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Also known as Diroximel Fumarate
Diroximel · 3 trials · 2 indications
An adverse event (AE) was any unfavorable and unintended sign, symptom, or disease temporally associated with the use of a medicinal (investigational) product, whether/not related to the medicinal (investigational) product. An AE was considered treatment-emergent if it starts or worsens on or after the date of the first dose of study drug in the study until 1 day after the last dose of treatment, or completion of the safety follow-up visit, if applicable.(i.e. for participants who prematurely discontinued study treatment or withdrew from the study) . An SAE is any untoward medical occurrence that at any dose results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in a significant disability/incapacity or congenital anomaly, is a medically important event. TEAEs included both serious and non-serious TEAEs.
Laboratory assessments included hematology, blood chemistry, and urinalysis parameters. Hematology parameters:basophils, hematocrit, red blood cell, total and differential leukocytes, platelets, lymphocytes, monocytes, neutrophils, eosinophils, hemoglobin. Blood chemistry parameters:sodium, potassium, chloride, glucose, calcium, uric acid, total protein, albumin, creatine phosphokinase, Lipid profile: blood cholesterol,high-density lipoprotein,low-density lipoprotein, and triglycerides,serum follicle-stimulating hormone,Vitamin D,thyroid stimulating hormone,serum pregnancy, HIV/Hepatitis screen,tuberculosis test,alanine aminotransferase,aspartate aminotransferase,Lactate Dehydrogenase,alkaline phosphatase,gamma glutamyl transferase,total bilirubin,blood urea nitrogen,creatinine and bicarbonate. Urinalysis parameters:color,pH,specific gravity,ketones,protein,glucose, bilirubin,nitrite,urobilinogen,occult blood,microscopic examination,urine albumin,beta-2-microglobulin,urine creatinine.
The number of participants with shifts to categorical values of ECG interpretation (e.g. abnormal) were presented by each cohort. Abnormal shift includes a shift from normal or unknown to abnormal.
Vital sign assessment included temperature, pulse rate systolic blood pressure, diastolic blood pressure, and respiratory rate. The criteria for determining potentially clinically relevant abnormalities in vital signs included: temperature \< 36 and \> 38 degrees Celsius (C), pulse rate \< 60 and \> 100 beats per minute (bpm), systolic blood pressure (\< 90, \> 140 and \> 160 millimeters of mercury \[mmHg\]), diastolic blood pressure \< 50, \> 90 and \> 100 mmHg, weight (7% or more increase from baseline, 7% or more decrease from baseline) and respiratory rate \< 12 and \> 20 breaths per minute.
The C-SSRS was an interview-based rating scale to systematically assess suicidal ideation, suicidal behavior and non-suicidal self-injurious behavior. The C-SSRS includes 'yes' or 'no' responses for assessment of suicidal behavior (preparatory acts or behavior, aborted attempt, interrupted attempt, Suicidal behavior, completed suicide), suicidal ideation (wish to be dead, non-specific active suicidal thoughts, active suicidal ideation with any methods, active suicidal ideation with some intent, active suicidal ideation with specific plan and intent) and non-suicidal self-injurious behavior.
An AE was any unfavorable and unintended sign, symptom, or disease temporally associated with use of medicinal (investigational) product, whether/not related to medicinal (investigational) product. AE was considered treatment-emergent if it starts or worsens on or after the date of the first dose of study drug in the study until 1 day after the last dose of treatment. SAE is any untoward medical occurrence that at any dose results in death, is a life-threatening event, requires inpatient hospitalization or prolongation of existing hospitalization, results in a significant disability/incapacity or congenital anomaly, is a medically important event. TEAEs included both serious and non-serious TEAEs. TEAEs and TESAEs for participants were collected cumulatively for Parts 1 and 2.
Laboratory assessments included hematology, blood chemistry, and urinalysis parameters. Hematology parameters:basophils, hematocrit, red blood cell, total and differential leukocytes, platelets, lymphocytes, monocytes, neutrophils, eosinophils, hemoglobin. Blood chemistry parameters:sodium, potassium, chloride, glucose, calcium, uric acid, total protein, albumin, creatine phosphokinase, Lipid profile: blood cholesterol,high-density lipoprotein,low-density lipoprotein, and triglycerides,serum follicle-stimulating hormone,Vitamin D,thyroid stimulating hormone,serum pregnancy, HIV/Hepatitis screen,tuberculosis test,alanine aminotransferase,aspartate aminotransferase,Lactate Dehydrogenase,alkaline phosphatase,gamma glutamyl transferase,total bilirubin,blood urea nitrogen,creatinine and bicarbonate. Urinalysis parameters:color,pH,specific gravity,ketones,protein,glucose, bilirubin,nitrite,urobilinogen,occult blood,microscopic examination,urine albumin,beta-2-microglobulin,urine creatinine.
The number of participants with shifts to categorical values of ECG interpretation (e.g. abnormal) were presented by each cohort. Abnormal shift includes a shift from normal or unknown to abnormal.
Vital sign assessment included temperature, pulse rate systolic blood pressure, diastolic blood pressure, and respiratory rate. The criteria for determining potentially clinically relevant abnormalities in vital signs included: temperature \< 36 and \> 38 degrees C, pulse rate \< 60 and \> 100 bpm, systolic blood pressure (\< 90, \> 140 and \> 160 mmHg), diastolic blood pressure \< 50, \> 90 and \> 100 mmHg, weight (7% or more increase from baseline, 7% or more decrease from baseline) and respiratory rate \< 12 and \> 20 breaths per minute.
The C-SSRS was an interview-based rating scale to systematically assess suicidal ideation, suicidal behavior and non-suicidal self-injurious behavior. The C-SSRS includes 'yes' or 'no' responses for assessment of suicidal behavior (preparatory acts or behavior, aborted attempt, interrupted attempt, Suicidal behavior, completed suicide), suicidal ideation (wish to be dead, non-specific active suicidal thoughts, active suicidal ideation with any methods, active suicidal ideation with some intent, active suicidal ideation with specific plan and intent) and non-suicidal self-injurious behavior.
| Arm | Type | Description |
|---|---|---|
| TP Cohort A | EXPERIMENTAL | Participants weighing ≤ 40 kilograms (kg) will receive DRF 231 milligrams (mg) orally, once daily (QD) (starting dose) on Days 1 to 7 followed by DRF 231 mg orally, BID (twice daily) (maintenance dose) on Day 8 through Week 96. |
| TP Cohort B: Sub-cohort 1: DRF | EXPERIMENTAL | Participants weighing \>40 kg will receive DRF/placebo matching fingolimod 231 mg orally, BID (starting dose) on Days 1 to 7 followed by DRF 462 mg orally, BID (maintenance dose) on Day 8 through Week 96. |
| TP Cohort B: Sub-cohort 1: Fingolimod | EXPERIMENTAL | Participants weighing \>40 kg will receive Fingolimod 0.5 mg orally, QD followed by placebo matching DRF 231 mg (starting dose) orally, BID on Days 1 to 7 followed by placebo matching DRF 462 mg (maintenance dose) orally, BID on Day 8 through Week 96. |
| TP Cohort B: Sub-cohort 2: DMF | EXPERIMENTAL | Participants weighing \>40 kg will receive DMF/placebo matching fingolimod 120 mg (starting dose) orally, BID on Days 1 to 7 followed by DMF 240 mg (maintenance dose) orally BID, on Day 8 through Week 96. |
| TP Cohort B: Sub-cohort 2: Fingolimod | EXPERIMENTAL | Participants weighing \>40 kg will receive Fingolimod 0.5 mg oral QD followed by placebo matching DMF 120 mg (starting dose) orally, BID on Days 1 to 7 followed by placebo matching DMF 240 mg (maintenance dose) orally, BID through Week 96. |
| Open-label Extension (OLE) Period: Participants from Cohort A | EXPERIMENTAL | Participants from Cohort A weighing ≤ 40 kg will receive DRF 231 mg orally, BID and participants weighing \>40 kg will receive DRF 462 mg orally, BID up to 192 weeks. |
| OLE Period: Participants from Cohort B | EXPERIMENTAL | Participants from Cohort B will receive DRF 231 mg orally, BID for 7 days, followed by the maintenance dose of DRF 462 mg orally, BID up to 192 weeks. |
| Diroximel Fumarate (DRF) | EXPERIMENTAL | Japanese and Chinese participants will initiate treatment with DRF 231 milligrams (mg), oral capsule, twice daily on Day 1 through Day 7, followed by DRF 462 mg, oral capsules, twice daily from Day 8 up to Week 48. |
| DRF: Chinese Participants | EXPERIMENTAL | Chinese participants will receive DRF Dose 1, oral capsules, twice daily (BID), from Day 1 to Day 4 and DRF Dose 1, oral capsules, once daily (QD), on Day 5. |
| DRF: Caucasian Participants | EXPERIMENTAL | Caucasian participants will receive DRF Dose 1, oral capsules, BID, from Day 1 to Day 4 and DRF Dose 1, oral capsules, QD, on Day 5. |
| Name | Type | Description |
|---|---|---|
| Diroximel Fumarate | DRUG | Oral capsule |
| Dimethyl Fumarate | DRUG | Oral capsule |
| Fingolimod | DRUG | Oral capsule |
| Placebo matching DRF | DRUG | Oral capsule |
| Placebo matching DMF | DRUG | Oral capsule |
| Placebo matching fingolimod | DRUG | Oral capsule |
Key Inclusion Criteria: Treatment Period: * Must have a diagnosis of pediatric MS (as defined by the revised consensus definition of pediatric MS). * Must have an Expanded Disability Status Scale (EDSS) score between 0 and 5.0, inclusive. * Must have experienced at least 1 of the following conditi...
Diroximel, also known as Diroximel Fumarate, is being studied for the treatment of relapsing forms of multiple sclerosis (RMS) in adult and pediatric participants. It is an investigational small molecule developed by Biogen Inc. (BIIB). Clinical trials are evaluating its safety and effects on relapses in this patient population.
Diroximel is a small molecule being developed for relapsing forms of multiple sclerosis. Its specific molecular target has not been disclosed in the available clinical trial information. The drug is being studied for its effects on disease activity and relapse rates in patients with RMS.
Diroximel is being developed by Biogen Inc., a biotechnology company traded on the NASDAQ under the ticker symbol BIIB. Biogen is conducting clinical trials to evaluate the safety and efficacy of Diroximel Fumarate in patients with relapsing forms of multiple sclerosis.
Diroximel is in clinical development, with trials listed in Phase 1 and Phase 3. A Phase 1 study in healthy volunteers has been completed, and a Phase 3 study in adult patients with relapsing forms of multiple sclerosis has also been completed. An additional Phase 3 study in pediatric patients is planned but has not yet started recruiting.
Diroximel has been studied in clinical trials including NCT05083923, a completed Phase 3 study in adults with relapsing forms of multiple sclerosis in China and Japan, and NCT05127564, a completed Phase 1 study in healthy Chinese and Caucasian participants. A third trial, NCT07483632, is a planned Phase 3 study in pediatric patients with RMS.
Yes, Diroximel is also known as Diroximel Fumarate (DRF). Clinical trials refer to the drug as Diroximel Fumarate, and it is being investigated for the treatment of relapsing forms of multiple sclerosis. Biogen Inc. is the developer of this investigational small molecule.