B-cell Chronic Lymphocytic LeukemiaPhase 1/2NCT00648739
Safety and Dose Ranging Study of Samalizumab to Treat Relapsing or Refractory CLL or MM
A Phase 1/2 study testing Samalizumab in people with B-cell Chronic Lymphocytic Leukemia and Multiple Myeloma, run by Alexion Pharmaceuticals, Inc. at 4 sites in United States.
Trial summary
The purpose of this study was to determine the safety and maximum tolerated dose (MTD) of ALXN6000 (samalizumab) in treating relapsing or refractory B-cell chronic lymphocytic leukemia (B-CLL) or multiple myeloma (MM) and to study how samalizumab may help the immune system fight tumors that express CD200. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- B-cell Chronic Lymphocytic Leukemia, Multiple Myeloma
- What is being tested?
- Samalizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 26 participants
- Where
- 4 sitesUnited States
- Study dates
- 19 Jun 2008to 14 Dec 2010
- Phase
- Phase 1/2
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Relapsing or Refractory B-CLL or MM
- Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
- Anticipated survival of greater than 6 months
- Female participants of childbearing potential must agree to use 2 forms of contraception
- Participants must have a standard indication for treatment of their malignancy
- Is willing and able to give written informed consent prior to any procedure not considered standard of care
Who cannot join
- Absolute neutrophil count (ANC) < 1000 x 10^9/liter (L)
- Platelet count < 50,000 x 10^9/L
- Pregnant or lactating women
- Prior history of autoimmune hemolysis requiring therapy
- Prior history of immune thrombocytopenia
- Active autoimmune disease requiring immunosuppressive therapy
Show all 12Show fewer
- Positive Coombs' Test (neither direct or indirect)
- Ongoing corticosteroid treatment equivalent to the mineralocorticoid potency of 10 milligrams (mg) /day of prednisone, or greater, for any condition
- Prior stem cell transplantation within 4 weeks prior to enrollment
- Prior chemotherapy for the applicable malignancy within 30 days of enrollment
- Neurosurgery or cranial radiation therapy within 1 year of enrollment
- Clinically significant renal, hepatic, or cardiopulmonary disease
Trial locations and contacts
4 sites in 1 country.
United States4 sites
Study site
Atlanta, Georgia, 30322
Study site
Durham, North Carolina, 27710
Study site
Morristown, New Jersey, 07962
Study site
Tucson, Arizona, 85719
About this study
Official title: A Phase I/II Open Label Study To Evaluate The Safety, Pharmacokinetics And Pharmacodynamics Of ALXN6000 In Patients With Relapsing Or Refractory B-Cell Chronic Lymphocytic Leukemia Or Multiple Myeloma
The purpose of this study was to determine the safety and maximum tolerated dose (MTD) of ALXN6000 (samalizumab) in treating relapsing or refractory B-cell chronic lymphocytic leukemia (B-CLL) or multiple myeloma (MM) and to study how samalizumab may help the immune system fight tumors that express CD200.
Read the detailed description
This was an open-label multicenter study for participants with relapsing or refractory B-CLL or MM. The study was planned to be conducted in 2 parts: Part A and Part B. Both parts were to evaluate safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy (to the extent possible) of samalizumab in the target participant population. Part A was designed as the open-label, intravenous (IV) single dose-escalation portion of the study to determine the MTD and to assess the overall safety of different dose levels of up to 4 IV doses of samalizumab in participants with either refractory or relapsing B-CLL or MM. Initially, at least 3 participants would be enrolled into a cohort until a dose-limiting toxicity (DLT) was reached. If any 1 of the initial 3 participants in the cohort experienced a DLT, the cohort would be expanded to at least 6 participants.
After determination of the MTD in Part A, the Sponsor was to review the safety, PK, and relevant PD data to determine the dosing administration schedule for Part B. However, no participants were enrolled for Part B, as the study was terminated by the Sponsor for administrative reasons.
Participant enrollment in Cohort 7 (600 milligrams per square meter [mg/m^2] dose level), Part A, was halted after enrollment of the first participant. The study was terminated by the Sponsor for administrative reasons and not due to any safety concerns. Participants who were on study at the time of study termination were allowed to continue until the expiry date of the drug lot being used and then were followed for 30 (±1) days per protocol. The study was terminated by the Sponsor at that time. Part B of the study was not conducted.
- Being tested
- Samalizumab
- Keywords
- B-cell Chronic Lymphocytic Leukemia, Leukemia, Multiple Myeloma, CD200, Anti-CD200
- Medical subject headings
- Leukemia, Lymphocytic, Chronic, B-Cell, Multiple Myeloma, Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SamalizumabDrug
Also called ALXN6000
Samalizumab is a humanized anti-CD200 monoclonal antibody.
| Study group | Role | What they receive |
|---|---|---|
| Samalizumab | Experimental | All doses of samalizumab were individualized based on the participant's body surface area in mg/m^2 based on screening height and weight. Participants were assigned to a dose cohort, ranging from 50 to 600 mg/m^2, and received a single IV dose of samalizumab. Participants who tolerated the drug and demonstrated at least stable disease received up to 3 additional cycles of samalizumab at the same dose originally received at a minimum of 28-day intervals and beginning no sooner than 6 weeks after the initial dose. If no participants enrolled into a cohort experienced a DLT, escalation to the next dose level occurred with a new cohort. If any 1 of the initial 3 participants in the cohort experienced a DLT, the cohort was expanded to at least 6 participants. Then, if less than one third of participants within the cohort experienced a DLT, escalation to the next dose level occurred with a new cohort. Dose cohorts were enrolled sequentially. Receives: Drug: Samalizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Maximum Tolerated Dose (MTD) Of Samalizumab
The MTD was to be defined as the dose level at which less than one-third of participants experienced a dose-limiting toxicity (DLT) during the first 28 days of treatment and immediately below the dose at which at least one-third of participants experienced a DLT.
A DLT was defined as any Grade 3 or greater toxicity according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 3.0 not related to the disease under study or its sequelae that occurred in the first 28 days after dosing in Cycle 1. The NCI CTCAE grading system for the organ of involvement was used to classify severity of adverse autoimmune reactions.
Measured: From first dose through 10 weeks after the last dose
Number Of Participants With Treatment-Emergent Adverse Events (TEAEs)
A TEAE was defined as any event not present prior to exposure to samalizumab or any event already present that worsened in either intensity or frequency following exposure to samalizumab. A treatment-emergent serious adverse event was defined as any event that resulted in death, was immediately life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
Measured: From first dose through 10 weeks after the last dose
Secondary outcomes (4)
Percent Of Bound Samalizumab On B-Cell Chronic Lymphocytic Leukemia (B-CLL) Cells In Participants With B-CLL At Baseline (Predose) And Day 1 (Postdose)
Bound samalizumab was detected using a monoclonal antibody (mAb) specific for samalizumab. The binding of samalizumab to CD200 on peripheral B-CLL tumor cells was evaluated in participants with B-CLL by flow cytometry and expressed as the percent of B-CLL cells bound by samalizumab. For participants with insufficient numbers of peripheral B-CLL for analysis, the samples were documented as "unevaluable" and an analysis was not performed.
Measured: Baseline (Predose) and Day 1 (Postdose)
Density Of B-CLL Cells Bound At Baseline (Predose) And Day 1 (Postdose)
Bound samalizumab was detected using a mAb specific for samalizumab. The binding of samalizumab to CD200 on peripheral B-CLL tumor cells was evaluated in participants with B-CLL by flow cytometry and expressed as mean channel fluorescence intensity (MFI) of bound samalizumab to provide an indication of the density of bound samalizumab on target B-CLL cells. For participants with insufficient numbers of peripheral B-CLL for analysis, the samples were documented as "unevaluable" and an analysis was not performed.
Measured: Baseline (Predose) and Day 1 (Postdose)
Clinical Response Of Participants With B-CLL Following Samalizumab Dosing Using Modified NCI Working Group Response Criteria For ORR Rate
Clinical responses assessed using the Modified NCI Working Group Response Criteria. Overall Response Rate (ORR)=percentage of participants who maintained best response for ≥1 month after achieving that best response and having complete response (CR), partial response (PR), nodular partial response (nPR), or stable disease (SD). CR=absence of lymphadenopathy, hepatomegaly, or splenomegaly and normal complete blood count (CBC). PR=≥50% decrease in peripheral lymphocyte count or ≥50% reduction in lymphadenopathy, hepatomegaly, or splenomegaly and normal or 50% improvement over baseline CBC. nPR=CR participants with lymphoid aggregates. Progressive disease (PD)=>50% increase for >2 lymph nodes, spleen and/or liver size, or absolute number of circulating lymphocytes; or new lymph nodes. SD=have not achieved CR, PR, or nPR or have not shown PD. Features must be exhibited for ≥1month for CR/PR. Number of participants with individual best response and ORR for each cohort is presented.
Measured: From first dose through 10 weeks after the last dose
Clinical Response Of Participants With Multiple Myeloma (MM) Following Dosing With Samalizumab
Clinical responses of samalizumab were assessed using the International Myeloma Working Group Uniform Response Criteria. ORR was defined as the percentage of participants who had either stringent complete response, complete response, very good partial response, or partial response on 2 consecutive assessments made at any time before the administration of any new therapy.
The number of participants with the individual best response and ORR for each cohort is presented.
Measured: From first dose through 10 weeks after the last dose
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2008 (estimated)
- Study start19 Jun 2008
- Primary completion14 Dec 2010
- Study completion14 Dec 2010
Why it stopped: The study was terminated by the Sponsor for administrative reasons and not due to any safety concerns.
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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for B-cell Chronic Lymphocytic LeukemiaStudies of the same condition, open and under way.
- More trials run by Alexion Pharmaceuticals, Inc.The 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 1/2
- Primary purpose
- Treatment
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- Alexion Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00648739
- Sponsor's protocol ID
- C07-003
- Status last verified
- Feb 2019
- First submitted
- 28 Mar 2008
- Last updated
- 5 Mar 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Mahadevan D, Lanasa MC, Farber C, Pandey M, Whelden M, Faas SJ, Ulery T, Kukreja A, Li L, Bedrosian CL, Zhang X, Heffner LT. Phase I study of samalizumab in chronic lymphocytic leukemia and multiple myeloma: blockade of the immune checkpoint CD200. J Immunother Cancer. 2019 Aug 23;7(1):227. doi: 10.1186/s40425-019-0710-1.
PubMed 31443741
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II Open Label Study To Evaluate The Safety, Pharmacokinetics And Pharmacodynamics Of ALXN6000 In Patients With Relapsing Or Refractory B-Cell Chronic Lymphocytic Leukemia Or Multiple Myeloma
- ClinicalTrials.gov ID
- NCT00648739
- Acronym / sponsor ID
- C07-003
- Keywords
- B-cell Chronic Lymphocytic Leukemia, Leukemia, Multiple Myeloma, CD200, Anti-CD200
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was terminated by the Sponsor for administrative reasons and not due to any safety concerns.
- Enrolment
- 26 participants (actual)
- Lead sponsor
- Alexion Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 19 Jun 2008 (actual)
- Primary completion
- 14 Dec 2010 (actual)
- Study completion
- 14 Dec 2010 (actual)
- First posted
- 1 Apr 2008 (estimated)
- Results
- Posted 5 Mar 2019
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 was to determine the safety and maximum tolerated dose (MTD) of ALXN6000 (samalizumab) in treating relapsing or refractory B-cell chronic lymphocytic leukemia (B-CLL) or multiple myeloma (MM) and to study how samalizumab may help the immune system fight tumors that express CD200.
Detailed description
This was an open-label multicenter study for participants with relapsing or refractory B-CLL or MM. The study was planned to be conducted in 2 parts: Part A and Part B. Both parts were to evaluate safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy (to the extent possible) of samalizumab in the target participant population. Part A was designed as the open-label, intravenous (IV) single dose-escalation portion of the study to determine the MTD and to assess the overall safety of different dose levels of up to 4 IV doses of samalizumab in participants with either refractory or relapsing B-CLL or MM. Initially, at least 3 participants would be enrolled into a cohort until a dose-limiting toxicity (DLT) was reached. If any 1 of the initial 3 participants in the cohort experienced a DLT, the cohort would be expanded to at least 6 participants.
After determination of the MTD in Part A, the Sponsor was to review the safety, PK, and relevant PD data to determine the dosing administration schedule for Part B. However, no participants were enrolled for Part B, as the study was terminated by the Sponsor for administrative reasons.
Participant enrollment in Cohort 7 (600 milligrams per square meter [mg/m^2] dose level), Part A, was halted after enrollment of the first participant. The study was terminated by the Sponsor for administrative reasons and not due to any safety concerns. Participants who were on study at the time of study termination were allowed to continue until the expiry date of the drug lot being used and then were followed for 30 (±1) days per protocol. The study was terminated by the Sponsor at that time. Part B of the study was not conducted.
Study design
- Primary purpose
- Treatment
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Samalizumab | Experimental | Drug: SamalizumabAll doses of samalizumab were individualized based on the participant's body surface area in mg/m^2 based on screening height and weight. Participants were assigned to a dose cohort, ranging from 50 to 600 mg/m^2, and received a single IV dose of samalizumab. Participants who tolerated the drug and demonstrated at least stable disease received up to 3 additional cycles of samalizumab at the same dose originally received at a minimum of 28-day intervals and beginning no sooner than 6 weeks after the initial dose. If no participants enrolled into a cohort experienced a DLT, escalation to the next dose level occurred with a new cohort. If any 1 of the initial 3 participants in the cohort experienced a DLT, the cohort was expanded to at least 6 participants. Then, if less than one third of participants within the cohort experienced a DLT, escalation to the next dose level occurred with a new cohort. Dose cohorts were enrolled sequentially. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Relapsing or Refractory B-CLL or MM
- Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2
- Anticipated survival of greater than 6 months
- Female participants of childbearing potential must agree to use 2 forms of contraception
- Participants must have a standard indication for treatment of their malignancy
Key exclusion criteria (12 listed)
- Absolute neutrophil count (ANC) < 1000 x 10^9/liter (L)
- Platelet count < 50,000 x 10^9/L
- Pregnant or lactating women
- Prior history of autoimmune hemolysis requiring therapy
- Prior history of immune thrombocytopenia
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 |
|---|---|---|
| SamalizumabAlso: ALXN6000Samalizumab is a humanized anti-CD200 monoclonal antibody. | Drug | Samalizumab |
Primary outcome
Maximum Tolerated Dose (MTD) Of Samalizumab
Time frame: From first dose through 10 weeks after the last dose
The MTD was to be defined as the dose level at which less than one-third of participants experienced a dose-limiting toxicity (DLT) during the first 28 days of treatment and immediately below the dose at which at least one-third of participants experienced a DLT. A DLT was defined as any Grade 3 or greater toxicity according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 3.0 not related to the disease under study or its sequelae that occurred in the first 28 days after dosing in Cycle 1. The NCI CTCAE grading system for the organ of involvement was used to classify severity of adverse autoimmune reactions.
Number Of Participants With Treatment-Emergent Adverse Events (TEAEs)
Time frame: From first dose through 10 weeks after the last dose
A TEAE was defined as any event not present prior to exposure to samalizumab or any event already present that worsened in either intensity or frequency following exposure to samalizumab. A treatment-emergent serious adverse event was defined as any event that resulted in death, was immediately life threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, or was a congenital anomaly/birth defect. A summary of serious and all other non-serious adverse events regardless of causality is located in the Reported Adverse Events module.
4 secondary outcomes
Percent Of Bound Samalizumab On B-Cell Chronic Lymphocytic Leukemia (B-CLL) Cells In Participants With B-CLL At Baseline (Predose) And Day 1 (Postdose)
Time frame: Baseline (Predose) and Day 1 (Postdose)
Density Of B-CLL Cells Bound At Baseline (Predose) And Day 1 (Postdose)
Time frame: Baseline (Predose) and Day 1 (Postdose)
Clinical Response Of Participants With B-CLL Following Samalizumab Dosing Using Modified NCI Working Group Response Criteria For ORR Rate
Time frame: From first dose through 10 weeks after the last dose
Clinical Response Of Participants With Multiple Myeloma (MM) Following Dosing With Samalizumab
Time frame: From first dose through 10 weeks after the last dose
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 31443741Mahadevan D, Lanasa MC, Farber C, Pandey M, Whelden M, Faas SJ, Ulery T, Kukreja A, Li L, Bedrosian CL, Zhang X, Heffner LT. Phase I study of samalizumab in chronic lymphocytic leukemia and multiple myeloma: blockade of the immune checkpoint CD200. J Immunother Cancer. 2019 Aug 23;7(1):227. doi: 10.1186/s40425-019-0710-1.
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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.
The record lists no contact. Use the official record to reach the sponsor.
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Samalizumab in people with B-cell Chronic Lymphocytic Leukemia and Multiple Myeloma, run by Alexion Pharmaceuticals, Inc. at 4 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 6 inclusion and 12 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: stopped early, as of the sponsor's last update on 5 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 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 combined 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.
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 5 Mar 2019). 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?
Maximum Tolerated Dose (MTD) Of Samalizumab (time frame: From first dose through 10 weeks after the last dose). The study lists 2 primary outcomes in all. 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 NCT00648739, which also lists 1 linked publication.
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 5 Mar 2019.