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This is a randomized, double-blind, vehicle-controlled, parallel group dose response study evaluating the safety and effectiveness of 2 concentrations of NFX-179 gel in participants with cNF. At Visit 1 (Screening visit), the investigator will identify 10 Target cNFs that fulfil the enrollment criteria. The Target cNFs must be located on the participant's face, anterior trunk, or upper extremities. Two Target cNFs must be on the face and 8 must be on the anterior trunk or upper extremities. The study medication will be applied topically once daily to the Target cNFs for 182 days (26 weeks). During the duration of the study participants will be evaluated for safety and efficacy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| NFX-179 Topical Gel 0.5% | Experimental | NFX-179 topical gel 0.5% will be applied once daily to the target cutaneous neurofibromas (cNFs) for 182 days. |
|
| NFX-179 Topical Gel 1.5% | Experimental | NFX-179 topical gel 1.5% will be applied once daily to the target cNFs for 182 days. |
|
| NFX-179 Topical Gel Vehicle | Placebo Comparator | NFX-179 topical gel vehicle will be applied once daily to the target cNFs for 182 days. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NFX-179 gel | Drug | NFX-179 topical gel is the active investigational product being studied |
|
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) | An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. A TEAE was an event that occurred or worsened on or after the first dose of study drug. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Baseline (Day 1) up to Day 211 |
| Percentage of Participants With At Least 50% of Target Lesions With 50% Reduction in cNF Volume Above the Surrounding Non-tumor Skin | Treatment effectiveness was measured by the percentage of treated participants with at least 50% of target lesions with 50% reduction in cNF volume after 6 months of treatment. Tumor dimensions were measured using a standardized ruler. | Day 182 |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants With At Least 50% of Target Lesions With 50% Reduction in cNF Height Above the Surrounding Non-tumor Skin | Treatment effectiveness was measured by the percentage of treated participants with at least 50% of target lesions with 50% reduction in cNF height after 6 months of treatment. Tumor dimensions were measured using a standardized ruler. | Day 182 |
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Inclusion Criteria:
Participant is at least 18 years of age
Participant must provide written informed consent prior to any study procedures
Participant must have a clinical diagnosis of NF1
Participant has 10 clinically diagnosed Target cNFs with preferably 2 Target cNFs located on the face and 8 Target cNFs located on the anterior trunk or upper extremities. Alternatively, at least 1 Target cNF is located on the face, in which case 9 Target cNFs must be located on the anterior trunk or upper extremities. Each Target cNF must meet the following criteria:
Participant agrees to avoid exposure of Target cNFs to excessive sunlight and to use her/his routine sunscreen if excessive exposure cannot be avoided
Participant agrees not to use tanning beds
Participant is willing to forego treatment of each Target cNF, except protocol specified therapy, during the study
Female participants who are women of childbearing potential must have a negative urine pregnancy test result and be willing to use a protocol approved, contraceptive method for the duration of the study
Participant is willing and able to follow all study instructions and to attend all study visits.
Exclusion Criteria:
Participant has used any of the following topical therapies within the specified period prior to Visit 1 (screening visit) on or in proximity to any Target cNF that, in the investigator's opinion, impairs evaluation of any the cNFs or which exposes the participant to an unacceptable risk by study participation:
The participant has used any of the following systemic medications therapies within the specified period prior to Visit 1 (screening visit):
Participant has a history of hypersensitivity to any of the ingredients in the study medications
Participant has any known intercurrent illness or physical condition that would, in the investigator's opinion, impair evaluation of a Target cNF or which exposes the participant to an unacceptable risk by study participation
Participant has, in the investigator's opinion, clinically relevant history of liver disease, including viral hepatitis, current alcohol abuse, or cirrhosis
Participant has a history of metastatic disease, or active cancer (excluding nonmelanoma skin cancer, Stage I cervical cancer, ductal carcinoma in situ of the breast, or Stage 0 chronic lymphocytic lymphoma) within the previous 5 years
Participant has any condition (for example, other skin conditions or diseases, metabolic dysfunction, physical examination findings, clinical laboratory findings) or situation (for example, vacation, scheduled surgery) that would, in the investigator's opinion, impair evaluation of a Target cNF or which exposes the participant to an unacceptable risk by study participation
Participant has participated in an investigational drug trial in which administration of an investigational study medication occurred within the previous 30 days
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Arkansas for Medical Sciences | Little Rock | Arkansas | 72205-7199 | United States | ||
| Northwest Arkansas Clinical Trials Center, PLLC |
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| ID | Title | Description |
|---|---|---|
| FG000 | NFX-179 Topical Gel Vehicle | NFX-179 topical gel vehicle was applied once daily to the target cutaneous neurofibromas (cNFs) for 182 days. |
| FG001 | NFX-179 Topical Gel 0.5% | NFX-179 topical gel 0.5% was applied once daily to the target cNFs for 182 days. |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Nov 6, 2023 |
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| Vehicle gel | Drug | NFX-179 vehicle gel is the placebo comparator for this study |
|
|
| Mean Percent Change From Baseline in cNF Volume at Day 182 Based on cNF Volume Derived From Ruler Measurements | Mean percent change of tumor volume was calculated from ruler measurements throughout the course of treatment. Presented here are data analyzed at the participant level. | Baseline, Day 182 |
| Change From Baseline in Tumor Severity Score Per Physician's Tumor Assessment (PTA) at Day 182 | Effect of treatment with the PTA was the investigator's assessment of the average overall severity of each Target cNF tumor at a particular time point. The PTA is a 5-point measuring tumor severity (0 = clear/none, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe). Presented here are data analyzed at the participant level. | Baseline, Day 182 |
| Change From Baseline in Tumor Severity Score Per Subject's Self-Assessment (SSA) at Day 182 | The SSA was the participant's assessment of the average overall severity of each Target cNF at a particular time point. The SSA is a 5-point measuring tumor severity (0 = clear/none, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe). | Baseline, Day 182 |
| Rogers |
| Arkansas |
| 72758 |
| United States |
| Center for Dermatology Clinical Research, Inc. | Fremont | California | 94538 | United States |
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | United States |
| University Clinical Trials, Inc. | San Diego | California | 92123 | United States |
| Children's National Hospital | Washington D.C. | District of Columbia | 20010 | United States |
| University of Florida | Gainesville | Florida | 32610 | United States |
| Northshore University HealthSystem | Evanston | Illinois | 60077 | United States |
| Dawes Fretzin Clinical Research Group | Indianapolis | Indiana | 46250 | United States |
| The Johns Hopkins School of Medicine | Baltimore | Maryland | 21287 | United States |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | United States |
| Minnesota Clinical Study Center | New Brighton | Minnesota | 55112 | United States |
| Skin Specialists, P.C. | Omaha | Nebraska | 68144 | United States |
| Sadick Research Group, LLC | New York | New York | 10075 | United States |
| Skin Search of Rochester, Inc. | Rochester | New York | 14623 | United States |
| Wake Forest School of Medicine | Winston-Salem | North Carolina | 27157 | United States |
| Apex Clinical Research Center, LLC | Mayfield Heights | Ohio | 44124 | United States |
| Oregon Health & Science University | Portland | Oregon | 97239 | United States |
| Derm Dox Center for Dermatology | Sugarloaf | Pennsylvania | 18249 | United States |
| Dermatology Treatment and Research Center | Dallas | Texas | 75230 | United States |
| UTHealth McGovern Medical School | Houston | Texas | 77030 | United States |
| University of Utah | Salt Lake City | Utah | 84108 | United States |
| The Education & Research Foundation, Inc. | Lynchburg | Virginia | 24501 | United States |
| FG002 | NFX-179 Topical Gel 1.5% | NFX-179 topical gel 1.5% was applied once daily to the target cNFs for 182 days. |
| Received at Least 1 Dose of Study Drug |
|
| COMPLETED |
|
| NOT COMPLETED |
|
|
Safety Population included all randomized participants who received at least 1 confirmed dose of study drug and had at least 1 post-baseline safety evaluation.
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| ID | Title | Description |
|---|---|---|
| BG000 | NFX-179 Topical Gel Vehicle | NFX-179 topical gel vehicle was applied once daily to the target cNFs for 182 days. |
| BG001 | NFX-179 Topical Gel 0.5% | NFX-179 topical gel 0.5% was applied once daily to the target cNFs for 182 days. |
| BG002 | NFX-179 Topical Gel 1.5% | NFX-179 topical gel 1.5% was applied once daily to the target cNFs for 182 days. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
| ||||||||||||||||
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) | An adverse event (AE) was defined as any untoward medical occurrence associated with the use of a drug in humans, whether or not considered drug related. A TEAE was an event that occurred or worsened on or after the first dose of study drug. A summary of all Serious Adverse Events and Other Adverse Events (nonserious) regardless of causality is located in the 'Reported Adverse Events' Section. | Safety Population included all randomized participants who received at least 1 confirmed dose of study drug, and had at least 1 post-baseline safety evaluation. | Posted | Count of Participants | Participants | Baseline (Day 1) up to Day 211 |
|
|
| ||||||||||||||||||||||||||||||||
| Primary | Percentage of Participants With At Least 50% of Target Lesions With 50% Reduction in cNF Volume Above the Surrounding Non-tumor Skin | Treatment effectiveness was measured by the percentage of treated participants with at least 50% of target lesions with 50% reduction in cNF volume after 6 months of treatment. Tumor dimensions were measured using a standardized ruler. | The observed cases (OC) population included all participants who were randomized and dispensed study medication and had at least 1 post-baseline visit observed efficacy data at Day 182. | Posted | Number | percentage of participants | Day 182 |
| ||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants With At Least 50% of Target Lesions With 50% Reduction in cNF Height Above the Surrounding Non-tumor Skin | Treatment effectiveness was measured by the percentage of treated participants with at least 50% of target lesions with 50% reduction in cNF height after 6 months of treatment. Tumor dimensions were measured using a standardized ruler. | The OC population included all participants who were randomized and dispensed study medication and had at least 1 post-baseline visit observed efficacy data at Day 182. | Posted | Number | percentage of participants | Day 182 |
| ||||||||||||||||||||||||||||||||||
| Secondary | Mean Percent Change From Baseline in cNF Volume at Day 182 Based on cNF Volume Derived From Ruler Measurements | Mean percent change of tumor volume was calculated from ruler measurements throughout the course of treatment. Presented here are data analyzed at the participant level. | The OC population included all participants who were randomized and dispensed study medication and had at least 1 post-baseline visit observed efficacy data at Day 182. Data are reported at participant-level, not tumor-level. | Posted | Mean | Standard Deviation | percent change | Baseline, Day 182 |
| |||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Tumor Severity Score Per Physician's Tumor Assessment (PTA) at Day 182 | Effect of treatment with the PTA was the investigator's assessment of the average overall severity of each Target cNF tumor at a particular time point. The PTA is a 5-point measuring tumor severity (0 = clear/none, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe). Presented here are data analyzed at the participant level. | The OC population included all participants who were randomized and dispensed study medication and had at least 1 post-baseline visit observed efficacy data at Day 182. 'Overall number of participants analyzed' = participants evaluable for this outcome measure. Data are reported at participant-level, not tumor-level. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Day 182 |
| |||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Tumor Severity Score Per Subject's Self-Assessment (SSA) at Day 182 | The SSA was the participant's assessment of the average overall severity of each Target cNF at a particular time point. The SSA is a 5-point measuring tumor severity (0 = clear/none, 1 = almost clear, 2 = mild, 3 = moderate, and 4 = severe). | The OC population included all participants who were randomized and dispensed study medication and had at least 1 post-baseline visit observed efficacy data at Day 182. 'Overall number of participants analyzed' = participants evaluable for this outcome measure. Data are reported at participant-level, not tumor-level. | Posted | Mean | Standard Deviation | units on a scale | Baseline, Day 182 |
|
Baseline (Day 1) up to Day 211
Safety Population included all randomized participants who received at least 1 confirmed dose of study drug and had at least 1 post-baseline safety evaluation.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | NFX-179 Topical Gel Vehicle | NFX-179 topical gel vehicle was applied once daily to the target cNFs for 182 days. | 0 | 67 | 3 | 67 | 35 | 67 |
| EG001 | NFX-179 Topical Gel 0.5% | NFX-179 topical gel 0.5% was applied once daily to the target cNFs for 182 days. | 1 | 66 | 4 | 66 | 36 | 66 |
| EG002 | NFX-179 Topical Gel 1.5% | NFX-179 topical gel 1.5% was applied once daily to the target cNFs for 182 days. | 1 | 66 | 3 | 66 | 49 | 66 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Influenza | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Hip fracture | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Intervertebral disc disorder | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Ischaemic stroke | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Acute respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Postoperative ileus | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Hypoxia | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Traumatic intracranial haemorrhage | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Central nervous system lesion | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Major depression | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Chest pain | Cardiac disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hydrocele | Congenital, familial and genetic disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Meningocele | Congenital, familial and genetic disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Neurofibromatosis | Congenital, familial and genetic disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Eye haemorrhage | Eye disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Retinal detachment | Eye disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Barrett"s oesophagus | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Chapped lips | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Pancreatic failure | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Umbilical hernia | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Gait disturbance | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Oedema | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Pain | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Procedural pain | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Vaccination site pain | General disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hepatitis alcoholic | Hepatobiliary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Seasonal allergy | Immune system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Body tinea | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| COVID-19 | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Conjunctivitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Cystitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Folliculitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Fungal skin infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Gastroenteritis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Herpes zoster | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Lip infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Oral herpes | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Otitis externa | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Otitis media | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Pharyngitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Pharyngitis streptococcal | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Respiratory tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Scrotal cellulitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Sepsis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Sinusitis bacterial | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 24.0 | Systematic Assessment |
| |
| Accidental exposure to product | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Animal bite | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Facial bones fracture | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Head injury | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Ligament sprain | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Limb injury | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Lip injury | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Meniscus injury | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Suture related complication | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Tooth fracture | Injury, poisoning and procedural complications | MedDRA 24.0 | Systematic Assessment |
| |
| Alanine aminotransferase increased | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| Blood bilirubin increased | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| Blood lactate dehydrogenase increased | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| Blood potassium increased | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| Brain scan abnormal | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| Urine analysis abnormal | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| White blood cell count decreased | Investigations | MedDRA 24.0 | Systematic Assessment |
| |
| Hypercholesterolaemia | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hyperglycaemia | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hyperuricaemia | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hypokalaemia | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hyponatraemia | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hypophosphataemia | Metabolism and nutrition disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Arthritis | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Musculoskeletal stiffness | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Osteoporosis | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Trigger finger | Musculoskeletal and connective tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Basal cell carcinoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Breast cancer | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Breast neoplasm | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Meningioma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Neurofibroma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Schwannoma | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Squamous cell carcinoma of skin | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 24.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hypoaesthesia | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Migraine | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Neurofibroma | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Neuropathy peripheral | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Sciatic nerve neuropathy | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Seizure | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Trigeminal neuralgia | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Vertebral artery occlusion | Nervous system disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Depression | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Bladder prolapse | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Polyuria | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Urinary incontinence | Renal and urinary disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Breast haematoma | Reproductive system and breast disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Prostatitis | Reproductive system and breast disorders | MedDRA 24.0 | Systematic Assessment | This is a sex-specific AE. Only male participants were at risk. |
|
| Scrotal pain | Reproductive system and breast disorders | MedDRA 24.0 | Systematic Assessment | This is a sex-specific AE. Only male participants were at risk. |
|
| Asthma | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Chronic obstructive pulmonary disease | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hiccups | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Rhinitis allergic | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Sleep apnoea syndrome | Respiratory, thoracic and mediastinal disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Application site discolouration | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Blister | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Cellulitis | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dermatitis | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dermatitis allergic | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dermatitis psoriasiform | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dry skin | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Eczema | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Eczema nummular | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Pain of skin | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Photosensitivity reaction | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Rash | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Rash macular | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Rash maculo-papular | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Rash pustular | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Scab | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Skin abrasion | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Skin burning sensation | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Skin erosion | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Skin exfoliation | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Skin haemorrhage | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Skin irritation | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Skin oedema | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Skin reaction | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Skin ulcer | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Swelling face | Skin and subcutaneous tissue disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Dental cosmetic procedure | Surgical and medical procedures | MedDRA 24.0 | Systematic Assessment |
| |
| Large intestinal polypectomy | Surgical and medical procedures | MedDRA 24.0 | Systematic Assessment |
| |
| Lipoma excision | Surgical and medical procedures | MedDRA 24.0 | Systematic Assessment |
| |
| Removal of foreign body | Surgical and medical procedures | MedDRA 24.0 | Systematic Assessment |
| |
| Skin lesion removal | Surgical and medical procedures | MedDRA 24.0 | Systematic Assessment |
| |
| Surgery | Surgical and medical procedures | MedDRA 24.0 | Systematic Assessment |
| |
| Bloody discharge | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Haematoma | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 24.0 | Systematic Assessment |
|
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Vice President, Clinical Operations | NFlection | 6513439370 | phorwath@nflection.com |
| Dec 16, 2025 |
| Prot_SAP_000.pdf |
Not provided
| ID | Term |
|---|---|
| D009456 | Neurofibromatosis 1 |
| ID | Term |
|---|---|
| D017253 | Neurofibromatoses |
| D009455 | Neurofibroma |
| D018317 | Nerve Sheath Neoplasms |
| D009380 | Neoplasms, Nerve Tissue |
| D009370 | Neoplasms by Histologic Type |
| D009369 | Neoplasms |
| D009386 | Neoplastic Syndromes, Hereditary |
| D020752 | Neurocutaneous Syndromes |
| D009422 | Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D010523 | Peripheral Nervous System Diseases |
| D009468 | Neuromuscular Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
Not provided
Not provided
| Male |
|
| Not Hispanic or Latino |
|
| Unknown or Not Reported |
|
| Asian |
|
| Native Hawaiian or Other Pacific Islander |
|
| Black or African American |
|
| White |
|
| More than one race |
|
| Unknown or Not Reported |
|
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