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

NCT04539340 | Completed Phase 1 DMC

• 1 other identifier: IDB-MPS-I
• Study Type: interventional
• Target: 20
• 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, Inborn; Genetic Diseases, Inborn; Neurobehavioral Manifestations; Neurologic Manifestations; Genetic Diseases, X-Linked

Outcome Measures

Primary Outcomes (1)

• Number of participants with Adverse Events

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

  Day 7 ± 1

Secondary Outcomes (8)

• Serum pharmacokinetic parameters of GNR-055

  Day 2

• Cmax - Maximum Serum Concentration

  Day 2

• AUC - Area Under the Curve

  Day 2

• Т1/2 - Half-life

  Day 2

• Kel - Elimination rate constant

  Day 2

Study Arms (5)

Cohort 1 GNR-055 (0.3 mg/kg)

OTHER

GNR-055 (0.3 mg/kg) Single intravenous administration GNR-055

Biological: GNR-055

Cohort 2 GNR-055 (0.5 mg/kg)

OTHER

GNR-055 (0.5 mg/kg) Single intravenous administration GNR-055

Biological: GNR-055

Cohort 3 GNR-055 (1 mg/kg)

OTHER

GNR-055 (1 mg/kg) Single intravenous administration GNR-055

Biological: GNR-055

Cohort 4 GNR-055 ( 2 mg/kg)

OTHER

GNR-055 ( 2 mg/kg) Single intravenous administration GNR-055

Biological: GNR-055

Cohort 5 GNR-055 (3 mg/kg)

OTHER

GNR-055 (3 mg/kg) Single intravenous administration GNR-055

Biological: GNR-055

Interventions

GNR-055 BIOLOGICAL

Single intravenous administration

Cohort 1 GNR-055 (0.3 mg/kg); Cohort 2 GNR-055 (0.5 mg/kg); Cohort 3 GNR-055 (1 mg/kg); Cohort 4 GNR-055 ( 2 mg/kg); Cohort 5 GNR-055 (3 mg/kg)

Eligibility Criteria

• Age: 18 Years - 50 Years
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Men aged 18 to 50 years (inclusive) at the time of signing the Informed Consent Form;
• Body mass index (BMI) 18.5 to 30 kg/m2, body weight of 50 to 90 kg;
• A verified diagnosis as "healthy" (the diagnosis "healthy" is established on the basis of 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 100 mm Hg or above 140 mm Hg; diastolic blood pressure (DBP) below 70 mm Hg or above 90 mm Hg; HR below 60 bpm or above 80 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)

FSAEI HE "I.M. Sechenov First Moscow State Medical University" of the Ministry of Health of Russian Federation

Moscow, 119991, 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 Design

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

Study Record Dates

• First Submitted: August 28, 2020
• First Posted: September 7, 2020
• Study Start: February 28, 2020
• Primary Completion: September 8, 2020
• Study Completion: September 8, 2020
• Last Updated: October 14, 2020

Record last verified: 2020-08

Data Sharing

• IPD Sharing: Will not share

Locations

RU (1)