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

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What is this study about?

This clinical trial is focused on studying a condition known as Spinal and Bulbar Muscular Atrophy (SBMA). SBMA is a rare genetic disorder that affects muscle strength and movement. The trial will test a new treatment called NIDO-361, which is taken as a tablet. The purpose of the study is to see if NIDO-361 can help restore muscle volume and to check its safety when taken once a day by mouth.

Participants in the study will be randomly assigned to receive either the NIDO-361 tablets or placebo tablets. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving the actual medication or the placebo. This helps ensure that the results are unbiased. The trial will last for a period of up to 12 months, during which participants will have regular check-ups to monitor their health and any changes in their muscle condition.

The main focus of the study is to observe changes in muscle volume using MRI scans, which are special imaging tests that provide detailed pictures of the muscles. The study will also track any side effects experienced by participants and assess their overall physical activity and muscle strength through various tests. This research aims to provide valuable information on the potential benefits and safety of NIDO-361 for individuals with SBMA.

1 joining the study

Upon joining the study, the patient will be required to provide signed written informed consent. This is necessary before any study procedures can begin.

2 initial assessments

The patient will undergo initial assessments to confirm eligibility. This includes a six-minute walk test and a whole-body MRI to evaluate muscle condition.

3 randomization

The patient will be randomly assigned to receive either the NIDO-361 tablet or a placebo. This process is double-blind, meaning neither the patient nor the researchers will know which treatment is being administered.

4 medication administration

The patient will take the assigned tablet orally once daily. The duration of this administration will continue throughout the study period.

5 regular monitoring

The patient will be monitored regularly for any changes in muscle volume using MRI scans. Additionally, the patient will be observed for any adverse events or side effects.

6 physical activity assessments

The patient will undergo various physical activity assessments, including the six-minute walk test, grip strength measurement, and timed up and go test, to evaluate changes from baseline.

7 study completion

Upon completion of the study, the patient will undergo final assessments to evaluate the overall effects of the treatment. This includes a final MRI scan and physical activity tests.

Who Can Join the Study?

Must be able to understand and sign the written consent form before any study procedures.
Must be a male who can walk on their own.
Must be between 18 and 70 years old.
Must have a Body Mass Index (BMI) between 18 and 32. BMI is a measure of body fat based on height and weight.
Must have a confirmed diagnosis of Spinal and Bulbar Muscular Atrophy (SBMA) through DNA genetic testing.
Must be able to complete a six-minute walk test, which measures how far you can walk in six minutes.
Must have a score between 25 and 45 on the Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS), which assesses the severity of the condition.
Must show evidence of muscle fat replacement on an initial whole-body Magnetic Resonance Imaging (MRI) scan. This means having at least 10% but no more than 50% muscle fat in affected muscles, with specific volume requirements.
If using supplements or vitamins, the dosage must have been stable for at least eight weeks before the screening visit.
If not sterilized and sexually active with a female partner who can have children, must agree to use adequate contraception during the study and for 90 days after the last dose. Must also agree not to donate sperm during the study and for three months after the last dose.

Who Cannot Join the Study?

Individuals who are not male cannot participate in the study.
Individuals who do not have Spinal and Bulbar Muscular Atrophy cannot participate in the study.
Individuals who are part of a vulnerable population cannot participate in the study. A vulnerable population includes groups of people who may need special protection, like children or those with certain disabilities.

Where you can join this trial?

Verified and Recommended Sites

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Verified Sites

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Other Sites

Site Name	City	Country
Azienda Ospedaliera di Padova	Padua	Italy
Rigshospitalet	Copenhagen	Denmark
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy

Trial status

Country	Status	Recruitment Start
Denmark	Not recruiting	31.01.2024
Italy	Not recruiting	31.01.2024

Trial locations

Investigated drugs:

Nido-361

NIDO-361 is a medication being studied for its potential to help patients with Spinal and Bulbar Muscular Atrophy (SBMA). The main goal of the trial is to see if NIDO-361 can increase muscle volume in these patients. Additionally, the study aims to assess how safe and tolerable NIDO-361 is when taken as a daily oral medication.

Spinal and Bulbar Muscular Atrophy – This is a rare genetic disorder that primarily affects males, characterized by progressive muscle weakness and wasting. It is caused by a mutation in the androgen receptor gene, leading to degeneration of motor neurons in the spinal cord and brainstem. Symptoms often begin in adulthood and include difficulty with speech and swallowing, muscle cramps, and tremors. Over time, individuals may experience increased difficulty with movement and coordination. The disease progresses slowly, and symptoms can vary widely among affected individuals.

Trial ID:

2023-507128-22-00

Protocol code:

NIDO-361-002

Trial Phase:

Therapeutic exploratory (Phase II)

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

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