A Study To Assess Distribution Of RO7248824 In The Central Nervous System Following Single Intrathecal Doses Of [89zr] Labeled RO7248824 In Healthy Male Participants

NCT04863794 | Completed Phase 1

• 1 other identifier: BP41660
• Study Type: interventional
• Target: 21
• Locations: 1 country
• Sites: 1

Brief Summary

The aim of Study BP41660 is to quantify the amount and concentration of \[89Zr\]DFO-RO7248824 in the brain with positron emission tomography (PET) following a single sub-pharmacological dose of RO7248824 and \[89Zr\]DFO-RO7248824 administered via IT injection to healthy participants.

Conditions

Angelman Syndrome

Outcome Measures

Primary Outcomes (1)

• Quantification of [89Zr]DFO-RO7248824 distribution within the central nervous system (CNS)

  Baseline up to 6 weeks

Secondary Outcomes (1)

• Percentage of participants with adverse events (AEs)

  Baseline up to 6 weeks

Study Arms (1)

RO7248824

EXPERIMENTAL

In Part 1 of the study RO7248824 and \[89Zr\]-labeled RO7248824 will be administered as a single bolus IT injection following a standard IT Administration procedure. In Part 2 of the study, it is planned to test up to 3 additional IT procedures. This part is tentative with regard to its conduct and the number of procedures that may be tested.

Drug: RO7248824

Interventions

RO7248824 DRUG

A single dose of 10 mg RO7248824 will be used for this PET study in healthy participants.

RO7248824

Eligibility Criteria

• Age: 25 Years - 55 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Informed Consent
• Able and willing to provide written informed consent and to comply with the study protocol according to ICH and local regulations
• Age
• Aged from 25 to to 55 years at the time of dosing
• Type of Participants and Disease Characteristics
• Overtly healthy (defined by absence of evidence of any active or chronic disease) as determined by medical evaluation including:
• A detailed medical and surgical history
• A complete physical and neurological examination
• Vital signs
• lead ECG
• Hematology
• Coagulation
• Blood chemistry
• Serology and urinalysis
• Fluent in the language of the Investigator and study staff, and able to communicate with the study staff

You may not qualify if:

• Medical Conditions
• Any condition or disease detected during the medical interview/physical examination that would render the participant unsuitable for the study, place the participant at undue risk or interfere with the ability of the participant to complete the study, as determined by the Investigator
• History or presence of cardiovascular, respiratory, hepatic, renal, gastrointestinal, endocrine, hematological, or neurological disorders capable of significantly altering the absorption, metabolism, or elimination of drugs; constituting a risk when taking the study treatment; or interfering with the interpretation of data
• History or presence of clinically significant cardiovascular disease in the opinion of the Investigator
• History or presence of an abnormal ECG that is clinically significant in the Investigator's opinion
• Uncontrolled arrhythmias or history of clinically significant arrhythmias
• Confirmed abnormal blood pressure
• Abnormal pulse rate
• Abnormalities in brain and
• Evidence or history of clinically significant back pain, back pathology and/or back injury
• Evidence or history of significant active bleeding or coagulation disorder
• Allergy to lidocaine (Xylocaine) or its derivatives
• Medical or surgical conditions for which LP or associated procedures is contraindicated
• Alanine transaminase (ALT) and bilirubin \> 1.5 x upper limit of normal (ULN)
• Current or chronic history of liver disease, or known hepatic or biliary abnormalities

Sponsors & Collaborators

• Hoffmann-La Roche: lead

Study Sites (1)

Pra International Group B.V

Groningen, 9728 NZ, Netherlands

Location

MeSH Terms

Conditions

Angelman Syndrome

Condition Hierarchy (Ancestors)

Movement Disorders; Central Nervous System Diseases; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders

Study Officials

• Clinical Trials

  Hoffmann-La Roche

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 26, 2021
• First Posted: April 28, 2021
• Study Start: April 29, 2021
• Primary Completion: August 1, 2022
• Study Completion: August 1, 2022
• Last Updated: September 8, 2022

Record last verified: 2022-09

Locations

NL (1)