Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Napazimone · 1 trial · 1 indication
Number of adverse events will be monitored throughout the study for all subjects
The following parameters and body systems will be examined and any abnormalities described: height and weight; general appearance; skin; head, ears, eyes, nose, and throat; lungs; heart; lower extremity examination; abdomen; neurologic and lymph nodes. Any clinically significant changes from baseline should be recorded as AEs.
Body temperature, systolic and diastolic cuff blood pressure, pulse rate and pulse oximetry will be measured and any clinically significant changes from baseline should be recorded as AEs.
Changes from baseline of ECG parameters will be evaluated.
Monitoring of the clinically significant laboratory results for sodium, potassium, chloride, bicarbonate/carbon dioxide;, blood urea nitrogen, serum creatinine, glucose, albumin, total protein, alkaline phosphatase, aspartate aminotransferase, alanine aminotransferase, total bilirubin, direct bilirubin, indirect bilirubin, calcium, gamma-glutamyl transferase, creatine kinase
Monitoring of the clinically significant laboratory results for specific gravity, pH, semi-quantitative "dipstick" evaluation of glucose, protein, bilirubin, ketones, leukocytes, blood microscopy and/or culture to be performed if clinically indicated or if urinalysis results positive.
Monitoring of the clinically significant laboratory results for Hemoglobin, hematocrit, white blood cell with differentials (monocytes, eosinophils, basophils, neutrophils, lymphocytes) as an absolute value, red blood cell count, platelet count, C-reactive protein
These events will be monitored throughout the study.
C-SSRS assesses suicidal ideation and behavior risk through a series of questions to assess for suicidal ideation and behavior, the severity and immediacy of the risk, and the level of support the subject may need. C-SSRS Severity of Ideation scores of 4 or 5 are considered SAEs.
| Arm | Type | Description |
|---|---|---|
| Open label extension | EXPERIMENTAL | Subjects will receive medication twice a day for 48 weeks minimum |
| Name | Type | Description |
|---|---|---|
| Napazimone | DRUG | Product: KL1333 (international nonproprietary name: napazimone) Dose: Each subject will be up-titrated to his/her maximum well tolerated dose. The starting dose will be 25 mg KL1333 twice daily (BID; total daily dose of 50 mg). If KL1333 is considered to be well tolerated after 4 weeks of treatment, the dose will be increased to 50 mg KL1333 BID (total daily dose of 100 mg). The dose may be lowered from 50 mg BID to 25 mg BID at the investigator's discretion throughout the study in case of tolerability issues. Frequency: Twice daily Route: Oral |
Inclusion Criteria: * Completed the FALCON study (age 18 years or older), and in the opinion of the investigator and sponsor has been compliant with the study requirements * Willingness and ability to attend study appointments within the specified time windows * Willingness and ability to complete ...
Napazimone is an investigational small molecule being developed for the treatment of mitochondrial diseases. It is currently in Phase 2 clinical development and has not been approved by regulatory authorities. The drug is being studied in patients with primary mitochondrial disease to assess its long-term safety and efficacy.
Napazimone is being developed by Pharming Group N.V., a biopharmaceutical company listed on the stock exchange under the ticker symbol PHAR. The company is conducting clinical trials to evaluate the drug's potential in treating mitochondrial diseases.
Napazimone is currently in Phase 2 clinical development. It is an investigational drug and has not received regulatory approval. The ongoing Phase 2 trial is designed to evaluate the long-term safety and efficacy of Napazimone in patients with primary mitochondrial disease.
Napazimone is being studied in a Phase 2 clinical trial registered as NCT07514338. This open-label extension study aims to assess the long-term safety and efficacy of Napazimone in patients with primary mitochondrial disease. The trial is not yet recruiting and plans to enroll approximately 140 participants aged 18 years and older.
Napazimone is not FDA approved. It is an investigational drug currently in Phase 2 clinical development for mitochondrial diseases. The drug has not completed clinical trials or received marketing authorization from regulatory agencies, and its safety and efficacy have not been established.