POEMS SyndromePhase 2NCT04396496
Treatment of POEMS Syndrome With Daratumumab
A Phase 2 study testing Daratumumab Injection in people with POEMS Syndrome, run by University of Arkansas at 1 site in United States.
Trial summary
This trial investigates the use of Daratumumab (DARA), an antibody directed at the human cluster of differentiation 38 (CD38) molecule, for the treatment of patients with Polyneuropathy, Organomegaly, Endocrinopathy, m Protein Component, Skin Changes (POEMS) syndrome. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- POEMS Syndrome
- What is being tested?
- Daratumumab Injection
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 1 siteUnited States
- Study dates
- 22 Sep 2020to 16 Oct 2023
- 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
- Patients must have POEMS syndrome and meet the diagnostic criteria for POEMS syndrome as described in Appendix I.
- Both newly-diagnosed and relapsed POEMS syndrome will be eligible for inclusion.
- Patients must have a platelet count of ≥ 50,000/μL.
- Patients must be at least 18 years of age.
- Participants must have preserved renal function as defined by a serum creatinine level of < 3 mg/dL.
- Participants must have an ejection fraction by echocardiogram (ECHO) or multigated acquisition (MUGA) scan ≥ 40 percent.
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- Eastern Cooperative Oncology Group ≥ 1,
- Overall Neuropathy Limitations Scale (ONLS) ≥ 1.
- Patients must have signed an institutional review board (IRB)-approved informed consent indicating their understanding of the proposed treatment and understanding that the protocol has been approved by the IRB.
Who cannot join
- Documented allergy to lenalidomide, DARA, mannitol, other monoclonal antibodies or human proteins and mammalian-derived products.
- Prior treatment with DARA or other CD38 monoclonal antibodies
- Patients with central nervous system (CNS) Multiple Myeloma (MM) involvement.
- Patients who have received an investigational drug or device within 4 weeks prior to enrollment or received live attenuated vaccine within 4 weeks prior to enrollment.
- Poorly controlled hypertension, diabetes mellitus, or other serious medical illness or psychiatric illness that could potentially interfere with the completion of treatment according to this protocol.
- Poor performance status will not be an exclusion criterion since POEMS patients can be expected to have significant limitations.
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- Patients must not have prior malignancy, except for adequately treated basal cell or squamous cell skin cancer, in situ cervical cancer, or other cancer for which the patient has not received treatment for one year prior to enrollment. Other cancers will only be accepted if the patient's life expectancy exceeds five years.
- Male and Female subjects and their partners of reproductive potential may not participate unless they have agreed to use an effective contraceptive while on study and for 3 months after cessation of DARA.
- Males who are unwilling to abstain from sperm donation while on study and for 3 months after cessation of DARA.
- Females of childbearing potential must have a negative pregnancy test documented within one week of registration.
- Females who are pregnant or nursing women may not participate.
- Patients with POEMS syndrome, who do not have disseminated bone marrow (BM) involvement and have an isolated plasmacytoma; these patients should be considered for irradiation.
- Subjects has had major surgery within 2 weeks prior to enrollment.
- Clinically significant cardiac disease, including myocardial infarction within the past 6 months or unstable or uncontrolled conditions (e.g., unstable angina or congestive heart failure) or other cardiac disease which in the opinion of the investigator would constitute a hazard for participating in the study. Some cardiac dysfunction is expected in this population.
- Participant has known chronic obstructive pulmonary disease (COPD) [defined as a forced expiratory volume in 1 second (FEV1) <50% of predicted normal, forced vital capacity (FVC), etc.] and diffusion capacity (DLCO) < 40% of predicted.), known moderate or severe persistent asthma within the last 2 years or currently has uncontrolled asthma of any classification (controlled intermittent asthma or controlled mild persistent asthma is allowed). Exception may be granted if the principal investigator documents that the patient is a candidate for therapy since alternative therapies will at least have similar or not more toxicity.
- Participant with known or suspected COPD must have an FEV1 test during screening.
- Subjects who are seropositive for human immunodeficiency virus (HIV).
- Subjects who are seropositive for hepatitis B [defined by a positive test for hepatitis B surface antigen (HBsAg)]. Subjects with resolved infection [i.e., subjects who are HBsAg negative but positive for antibodies to hepatitis B core antigen (anti-HBc)] and/or antibodies to hepatitis B surface antigen (anti-HBs) must be screened using real-time polymerase chain reaction (PCR) measurement of hepatitis B virus (HBV) DNA levels. Those who are PCR positive will be excluded. EXCEPTION: Subjects with serologic findings suggestive of HBV vaccination (anti-HBs positivity as the only serologic marker) AND a known history of prior HBV vaccination, do not need to be tested for HBV DNA by PCR.
- Subjects who are seropositive for hepatitis C except in the setting of a sustained virologic response (SVR), defined as aviremia at least 12 weeks after completion of antiviral therapy).
Trial locations and contacts
1 site in 1 country.
United States1 site
University of Arkansas for Medical Sciences
Little Rock, Arkansas, 72205
About this study
Official title: A Phase II Trial for the Treatment of POEMS Syndrome With Daratumumab
This trial investigates the use of Daratumumab (DARA), an antibody directed at the human cluster of differentiation 38 (CD38) molecule, for the treatment of patients with Polyneuropathy, Organomegaly, Endocrinopathy, m Protein Component, Skin Changes (POEMS) syndrome. This trial will enroll ten subjects, who will complete 12 four-week cycles of DARA, in combination with the immunomodulatory drug (IMiD) lenalidomide. Objectives of this study include improvement in neuropathy and performance status, as well as improvement in laboratory values and survival.
Read the detailed description
This trial investigates the use of DARA, an antibody directed at the human CD38 molecule, for the treatment of patients with POEMS syndrome. This trial will enroll ten subjects, who will complete 12 four-week cycles of DARA, in combination with the IMiD lenalidomide. Objectives of this study include improvement in neuropathy and performance status, as well as improvement in laboratory values and survival.
Patients will receive DARA in the outpatient setting, with infusions being administered in Infusion 4. Safety and response assessments will occur prior to Cycles 1 through 6, and at the end of DARA treatment. Once DARA is completed, response assessments are completed every 6 months for the first year, then annually until disease progression. Subjects will also be followed for overall survival.
- Conditions
- POEMS Syndrome
- Being tested
- Daratumumab Injection
- Medical subject headings
- POEMS Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Daratumumab InjectionDrug
Also called Darzalex
DARA is a human monoclonal antibody that specifically recognizes the CD38 molecule that is expressed at a high level in a variety of hematological malignancies, including myeloma cells.
DARA injection will be given with oral lenalidomide for up to 12 four week cycles.
| Study group | Role | What they receive |
|---|---|---|
| Daratumumab Injection | Experimental | Up to 12 four-week cycles of Daratumumab (DARA), in combination with the immunomodulatory drug (IMiD) lenalidomide.
DARA is injected. Dosage calculated by weight.On Cycles 1 and 2, DARA is given on Days 1, 8,15 and 22. On Cycles 3-6,DARA is given on Days 1 and 15. On Cycles 7-12, DARA is given on Day 1.
Lenalidomide is taken by mouth. 15 mg on Days 1-21 of each cycle. Receives: Drug: Daratumumab Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment Success
There is a one-point improvement in ECOG performance score; and/or ≥ 50% reduction in ONLS score at Day 360.
Measured: 360 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2020
- Study start22 Sep 2020
- Primary completion29 Aug 2023
- Study completion16 Oct 2023
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 POEMS SyndromeStudies of the same condition, open and under way.
- More trials run by University of ArkansasThe 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
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Arkansas
- Sponsor type
- University, hospital or other
- Collaborators
- Janssen Scientific Affairs, LLC
- Responsible party
- Sponsor
- Investigators
- Frits van Rhee, MD, Principal investigator, University of Arkansas
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04396496
- Sponsor's protocol ID
- 207442
- Status last verified
- Oct 2023
- First submitted
- 15 May 2020
- Last updated
- 13 Nov 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Trial for the Treatment of POEMS Syndrome With Daratumumab
- ClinicalTrials.gov ID
- NCT04396496
- Acronym / sponsor ID
- 207442
- Conditions
- POEMS Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- University of ArkansasUniversity, hospital or other
- Collaborators
- Janssen Scientific Affairs, LLC
- Study start
- 22 Sep 2020 (actual)
- Primary completion
- 29 Aug 2023 (actual)
- Study completion
- 16 Oct 2023 (actual)
- First posted
- 20 May 2020 (actual)
- Results
- Posted 13 Nov 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This trial investigates the use of Daratumumab (DARA), an antibody directed at the human cluster of differentiation 38 (CD38) molecule, for the treatment of patients with Polyneuropathy, Organomegaly, Endocrinopathy, m Protein Component, Skin Changes (POEMS) syndrome. This trial will enroll ten subjects, who will complete 12 four-week cycles of DARA, in combination with the immunomodulatory drug (IMiD) lenalidomide. Objectives of this study include improvement in neuropathy and performance status, as well as improvement in laboratory values and survival.
Detailed description
This trial investigates the use of DARA, an antibody directed at the human CD38 molecule, for the treatment of patients with POEMS syndrome. This trial will enroll ten subjects, who will complete 12 four-week cycles of DARA, in combination with the IMiD lenalidomide. Objectives of this study include improvement in neuropathy and performance status, as well as improvement in laboratory values and survival.
Patients will receive DARA in the outpatient setting, with infusions being administered in Infusion 4. Safety and response assessments will occur prior to Cycles 1 through 6, and at the end of DARA treatment. Once DARA is completed, response assessments are completed every 6 months for the first year, then annually until disease progression. Subjects will also be followed for overall survival.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Daratumumab Injection | Experimental | Drug: Daratumumab InjectionUp to 12 four-week cycles of Daratumumab (DARA), in combination with the immunomodulatory drug (IMiD) lenalidomide. DARA is injected. Dosage calculated by weight.On Cycles 1 and 2, DARA is given on Days 1, 8,15 and 22. On Cycles 3-6,DARA is given on Days 1 and 15. On Cycles 7-12, DARA is given on Day 1. Lenalidomide is taken by mouth. 15 mg on Days 1-21 of each cycle. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Patients must have POEMS syndrome and meet the diagnostic criteria for POEMS syndrome as described in Appendix I.
- Both newly-diagnosed and relapsed POEMS syndrome will be eligible for inclusion.
- Patients must have a platelet count of ≥ 50,000/μL.
- Patients must be at least 18 years of age.
- Participants must have preserved renal function as defined by a serum creatinine level of < 3 mg/dL.
Key exclusion criteria (19 listed)
- Documented allergy to lenalidomide, DARA, mannitol, other monoclonal antibodies or human proteins and mammalian-derived products.
- Prior treatment with DARA or other CD38 monoclonal antibodies
- Patients with central nervous system (CNS) Multiple Myeloma (MM) involvement.
- Patients who have received an investigational drug or device within 4 weeks prior to enrollment or received live attenuated vaccine within 4 weeks prior to enrollment.
- Poorly controlled hypertension, diabetes mellitus, or other serious medical illness or psychiatric illness that could potentially interfere with the completion of treatment according to this protocol.
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 |
|---|---|---|
| Daratumumab InjectionAlso: DarzalexDARA is a human monoclonal antibody that specifically recognizes the CD38 molecule that is expressed at a high level in a variety of hematological malignancies, including myeloma cells. DARA injection will be given with oral lenalidomide for up to 12 four week cycles. | Drug | Daratumumab Injection |
Primary outcome
Treatment Success
Time frame: 360 days
There is a one-point improvement in ECOG performance score; and/or ≥ 50% reduction in ONLS score at Day 360.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Nov 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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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.
Frits van Rhee, MD principal investigator
University of Arkansas
1 site 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 Injection in people with POEMS Syndrome, run by University of Arkansas 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 9 inclusion and 19 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: finished, as of the sponsor's last update on 13 Nov 2023. 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 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 13 Nov 2023). 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?
Treatment Success (time frame: 360 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 13 Nov 2023.