Multiple Myeloma in RelapsePhase 2NCT02176213
Study of Pomalidomide, Cyclophosphamide, Dexamethasone in Relapsed/Refractory Multiple Myeloma
A Phase 2 study testing Pomalidomide, Cyclophosphamide and Dexamethasone in people with Multiple Myeloma in Relapse and Multiple Myeloma, Refractory, run by Ajai Chari at 1 site in United States.
Trial summary
This study is being done to learn more about the drug, pomalidomide and to gather data on its safety and side effects when used in combination with commercially available cyclophosphamide and dexamethasone. This combination is considered experimental and has not been approved by the FDA. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Multiple Myeloma in Relapse, Multiple Myeloma, Refractory
- What is being tested?
- Pomalidomide, Cyclophosphamide and Dexamethasone
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 35 participants
- Where
- 1 siteUnited States
- Study dates
- Jun 2014to 7 May 2019
- Phase
- Phase 2
- Are there results?
- None posted in the registry
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
- Disease related:
- Patients must have a history of symptomatic multiple myeloma according to the IMWG criteria
- Patients must have received at least two prior lines of therapy and also must be refractory to lenalidomide.
- Patient has relapsed or relapsed/refractory MM.
- Patients must currently have measurable disease, as defined as:
- Serum M-protein ≥ 0.5 g/dL
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- Urine M-protein ≥ 200 mg/24 hours
- Serum free light chain assay: involved FLC level ≥ 10 mg/dl provided serum FLC ratio is abnormal
- If no monoclonal protein is detected, then > 30% monoclonal bone marrow plasma cells
- Demographic:
- Male or female adults ≥ 18 years old
- Able to sign informed consent and to comply with the protocol
- Life expectancy > 12 weeks
- ECOG performance status ≤ 2
- All study participants must be registered into the mandatory POMALYST REMS program, and be willing and able to comply with the requirements of the POMALYST REMS program.
- Laboratory
- ANC ≥ 1000/µL
- Platelets ≥ 50,000/µL (Patients with plasma cells 50% of bone marrow nucleated cells, and platelets ≥ 30,000/µL will be permitted regardless of the baseline ANC)
- Cr < 3
- AST ≤ 2.5 x ULN
- ALT ≤ 2.5 x ULN
- Serum Bilirubin ≤ 1.5 x ULN (except patients with Gilbert's syndrome who must have a total bilirubin of <3 time ULN)
- Other
- Females of childbearing potential must have a negative serum or urine pregnancy test with a sensitivity of at least 25 mIU/mL within 10 - 14 days prior to and again within 24 hours of starting pomalidomide and must either commit to continued abstinence from heterosexual intercourse or begin TWO acceptable methods of birth control at least 28 days before taking pomalidomide.
Who cannot join
- Previous treatment with pomalidomide
- Patients who received chemotherapy or radiation therapy to 30% of marrow-bearing bone within ≤ 2 weeks or experimental agent/therapy within 4 weeks prior to starting study treatment; or who have not yet recovered from side effects of such therapies
- Known hypersensitivity to thalidomide or lenalidomide
- The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide, lenalidomide or similar drugs
- Other concurrent severe and/or uncontrolled medical conditions including abnormal laboratory values that could cause unacceptable safety risks or compromise compliance with the protocol
- Patients for whom prophylactic anticoagulation therapy is not an option unless due to thrombocytopenia
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- Patients who received allogenic stem cell transplantation < 12 months prior to entering the study or show evidence of active graft-versus-host disease that requires immunosuppressive therapy
- Patients with existing peripheral neuropathy grade > 2
- Patients with an active malignancy requiring treatment in the next 12 months (except for basal or squamous cell carcinoma, or in situ cancer of the cervix or breast, and asymptomatic prostate cancer)
- Patients with known positivity for HIV or active hepatitis B or C
- Corticosteroid therapies of > 20 mg/day prednisone, > 4 mg/day dexamethasone, > 80 mg/day hydrocortisone, or equivalent. Oral, inhaled, or topical steroids are allowed during study as long as it does not exceed 80 mg/day hydrocortisone.
Trial locations and contacts
1 site in 1 country.
United States1 site
Icahn School of Medicine at Mount Sinai
New York, New York, 10029
About this study
Official title: A Phase II Study of Pomalidomide, Daily Low Dose Oral Cyclophosphamide, and Dexamethasone in Relapsed/Refractory Multiple Myeloma
This study is being done to learn more about the drug, pomalidomide and to gather data on its safety and side effects when used in combination with commercially available cyclophosphamide and dexamethasone. This combination is considered experimental and has not been approved by the FDA.
Pomalidomide is a third generation immunomodulatory (IMiDs) agent, which is a more potent version of thalidomide and lenalidomide drugs that have been approved by the United States Food and Drug Administration [FDA] for the treatment of MM. In February 2013, pomalidomide was also approved by the FDA for patients with MM who have had more than 2 types of therapy.
Pomalidomide is taken orally as capsules, and cyclophosphamide and dexamethasone are also taken orally as tablets in this study. Cyclophosphamide and dexamethasone are commercially available and are often used in combination with other drugs to treat Multiple Myeloma. Preliminary data from both the laboratory and patient studies suggest that this combination of drugs is more effective than pomalidomide and dexamethasone alone. However, the regimen being used in this study, which consists of daily cyclophosphamide, also permits support of low blood counts with either injections or transfusions as needed.
Read the detailed description
This is an open label, single center, phase II study of a combination of pomalidomide, daily low dose oral cyclophosphamide, and dexamethasone in patients with relapsed/refractory multiple myeloma. The three oral drugs will be given in 28-day cycles: Pomalidomide 4 mg daily x 21 days; cyclophosphamide 50 mg BID x 21 days; and dexamethasone 40 mg weekly x 3 (20 mg weekly if the patient aged ≥ 75 years old). Subjects meeting eligibility criteria with ANC < 1000/µL and platelet count < 50,000/µL will start at dose level -1 of both pomalidomide (3 mg daily) and cyclophosphamide (50 mg daily). G-CSF and platelet transfusion support is permitted if needed.
Dose reduction for hematologic toxicity will begin with cyclophosphamide and then subsequently alternate with pomalidomide until a subject cannot tolerate dose level -2 of both agents - in which case subject would come off study. If subject has 2 or more concurrent toxicities that are potentially attributable to both agents (e.g. hematologic toxicity) then dose modification guidelines will be followed with dose reduction being done sequentially with one agent at a time, unless in the opinion of the investigator, both agents required concurrent dose reduction.
- Being tested
- Pomalidomide, Cyclophosphamide, Dexamethasone
- Keywords
- Relapsed, Refractory, Multiple Myeloma, Pomalidomide, Cyclophosphamide, Dexamethasone
- Medical subject headings
- Multiple Myeloma, Recurrence
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PomalidomideDrug
Also called pomalyst
4 mg PO x 21 days
CyclophosphamideDrug
Also called cytoxan
low dose cyclophosphamide 50 mg PO BID x 21 days
DexamethasoneDrug
dexamethasone 40 mg PO weekly (or 20 mg if ≥ 75 years old).
| Study group | Role | What they receive |
|---|---|---|
| Pomalidomide, Cyclophosphamide, Dexamethasone | Experimental | Three oral drugs will be given in 28-day cycles: Pomalidomide 4 mg daily x 21 days; cyclophosphamide 50 mg BID x 21 days; and dexamethasone 40 mg weekly x 3 (20 mg weekly if the patient aged ≥ 75 years old) Receives: Drug: Pomalidomide; Drug: Cyclophosphamide; Drug: Dexamethasone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Best overall response rate (ORR)
disease response
Measured: up to 24 months
Secondary outcomes (9)
Stringent complete response (sCR)
Measured: up to 24 months
Complete response (CR)
Measured: up to 24 months
Very good partial response (VGPR)
Measured: up to 24 months
Partial response (PR)
Measured: up to 24 months
Time to progression (TTP)
TTP is defined as beginning with the time the first dose of PCD regimen is administered until disease progression on PCD regimen.
Measured: up to 24 months
Duration of response (DOR)
DOR is defined as the time from first evidence of PR or better to confirmation of disease progression.
Measured: up to 24 months
Clinical benefit response (CBR)
CBR is the combination of the ORR and minimal response (MR) i.e. this includes sCR, CR, VGPR, PR, and MR.
Measured: up to 24 months
Progression free survival (PFS)
PFS is defined as the duration of time from start of treatment to the first occurrence of disease progression or death on study from any cause, whichever occurs earlier.
Measured: up to 24 months
Overall survival (OS)
OS is defined as the time from the first dose of study treatment to the date of death (whatever the cause).
Measured: up to 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2014 (estimated)
- Study startJun 2014
- Primary completion7 May 2019
- Study completion7 May 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Multiple Myeloma in RelapseStudies of the same condition, open and under way.
- 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
- 35 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ajai Chari
- Sponsor type
- University, hospital or other
- Collaborators
- Celgene Corporation
- Responsible party
- Sponsor-investigator, Ajai Chari, Icahn School of Medicine at Mount Sinai
- Investigators
- Ajai Chari, MD, Principal investigator, Icahn School of Medicine at Mount Sinai
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02176213
- Sponsor's protocol ID
- GCO 14-0825
- Other IDs
- PO-CL-MM-PI-002404
- Status last verified
- Jun 2019
- First submitted
- 25 Jun 2014
- Last updated
- 18 Jun 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase II Study of Pomalidomide, Daily Low Dose Oral Cyclophosphamide, and Dexamethasone in Relapsed/Refractory Multiple Myeloma
- ClinicalTrials.gov ID
- NCT02176213
- Acronym / sponsor ID
- GCO 14-0825
- Keywords
- Relapsed, Refractory, Multiple Myeloma, Pomalidomide, Cyclophosphamide, Dexamethasone
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 35 participants (actual)
- Lead sponsor
- Ajai ChariUniversity, hospital or other
- Collaborators
- Celgene Corporation
- Study start
- Jun 2014
- Primary completion
- 7 May 2019 (actual)
- Study completion
- 7 May 2019 (actual)
- First posted
- 27 Jun 2014 (estimated)
- 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 study is being done to learn more about the drug, pomalidomide and to gather data on its safety and side effects when used in combination with commercially available cyclophosphamide and dexamethasone. This combination is considered experimental and has not been approved by the FDA.
Pomalidomide is a third generation immunomodulatory (IMiDs) agent, which is a more potent version of thalidomide and lenalidomide drugs that have been approved by the United States Food and Drug Administration [FDA] for the treatment of MM. In February 2013, pomalidomide was also approved by the FDA for patients with MM who have had more than 2 types of therapy.
Pomalidomide is taken orally as capsules, and cyclophosphamide and dexamethasone are also taken orally as tablets in this study. Cyclophosphamide and dexamethasone are commercially available and are often used in combination with other drugs to treat Multiple Myeloma. Preliminary data from both the laboratory and patient studies suggest that this combination of drugs is more effective than pomalidomide and dexamethasone alone. However, the regimen being used in this study, which consists of daily cyclophosphamide, also permits support of low blood counts with either injections or transfusions as needed.
Detailed description
This is an open label, single center, phase II study of a combination of pomalidomide, daily low dose oral cyclophosphamide, and dexamethasone in patients with relapsed/refractory multiple myeloma. The three oral drugs will be given in 28-day cycles: Pomalidomide 4 mg daily x 21 days; cyclophosphamide 50 mg BID x 21 days; and dexamethasone 40 mg weekly x 3 (20 mg weekly if the patient aged ≥ 75 years old). Subjects meeting eligibility criteria with ANC < 1000/µL and platelet count < 50,000/µL will start at dose level -1 of both pomalidomide (3 mg daily) and cyclophosphamide (50 mg daily). G-CSF and platelet transfusion support is permitted if needed.
Dose reduction for hematologic toxicity will begin with cyclophosphamide and then subsequently alternate with pomalidomide until a subject cannot tolerate dose level -2 of both agents - in which case subject would come off study. If subject has 2 or more concurrent toxicities that are potentially attributable to both agents (e.g. hematologic toxicity) then dose modification guidelines will be followed with dose reduction being done sequentially with one agent at a time, unless in the opinion of the investigator, both agents required concurrent dose reduction.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pomalidomide, Cyclophosphamide, Dexamethasone | Experimental | Drug: Pomalidomide, Drug: Cyclophosphamide, Drug: DexamethasoneThree oral drugs will be given in 28-day cycles: Pomalidomide 4 mg daily x 21 days; cyclophosphamide 50 mg BID x 21 days; and dexamethasone 40 mg weekly x 3 (20 mg weekly if the patient aged ≥ 75 years old) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (24 listed)
- Patients must have a history of symptomatic multiple myeloma according to the IMWG criteria
- Patients must have received at least two prior lines of therapy and also must be refractory to lenalidomide.
- Patient has relapsed or relapsed/refractory MM.
- Patients must currently have measurable disease, as defined as:
- Male or female adults ≥ 18 years old
Key exclusion criteria (11 listed)
- Previous treatment with pomalidomide
- Patients who received chemotherapy or radiation therapy to 30% of marrow-bearing bone within ≤ 2 weeks or experimental agent/therapy within 4 weeks prior to starting study treatment; or who have not yet recovered from side effects of such therapies
- Known hypersensitivity to thalidomide or lenalidomide
- The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide, lenalidomide or similar drugs
- Other concurrent severe and/or uncontrolled medical conditions including abnormal laboratory values that could cause unacceptable safety risks or compromise compliance with the 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 |
|---|---|---|
| PomalidomideAlso: pomalyst4 mg PO x 21 days | Drug | Pomalidomide, Cyclophosphamide, Dexamethasone |
| CyclophosphamideAlso: cytoxanlow dose cyclophosphamide 50 mg PO BID x 21 days | Drug | Pomalidomide, Cyclophosphamide, Dexamethasone |
| Dexamethasonedexamethasone 40 mg PO weekly (or 20 mg if ≥ 75 years old). | Drug | Pomalidomide, Cyclophosphamide, Dexamethasone |
Primary outcome
Best overall response rate (ORR)
Time frame: up to 24 months
disease response
9 secondary outcomes
Stringent complete response (sCR)
Time frame: up to 24 months
Complete response (CR)
Time frame: up to 24 months
Very good partial response (VGPR)
Time frame: up to 24 months
Partial response (PR)
Time frame: up to 24 months
Time to progression (TTP)
Time frame: up to 24 months
Duration of response (DOR)
Time frame: up to 24 months
Clinical benefit response (CBR)
Time frame: up to 24 months
Progression free survival (PFS)
Time frame: up to 24 months
Overall survival (OS)
Time frame: up to 24 months
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2019.
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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.
Ajai Chari, MD principal investigator
Icahn School of Medicine at Mount Sinai
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 Pomalidomide, Cyclophosphamide and Dexamethasone in people with Multiple Myeloma in Relapse and Multiple Myeloma, Refractory, run by Ajai Chari 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 24 inclusion and 11 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 18 Jun 2019. 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Best overall response rate (ORR) (time frame: up to 24 months). 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 18 Jun 2019.