A Phase 2 interventional study of PF-03084014 in Desmoid Tumors and Aggressive Fibromatosis, sponsored by National Cancer Institute (NCI). Completed at 1 site in United States. Open to participants aged 18 Years to 120 Years. Per ClinicalTrials.gov, last updated 2024-02-06.
Sponsored by National Cancer Institute (NCI) · Phase 2, Interventional, and Treatment
Among 33 studies in Desmoid Tumors with a site in United States — 6 open to participants now, 9 under way, 1 of them phase 3. Desmoid Tumors · United States.
Among the 33 studies in Desmoid Tumors with a site in United States, this one is the 19th largest by enrollment of the 24 interventional ones that give a count, is the only one that started in 2013, is one of 8 in phase 2, and was the 11th registered.
71 studies on the registry are indexed under Desmoid Tumors; 21 are open to participants now.
Browse Desmoid Tumors studies →6 of the 33 studies in Desmoid Tumors with a site in United States are open now. Recruiting first, the newest first:
Nam Bui · Sites in United States
Ages 18+
Parabilis Medicines, Inc. · Sites in United States, Australia
Ages 18+
Children's Oncology Group · Sites in United States
Ages 12 mo–30 yrs
Memorial Sloan Kettering Cancer Center · Sites in United States
Ages 18+
Children's Oncology Group · Sites in United States, Australia, Canada and 3 more countries
Ages up to 25
A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →
Of the 17 Desmoid Tumors studies recruiting now, 13 (76%) set no upper age limit and 4 (24%) stop at 75 or younger; 6 take children under 18; none take healthy volunteers.
This study: 18 Years to 120 Years · all sexes · no healthy volunteers.
The full table on the hub →This tab as a report to print or save as PDF → With an account.
33 studies; the count open now beside each. This study's row is marked.
A study in two phases is counted once above. Listed by phase, each with its combined phases: Phase 1 10 · Phase 2 13 · Phase 3 4
First in 1991; this study’s year is marked. 1 started in 2013.
By drug class, each counting the studies that test any of its medicines:
This study's enrollment of 17 is below the median of 44 across 47 interventional studies indexed under Desmoid Tumors.
Median enrollment 43 across the 24 interventional studies, 150 across the 7 observational. Of the 20 that completed or were terminated, 9 (45%) have results posted.
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6 of 33 have an industry lead sponsor. The 10 most frequent:
National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.
Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) have results posted.
National Cancer Institute (NCI) has 4 studies filed under Desmoid Tumors — fewer than ten, so there is no comparison to draw.
2 of the studies in Desmoid Tumors with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT04281381Memorial Sloan Kettering Cancer CenterObserving People With Desmoid-Type Fibromatosis | 2027-02-19 |
| NCT05919264Parabilis Medicines, Inc.FOG-001 in Locally Advanced or Metastatic Solid Tumors | 2027-04-01 |
Add all 2 to your calendar (.ics) → With an account.
Among the 33 studies in Desmoid Tumors with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
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Background:
Objectives:
Eligibility:
Study Design:
Background:
Objectives:
Eligibility:
Study Design:
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.
2.1.1.1 Patients must have confirmed desmoid tumor confirmed by the Laboratory of Pathology, National Cancer Institute (NCI), that has progressed after receiving at least one line of standard treatment and that is not amenable to surgical resection or definitive radiation therapy.
2.1.1.2 Willingness to provide blood samples and 10 unstained slides or a tumor block for genetic research studies.
2.1.1.3 Any with prior desmoid therapy, including radiotherapy, should have been completed at least 2 weeks before study entry and all toxicities must have resolved at least to eligibility levels.
2.1.1.4 Age greater than or equal 18 years; because no dosing or adverse event data are currently available on the use of PF-03084014 in patients \<18 years of age, children are excluded from this study, but may be eligible for future pediatric trials.
2.1.1.5 (ECOG) performance status less than or equal 2.
2.1.1.6 > 3 months.
2.1.1.7 Patients must have normal organ and marrow function as defined below:
OR
2.1.1.8 Patients must be able to swallow whole tablets or capsules with no gastrointestinal (GI) condition affecting absorption; nasogastric or G-tube administration is not allowed.
2.1.1.9 The effects of PF-03084014 on the developing human fetus are unknown. For this reason and because >=-secretase inhibitors are known to be teratogenic, women of and men must agree to use adequate (hormonal or barrier method of birth control; abstinence) for the duration of study participation and for at least 6 months after dosing with study drugs ceases. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately. Men treated or enrolled on this protocol must also agree to use adequate contraception for the duration of study participation, and 6 months after completion of study drug administration.
2.1.1.10 Ability to understand and the willingness to sign a written document.
2.1.1.11 Evidence of by computed tomography (CT) scan. Measurable lesions are defined as those that can be accurately measured in at least one dimension (longest diameter to be recorded) as greater than or equal 20 mm by chest x-ray, as greater than or equal 10 mm with CT scan, or greater than or equal 10 mm with calipers by clinical exam.
EXCLUSION CRITERIA:
2.1.2.1 Patients who are receiving any other agents. Concurrent mediations that the patient is taking will be reviewed by the principal investigator (PI) to assess safety and eligibility.
2.1.2.2 Prior treatment with Gamma-secretase inhibitors or anti-notch antibody therapy.
2.1.2.3 Uncontrolled intercurrent illness including, but not limited to, serious infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
2.1.2.4 interval (QTc) interval of >470 msec at study entry; congenital long QT syndrome.
2.1.2.5 Pregnant women are excluded from this study because PF-03084014 is a Gamma-secretase inhibitor with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with PF-03084014, breastfeeding should be discontinued if the mother is treated with PF-03084014.
2.1.2.6 Patients with gastrointestinal conditions that might predispose for drug intolerability or poor drug absorption (e.g., inability to take oral medication or a requirement for intravenous (IV) alimentation, prior surgical procedures affecting absorption, malabsorption syndrome, and active peptic ulcer disease) are excluded. Subjects with ulcerative colitis, inflammatory bowel disease, or a partial or complete small bowel obstruction are also excluded, as are any patients who cannot swallow the tablet whole. Tablets must not be crushed or chewed; nasogastric or gastrostomy (G)-tube administration is not allowed.
2.1.2.7 (HIV)-positive patients on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with PF-03084014.
2.1.3 Recruitment Strategies
We have had multiple discussions with the Desmoid Tumor Research Foundation, Inc., who have various outreach efforts including patient meetings and webcasts. Senior executives of the Foundation have indicated strong interest in this trial and willingness to inform their members. We also have a network of referring physicians nationally that refers patients with solid tumors to our clinical program. Given our interest and trials for sarcomas, we have formed connections with centers that treat patients with mesenchymal . We will be informing all of these of the availability of this trial once it is open.
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.
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
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.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
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.
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.
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.
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — 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.
Line of therapy
A line of therapy is one course of treatment, which may combine several drugs. First-line is the first course given; second-line follows when that stops working or is not tolerated.
“Two prior lines” means two courses that count separately under the study’s rules — not simply two drugs. Ask the study team if you are unsure how yours are counted.
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.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Absolute neutrophil count (ANC)
The number of neutrophils, white blood cells that help fight infection, in a given amount of blood. Too few raises the risk of infection.
It is a count, not a percentage of the white cells. Limits are written in units that mean the same: ≥ 1.5 × 10⁹/L is ≥ 1,500 per mm³ (µL). Each lab has its own reference range.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Platelets
Blood cells that help blood clot. Too few raises the risk of bleeding and bruising. A usual adult range is about 150–400 × 10⁹/L (150,000–400,000 per µL); each lab has its own reference range.
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.
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.
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.
Creatinine clearance (CrCl)
How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hemoglobin (Hb)
The protein in red blood cells that carries oxygen. A low level is anaemia, which can cause tiredness and breathlessness. Measured in g/dL or g/L (9 g/dL is 90 g/L).
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Childbearing potential
Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).
Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Measurable disease
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
QT and QTc
On an ECG, the QT interval is the time the heart’s lower chambers take to activate electrically and recover in each beat. QTc is that time adjusted for heart rate (QTcF by Fridericia’s formula, QTcB by Bazett’s). Some medicines lengthen it, and a long QTc can raise the risk of a dangerous heart rhythm.
The study may name a correction formula and a limit in milliseconds, often around 450–480.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
PF-03084014 will be administered orally at 150 mg twice a day in 21-day cycles
Drug: PF-03084014
Treatment with the selective small-molecule \>=-secretase inhibitor PF- 3084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early-phase clinical trial.
Number of Participants With a Complete Response (CR) + Partial Response (PR)
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 that is the smallest on study). The sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered progressions. Stable Disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.
Time frame: 20 months
Number of Participants With Serious and Non-serious Adverse Events
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 of the previous outcomes mentioned.
Time frame: Date treatment consent signed to date off study, approximately 66 months and 27 days.
Number of Participants With Somatic or Germline Mutations Identified in Adenomatous Polyposis Coli Gene (APC) or Catenin Beta-1 (CTNNB1) Genes
Tumor and blood samples were obtained from participants and genotyped for somatic and germline mutations.
Time frame: Baseline
Mean MD Anderson Symptom Inventory Scores After 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 symptom distress is obtaining by averaging the 6 symptom interference items (e.g., general activity, mood, etc.).
Time frame: At baseline prior to drug administration and at least every 6 cycles of drug (18 weeks) up to 20 months of treatment.
| Milestone | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| Started | 17 |
| Completed | 8 |
| Not completed | 9 |
| Withdrew: Refused further treatment | 8 |
| Withdrew: Adverse event | 1 |
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 that is the smallest on study). The sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered progressions. Stable Disease (SD) is neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, taking as reference the smallest sum diameters while on study.
| Participants | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| Complete Response | 0 |
| Partial Response | 5 |
| Not Evaluable | 1 |
| Stable Disease | 11 |
| Progressive Disease | 0 |
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 of the previous outcomes mentioned.
| Participants | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| Number of Participants With Serious and Non-serious Adverse Events | 17 |
Tumor and blood samples were obtained from participants and genotyped for somatic and germline mutations.
| Participants | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| Number of Participants With Somatic or Germline Mutations Identified in Adenomatous Polyposis Coli Gene (APC) or Catenin Beta-1 (CTNNB1) Genes | 15 |
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 symptom distress is obtaining by averaging the 6 symptom interference items (e.g., general activity, mood, etc.).
| Scores on a scale | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| Symptom Severity Score | 1.37 ± 1.46 |
| Symptom Interference Score | 1.57 ± 2.69 |
Collected over Date treatment consent signed to date off study, approximately 66 months and 27 days.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis | 1/17 (5.9%) | 9/17 (52.9%) | 17/17 (100%) |
| Event | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| Allergic reactionImmune system disorders | 1/17 |
| Back painMusculoskeletal and connective tissue disorders | 1/17 |
| Blood bilirubin increasedInvestigations | 1/17 |
| BronchospasmRespiratory, thoracic and mediastinal disorders | 1/17 |
| ConstipationGastrointestinal disorders | 1/17 |
| FeverGeneral disorders | 1/17 |
| HematomaVascular disorders | 1/17 |
| HematuriaRenal and urinary disorders | 1/17 |
| Injury, poisoning and procedural complications - Other, specifyInjury, poisoning and procedural complications | 1/17 |
| Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, squamous cell carcinomaNeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/17 |
| Event | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| DiarrheaGastrointestinal disorders | 15/17 |
| HeadacheNervous system disorders | 13/17 |
| HypophosphatemiaMetabolism and nutrition disorders | 13/17 |
| NauseaGastrointestinal disorders | 13/17 |
| Aspartate aminotransferase increasedInvestigations | 12/17 |
| Lymphocyte count decreasedInvestigations | 12/17 |
| Rash maculo-papularSkin and subcutaneous tissue disorders | 10/17 |
| Alanine aminotransferase increasedInvestigations | 9/17 |
| AnemiaBlood and lymphatic system disorders | 9/17 |
| FatigueGeneral disorders | 9/17 |
| Age, Categorical(Participants) | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| <=18 years | 0 |
| Between 18 and 65 years | 16 |
| >=65 years | 1 |
| Age, Continuous(years) | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| Mean | 39.02 ± 14.47 |
| Sex: Female, Male(Participants) | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| Female | 14 |
| Male | 3 |
| Ethnicity (NIH/OMB)(Participants) | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| Hispanic or Latino | 0 |
| Not Hispanic or Latino | 17 |
| Unknown or Not Reported | 0 |
| Race (NIH/OMB)(Participants) | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| American Indian or Alaska Native | 0 |
| Asian | 0 |
| Native Hawaiian or Other Pacific Islander | 0 |
| Black or African American | 2 |
| White | 15 |
| More than one race | 0 |
| Unknown or Not Reported | 0 |
| Region of Enrollment(participants) | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| United States | 17 |
| Mean MD Anderson Symptom Inventory Scores at Baseline(Scores on a scale) | PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis |
|---|---|
| Symptom Severity Score | 2.0 ± 1.39 |
| Symptom Interference Score | 2.94 ± 2.50 |
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: Yes — All individual participant data (IPD) recorded in the medical record will be shared with intramural investigators upon request.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form
Time frame: Clinical data available during the study and indefinitely.
Access criteria: Clinical data will be made available via subscription to Biomedical Translational Research Information System (BTRIS) and with the permission of the study principal investigator (PI).
Ran from Oct 2013 to primary completion in Dec 2018, 61 months.
Results were first posted in Feb 2020, 1 year 1 month after primary completion.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Jan 2024. 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.
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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: Nirogacestat
National Cancer Institute (NCI) →