A Phase 2 interventional study of PCI-32765 in Waldenstrom's Macroglobulinemia, sponsored by Dana-Farber Cancer Institute. Completed at 4 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2020-01-21.
Sponsored by Dana-Farber Cancer Institute · Phase 2, Interventional, and Treatment
This research study is a Phase II clinical trial. Phase II clinical trials test the effectiveness of an investigational drug, PCI-32765, to learn whether PCI-32765 works in treating a specific cancer. "Investigational" means that PCI-32765 is still being studied and that research doctors are trying to find out more about it-such as the safest dose to use, the side effects it may cause, and if PCI-32765 is effective for treating different types of cancer. It also means that the FDA has not yet approved PCI-32765 for use in patients, including people with Waldenstrom's Macroglobulinemia.
PCI-32765 is a newly discovered drug that is being developed as an anti-cancer agent. PCI-32765 is a Bruton's tyrosine kinase (Btk) inhibitor drug which interrupts B cell receptor (BCR) signaling in lymphomas by selectively and irreversibly binding to the Btk protein, which then results in malignant cell death. This drug has been used in laboratory experiments and other research studies in B-cell malignancies and information from those other research studies suggests that PCI-32765 may be a treatment strategy for B-cell malignancies, including Waldenstrom's Macroglobulinemia.
In this research study, the investigators are testing the safety and efficacy of PCI-32765 as a treatment option for relapsed or refractory Waldenstrom's Macroglobulinemia.
Patients in this research study will receive up to 40 cycles of treatment. Each treatment cycle lasts 4 weeks. Patients will take PCI-32765 by mouth, once a day in the morning.
During each cycle patients will be asked to visit the clinic for scheduled tests and exams and to receive a supply of PCI-32765 to take at home every day. Patients will visit the clinic on the first day of each of the first 3 cycles, and then just once at the beginning of every three cycles.
During study visits, patients will have a physical exam where they will be asked questions about their general health and specific questions about any problems that they might be having and any medications they may be taking. Patients will have blood tests to see how their disease is responding to the study treatment and how they are tolerating the study drug. Patients may also have CT scans of the chest, abdomen and pelvis as well as a bone marrow aspirate and biopsy. If a patient's disease stays the same or is helped, he/she will continue to get study treatment. If disease worsens, he/she will be taken off study treatment at that time.
After completion of the treatment and as part of standard of care, follow-up tests will include a physical exam, review of symptoms and medications, blood tests, bone marrow aspirate and biopsy, CT scans of the chest, abdomen and pelvis. The investigators would like to continue to monitor progress by following-up every three months for up to two years after completion of study treatment or until next treatment, whichever comes first.
372 studies on the registry are indexed under Waldenstrom Macroglobulinemia; 63 are open to participants now.
This study's enrollment of 63 is above the median of 40 across 322 interventional studies indexed under Waldenstrom Macroglobulinemia.
Browse Waldenstrom Macroglobulinemia studies →Dana-Farber Cancer Institute is the lead sponsor of 832 studies on the registry; 122 are open to participants now.
Of its 113 completed or terminated interventional studies of FDA-regulated products, 77 (68%) have results posted.
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Exclusion Criteria:
PCI-32765; ibrutinib
Drug: PCI-32765
Taken orally, once daily in the morning
Also known as: ibrutinib
Overall Response Rate
To assess the overall response rate (\>25% reduction in serum IgM from baseline).
Time frame: 4 years
Safety and Tolerability of PCI-32765
To assess the safety and tolerability of PCI-32765 in symptomatic WM patients with relapsed/refractory disease. Grade \> or = 2 Adverse Events determined to be associated with PCI-32765 and subsequent outcomes will constitute the safety profile of PCI-32765 in WM. Percent of participants who experienced at least 1 grade 2 or higher treatment emergent adverse event.
Time frame: 4 years
Determine Progression Free Survival
To determine Progression Free Survival (PFS in symptomatic WM patients with relapsed/refractory disease. Participants were treated for 40 cycles and then followed for 2 years or until next therapy or death. 40 participants were censored prior to disease progression.
Time frame: 6 years
To Determine Time to Next Therapy (TTNT) of PCI-32765 in Symptomatic WM Patients With Relapsed/Refractory Disease
Time to Next Therapy is the duration of time from of starting ibrutinib until next therapy. Participants were treated for 40 cycles and then followed for 2 years or until next therapy or death. Participants had the option to continue ibrutinib commercially. 40 participants were censored while still on commercial ibrutinib therapy.
Time frame: 6 years
Major Response Rates
To assess the major response rate (\>50% reduction in serum IgM from baseline)
Time frame: 4 years
Very Good Partial Response Rate
To assess the very good partial response rate (\>90% reduction in serum IgM from baseline)
Time frame: 4 years
| Milestone | Treatment Arm |
|---|---|
| Started | 63 |
| Completed | 63 |
| Not completed | 0 |
To assess the overall response rate (\>25% reduction in serum IgM from baseline).
| Participants | Treatment Arm |
|---|---|
| Overall Response Rate | 57 |
To assess the safety and tolerability of PCI-32765 in symptomatic WM patients with relapsed/refractory disease. Grade \> or = 2 Adverse Events determined to be associated with PCI-32765 and subsequent outcomes will constitute the safety profile of PCI-32765 in WM. Percent of participants who experienced at least 1 grade 2 or higher treatment emergent adverse event.
| Participants | Treatment Arm |
|---|---|
| Safety and Tolerability of PCI-32765 | 62 |
To determine Progression Free Survival (PFS in symptomatic WM patients with relapsed/refractory disease. Participants were treated for 40 cycles and then followed for 2 years or until next therapy or death. 40 participants were censored prior to disease progression.
| months | Treatment Arm |
|---|---|
| Determine Progression Free Survival | 39 (0 to 67) |
Time to Next Therapy is the duration of time from of starting ibrutinib until next therapy. Participants were treated for 40 cycles and then followed for 2 years or until next therapy or death. Participants had the option to continue ibrutinib commercially. 40 participants were censored while still on commercial ibrutinib therapy.
| months | Treatment Arm |
|---|---|
| To Determine Time to Next Therapy (TTNT) of PCI-32765 in Symptomatic WM Patients With Relapsed/Refractory Disease | 49 (1 to 67) |
To assess the major response rate (\>50% reduction in serum IgM from baseline)
| Participants | Treatment Arm |
|---|---|
| Major Response Rates | 49 |
To assess the very good partial response rate (\>90% reduction in serum IgM from baseline)
| Participants | Treatment Arm |
|---|---|
| Very Good Partial Response Rate | 17 |
Collected over Adverse events were collected from Cycle 1 Day 1 through 30 days after discontinuing protocol ibrutinib therapy, an average of 3 years.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Treatment Arm | 1/63 (1.6%) | 30/63 (47.6%) | 62/63 (98.4%) |
| Event | Treatment Arm |
|---|---|
| PneumoniaInfections and infestations | 11/63 |
| FeverGeneral disorders | 3/63 |
| Atrial FibrillationCardiac disorders | 2/63 |
| Pleural effusionRespiratory, thoracic and mediastinal disorders | 2/63 |
| Disease Transformation DLBCLNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 2/63 |
| AnemiaBlood and lymphatic system disorders | 2/63 |
| Platelet Count DecreasedInvestigations | 2/63 |
| Squamous cell carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 2/63 |
| Fever and TachycardiaGeneral disorders | 1/63 |
| FluInfections and infestations | 1/63 |
| Event | Treatment Arm |
|---|---|
| AnemiaBlood and lymphatic system disorders | 40/63 |
| FatigueGeneral disorders | 33/63 |
| Platelet count decreasedInvestigations | 29/63 |
| Neutrophil count decreasedInvestigations | 26/63 |
| SinusitisInfections and infestations | 21/63 |
| DiarrheaGastrointestinal disorders | 20/63 |
| Upper respiratory infectionInfections and infestations | 19/63 |
| ArthralgiaMusculoskeletal and connective tissue disorders | 17/63 |
| Lung infectionInfections and infestations | 14/63 |
| MyalgiaMusculoskeletal and connective tissue disorders | 14/63 |
| Age, Categorical(Participants) | Treatment Arm |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 32 |
| >=65 years | 31 |
| Age, Continuous(years) | Treatment Arm |
|---|---|
| Median | 63 ± 10.6 |
| Sex: Female, Male(Participants) | Treatment Arm |
|---|---|
| Female | 15 |
| Male | 48 |
| Race and Ethnicity Not Collected(Participants) | Treatment Arm |
|---|
| Region of Enrollment(participants) | Treatment Arm |
|---|---|
| United States | 63 |
This study is completed, as verified in Jan 2020. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Ibrutinib
Waldenstrom Macroglobulinemia →
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