A Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers

NCT06475404 | Completed Phase 1

• 1 other identifier: IDB-MPS-I02
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

It is a phase I open-label single-dose, dose-escalation cohort study to evaluate of the tolerance, safety, and pharmacokinetics of GNR-055 in healthy volunteers

Conditions

Mucopolysaccharidosis Type II; Metabolic Diseases

Keywords

Mucopolysaccharidosis type II; Mucopolysaccharidoses; Metabolic Diseases; Lysosomal Storage Diseases; Carbohydrate Metabolism; Inborn; Metabolism; Genetic Diseases; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked

Outcome Measures

Primary Outcomes (1)

• Proportion of participants with Adverse Events

  Adverse events, Laboratory tests, Vital signs, Physical examination, 12-lead electrocardiogram, Allergic reactions, Infusion reactions, Antidrug antibody

  Day 28

Secondary Outcomes (2)

• Pharmacokinetics: Area under the plasma concentration versus time curve (AUC)

  Day 4

• Pharmacokinetics: Peak Plasma Concentration (Cmax)

  Day 4

Study Arms (3)

verenafusp alfa 6 mg/kg intravenously

EXPERIMENTAL

Single intravenous administration

Biological: verenafusp alfa 6 mg/kg

verenafusp alfa 9 mg/kg intravenously

EXPERIMENTAL

Single intravenous administration

Biological: verenafusp alfa 9 mg/kg

verenafusp alfa 12 mg/kg intravenously

EXPERIMENTAL

Single intravenous administration

Biological: verenafusp alfa 12 mg/kg

Interventions

verenafusp alfa 6 mg/kg BIOLOGICAL

Single intravenous administration of 6 mg/kg of verenafusp alfa

Also known as: GNR-055

verenafusp alfa 6 mg/kg intravenously

verenafusp alfa 9 mg/kg BIOLOGICAL

Single intravenous administration of 9 mg/kg of verenafusp alfa

Also known as: GNR-055

verenafusp alfa 9 mg/kg intravenously

verenafusp alfa 12 mg/kg BIOLOGICAL

Single intravenous administration of 12 mg/kg of verenafusp alfa

Also known as: GNR-055

verenafusp alfa 12 mg/kg intravenously

Eligibility Criteria

• Age: 18 Years - 45 Years
• Gender Eligibility Details: As a rule, boys suffer from Mucopolysaccharidosis type II
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Men aged 18 to 45 years (inclusive) at the time of signing the Informed Consent Form;
• Body mass index (BMI) 18.5 to 29.9 kg/m2, body weight of 50 to 90 kg;
• A verified diagnosis as "healthy" (the diagnosis "healthy" is established based on a detailed medical history, in the absence of deviations from normal values during a clinical examination, including measurement of blood pressure, respiratory rate, heart rate, body temperature, laboratory and instrumental (ECG) examination data);
• A written informed consent to participate in the study in accordance with applicable laws in place and compliance with all the procedures and requirements/restrictions provided for by the study protocol;
• Consent to use adequate methods of contraception (double barrier method-male or female (for partners of male research volunteers), condom with spermicide, intrauterine device (IUD), diaphragm with spermicide, contraceptive sponge, cervical cap with spermicide).

You may not qualify if:

• Known hypersensitivity to idursulfase and/or to the medicinal product excipients;
• Burdened allergy history;
• Drug intolerance;
• History of seizures;
• Deposit injections or implants use of any other medicinal product three months before the Screening Visit;
• An unusual way of living (night working, extreme physical activity);
• Diarrhea dehydration, vomiting, or other causes within 24 hours before the Screening Visit;
• Deviations from the normal values of the clinical, laboratory, and ECG examinations;
• If there are acute or chronic diseases of the cardiovascular, bronchopulmonary, nervous, immune, and endocrine systems, diseases of the gastrointestinal tract, liver and biliary tract, kidney and urinary tract, blood and lymphatic system, a history of mental illness;
• Positive results for hepatitis B or C markers, human immunodeficiency virus (HIV), and syphilis;
• Acute infectious diseases less than four weeks before the Screening Visit;
• Regular administration of medicinal products less than two weeks before the Screening Visit;
• Systolic blood pressure (SBP) below 90 mm Hg or above 139 mm Hg; diastolic blood pressure (DBP) below 50 mm Hg or above 89 mm Hg; heart rate (HR) below 60 bpm or above 90 bpm;
• Blood donation (450 mL or more of blood or plasma) less than three months before the Screening Visit;
• Participation in human clinical studies of medicinal products less than three months before the Screening Visit;

Sponsors & Collaborators

• AO GENERIUM: lead

Study Sites (1)

State budgetary healthcare institution of the city of Moscow "City Clinic No. 2 of the Moscow Health Department"

Moscow, 117556, Russia

Location

MeSH Terms

Conditions

Mucopolysaccharidosis II; Metabolic Diseases; Mucopolysaccharidoses; Lysosomal Storage Diseases; Genetic Diseases, Inborn; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Nervous System Diseases; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Mucinoses; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Nutritional and Metabolic Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms

Study Officials

• Oksana A. Markova, MD

  JSC GENERIUM

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 20, 2024
• First Posted: June 26, 2024
• Study Start: April 6, 2023
• Primary Completion: September 20, 2023
• Study Completion: September 20, 2023
• Last Updated: July 3, 2024

Record last verified: 2024-06

Data Sharing

• IPD Sharing: Will not share

Locations

RU (1)