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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Nido Biosciences Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male

Therapeutic area

Diseases [C] - Musculoskeletal Diseases [C05]

Trial duration

1 Mar 2024 → 22 Oct 2025

Decision date (initial)

2024-01-31

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

Nido Biosciences, Inc.

External identifiers

EU CT number

2023-507128-22-00

WHO UTN

U1111-1297-0638

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Dose response, Pharmacokinetic, Efficacy, Pharmacodynamic, Safety, Therapy

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.

Secondary objectives 1

1. To determine the efficacy of NIDO-361 in restoring muscle strength and muscle endurance.

Conditions and MedDRA coding

Spinal and Bulbar Muscular Atrophy

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 10

1. 1. Ability to understand the written study informed consent form(s) (ICF(s)) and provide signed written informed consent prior to any study procedures
2. 2. Ambulatory male
3. 3. Age ≥18 to ≤70 years old
4. 4. Body mass index (BMI) ≥18 kg/m2 to ≤32 kg/m2
5. 5. Documented SBMA diagnosis confirmed by DNA genetic testing
6. 6. Able to complete six-minute walk test (6MWT)
7. 7. Spinal and Bulbar Muscular Atrophy Functional Rating Scale (SBMAFRS) scores ≥25 and ≤45
8. 8. 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
9. 9. If using supplements or vitamins, dosing must be stable for at least eight weeks prior to Screening Visit
10. 10. 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.

Exclusion criteria 15

1. 1. Use of other investigational drugs within 30 days or 5 half-lives prior to the planned first drug administration, whichever is longer
2. 2. 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
3. 3. 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
4. 4. 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
5. 5. 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
6. 6. Clinically significant laboratory abnormality at Screening Visit
7. 7. History of substance abuse disorder, (except nicotine) within 6 months prior to the Screening Visit
8. 8. History of epilepsy or previous seizure within 10 years prior to the Screening Visit
9. 9. Positive for opioids (unprescribed), cocaine, amphetamines, methadone, barbiturates, methamphetamine, or phencyclidine at the Screening Visit
10. 10. 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
11. 11. Positive for Hepatis B virus (HBV) or Hepatis C virus (HCV)
12. 12. Known to be positive for human immunodeficiency virus (HIV)
13. 13. Inability to undergo MRI (mild sedation may be allowed)
14. 14. 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
15. 15. History of hypersensitivity to the excipients of NIDO-361

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 4

1. 1. Change from baseline in thigh and total lean muscle volume (LMV) as assessed by whole-body MRI.
2. 2. Number of patients with adverse events (AEs) or serious adverse events (SAEs).
3. 3. Number of patients discontinuing study and number of deaths.
4. 4. Number of mild, moderate, and severe AEs

Secondary endpoints 6

1. 1. Change from baseline in 6MWT
2. 2. Change from baseline in modified-SBMAFRS (m-SBMAFRS)
3. 3. Change from baseline in actigraphy-derived physical activity
4. 4. Change from baseline in timed up and go (TUG) test
5. 5. Change from baseline in grip strength as measured by handheld dynamometer (HHD)
6. 6. Change from baseline in two-minute walk test (2MWT)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Nido Biosciences Inc.

Sponsor organisation

Nido Biosciences Inc.

Address

134 Coolidge Avenue Ste 2

City

Watertown

Postcode

02472-2972

Country

United States

Scientific contact point

Organisation

Nido Biosciences Inc.

Contact name

Regulatory Submissions

Public contact point

Organisation

Nido Biosciences Inc.

Contact name

Regulatory Submissions

Third parties 7

Locations

2 EU/EEA countries · 3 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 23 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

6 submissions — initial application plus substantial / non-substantial modifications