Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Interferon γ-1b · 3 trials · 1 indication
An adverse event (AE) is any untoward medical occurrence, whether or not the event is considered related to the investigational product. A TEAE is any adverse change from the subject's baseline condition, including any laboratory test value abnormality judged as clinically significant by the investigator, that occurs on or after the date of the first dose of study drug administered at home and throughout the duration of the clinical study, whether the adverse event is considered related to the treatment or not. A serious AE (SAE) is an AE that results in death, is life-threatening, results in persistent or significant disability or incapacity, inpatient hospitalization or prolongation of an existing hospitalization, is a congenital anomaly or birth defect, or other medically important event.
An adverse event (AE) is any untoward medical occurrence, whether or not the event is considered related to the investigational product. A TEAE is any adverse change from the subject's baseline condition, including any laboratory test value abnormality judged as clinically significant by the investigator, that occurs on or after the date of the first dose of study drug administered at home and throughout the duration of the clinical study, whether the adverse event is considered related to the treatment or not. An SAE is an AE that results in death, is life-threatening, results in persistent or significant disability or incapacity, inpatient hospitalization or prolongation of an existing hospitalization, is a congenital anomaly or birth defect, or other medically important event.
NAb testing only for those participants with a positive ADA test. Baseline is defined as the last non-missing measurement/assessment on the date of Week 26 Visit from study HZNP-ACT-301 (NCT02415127). If this measurement was missing or otherwise unavailable, it was the last non-missing measurement/assessment on or prior to first dose in this study. If the participant discontinued the study, then premature withdrawal assessments were mapped to the nearest scheduled visit based on schedule of the assessment and the study day. If the mapped visit was already available then the visit was mapped to the next schedule visit. Last on study assessment is the last non-missing post-baseline assessment for each participant.
The FARS assessment includes neurological signs that specifically reflect neural substrates affected in FA. Based on a neurological examination, bulbar, upper limb, lower limb, peripheral nerve, and upright stability/gait functions were assessed. The FARS-mNeuro score excludes the peripheral nervous system subscale score and the facial and tongue atrophy and fasciculations from the bulbar subscale score. Scores range from 0 (normal) to 93 (most impairment). A negative change from baseline is an improvement.
| Arm | Type | Description |
|---|---|---|
| interferon γ-1b | EXPERIMENTAL | ACTIMMUNE® will be administered 3 times per week (TIW) by subcutaneous (SC) injection. |
| Placebo | PLACEBO_COMPARATOR | Approximately 45 participants will receive SC doses of placebo TIW for a total of 26 weeks. |
| Name | Type | Description |
|---|---|---|
| interferon γ-1b | DRUG | ACTIMMUNE® will be administered three times per week by subcutaneous injection. The initial dose will be individualized for each participant and will be determined by the investigator, provided that the initial dose does not exceed the maximum tolerated dose in HZNP-ACT-302 (NCT02593773). The investigator may subsequently adjust the dose for any participant if deemed clinically appropriate, provided that the dose does not exceed 100 μg/m². |
| Placebo | DRUG | The volume of placebo is planned to correspond with volume of study drug that would be given to the participant if the participant was randomized to the study drug arm. |
Inclusion Criteria: * Written informed consent and child assent, if applicable. * Completed 26 weeks of treatment and the Week 28 Follow-Up visit in Study HZNP-ACT-302 (NCT02593773). * If female, the subject is not pregnant or lactating or intending to become pregnant during the study, or within 30...
Interferon γ-1b is an investigational treatment for Friedreich's Ataxia, a rare inherited disease that causes progressive nervous system damage. It is being studied to evaluate its safety, tolerability, and efficacy in patients with this condition. The drug is administered as a dose escalation regimen in clinical trials.
Interferon γ-1b is developed by Amgen Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol AMGN. Amgen is conducting clinical trials to assess the drug's potential as a treatment for Friedreich's Ataxia.
Interferon γ-1b is in Phase 3 clinical development for Friedreich's Ataxia. It is an investigational drug, meaning it has not been approved by regulatory authorities. All three Phase 3 trials have been completed, with a total of 216 participants enrolled across the studies.
Interferon γ-1b has been studied in three completed Phase 3 trials for Friedreich's Ataxia. These include NCT02415127 and NCT02593773, both evaluating safety, tolerability, and efficacy of dose escalation, and NCT02797080, a long-term safety extension study in children and young adults. All trials were conducted in the United States.
Yes, Interferon γ-1b is also known as ACTIMMUNE. The clinical trials for Friedreich's Ataxia reference ACTIMMUNE in their titles, indicating that ACTIMMUNE is the brand name for this investigational drug. Both names refer to the same product being developed by Amgen.
Interferon γ-1b is a small molecule that works by modulating the immune system. It is designed to target pathways involved in inflammation and cellular stress, which are thought to contribute to the progression of Friedreich's Ataxia. The exact mechanism in this condition is still under investigation.