A Study of NIDO-361 in Patients With SBMA (PIONEER KD)

Nido Biosciences, Inc.

Status and phase: Active, not recruiting; Phase 2

Conditions: Spinal and Bulbar Muscular Atrophy; Kennedy's Disease

Treatments: Drug: Placebo; Drug: NIDO-361

Study type: Interventional

Funder types: Industry

Identifiers

NCT06411912

NIDO-361-002

Details and patient eligibility

About

The purpose of this study is to evaluate the safety, tolerability, and efficacy of NIDO-361 in adult patients with Spinal and Bulbar Muscular Atrophy (SBMA).

Enrollment: 54 estimated patients

Sex: Male

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Ambulatory male
• Documented SBMA diagnosis confirmed by DNA genetic testing
• Able to complete six-minute walk test (6MWT)

Exclusion criteria

• 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
• Inability to undergo MRI (mild sedation may be allowed)

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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54 participants in 2 patient groups, including a placebo group

NIDO-361

Experimental group

Description:

Participants receive 100 mg of NIDO-361 given orally once daily.

Treatment:

Drug: NIDO-361

Placebo

Placebo Comparator group

Description:

Participants receive matched dose placebo given orally once daily.

Treatment:

Drug: Placebo