/dL
- ---≥ 10% Bone Marrow Plasma Cells
- FLC ratio < 0.125 or > 8
- -No evidence of CRAB criteria† or new criteria of active MM which including the following:
- Increased calcium levels (corrected serum calcium >0.25 mmol/dL above the upper limit of normal or >0.275 mmol/dL)
- Renal insufficiency (attributable to myeloma)
- Anemia (Hb 2 g/dL below the lower limit of normal or <10 g/dL)
- Bone lesions (lytic lesions or generalized osteoporosis with compression fractures)
- No evidence of the following new criteria for active MM including the following: Bone marrow plasma cells >60%, Serum involved/uninvolved FLC ratio ≥100, and MRI with more than one focal lesion
- Participants with CRAB criteria that are attributable to conditions other than the disease under study may be eligible
- ECOG Performance Status (PS) 0, 1, or 2 (Appendix A)
- The following laboratory values obtained ≤ 21 days prior to registration:
- ANC ≥ 1000/uL
- PLT ≥ 50,000/uL
- Total bilirubin ≤ 2.0 mg/dL (If total is elevated check direct and if normal patient is eligible.)
- AST ≤ 3 x institutional upper limit of normal (ULN)
- ALT ≤ 3 x institutional upper limit of normal (ULN)
- Creatinine ≤ 2 mg/dL or Creatinine Clearance ≥ 40 mL/min
- Ability to understand and the willingness to sign an informed consent before performance of any study-related procedure not part of normal medical care, with the understanding that consent may be withdrawn by the subject at any time without prejudice to future medical care.
- Female patients who are postmenopausal for at least 1 year before the screening visit or are surgically sterile are eligible. Females of childbearing potential (as defined below) may also be eligible but must have a negative serum or urine pregnancy test with a sensitivity of at least 25 mIU/mL within 21 days of registration.
- A female of childbearing potential is a sexually mature female who:
- Has not undergone a hysterectomy (the surgical removal of the uterus) or bilateral oophorectomy (the surgical removal of both ovaries)
- OR
- ---Has not been naturally postmenopausal (amenorrhea following cancer therapy does not rule out childbearing potential) for at least 24 consecutive months (i.e., has had menses at any time during the preceding 24 consecutive months)
Who cannot join
- Any prior therapy for symptomatic Multiple Myeloma or smoldering Multiple Myeloma should also be excluded, including prior use of IMIDs, proteasome inhibitors, or CD138 inhibitors. Prior therapy for smoldering Multiple Myeloma with agents that are not therapeutically active against MM is not an exclusion criterion.
- Other concurrent chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy considered investigational. Prior therapy with bisphosphonates is allowed. Prior radiation therapy to a solitary plasmacytoma is allowed.
- Concurrent exposure to any commercially available agents known to be active against SMM and MM.
- Serious medical or psychiatric illness likely to interfere with participation in this clinical study.
- Diagnosed or treated for another malignancy within 2 years of enrollment, with the exception of complete resection of basal cell carcinoma or squamous cell carcinoma of the skin, an in situ malignancy, or low-risk prostate cancer after curative therapy.
- Subject has known chronic obstructive pulmonary disease (COPD) with a Forced Expiratory Volume in 1 second (FEV1) < 50% of predicted normal.
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- Note that FEV1 testing is required for patients suspected of having COPD and subjects must be excluded if FEV1 <50% of predicted normal.
- Subject has known moderate or severe persistent asthma within the past 2 years or currently has uncontrolled asthma of any classification.
- Subjects who currently have controlled intermittent asthma or controlled mild persistent asthma are allowed in the study
- Uncontrolled intercurrent illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, uncontrollable cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
- Pregnant or nursing women will be excluded from the study.
- History of allergic reactions attributed to compounds of similar chemical or biologic composition to Daratumumab.
- Known seropositive for or active viral infection with human immunodeficiency virus (HIV), hepatitis B virus (HBV) or hepatitis C virus (HCV). Patients who are seropositive because of hepatitis B virus vaccine are eligible. Patients who are positive for hepatitis B core antibody or hepatitis B surface antigen must have a negative polymerase chain reaction (PCR) result before enrollment. Those who are PCR positive will be excluded.
- Major surgery within 4 weeks before enrollment.
- Subject is known or suspected of not being able to comply with the study protocol (eg, because of alcoholism, drug dependency, or psychological disorder). Subject has any condition for which, in the opinion of the investigator, participation would not be in the best interest of the subject (eg, compromise the well-being) or that could prevent, limit, or confound the protocol-specified assessments.
- Vaccination with live attenuated vaccines within 4 weeks of first study agent administration
- Subject has clinically significant cardiac disease, including significant ischemic coronary disease, congestive heart failure (New York Heart Association [NYHA] Class III or IV), unstable arrhythmias, myocardial infarction or unstable angina within 6 months before randomization, a history of additional risk factors for torsades de pointes (eg, electrolyte abnormalities, family history of Long QT Syndrome), or a family history of sudden cardiac death before age 40.
- Participation in other therapeutic clinical trials, including those with other investigational agents not included in this trial, within 30 days of the start of this trial and throughout the duration of this trial
Trial locations and contacts
6 sites in 1 country.
6 sites in 1 country
United States6 sites
Massachusetts General Hospital
Boston, Massachusetts, 02214
Beth Israel Deaconess Medical Center
Boston, Massachusetts, 02215
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
Colorado Blood Cancer Institute
Denver, Colorado, 80218
Karmanos Cancer Insitute
Detroit, Michigan, 48201
Pacific Cancer Care
Monterey, California, 93940
About this study
Official title: A Phase II Study of the CD38 Antibody Daratumumab in Patients With High-Risk MGUS and Low-Risk Smoldering Multiple Myeloma
This research study is studying a drug as a possible treatment for Monoclonal Gammopathy of Unknown Significance (MGUS) or Smoldering Multiple Myeloma (SMM).
The drug involved in this study is:
-Daratumumab
Read the detailed description
This research study is a Phase II clinical trial, which tests the effectiveness of an investigational drug. Preliminary experience suggests that daratumumab may prevent or postpone SMM from becoming active multiple myeloma. The purpose of this research study is to determine if the this drug may improve the rate of prevention of multiple myeloma.
Multiple myeloma is a cancer of the plasma cell, which is an important part of the immune system. Patients with active multiple myeloma generally require treatment. There are currently no approved therapies for smoldering multiple myeloma or Monoclonal Gammopathy of Unknown Significance.
Daratumumab is a drug that may kill or stop cancer cells from growing through a variety of mechanisms by attaching to the CD38 molecule, which is over-expressed in multiple myeloma cells. This type of drug is called a monoclonal antibody. The FDA (the U.S. Food and Drug Administration) has not approved Daratumumab for the participant specific disease but it has been approved for use in active Multiple Myeloma.
- Conditions
- Monoclonal Gammopathy, Smoldering Multiple Myeloma
- Being tested
- Daratumumab
- Keywords
- Smoldering Multiple Myeloma, Monoclonal Gammopathy
- Medical subject headings
- Paraproteinemias, Smoldering Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DaratumumabDrug
Also called Darzalex
Daratumumab is a drug that may kill or stop cancer cells from growing through a variety of mechanisms by attaching to the CD38 molecule
| Study group | Role | What they receive |
|---|---|---|
| Daratumumab | Experimental | Daratumumab will be administered by IV infusion weekly during cycles 1 and 2 Daratumumab will be administered by IV infusion every other week during cycles 3 through 6 Daratumumab will be administered by IV infusion monthly during cycles 7 through 20 Receives: Drug: Daratumumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Proportion Of Patients In Deep Response
To determine the proportion of patients who are in VGPR or better after twenty cycles of therapy with Daratumumab. This will be determined at the time of best overall response.
Measured: 2 years
Secondary outcomes (6)
Objective Response Rate
The objective response rate (partial response or better according to the modified IMWG criteria) and the proportion of patients with a MRD, CR, PR or MR will be reported with 90% exact binominal confidence interval (CI). The exact two-sided 90% CI around response rate will be no wider than 28% with 40 eligible patients.
Measured: 2 years
Duration of Response
To estimate the duration of response (time from objective response to disease progression or death, or date last known progression-free and alive for those who have not progressed or died) the Kaplan-Meier method will be used.
Measured: 2 years
Complete Response Rate
The duration of overall CR is progression or death. Patients who have not progressed or died are censored at the date last known progression-free.
Measured: 2 years
Overall Response Rate
The duration of overall response is measured as the time from initiation of first response to first documentation of disease progression or death. Patients who have not progressed or died are censored at the date last known progression-free.
Measured: 2 years
Progression Free Survival
PFS is defined as the time from first-dose to the disease progression or death from any cause. Patients who have not progressed or died are censored at the date last known progression-free.
Measured: 2 years
Incidence of Treatment Emergent Adverse Events (Safety and Tolerability)
Safety analysis will be conducted using the Safety Population defined as any patient receiving one dose of study treatment. For toxicity reporting, all adverse events and laboratory abnormalities will be graded and analyzed using CTCAE version 4 as appropriate.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Aug 2017
- Study start24 Nov 2017
- Primary completion4 Oct 2021
- Study completion4 Oct 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Monoclonal GammopathyStudies of the same condition, open and under way.
- More trials run by Dana-Farber Cancer InstituteThe 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 42 participants (actual)
Sponsor and investigators
- Lead sponsor
- Dana-Farber Cancer Institute
- Sponsor type
- University, hospital or other
- Collaborators
- Janssen Pharmaceuticals, Multiple Myeloma Research Consortium, Blood Cancer Research Partnership, The Leukemia and Lymphoma Society
- Responsible party
- Principal investigator, Irene Ghobrial, MD, Dana-Farber Cancer Institute
- Investigators
- Irene Ghobrial, MD, Principal investigator, Dana-Farber Cancer Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03236428
- Sponsor's protocol ID
- 17-212
- Status last verified
- Mar 2026
- First submitted
- 11 Jul 2017
- Last updated
- 27 Mar 2026
- Results posted
- Yes, on ClinicalTrials.gov
- 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 27 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of the CD38 Antibody Daratumumab in Patients With High-Risk MGUS and Low-Risk Smoldering Multiple Myeloma
- ClinicalTrials.gov ID
- NCT03236428
- Acronym / sponsor ID
- 17-212
- Conditions
- Monoclonal Gammopathy, Smoldering Multiple Myeloma
- Keywords
- Smoldering Multiple Myeloma, Monoclonal Gammopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 42 participants (actual)
- Lead sponsor
- Dana-Farber Cancer InstituteUniversity, hospital or other
- Collaborators
- Janssen Pharmaceuticals, Multiple Myeloma Research Consortium, Blood Cancer Research Partnership, The Leukemia and Lymphoma Society
- Study start
- 24 Nov 2017 (actual)
- Primary completion
- 4 Oct 2021 (actual)
- Study completion
- 4 Oct 2026 (estimated)
- First posted
- 1 Aug 2017 (actual)
- Results
- Posted 27 Mar 2026
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This research study is studying a drug as a possible treatment for Monoclonal Gammopathy of Unknown Significance (MGUS) or Smoldering Multiple Myeloma (SMM).
The drug involved in this study is:
-Daratumumab
Detailed description
This research study is a Phase II clinical trial, which tests the effectiveness of an investigational drug. Preliminary experience suggests that daratumumab may prevent or postpone SMM from becoming active multiple myeloma. The purpose of this research study is to determine if the this drug may improve the rate of prevention of multiple myeloma.
Multiple myeloma is a cancer of the plasma cell, which is an important part of the immune system. Patients with active multiple myeloma generally require treatment. There are currently no approved therapies for smoldering multiple myeloma or Monoclonal Gammopathy of Unknown Significance.
Daratumumab is a drug that may kill or stop cancer cells from growing through a variety of mechanisms by attaching to the CD38 molecule, which is over-expressed in multiple myeloma cells. This type of drug is called a monoclonal antibody. The FDA (the U.S. Food and Drug Administration) has not approved Daratumumab for the participant specific disease but it has been approved for use in active Multiple Myeloma.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Daratumumab | Experimental | Drug: DaratumumabDaratumumab will be administered by IV infusion weekly during cycles 1 and 2 Daratumumab will be administered by IV infusion every other week during cycles 3 through 6 Daratumumab will be administered by IV infusion monthly during cycles 7 through 20 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (33 listed)
- Age ≥ 18 years
- Must meet criteria for high-risk MGUS or low-risk smoldering myeloma as described below:
- High-Risk MGUS
- Must have <10% plasma cells and <3.0g/dL M-spike and at least 2 of the following 3 criteria:
- Abnormal free light-chain (FLC) ratio (<0.26 or >1.65)
Key exclusion criteria (18 listed)
- Any prior therapy for symptomatic Multiple Myeloma or smoldering Multiple Myeloma should also be excluded, including prior use of IMIDs, proteasome inhibitors, or CD138 inhibitors. Prior therapy for smoldering Multiple Myeloma with agents that are not therapeutically active against MM is not an exclusion criterion.
- Other concurrent chemotherapy, immunotherapy, radiotherapy, or any ancillary therapy considered investigational. Prior therapy with bisphosphonates is allowed. Prior radiation therapy to a solitary plasmacytoma is allowed.
- Concurrent exposure to any commercially available agents known to be active against SMM and MM.
- Serious medical or psychiatric illness likely to interfere with participation in this clinical study.
- Diagnosed or treated for another malignancy within 2 years of enrollment, with the exception of complete resection of basal cell carcinoma or squamous cell carcinoma of the skin, an in situ malignancy, or low-risk prostate cancer after curative therapy.
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 |
|---|---|---|
| DaratumumabAlso: DarzalexDaratumumab is a drug that may kill or stop cancer cells from growing through a variety of mechanisms by attaching to the CD38 molecule | Drug | Daratumumab |
Primary outcome
The Proportion Of Patients In Deep Response
Time frame: 2 years
To determine the proportion of patients who are in VGPR or better after twenty cycles of therapy with Daratumumab. This will be determined at the time of best overall response.
6 secondary outcomes
Objective Response Rate
Time frame: 2 years
Duration of Response
Time frame: 2 years
Complete Response Rate
Time frame: 2 years
Overall Response Rate
Time frame: 2 years
Progression Free Survival
Time frame: 2 years
Incidence of Treatment Emergent Adverse Events (Safety and Tolerability)
Time frame: 2 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Mar 2026.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
Irene Ghobrial, MD principal investigator
Dana-Farber Cancer Institute
6 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Daratumumab in people with Monoclonal Gammopathy and Smoldering Multiple Myeloma, run by Dana-Farber Cancer Institute at 6 sites 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 33 inclusion and 18 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 27 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Mar 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
The Proportion Of Patients In Deep Response (time frame: 2 years). 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 27 Mar 2026.