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Nido-361

A groundbreaking clinical trial is underway to assess the potential of NIDO-361 in treating Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy’s Disease. This Phase 2 study aims to investigate the safety, tolerability, and effectiveness of NIDO-361 in adult patients with SBMA. The trial involves a randomized, double-blind, placebo-controlled approach, offering hope for those affected by this rare genetic disorder.

Table of Contents

What is NIDO-361?

NIDO-361 is a new medication being studied for the treatment of Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy’s Disease[1]. It is currently undergoing clinical trials to evaluate its safety, how well patients tolerate it, and how effective it is in treating SBMA[1].

The medication comes in the form of tablets containing 100mg of NIDO-361, which are taken orally (by mouth) once daily[1]. This makes it a convenient option for patients, as it doesn’t require injections or hospital visits for administration.

Target Conditions: SBMA and Kennedy’s Disease

NIDO-361 is being developed to treat Spinal and Bulbar Muscular Atrophy (SBMA), which is also referred to as Kennedy’s Disease[1]. These are two names for the same condition. SBMA is a rare genetic disorder that primarily affects males. It causes progressive muscle weakness and wasting, particularly in the arms, legs, and facial muscles. The condition also affects the bulbar muscles, which are involved in speech and swallowing.

Clinical Trial Details

The clinical trial for NIDO-361 is currently in Phase 2[1]. This means that the drug has already passed initial safety tests in Phase 1 and is now being tested on a larger group of patients with SBMA to further evaluate its effectiveness and safety.

The official title of the study is “A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study of NIDO-361 in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)”[1]. Let’s break down what this means:

  • Randomized: Patients are randomly assigned to either receive NIDO-361 or a placebo. This helps ensure that the results are not biased.
  • Double-Blind: Neither the patients nor the doctors directly involved in the study know who is receiving the real drug and who is receiving the placebo. This further reduces bias in the study.
  • Placebo-Controlled: Some patients will receive a placebo (a tablet that looks like NIDO-361 but contains no active ingredient) instead of the actual drug. This allows researchers to compare the effects of NIDO-361 against no treatment.

Study Design

The study is designed to test NIDO-361 at what researchers call the “recommended Phase 2 dose” (RP2D) of 100 mg once daily[1]. Here’s how the study is set up:

  • Patients are randomly assigned to one of two groups:
  • Group 1: Receives 100 mg of NIDO-361 given orally (by mouth) once daily[1].
  • Group 2: Receives a matching placebo tablet given orally once daily[1].
  • The treatment period lasts for 12 months[1].

Primary Outcomes

The researchers are looking at two main outcomes to determine if NIDO-361 is effective and safe:

  1. Change in muscle volume: They will measure the change from baseline in thigh and total lean muscle volume using whole-body MRI (Magnetic Resonance Imaging)[1]. This will help determine if NIDO-361 can slow down or reverse muscle loss in SBMA patients.
  2. Safety and tolerability: They will track the number of patients who experience side effects (adverse events) or serious side effects, how many patients stop the study, and any deaths that occur[1]. They will also categorize the side effects as mild, moderate, or severe.

These outcomes will be measured throughout the study, which is expected to last an average of 1 year[1].

Safety and Side Effects

As with any new medication, monitoring the safety of NIDO-361 is a crucial part of this clinical trial. The researchers will be carefully tracking any side effects that patients experience[1]. This includes:

  • The number of patients who experience any side effects
  • The severity of these side effects (mild, moderate, or severe)
  • Any serious side effects that occur
  • The number of patients who have to stop taking the medication due to side effects
  • Any deaths that occur during the study

It’s important to note that at this stage, we don’t have information about specific side effects of NIDO-361. This is one of the key things that this clinical trial aims to determine.

Aspect Details
Study Type Phase 2, randomized, double-blind, placebo-controlled
Drug Name NIDO-361
Condition Studied Spinal and Bulbar Muscular Atrophy (SBMA) / Kennedy’s Disease
Dosage 100 mg once daily, oral administration
Duration 12 months
Primary Outcomes 1. Change in thigh and total lean muscle volume (MRI)
2. Safety assessment (adverse events, discontinuations, deaths)
Control Group Placebo oral tablets

FAQ

  • What is the purpose of the NIDO-361 clinical trial? The main purpose of this clinical trial is to evaluate the safety, tolerability, and efficacy of NIDO-361 in adult patients with Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy's Disease.
  • How is the study designed? The study is a Phase 2, randomized, double-blind, placebo-controlled trial. Patients will be randomly assigned to receive either NIDO-361 or a placebo. The recommended Phase 2 dose (RP2D) is 100 mg once daily, taken orally for 12 months.
  • What are the primary outcomes being measured in this trial? The primary outcomes include changes in thigh and total lean muscle volume (assessed by whole-body MRI) and the number of patients experiencing adverse events or serious adverse events. The study will also track the number of patients discontinuing the study, deaths, and the severity of adverse events.
  • How long will participants be involved in the study? Participants will receive daily oral doses of NIDO-361 or placebo for 12 months. The primary outcomes will be measured through study completion, which is expected to be an average of 1 year.
  • What is SBMA or Kennedy's Disease? Spinal and Bulbar Muscular Atrophy (SBMA), also known as Kennedy's Disease, is a rare genetic disorder that primarily affects males. It causes progressive muscle weakness and wasting, particularly in the arms, legs, and bulbar muscles (those involved in speech and swallowing).

Ongoing Clinical Trials on Nido-361

  • Study of NIDO-361 for Patients with Spinal and Bulbar Muscular Atrophy (SBMA)

    Not recruiting

    1
    Investigated drugs:
    Denmark Italy

Glossary

  • Spinal and Bulbar Muscular Atrophy (SBMA): A rare genetic disorder, also known as Kennedy's Disease, that causes progressive muscle weakness and wasting, primarily affecting males.
  • Phase 2 clinical trial: A stage of drug testing in humans that aims to evaluate the effectiveness and safety of a medication for a specific medical condition.
  • Randomized: A method of assigning participants to different treatment groups by chance, reducing bias in the study.
  • Double-blind: A study design where neither the participants nor the researchers know who is receiving the actual treatment or placebo.
  • Placebo-controlled: A study that includes a group receiving an inactive substance (placebo) to compare with the group receiving the actual treatment.
  • Efficacy: The ability of a treatment to produce the desired beneficial effect.
  • Adverse event: Any unfavorable or unintended sign, symptom, or disease that occurs during a clinical trial, whether or not it is related to the treatment being studied.
  • MRI (Magnetic Resonance Imaging): A medical imaging technique that uses magnetic fields and radio waves to create detailed images of the body's internal structures.
  • Lean muscle volume: The amount of muscle tissue in the body, excluding fat and other tissues.
  • Oral administration: A method of taking medication by mouth, typically in the form of tablets or capsules.

References

  1. https://clinicaltrials.gov/study/NCT06411912