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Gamma Secretase Inhibitor
Nirogacestat for Aggressive Fibromatosis
Phase 2
Waitlist Available
Led By Keila Torres, MD,PHD
Research Sponsored by M.D. Anderson Cancer Center
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up through study completion; an average of 1 year.
Awards & highlights
Study Summary
This trial examines the safety and effects of a new drug on people with a rare tumor.
Who is the study for?
This trial is for adults with desmoid tumors/aggressive fibromatosis who have either not been treated or whose tumors grew after treatment. They must be able to swallow tablets, sign consent, and have a tumor that can be measured. Participants need proper liver, kidney, and bone marrow function and agree to use contraception. Excluded are pregnant/breastfeeding women, those with certain health conditions or treatments that could interfere with the study.Check my eligibility
What is being tested?
The trial is testing nirogacestat's safety and effects on patients with desmoid tumors. About 40 participants will receive this investigational drug to see how well it works against their tumors which have shown growth despite previous treatments or in those untreated.See study design
What are the potential side effects?
While specific side effects of nirogacestat aren't listed here, similar drugs may cause digestive issues, fatigue, liver problems, blood disorders and increase infection risk. Animal studies suggest potential risks during pregnancy.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ through study completion; an average of 1 year.
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~through study completion; an average of 1 year.
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0
Side effects data
From 2023 Phase 2 trial • 17 Patients • NCT0198155188%
Diarrhea
76%
Headache
76%
Hypophosphatemia
76%
Nausea
71%
Aspartate aminotransferase increased
71%
Lymphocyte count decreased
59%
Rash maculo-papular
53%
Fatigue
53%
Alanine aminotransferase increased
53%
Anemia
53%
Hypocalcemia
53%
Hypokalemia
47%
Hyponatremia
47%
Hot flashes
47%
Pain
47%
Abdominal pain
41%
Rash acneiform
41%
Hypertension
41%
Skin infection
41%
White blood cell decreased
41%
Dry mouth
35%
Vomiting
35%
Myalgia
35%
Fever
35%
Flu like symptoms
35%
Cough
35%
Mucositis oral
35%
Edema limbs
29%
Constipation
29%
Bruising
29%
Alkaline phosphatase increased
29%
Upper respiratory infection
29%
Back pain
29%
Pain in extremity
29%
Hypoalbuminemia
24%
Irregular menstruation
24%
Nasal congestion
24%
Skin and subcutaneous tissue disorders - Other, folliculitis
24%
Epistaxis
24%
Fracture
24%
Activated partial thromboplastin time prolonged
24%
Peripheral sensory neuropathy
24%
Chills
24%
Hyperhidrosis
24%
Memory impairment
24%
Platelet count decreased
24%
Sore throat
24%
Weight loss
24%
Dizziness
24%
Dry skin
18%
Hyperkalemia
18%
Creatinine increased
18%
Infections and infestations - Other, upper respiratory infection
18%
Libido decreased
18%
Menorrhagia
18%
Gastroesophageal reflux disease
18%
Blurred vision
18%
Anorexia
18%
Blood bilirubin increased
18%
Hypercalcemia
18%
Hyperglycemia
18%
Reproductive system and breast disorders - Other, amenorrhea
18%
Anxiety
18%
Neutrophil count decreased
18%
Insomnia
18%
Dehydration
18%
Depression
18%
Dysgeusia
12%
Pharyngitis
12%
Postnasal drip
12%
Vertigo
12%
Weight gain
12%
Productive cough
12%
Sinusitis
12%
Pruritus
12%
Flatulence
12%
Arthralgia
12%
Palpitations
12%
Paresthesia
12%
Thromboembolic event
12%
Urinary tract infection
12%
Allergic rhinitis
12%
Alopecia
12%
Hypermagnesemia
12%
Hypernatremia
12%
Vaginal infection
12%
Skin hyperpigmentation
12%
Cognitive disturbance
12%
Dysmenorrhea
12%
Dyspnea
12%
Ear and labyrinth disorders - Other, eustachian tube dysfunction
6%
Oral hemorrhage
6%
Gastrointestinal disorders - Other, abdominal cramping
6%
Metabolism and nutrition disorders - Other, hyperphosphatemia
6%
Musculoskeletal and connective tissue disorder - Other, right deltoid muscle tenderness
6%
Musculoskeletal and connective tissue disorder - Other, stress injury
6%
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, R paraspinal form tumor
6%
Gingival pain
6%
Hip fracture
6%
Hirsutism
6%
Hypomagnesemia
6%
Injury, poisoning and procedural complications - Other, injury, abdomen contusion
6%
Infections and infestations - Other, rhinitis infective
6%
Infections and infestations - Other, viral gastroenteritis
6%
Postoperative hemorrhage
6%
Reproductive system and breast disorders - Other, menstruation
6%
Skin and subcutaneous tissue disorders - Other, skin lesions
6%
Infections and infestations - Other, fungal infection (ear)
6%
Vaginal dryness
6%
Infections and infestations - Other, oral infections
6%
Infusion site extravasation
6%
Skin and subcutaneous tissue disorders - Other, small hard lump/Rt upper thigh
6%
Infections and infestations - Other, sinus infection
6%
Injury, poisoning and procedural complications - Other, injury, swollen mouth
6%
Reproductive system and breast disorders - Other, elevated LH
6%
Seizure
6%
Supraventricular tachycardia
6%
Infusion related reaction
6%
Investigations - Other, hypophosphatemia
6%
Psychiatric disorders - Other, Mood swings
6%
Injection site reaction
6%
Laryngitis
6%
Joint effusion
6%
Skin and subcutaneous tissue disorders - Other, left groin cystic lesion
6%
Skin and subcutaneous tissue disorders - Other, cyst
6%
Oral pain
6%
Renal and urinary disorders - Other, kidney stones
6%
Respiratory, thoracic and mediastinal disorders - Other, sinus congestion
6%
Skin and subcutaneous tissue disorders - Other, skin keratosis
6%
Periodontal disease
6%
Blood gonadotrophin abnormal
6%
Concentration impairment
6%
Enterocolitis
6%
Reproductive system and breast disorders - Other, elevated FSH
6%
Reproductive system and breast disorders - Other, genital infection
6%
Vaginal pain
6%
Electrocardiogram QT corrected interval prolonged
6%
Gastrointestinal disorders - Other, abdominal discomfort
6%
Gastrointestinal disorders - Other, gastroenteritis
6%
Hematoma
6%
Confusion
6%
Hematuria
6%
Stroke
6%
Atrial fibrillation
6%
Bronchial infection
6%
Bullous dermatitis
6%
Floaters
6%
Endocrine disorders - Other, elevated glucose level
6%
Erythema multiforme
6%
Eye disorders - Other, eye injury
6%
Eye disorders - Other, left eye swelling & discharge
6%
Cardiac disorders - Other, potential murmur/"whooshing"
6%
Conjunctivitis
6%
Cystitis noninfective
6%
Gastrointestinal disorders - Other, canker sores
6%
Hemoglobin increased
6%
Hemorrhoids
6%
Injury, poisoning and procedural complications - Other, specify
6%
Surgical and medical procedures - Other, hysterectomy
6%
Vaginal inflammation
6%
Arthritis
6%
Eye disorders - Other, visual disturbance
6%
Eye disorders - Other, exopthalmos bilateral
6%
Hoarseness
6%
Lung infection
6%
Musculoskeletal and connective tissue disorder - Other, tendinitis left groin/thigh
6%
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, cyst
6%
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, popliteal cysts
6%
Neoplasms benign, malignant and unspecified
6%
Pain of skin
6%
Pelvic pain
6%
Reproductive system and breast disorders - Other, breast tenderness
6%
Reproductive system and breast disorders - Other, R breat lump
6%
Respiratory, thoracic and mediastinal disorders - Other, cold
6%
Respiratory, thoracic and mediastinal disorders - Other, dry nasal passages
6%
Respiratory, thoracic and mediastinal disorders - Other, pneumonia
6%
Sinus tachycardia
6%
Surgical and medical procedures - Other, esophageal dilation
6%
Tinnitus
6%
Urinary frequency
6%
Urticaria
6%
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, squamous cell carcinoma
6%
Amnesia
6%
Bloating
6%
Chest wall pain
6%
Eye disorders - Other, macular degeneration - left eye
6%
Eye disorders - Other, vision changes
6%
Musculoskeletal and connective tissue disorder - Other, tendinitis
6%
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, rt groin area
6%
Nervous system disorders - Other, lightheadedness
6%
Non-cardiac chest pain
6%
Osteoporosis
6%
Renal and urinary disorders - Other, elevated BUN
6%
Surgical and medical procedures - Other, splenectomy
6%
Watering eyes
6%
Lip infection
6%
Lymphocyte count increased
6%
Malaise
6%
Middle ear inflammation
6%
Musculoskeletal and connective tissue disorder - Other, bone spur
6%
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, cyst - right arm
6%
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, specify
6%
Tooth infection
6%
Allergic reaction
6%
Bronchospasm
6%
Facial pain
6%
Nail infection
6%
Pleural effusion
6%
Portal vein thrombosis
6%
Reproductive system and breast disorders - Other, menstrual spotting
6%
Skin and subcutaneous tissue disorders - Other, subcutaneous cysts
6%
Skin ulceration
6%
Soft tissue necrosis upper limb
6%
Stomach pain
6%
Wheezing
6%
Wound infection
6%
Psychiatric disorders - Other, Dreams
6%
Dry eye
6%
Hypoglycemia
6%
Cholesterol high
6%
Dyspepsia
6%
Dysphagia
6%
Ear pain
6%
Edema trunk
100%
80%
60%
40%
20%
0%
Study treatment Arm
PF-03084014 in Desmoid Tumors/Aggressive Fibromatosis
Trial Design
1Treatment groups
Experimental Treatment
Group I: nirogacestatExperimental Treatment1 Intervention
Participants will take nirogacestat by mouth every day of each 28-day study cycle. Based on when you enroll in this study, you will take nirogacestat either 1 time a day at about the same time each day OR 2 times a day, about 12 hours apart.
Find a Location
Who is running the clinical trial?
M.D. Anderson Cancer CenterLead Sponsor
2,971 Previous Clinical Trials
1,787,197 Total Patients Enrolled
9 Trials studying Tumors
8,522 Patients Enrolled for Tumors
SpringWorks Therapeutics, Inc.Industry Sponsor
12 Previous Clinical Trials
926 Total Patients Enrolled
Keila Torres, MD,PHDPrincipal InvestigatorM.D. Anderson Cancer Center
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- My organs and bone marrow are working well.I have a known heart condition or symptoms.I can understand and am willing to sign the consent form.I am experiencing symptoms or health issues due to my condition.I cannot have repeated tissue samples taken.My condition has come back or is newly diagnosed.I have another cancer that won't affect this treatment's safety or results.I have been diagnosed with familial adenomatous polyposis.I can take care of myself but might not be able to do heavy physical work.I need someone legally authorized to make decisions for me due to cognitive impairment.I am 18 years old or older.I am not on any medications that are not allowed in the study.I have had significant health events in the last 6 months.I have not been treated or my condition worsened after treatment for Desmoid Tumor.My tumor can be easily reached for multiple biopsy samples.I agree to provide a new sample of my tumor for testing.I can swallow pills and don't have stomach issues affecting drug absorption.I have recovered from side effects of previous cancer treatments, except for hair loss.My doctor recommends treatment that affects my whole body.I have cancer that has spread to my brain and another type of cancer.I can communicate in English or another language.My tumor has grown in the last 18 months, confirmed by tests.I am HIV positive and on treatment, but still have a detectable viral load.I have been diagnosed with Desmoid Tumors (DT).
Research Study Groups:
This trial has the following groups:- Group 1: nirogacestat
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Is nirogacestat a safe treatment option for patients?
"Due to the lack of efficacy data, nirogacestat was given a rating of 2 on our risk scale. Nevertheless, there is some evidence indicating that this substance does not pose an unreasonable safety concern."
Answered by AI
Is this clinical trial currently accepting participants?
"Data hosted on clinicaltrials.gov suggests that enrollment for this medical trial is currently suspended as the last update was posted on May 18th, 2023. Despite this, there are 45 other trials recruiting candidates at present."
Answered by AI
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