A Randomized, Double-Blind, Placebo-Controlled Study of NIDO-361 in Patients with SBMA

Trial details

Who runs it

Nido Biosciences Inc.

Trial code

EU 2023-507128-22-00

Record updated

2025. szeptember 16.

Original description

To determine the efficacy of NIDO-361 in restoring muscle volume. To determine the safety and tolerability of NIDO-361 when administered as once daily oral doses in patients with SBMA.Read full original descriptionShow less

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of NIDO-361 in Patients with Spinal and Bulbar Muscular Atrophy (SBMA)

To determine the efficacy of NIDO-361 in restoring muscle volume. To determine the safety and tolerability of NIDO-361 when administered as once daily oral doses in patients with SBMA.

Inclusion: Ability to understand the written study informed consent form(s) (ICF(s)) and provide signed written informed consent prior to any study procedures; A participant, who is non-sterilized and sexually active with a female partner of childbearing potential, agrees to use adequate contraception from the signing of the informed consent throughout the duration of the study and 90 days from the last dose. Adequate methods of contraception are described in Section 5.3. In addition, participants must be willing to forgo sperm donation for the duration of the study and 3 months after last dose of study drug; Ambulatory male; Age ≥18 to ≤70 years old; Body mass index (BMI) ≥18 kg/m2 to ≤32 kg/m2; Documented SBMA diagnosis confirmed by DNA genetic testing; Able to complete six-minute walk test (6MWT); Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS) scores ≥25 and ≤45; On initial whole-body magnetic resonance imaging (MRI), patient has evidence of muscle fat replacement such that the total volume of disease affected muscle (i.e., muscle with at least 10% muscle fat infiltration (MFI) and no more than 50% muscle fat fraction (MFF)is at least: - 500 cm3 if only 1 muscle is eligible, or - 250 cm3 if more than one muscle meets the criteria; If using supplements or vitamins, dosing must be stable for at least eight weeks prior to Screening Visit.

Exclusion: Use of other investigational drugs within 30 days or 5 half-lives prior to the planned first drug administration, whichever is longer; History of a prior treatment with androgen reducing agents including luteinizing hormone-releasing hormone (LHRH) agonists or antagonists, androgen receptor antagonists, and selective androgen receptor modifiers (SARMs) within the past 3 months or 5 half-lives of the treatment, whichever is longer; History of use of medicines that are known to increase the risk of seizures within 90 days prior to Day 1 and until 90 days after the last study dose; History of substance abuse disorder, (except nicotine) within 6 months prior to the Screening Visit; History of epilepsy or previous seizure within 10 years prior to the Screening Visit; History of malignancy or has received treatment for malignancy, other than treatment for basal cell or squamous cell carcinoma of the skin, within the previous 5 years; Positive for Hepatis B virus (HBV) or Hepatis C virus (HCV); Known to be positive for human immunodeficiency virus (HIV); History of hypersensitivity to the excipients of NIDO-361; Clinically significant cardiovascular, endocrine, hepatic, renal, pulmonary, gastrointestinal, neurologic, immunologic, malignant, metabolic, psychiatric, or other condition that, in the opinion of the Investigator, precludes the participant’s safe participation in the study or would interfere with the study assessments; Clinically significant abnormality at Screening electrocardiogram (ECG), including but not necessarily limited to a confirmed QT interval corrected for heart rate (QTc) ≥450 msec for males; Clinically significant laboratory abnormality at Screening Visit; Inability to undergo MRI (mild sedation may be allowed); Involved directly or indirectly in the conduct and administration of this study as an Investigator, sub-investigator, study coordinator, or other study staff member, or the patient is a first-degree family member, significant other, or relative residing with one of the above persons involved directly or indirectly in the study; Positive for opioids (unprescribed), cocaine, amphetamines, methadone, barbiturates, methamphetamine, or phencyclidine at the Screening Visit.

EU CTIS 2023-507128-22-00