A Phase 1/2 interventional study of FPA008 in Pigmented Villonodular Synovitis and Tenosynovial Giant Cell Tumor, sponsored by Five Prime Therapeutics, Inc.. Completed at 12 sites in 6 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2021-08-31.
Sponsored by Five Prime Therapeutics, Inc. (part of Amgen) · Phase 1/2, Interventional, and Treatment
This is a phase 1/2 single arm, open-label, safety, tolerability, and PK study of cabiralizumab in PVNS/dt-TGCT patients.
A Phase 1/2 study was an open-label, dose escalation and dose expansion study designed to evaluate the pharmacokinetics, pharmacodynamics, safety and preliminary efficacy of cabiralizumab, a CSF1-R monoclonal antibody, inpatients with unresectable diffuse tenosynovial giant cell tumors (TGCT).
20 studies on the registry are indexed under Synovitis, Pigmented Villonodular; 2 are open to participants now.
This study's enrollment of 66 is above the median of 36 across 17 interventional studies indexed under Synovitis, Pigmented Villonodular.
Browse Synovitis, Pigmented Villonodular studies →Five Prime Therapeutics, Inc. is the lead sponsor of 11 studies on the registry; none are open to participants now.
First submitted before Five Prime Therapeutics, Inc. became part of Amgen.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Histologically or cytologically confirmed
The diagnosis was confirmed by looking at a sample under a microscope: tissue from a biopsy or surgery (histology), or cells from a fluid or needle sample (cytology).
Your pathology report is the document that shows this.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
RECIST
The standard rules for measuring tumours on CT or MRI scans, to tell whether a cancer is shrinking, stable or growing during treatment.
“Measurable disease per RECIST 1.1” usually means at least one tumour 10 mm or more at its longest on a CT scan, or a lymph node 15 mm or more across its short axis.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECG (electrocardiogram)
A recording of the heart’s electrical activity, taken with stickers on the chest, arms and legs. It takes a few minutes and does not hurt.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
IV infusion; safety data will be reviewed prior to dose escalation decision. Dose escalation will complete when recommended dose (RD) is determined. RD will be the maximum tolerated dose or lower dose that provide adequate PK exposure and biologic activity with tolerability.
Biological: FPA008
IV infusion; once MTD and/or RD has been determined in Phase 1, expansion cohorts of approximately 30 patients (each cohort) with PVNS or dt-TGCT will be enrolled to characterize clinical activity and safety profile of the RD. Treatment is planned to continue for up to 24 weeks or 56 weeks.
Biological: FPA008
FPA008 will be administered by IV infusion over approximately 30 minutes every 2 or 4 weeks
Also known as: Cabiralizumab
The Incidence of Grade 3 and Grade 4 Adverse Events (AEs) and Defined as Dose-limiting Toxicities (DLTs) in Phase 1
Number of participants with grade 3 and grade 4 adverse events (AE) defined as dose limiting toxicities (DLTs) in Phase 1
Time frame: 52 weeks
The Incidence of Investigator-assessed, Confirmed Objective Responses (ORR) Per RECIST 1.1 (Phase 2)
Number of confirmed objective responses (ORR) as assessed by the investigator per RECIST 1.1 (Phase 2)
Time frame: 52 weeks
PK Parameters of Cabiralizumab: Area Under Concentration-time Curve (AUC)
Area under serum concentration-time curve (AUC) for cabiralizumab as a PK parameter
Time frame: 52 weeks
Maximum Serum Concentration (Cmax).
Composite PK parameters of cabiralizumab: Maximum observed serum concentration
Time frame: 52 weeks
Minimum Serum Concentration (Cmin).
Composite PK parameters of cabiralizumab: minimum serum concentration (Cmin).
Time frame: 52 weeks
Pharmacokinetic Clearance (CL).
Composite PK parameters of cabiralizumab: clearance (CL)
Time frame: 52 weeks
The Incidence of AEs.
treatment-emergent adverse events (TEAEs) by incidence for the Safety Population. Patients with at lease 1 TEAE.
Time frame: 52 weeks
The Incidence of Clinical Laboratory Abnormalities.
The number of patients with a clinical laboratory that is outside the normal range at some time point during the study
Time frame: 52 weeks
The Incidence of ECG Abnormalities.
The number of patients who had a change in their ECG that were clinically significant
Time frame: 52 weeks
Duration of Response Per RECIST 1.1 in Phase 2
The length of response per RECIST 1.1 from the time of first response to progression or going off study in Phase 2
Time frame: 52 weeks
| Milestone | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B |
|---|---|---|---|---|---|
| Started | 3 | 3 | 3 | 33 | 24 |
| Completed | 3 | 2 | 3 | 18 | 8 |
| Not completed | 0 | 1 | 0 | 15 | 16 |
| Withdrew: Withdrawal by subject | 0 | 1 | 0 | 2 | 5 |
| Withdrew: Study terminiated by sponsor | 0 | 0 | 0 | 1 | 0 |
| Withdrew: Lost to follow-up | 0 | 0 | 0 | 3 | 2 |
| Withdrew: Death | 0 | 0 | 0 | 1 | 0 |
| Withdrew: Other | 0 | 0 | 0 | 8 | 9 |
Number of participants with grade 3 and grade 4 adverse events (AE) defined as dose limiting toxicities (DLTs) in Phase 1
| Participants | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg |
|---|---|---|---|
| The Incidence of Grade 3 and Grade 4 Adverse Events (AEs) and Defined as Dose-limiting Toxicities (DLTs) in Phase 1 | 0 | 0 | 0 |
Number of confirmed objective responses (ORR) as assessed by the investigator per RECIST 1.1 (Phase 2)
| Participants | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B |
|---|---|---|
| The Incidence of Investigator-assessed, Confirmed Objective Responses (ORR) Per RECIST 1.1 (Phase 2) | 8 | 8 |
Area under serum concentration-time curve (AUC) for cabiralizumab as a PK parameter
| ug x day/mL | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B |
|---|---|---|---|---|---|
| PK Parameters of Cabiralizumab: Area Under Concentration-time Curve (AUC) | 102 ± 10.2 | 283 ± 26.3 | 593 ± 31.3 | 660 ± 17.7 | 593 ± 12.6 |
Composite PK parameters of cabiralizumab: Maximum observed serum concentration
| ug/mL | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B |
|---|---|---|---|---|---|
| Maximum Serum Concentration (Cmax). | 22.5 ± 14.3 | 52.1 ± 22 | 91.2 ± 15 | 91.8 ± 24.8 | 87 ± 12.6 |
Composite PK parameters of cabiralizumab: minimum serum concentration (Cmin).
| ug/mL | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B |
|---|---|---|---|---|---|
| Minimum Serum Concentration (Cmin). | 2.18 ± 117 | 7.26 ± 41.8 | 21.2 ± 59.2 | 23.9 ± 38.3 | 22.6 ± 28.2 |
Composite PK parameters of cabiralizumab: clearance (CL)
| L/d | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B |
|---|---|---|---|---|---|
| Pharmacokinetic Clearance (CL). | .663 ± 16.6 | .416 ± 24.5 | .694 ± 0 | .307 ± 44.9 | .525 ± 8.42 |
treatment-emergent adverse events (TEAEs) by incidence for the Safety Population. Patients with at lease 1 TEAE.
| Participants | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B |
|---|---|---|---|---|---|
| The Incidence of AEs. | 3 | 3 | 3 | 33 | 24 |
The number of patients with a clinical laboratory that is outside the normal range at some time point during the study
| participants | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B |
|---|---|---|---|---|---|
| The Incidence of Clinical Laboratory Abnormalities. | 3 | 2 | 3 | 33 | 24 |
The number of patients who had a change in their ECG that were clinically significant
| Participants | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B |
|---|---|---|---|---|---|
| The Incidence of ECG Abnormalities. | 0 | 0 | 0 | 0 | 0 |
The length of response per RECIST 1.1 from the time of first response to progression or going off study in Phase 2
| months | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B |
|---|---|---|
| Duration of Response Per RECIST 1.1 in Phase 2 | 4.4 (3.2 to NA) | NA (11.5 to NA) |
Collected over Event reporting commences at the time of signing the informed consent. A treatment-emergent AE was defined as an AE that began or worsened in severity after at least 1 dose of study drug had been administered. Adverse event reporting continued through the End-of-Treatment Follow-Up Period or until 90 days (± 7 days) after the last dose of study drug, the patient underwent local therapy (e.g., resection, radiation) or a new systemic therapy was initiated.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Phase 1 FPA008 Dose Escalation 1mg/kg | 0/3 (0%) | 0/3 (0%) | 3/3 (100%) |
| Phase 1 FPA008 Dose Escalation 2mg/kg | 0/3 (0%) | 0/3 (0%) | 3/3 (100%) |
| Phase 1 FPA008 Dose Escalation 4mg/kg | 0/3 (0%) | 2/3 (66.7%) | 3/3 (100%) |
| Phase 2 FPA008 Dose Expansion Cohort 2A | 1/32 (3.1%) | 8/33 (24.2%) | 33/33 (100%) |
| Phase 2 FPA008 Dose Expansion Cohort 2B | 0/24 (0%) | 6/24 (25%) | 24/24 (100%) |
| Event | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B |
|---|---|---|---|---|---|
| Abdominal pain upperGastrointestinal disorders | — | — | 1/3 | 0/33 | 0/24 |
| Streptococcal sepsisInfections and infestations | — | — | 1/3 | 0/33 | 0/24 |
| Intervertebral discitisInfections and infestations | — | — | 1/3 | 0/33 | 0/24 |
| EndocarditisInfections and infestations | — | — | 1/3 | 0/33 | 0/24 |
| HypertensionVascular disorders | — | — | 1/3 | 0/33 | 0/24 |
| MyocarditisCardiac disorders | — | — | 0/3 | 2/33 | 0/24 |
| Abdominal painGastrointestinal disorders | — | — | 0/3 | 1/33 | 1/24 |
| ColitisGastrointestinal disorders | — | — | 0/3 | 0/33 | 1/24 |
| DiverticulumGastrointestinal disorders | — | — | 0/3 | 0/33 | 1/24 |
| PyrexiaGeneral disorders | — | — | 0/3 | 1/33 | 1/24 |
| Event | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B |
|---|---|---|---|---|---|
| FatigueGeneral disorders | 2/3 | 1/3 | 3/3 | 11/33 | 8/24 |
| Blood creatine phosphokinase increasedInvestigations | 0/3 | 1/3 | 3/3 | 21/33 | 16/24 |
| Periorbital oedemaEye disorders | 2/3 | 2/3 | 2/3 | 20/33 | 13/24 |
| NauseaGastrointestinal disorders | 2/3 | 0/3 | 1/3 | 5/33 | 3/24 |
| Face oedemaGeneral disorders | 0/3 | 1/3 | 2/3 | 22/33 | 12/24 |
| Oedema peripheralGeneral disorders | 2/3 | 1/3 | 2/3 | 19/33 | 8/24 |
| Alanine aminotransferase increasedInvestigations | 0/3 | 0/3 | 2/3 | 6/33 | 0/24 |
| Aspartate aminotransferase increasedInvestigations | 0/3 | 0/3 | 2/3 | 10/33 | 3/24 |
| DermatitisSkin and subcutaneous tissue disorders | 2/3 | 0/3 | 0/3 | 0/33 | 0/24 |
| Dry skinSkin and subcutaneous tissue disorders | 0/3 | 1/3 | 2/3 | 2/33 | 4/24 |
| Age, Categorical(Participants) | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B | Total |
|---|---|---|---|---|---|---|
| <=18 years | 0 | 0 | 0 | 0 | 0 | 0 |
| Between 18 and 65 years | 3 | 2 | 3 | 32 | 22 | 62 |
| >=65 years | 0 | 1 | 0 | 1 | 2 | 4 |
| Age, Continuous(years) | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B | Total |
|---|---|---|---|---|---|---|
| Mean | 37.7 ± 11.24 | 40.7 ± 23.07 | 41.7 ± 7.51 | 38 ± 12.77 | 44.1 ± 15.32 | 40.8 ± 14.03 |
| Sex: Female, Male(Participants) | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B | Total |
|---|---|---|---|---|---|---|
| Female | 3 | 3 | 1 | 23 | 10 | 40 |
| Male | 0 | 0 | 2 | 10 | 14 | 26 |
| Race/Ethnicity, Customized(Participants) | Phase 1 FPA008 Dose Escalation 1mg/kg | Phase 1 FPA008 Dose Escalation 2mg/kg | Phase 1 FPA008 Dose Escalation 4mg/kg | Phase 2 FPA008 Dose Expansion Cohort 2A | Phase 2 FPA008 Dose Expansion Cohort 2B | Total |
|---|---|---|---|---|---|---|
| American Indian/alaska Native | 0 | 0 | 0 | 0 | 0 | 0 |
| Black | 0 | 0 | 0 | 0 | 2 | 2 |
| Native Hawaiian/Pacific Islander | 0 | 0 | 0 | 0 | 0 | 0 |
| White | 3 | 2 | 2 | 9 | 9 | 25 |
| Asian | 0 | 1 | 0 | 5 | 1 | 7 |
| Other | 0 | 0 | 0 | 19 | 8 | 27 |
| Unknown | 0 | 0 | 1 | 0 | 4 | 5 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Jun 2015 to primary completion in Apr 2020, 58 months.
Results were first posted in Aug 2021, 1 year 4 months after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jul 2020. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Studies of the drug tested here: Cabiralizumab
Synovitis, Pigmented Villonodular →
Five Prime Therapeutics, Inc. →