Multiple MyelomaPhase 1NCT01568294
Japanese Phase 1 Study to Evaluate Tolerated Dose, Safety, and Efficacy of Pomalidomide in Patients With Refractory or Relapsed and Refractory Multiple Myeloma
A Phase 1 study testing pomalidomide in people with Multiple Myeloma, run by Celgene at 8 sites in Japan.
Trial summary
The purpose of this study is to determine the tolerated dose of pomalidomide and also to evaluate the pharmacokinetics, safety and efficacy of pomalidomide in patients with refractory or relapsed and refractory multiple myeloma. Read more about this study
- Who is it for?
- Ages 20 years and older
- Conditions the study is for
- Multiple Myeloma
- What is being tested?
- pomalidomide
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 8 sitesJapan
- Study dates
- 1 Apr 2012to 8 Jul 2015
- Phase
- Phase 1
- 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 20 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Must be ≥ 20 years of age at the time of signing the informed consent document
- The subject must understand and voluntarily sign an informed consent document prior to any study related assessments/procedures are conducted.
- Must be able to adhere to the study visit schedule and other protocol requirements
- Subjects must have documented diagnosis of multiple myeloma and have measurable disease
- All subjects must have had at least 2 prior lines of anti-myeloma therapy. Induction therapy followed by stem cell transplant and consolidation/maintenance will be considered as one line
- All subjects must have either refractory or relapsed and refractory disease defined as documented disease progression during or within 60 days of completing their last anti-myeloma therapy.
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- Primary refractory: Subjects who have never achieved any response better than progressive disease (PD) to any previous line of anti-myeloma therapy.
- Relapsed and refractory: Subjects who have relapsed after having achieved at least stable disease (SD) to at least one prior regimen and then developed progressive disease (PD) on or within 60 days of completing their last anti-myeloma therapy.
- Subjects must have also undergone prior treatment with at least 2 cycles of lenalidomide and at least 2 cycles of bortezomib (either in separate regimens or within the same regimen).
- All subjects must have received adequate prior alkylator therapy.
- Eastern Cooperative Oncology Group (ECOG) performance status score of 0, 1, or 2.
Who cannot join
- Pregnant or breastfeeding females
- Hypersensitivity to thalidomide, lenalidomide, or dexamethasone
- ≥ Grade 3 rash during prior thalidomide or lenalidomide therapy
- Patients unable or unwilling to undergo antithrombotic prophylactic treatment will not be eligible to participate in this study
- Any of the following laboratory abnormalities:
- Absolute neutrophil count (ANC) < 1,000/µL
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- Platelet count < 75,000/µL for patients in whom < 50% of bone marrow nucleated cells are plasma cells; or a platelet count < 30,000/µL for patients in whom ≥ 50% of bone marrow nucleated cells are plasma cells
- Creatinine Clearance < 45 mL/min according to Cockcroft-Gault formula
- Corrected serum calcium > 14 mg/dL (> 3.5 mmol/L)
- Hemoglobin < 8 g/dL (< 4.9 mmol/L; prior RBC transfusion or recombinant human erythropoietin use is permitted)
- Serum glutamic oxaloacetic transaminase (SGOT) /aspartate aminotransferase (AST) or serum glutamic pyruvic transaminase (SGPT) /alanine aminotransferase (ALT) > 3.0 x upper limit of normal (ULN)
- Serum total bilirubin > 2.0 mg/dL (34.2 μmol/L); or ≥ 3.0 x upper limit of normal (ULN) for subjects with hereditary benign hyperbilirubinaemia
- Peripheral neuropathy ≥ Grade 2
- Patients who received any of the following within the last 14 days of initiation of study treatment:
- Plasmapheresis
- Major surgery (kyphoplasty is not considered major surgery)
- Radiation therapy
- Use of any anti-myeloma drug therapy
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
Japan8 sites
Kyusyu University Hospital
Fukuoka, 812-8582
Tokai University Hospital
Isehara, Kanagawa, 259-1193
Kameda General Hospital
Kamogawa, 296-1602
Saitama Medical Center, Saitama Medical University
Kawagoe, Saitama, 350-8550
Nagoya City University Hospital
Nagoya, Aichi-ken, 467-8602
Niigata Cancer Center Hospital
Niigata, 951-8566
Okayama Medical Center
Okayama, 701-1192
National Cancer Center Hospital
Tyuuou, Tokyo, 104-0045
About this study
Official title: A Phase 1, Multicenter, Open-label, Dose-escalation Study in Japan to Determine the Tolerated Dose and to Evaluate the Safety, Efficacy, and Pharmacokinetics of Pomalidomide Alone or in Combination With Dexamethasone in Patients With Refractory or Relapsed and Refractory Multiple Myeloma
The purpose of this study is to determine the tolerated dose of pomalidomide and also to evaluate the pharmacokinetics, safety and efficacy of pomalidomide in patients with refractory or relapsed and refractory multiple myeloma.
- Conditions
- Multiple Myeloma
- Being tested
- pomalidomide
- Medical subject headings
- Multiple Myeloma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
pomalidomideDrug
2 mg or 4mg oral pomalidomide once per day on Days 1-21 of a 28-day cycle
| Study group | Role | What they receive |
|---|---|---|
| pomalidomide | Experimental | Patients will receive pomalidomide orally on Days 1-21 of each 28-day cycle until when/if a discontinuation criterion, e.g., disease progression, development of an unacceptable toxicity, voluntary withdrawal, or pomalidomide is in market for the target indication. Receives: Drug: pomalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of dose-limiting toxicity in accordance with Common Terminology Criteria for Adverse Events
Incidence of dose-limiting toxicity in accordance with Common Terminology Criteria for Adverse Events
Measured: Up to 28 Days
Secondary outcomes (11)
Maximum observed plasma concentration (Cmax)
Maximum observed plasma concentration (Cmax)
Measured: Up to 28 days
Time to maximum observed plasma concentration (tmax)
Time to maximum observed plasma concentration (tmax)
Measured: Up 28 days
Area under the plasma concentration-time curve (AUC0-t)
Area under the plasma concentration-time curve (AUC0-t)
Measured: Up to 28 days
Apparent total plasma clearance (CL/F)
Apparent total plasma clearance (CL/F)
Measured: Up to 28 days
Apparent total volume of distribution (Vz/F)
Apparent total volume of distribution (Vz/F)
Measured: Up to 28 days
Estimate of the terminal elimination half-life in plasma (t1/2)
Estimate of the terminal elimination half-life in plasma (t1/2)
Measured: Up to 28 days
Safety (the number of participants with adverse events, incidence, severity, causality)
Safety (the number of participants with adverse events, incidence, severity, causality)
Measured: Up to 2 years
Progression-free survival
Progression-free survival
Measured: Up to 28 days
Myeloma response
Myeloma response
Measured: Up to 28 days
Time to Response
Time to Response
Measured: Up to 28 days
Duration of Response
Duration of Response
Measured: Up to 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 Apr 2012 (estimated)
- Study start1 Apr 2012
- Primary completion8 Jul 2015
- Study completion8 Jul 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Multiple MyelomaStudies of the same condition, open and under way.
- More trials run by CelgeneThe 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
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Celgene
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Toru Sasaki, Study director, Celgene K.K
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01568294
- Sponsor's protocol ID
- CC-4047-MM-004
- Status last verified
- Nov 2019
- First submitted
- 29 Mar 2012
- Last updated
- 12 Nov 2019
- Results posted
- No
- Data monitoring committee
- No
Publications
Matsue K, Iwasaki H, Chou T, Tobinai K, Sunami K, Ogawa Y, Kurihara M, Midorikawa S, Zaki M, Doerr T, Iida S. Pomalidomide alone or in combination with dexamethasone in Japanese patients with refractory or relapsed and refractory multiple myeloma. Cancer Sci. 2015 Nov;106(11):1561-7. doi: 10.1111/cas.12772. Epub 2015 Nov 4.
PubMed 26292221Mark TM, Forsberg PA, Rossi AC, Pearse RN, Pekle KA, Perry A, Boyer A, Tegnestam L, Jayabalan D, Coleman M, Niesvizky R. Phase 2 study of clarithromycin, pomalidomide, and dexamethasone in relapsed or refractory multiple myeloma. Blood Adv. 2019 Feb 26;3(4):603-611. doi: 10.1182/bloodadvances.2018028027.
PubMed 30792190
Study at a glance
From the registry record, last updated by the sponsor on 12 Nov 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Multicenter, Open-label, Dose-escalation Study in Japan to Determine the Tolerated Dose and to Evaluate the Safety, Efficacy, and Pharmacokinetics of Pomalidomide Alone or in Combination With Dexamethasone in Patients With Refractory or Relapsed and Refractory Multiple Myeloma
- ClinicalTrials.gov ID
- NCT01568294
- Acronym / sponsor ID
- CC-4047-MM-004
- Conditions
- Multiple Myeloma
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- CelgeneIndustry (pharma, biotech, devices)
- Study start
- 1 Apr 2012 (actual)
- Primary completion
- 8 Jul 2015 (actual)
- Study completion
- 8 Jul 2015 (actual)
- First posted
- 2 Apr 2012 (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.
The purpose of this study is to determine the tolerated dose of pomalidomide and also to evaluate the pharmacokinetics, safety and efficacy of pomalidomide in patients with refractory or relapsed and refractory 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 |
|---|---|---|
| pomalidomide | Experimental | Drug: pomalidomidePatients will receive pomalidomide orally on Days 1-21 of each 28-day cycle until when/if a discontinuation criterion, e.g., disease progression, development of an unacceptable toxicity, voluntary withdrawal, or pomalidomide is in market for the target indication. |
Who may qualify
- Ages
- 20 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- Must be ≥ 20 years of age at the time of signing the informed consent document
- The subject must understand and voluntarily sign an informed consent document prior to any study related assessments/procedures are conducted.
- Must be able to adhere to the study visit schedule and other protocol requirements
- Subjects must have documented diagnosis of multiple myeloma and have measurable disease
- All subjects must have had at least 2 prior lines of anti-myeloma therapy. Induction therapy followed by stem cell transplant and consolidation/maintenance will be considered as one line
Key exclusion criteria (18 listed)
- Pregnant or breastfeeding females
- Hypersensitivity to thalidomide, lenalidomide, or dexamethasone
- ≥ Grade 3 rash during prior thalidomide or lenalidomide therapy
- Patients unable or unwilling to undergo antithrombotic prophylactic treatment will not be eligible to participate in this study
- Any of the following laboratory abnormalities:
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 |
|---|---|---|
| pomalidomide2 mg or 4mg oral pomalidomide once per day on Days 1-21 of a 28-day cycle | Drug | pomalidomide |
Primary outcome
Incidence of dose-limiting toxicity in accordance with Common Terminology Criteria for Adverse Events
Time frame: Up to 28 Days
Incidence of dose-limiting toxicity in accordance with Common Terminology Criteria for Adverse Events
11 secondary outcomes
Maximum observed plasma concentration (Cmax)
Time frame: Up to 28 days
Time to maximum observed plasma concentration (tmax)
Time frame: Up 28 days
Area under the plasma concentration-time curve (AUC0-t)
Time frame: Up to 28 days
Apparent total plasma clearance (CL/F)
Time frame: Up to 28 days
Apparent total volume of distribution (Vz/F)
Time frame: Up to 28 days
Estimate of the terminal elimination half-life in plasma (t1/2)
Time frame: Up to 28 days
Safety (the number of participants with adverse events, incidence, severity, causality)
Time frame: Up to 2 years
Progression-free survival
Time frame: Up to 28 days
Myeloma response
Time frame: Up to 28 days
Time to Response
Time frame: Up to 28 days
Duration of Response
Time frame: Up to 28 days
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 12 Nov 2019.
- Background: PubMed 26292221Matsue K, Iwasaki H, Chou T, Tobinai K, Sunami K, Ogawa Y, Kurihara M, Midorikawa S, Zaki M, Doerr T, Iida S. Pomalidomide alone or in combination with dexamethasone in Japanese patients with refractory or relapsed and refractory multiple myeloma. Cancer Sci. 2015 Nov;106(11):1561-7. doi: 10.1111/cas.12772. Epub 2015 Nov 4.
- Background: PubMed 30792190Mark TM, Forsberg PA, Rossi AC, Pearse RN, Pekle KA, Perry A, Boyer A, Tegnestam L, Jayabalan D, Coleman M, Niesvizky R. Phase 2 study of clarithromycin, pomalidomide, and dexamethasone in relapsed or refractory multiple myeloma. Blood Adv. 2019 Feb 26;3(4):603-611. doi: 10.1182/bloodadvances.2018028027.
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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.
Toru Sasaki study director
Celgene K.K
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing pomalidomide in people with Multiple Myeloma, run by Celgene at 8 sites in Japan.
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 11 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: finished, as of the sponsor's last update on 12 Nov 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Japan. 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?
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?
Incidence of dose-limiting toxicity in accordance with Common Terminology Criteria for Adverse Events (time frame: Up to 28 Days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01568294, which also lists 2 linked publications.
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 12 Nov 2019.