Adults 18 to 120, any sex, with Desmoid Tumors or Aggressive Fibromatosis. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Number of Participants With a Complete Response (CR) + Partial Response (PR)Primary· 20 months
Complete Response + Partial Response was determined by the Response Evaluation Criteria in Solid Tumors (RECIST). Complete Response is disappearance of all target lesions. Any pathological lymph nodes (whether target or non-target) must have reduction in short axis to \<10 mm. Partial Response is at least a 30% decrease in the sum of the diameters of target lesions, taking as reference the baseline sum diameters. Progressive Disease (PD) is at least a 20% increase in the sum of the diameters of target lesions, taking as reference the smallest sum on study (this includes the baseline sum if tha
Complete Response
Group
Value
95% CI
PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis
0
Partial Response
Group
Value
95% CI
PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis
5
Not Evaluable
Group
Value
95% CI
PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis
1
Stable Disease
Group
Value
95% CI
PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis
11
Progressive Disease
Group
Value
95% CI
PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis
0
Number of Participants With Serious and Non-serious Adverse EventsSecondary· Date treatment consent signed to date off study, approximately 66 months and 27 days.
Here is the count of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria in Adverse Events (CTCAE v4.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one o
Group
Value
95% CI
PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis
17
Number of Participants With Somatic or Germline Mutations Identified in Adenomatous Polyposis Coli Gene (APC) or Catenin Beta-1 (CTNNB1) GenesSecondary· Baseline
Tumor and blood samples were obtained from participants and genotyped for somatic and germline mutations.
Group
Value
95% CI
PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis
15
Mean MD Anderson Symptom Inventory Scores After TreatmentSecondary· At baseline prior to drug administration and at least every 6 cycles of drug (18 weeks) up to 20 months of treatment.
The MD Anderson Symptoms Inventory (MDSAI) questionnaire includes questions regarding 13 symptoms commonly experienced by patients with cancer and 6 additional items that assess the extent to which these symptoms interfered with how patients felt and were able to function. The 0-10 scale (0=none, 1-4=mild, 5-6=moderate, and 7-10=severe) assesses how patients felt and were able to function over the previous 24 hours. A component score representing symptom severity is obtained by taking the average of the 13 symptom items (e.g., pain, fatigue, etc.) together. A component score representing sympt
Symptom Severity Score
Group
Value
95% CI
PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis
1.37
± 1.46
Symptom Interference Score
Group
Value
95% CI
PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis
1.57
± 2.69
Adverse events — posted to ClinicalTrials.gov
Time frame: Date treatment consent signed to date off study, approximately 66 months and 27 days..
Reporting threshold: 0%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis
Serious: 9/17 (53%)
Deaths: 1/17
Serious adverse events (13 terms)
Reaction
System
PF-03084014 in Desmoid Tum…
Allergic reaction
Immune system disorders
—
Back pain
Musculoskeletal and connective tissue disorders
—
Blood bilirubin increased
Investigations
—
Bronchospasm
Respiratory, thoracic and mediastinal disorders
—
Constipation
Gastrointestinal disorders
—
Fever
General disorders
—
Hematoma
Vascular disorders
—
Hematuria
Renal and urinary disorders
—
Injury, poisoning and procedural complications - Other, specify
Injury, poisoning and procedural complications
—
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, squamous cell carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
—
Stroke
Nervous system disorders
—
Surgical and medical procedures - Other, hysterectomy
Surgical and medical procedures
—
Vaginal inflammation
Reproductive system and breast disorders
—
Other adverse events (217 terms — click to expand)
Background:
* Desmoid tumors (also known as aggressive fibromatosis), are rare, locally invasive, slow-growing soft-tissue tumors. The disease can be either asymptomatic or be associated with severe loss of organ function and significant morbidity.
* Treatment with the selective small-molecule Gamma-secretase inhibitor PF-03084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early phase clinical trial.
* The Notch pathway is a key regulator of cell differentiation, proliferation and apoptosis; aberrant signaling via the Notch pathway is associated with carcinogenesis.
Objectives:
* Primary: Determine the response rate (Complete Response (CR)+Partial Response (PR)) of PF-03084014 in patients with desmoid tumors/aggressive fibromatosis
* Secondary: Assess symptom measures at baseline and on study; perform genotyping for germline and somatic mutations in adenomatous polyposis coli gene (APC) and catenin-beta 1 (CTNNB1) genes; correlate clinical response to therapy with genotyping data; and assess modulation of the Notch pathway by evaluating notch response genes in tumor biopsies at baseline and after drug administration
Eligibility:
* Age greater than or equal to18; histologically confirmed desmoid tumor not amenable to curative resection or definitive radiation therapy that has progressed after receiving at least one line of standard treatment; adequate organ function
* Willingness to provide blood samples and 10 unstained slides or a tumor block for genetic research studies
Study Design:
* This is an open-label Phase II trial of PF-03084014; study drug will be administered orally at 150 mg twice a day in 21-day cycles
* Optional tumor biopsies for research purposes will be performed at baseline prior to study treatment and at the beginning of cycle 7 (+/- one cycle)
* Restaging scans (magnetic resonance imaging (MRI) with diffusion weighting) will be performed at baseline, at the end of cycles 1 and 6, and then every 6 cycles
* Health-related quality of life (HRQOL)/symptom questionnaires will be administered at baseline and at each Clinical Center visit
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
NCT02299635 — A Study Evaluating PF-03084014 In Patients With Advanced Breast Cancer With Or Without Notch Alterations
· Phase 2
· terminated
NCT02109445 — Study Of PF-03084014 In Combination With Gemcitabine And Nab-Paclitaxel In Patients With Metastatic Pancreatic Adenocarc
· Phase 2
· terminated
NCT01876251 — A Study Evaluating The PF-03084014 In Combination With Docetaxel In Patients With Advanced Breast Cancer
· Phase 1
· terminated
NCT00878189 — A Trial In Patients With Advanced Cancer And Leukemia
· Phase 1
· completed
NCT02955446 — Compassionate Use Protocol for PF-03084014 in Patients With Advanced Solid Tumor Malignancies
· no longer available
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Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by National Cancer Institute (NCI)
Last refreshed: 6 February 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT01981551.