In short
A groundbreaking clinical trial is underway to investigate the effectiveness and safety of 4-Hydroxy-N-Desmethyltamoxifen, also known as Endoxifen, in treating adults with Bipolar I Disorder experiencing acute mania or manic episodes. This Phase III study aims to evaluate the drug's potential as a new treatment option for this challenging mental health condition. The trial involves a double-blind, placebo-controlled approach to assess Endoxifen's efficacy and establish its superiority over placebo in managing manic symptoms.
At a glance
- Drug Name
- 4-Hydroxy-N-Desmethyltamoxifen (Endoxifen)
- Trial Phase
- Phase III
- Condition Studied
- Bipolar I Disorder with Acute Mania/Manic Episodes
- Study Design
- Double-blind, oral, placebo-controlled, multiple-dose, parallel, randomized
- Primary Objective
- Evaluate efficacy and establish superiority of Endoxifen 8 mg against placebo
- Treatment Duration
- 21 days
- Primary Endpoint
- Mean change from baseline to Day 21 on YMRS Total Score
- Key Inclusion Criteria
- Adults 18-65, YMRS score >25, CGI-BP score >4
- Key Exclusion Criteria
- Certain cardiac conditions, active substance abuse, suicidal/homicidal ideation
- Safety Assessments
- Adverse events, laboratory tests, ECG, C-SSRS
What is Endoxifen?
Endoxifen, also known by its chemical name 4-hydroxy-N-desmethyltamoxifen, is a medication currently being studied for the treatment of Bipolar I Disorder. It is classified as an antipsychotic and a nonsteroidal selective estrogen receptor modulator (SERM). Endoxifen is being developed as a tablet to be taken orally.
Medical Condition: Bipolar I Disorder
Bipolar I Disorder is a mental health condition characterized by episodes of mania (extremely elevated mood and energy) and depression. In this clinical trial, researchers are specifically focusing on patients experiencing acute mania or manic episodes, which may or may not include mixed features (symptoms of both mania and depression occurring simultaneously).
How Endoxifen Works
While the exact mechanism of action for Endoxifen in treating Bipolar I Disorder is not fully explained in the provided information, it is known to be a selective estrogen receptor modulator. This means it can interact with estrogen receptors in the body, potentially influencing brain chemistry and mood regulation.
Clinical Trial Details
The clinical trial for Endoxifen is a Phase III study, which means it is in an advanced stage of testing. The main goal of this trial is to evaluate how effective and safe Endoxifen is for treating adults with Bipolar I Disorder who are experiencing acute mania or manic episodes.
Key details of the trial include:
- It is a double-blind study, meaning neither the patients nor the researchers know who is receiving Endoxifen or a placebo.
- Participants will be randomly assigned to either the Endoxifen group or the placebo group.
- The dosage being tested is 8 mg of Endoxifen.
- The treatment period lasts for 21 days.
- Patients will be hospitalized for at least 2 days and up to 21 days during the treatment period.
Eligibility Criteria
To participate in this clinical trial, patients must meet specific criteria. Some key eligibility requirements include:
- Male patients aged 18 to 65 years or postmenopausal female patients
- Diagnosed with Bipolar I Disorder and currently experiencing acute manic episodes
- A Young Mania Rating Scale (YMRS) total score of more than 25
- Willing to be hospitalized for the treatment period
- Not taking certain medications that could interfere with the study
There are also several exclusion criteria, such as certain heart conditions, history of blood clots, or other serious health issues. It's important to discuss all medical history with the study team to determine eligibility.
Potential Benefits
The researchers hope to see improvements in several areas, including:
- Reduction in manic symptoms, as measured by the Young Mania Rating Scale (YMRS)
- Improvement in overall bipolar disorder symptoms, assessed using the Clinical Global Impression-Bipolar (CGI-BP) scale
- Reduction in depressive symptoms, measured by the Montgomery-Åsberg Depression Rating Scale (MADRS)
- Decreased need for additional medications to control agitation or anxiety
Safety Considerations
As with any clinical trial, patient safety is a top priority. The researchers will be monitoring several aspects of participants' health, including:
- Heart health, including any changes in heart rhythm
- Risk of blood clots
- Liver function
- Thyroid function
- Mental health, including any thoughts of self-harm or suicide
It's important to note that Endoxifen is still being studied, and not all potential side effects may be known. Patients considering participation in this trial should discuss the potential risks and benefits with their healthcare provider and the study team.