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A Study to Investigate the Safety, Tolerability, and Processing by the Body of Intravenous and Subcutaneous RO7121932 in Participants With Multiple Sclerosis.

Phase 1
Recruiting
Conditions
Multiple Sclerosis
Interventions
Drug: RO7121932 IV - SAD
Drug: RO7121932 SC - SAD
Drug: RO7121932 - MAD
Registration Number
NCT05704361
Lead Sponsor
Hoffmann-La Roche
Brief Summary

The primary purpose of the study is to evaluate the safety and tolerability of single ascending intravenous (IV) (Part 1) and subcutaneous (SC) (Part 2) doses of RO7121932 and multiple ascending SC (Part 3) doses of RO7121932 in participants with multiple sclerosis (MS).

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
129
Inclusion Criteria
  • Expanded Disability Status Scale (EDSS) score ≤7.0 at Screening
  • Participants with relapsing multiple sclerosis (RMS) or progressive multiple sclerosis (PMS) who fulfil international panel criteria for diagnosis (McDonald 2017 criteria)
  • Participants not treated with any approved MS treatment at Screening and not planning to start on any MS therapy during the study (including follow-up)
  • Female participants must practice abstinence or otherwise use contraception
Exclusion Criteria
  • Evidence of clinical disease activity as defined by any clinical relapse within 3 months prior to screening, or by >1 clinical relapse within 12 months prior to screening
  • Evidence of magnetic resonance imaging (MRI) activity as defined by the presence of ≥ 1 Gadolinium-enhancing T1 lesion in the screening MRI scan or by ≥ 4 new or enlarging T2 lesions in the screening scan as compared to a reference scan
  • Participants who have active progressive multifocal leukoencephalopathy (PML), have had confirmed PML, or have a high degree of suspicion for PML
  • Known presence of other neurological disorders that may mimic MS including but not limited to: neuromyelitis optica spectrum disease, Lyme disease, untreated Vitamin B12 deficiency, neurosarcoidosis, cerebrovascular disorders, and untreated hypothyroidism
  • Known active or uncontrolled bacterial, viral, fungal, mycobacterial infection or other infection, excluding fungal infection of nail beds, including participants exhibiting symptoms consistent with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) within 6 weeks prior to Day 1
  • Participants with a current diagnosis of epilepsy
  • Clinically significant cardiac, metabolic, hematologic, hepatic, immunologic, urologic, endocrinologic, neurologic, pulmonary, psychiatric, dermatologic, allergic, renal, or other major diseases
  • History of cancer, including hematologic malignancy and solid tumors, within 10 years of screening. Basal or squamous cell carcinoma of the skin that has been excised and is considered cured and in situ carcinoma of the cervix treated with apparent success by curative therapy >1 year prior to screening is not exclusionary
  • Any concomitant disease that may require treatment with systemic corticosteroids or immunosuppressants during course of the study
  • History of currently active primary or secondary (non-drug-related) immunodeficiency
  • History of hypersensitivity to biologic agents or any of the excipients in the formulation
  • Only for cohorts where CSF samples are planned to be collected: Participants with a history of spinal cord compression, raised intra-cerebral pressure, clinically significant vertebral joint pathology or any other current abnormalities in the lumbar region which could prevent the lumbar puncture procedure.

Prior/Concomitant Therapy:

  • Treatment with any approved MS treatment at Screening. Participants may become eligible after completion of a washout period prior to acquiring any screening laboratory tests but should not be withdrawn from therapies for the sole purpose of meeting eligibility for the trial

  • Previous treatment with RO7121932, alemtuzumab, cladribine, mitoxantrone, cyclophosphamide, total body irradiation, bone marrow transplantation, and hematopoietic stem cell transplantation. For the USA only, previous treatment with daclizumab

  • Previous treatment with anti-CD20 B-cell-depleting therapies (e.g., rituximab, ocrelizumab, or ofatumumab)

    • <12 months prior to acquiring any screening laboratory tests,
    • ≥12 months prior to acquiring any screening laboratory tests, if B-cells are outside the normal range, or not back to individual baseline ± 20% (if data are available),
    • If discontinuation of a prior B-cell depletion therapy was motivated by safety reasons
  • Current or prior treatment with natalizumab (if <24 months prior to acquiring any screening laboratory tests)

Prior/Concurrent Clinical Study Experience:

  • Participation in an investigational drug medicinal product or medical device study within 30 days before Screening or within five times the pharmacodynamic (PD) or pharmacokinetic (PK) half-life (if known), whichever is longer

Diagnostic Assessments:

  • Positive result on human immunodeficiency virus (HIV1) and HIV2, hepatitis C, or hepatitis B
  • Participants with suicidal ideation or behavior within 6 months prior to Screening or participants who, in the Investigator's judgment, pose a suicidal or homicidal risk
  • Vaccination with a live or live-attenuated vaccine within 6 weeks prior to Day 1

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Part 1: Single Ascending Dose (SAD) IV: RO7121932- Dose Escalation Cohorts 1 to 6 and Later CohortsRO7121932 IV - SADParticipants will receive a single IV dose of RO7121932 on treatment Day 1. The planned starting dose of RO7121932 is 7 milligrams (mg) and will be escalated up to 2000 mg. The maximum dose will not exceed 4000 mg. Doses may be repeated, adjusted downwards, or intermediate doses may be investigated based on emerging data.
Part 2: SAD SC: RO7121932- Dose Escalation Cohorts 1 to 2RO7121932 SC - SADParticipants will receive a single SC dose of RO7121932 on treatment Day 1. The planned starting dose of RO7121932 is 70 mg and will be escalated up to 200 mg. Doses may be repeated, adjusted downwards, or intermediate doses may be investigated based on emerging data.
Part 3: Multiple Ascending Dose (MAD) SC: RO7121932- Dose Escalation Cohorts 1 to 3RO7121932 - MADParticipants will receive multiple SC doses of RO7121932, once weekly on treatment Day 1 through Day 22. The planned starting dose of RO7121932 is 70 mg and will be escalated up to 700 mg. Doses may be repeated, adjusted downwards, or intermediate doses may be investigated based on emerging data.
Primary Outcome Measures
NameTimeMethod
Part 1, Part 2 and 3: Percentage of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs) with Severity of AEs Measured According to National Cancer Institute Common Terminology Criteria for Adverse Events version 5 (NCI CTCAE v5)Day 1 to Day 169 for part 1 and part 2; Day 1 to Day 197 for part 3
Part 1, Part 2 and Part 3: Percentage of Participants With Abnormal Laboratory FindingsDay 1 to Day 169 for part 1 and part 2; Day 1 to Day 197 for part 3
Part 1, Part 2 and Part 3: Percentage of Participants With Abnormal Vital Signs and Electrocardiogram (ECG) ParametersDay 1 to Day 169 for part 1 and part 2; Day 1 to Day 197 for part 3
Part 1, Part 2 and Part 3: Change From Baseline in Suicide Risk as Assessed Using the Columbia-Suicide Severity Rating Scale (C-SSRS)Day 1 to Day 169 for part 1 and part 2; Day 1 to Day 197 for part 3

The C-SSRS is an interview-based instrument used to assess baseline incidence of suicidal ideation and behavior. The assessment includes "yes" or "no" responses for 5 questions, each related to suicidal ideation (wish to be dead, non-specific active suicidal thoughts, active suicidal ideation with any methods, active suicidal ideation with some intent, active suicidal ideation with specific plan) and suicidal behavior (preparatory acts or behavior, aborted attempt, interrupted attempt, actual attempt, suicide). Numeric ratings are provided for severity of ideation (if present), from 0 to 5. A score of 0 is assigned if no suicide risk is present. A score of 1 or higher indicates suicidal ideation or behavior, with 5 being the most severe.

Part 2 and Part 3: Percentage of Participants with Local Pain at the Site of Injection Assessed Using the Visual Analog Scale (VAS)Day 1, 2, 5, 8 for part 2; Day 1, 2, 5, 8, 15, 22, 29, 36 for part 3

VAS is a 100 millimetre (mm) horizontal visual analog scale with values 0 to 100 mm to indicate pain. The following cutpoints on the pain VAS will be considered in the interpretation of the pain data: no pain (0-4 mm), mild pain (5-44 mm), moderate pain (45-74 mm), and severe pain (75-100 mm). A higher score indicates greater pain intensity.

Part 2 and Part 3: Percentage of Participants with Local Injection Site Reaction Using Local Injection-site Symptom Assessment (LISSA)Day 1,2, 5, 8 for part 2; Day 1, 2, 5, 8, 15, 22, 29, 36 for part 3

LISSA form will be used to assess the participant's injection site for the following categories of reactions: Burning, Itching, Bruising, Erythema (redness), Hive formation, Induration, Swelling, Ecchymosis, Sensitivity, Papules, Stinging, Blister, Cold sensation, and Other. These reactions will be described using the following scale: Unable to assess, less than a dime (\<18 mm/\<0.7 inches), a dime (18 mm/0.7 inches), a nickel (21 mm/0.8 inches), a quarter (24 mm/1 inch), a half dollar (31 mm/1.2 inches), larger than a half dollar (\>31 mm/\>1.2 inches). A higher score indicate high injection site reactions.

Secondary Outcome Measures
NameTimeMethod
Part 3: Trough Concentrations (Ctrough) of RO7121932Day 1 to Day 197
Part 1, Part 2 and Part 3 (Week 1 and Week 4): Time to Maximum Observed Concentration (Tmax) of RO7121932Day 1 to Day 169 for part 1 and part 2; Days 1, 2, 5 and, 22 for part 3
Part 1, Part 2 and Part 3 (Week 1 and Week 4): Maximum Observed Serum Concentration (Cmax) of RO7121932Day 1 to Day 169 for part 1 and part 2; Days 1, 2, 5 and, 22 for part 3
Part 1, Part 2 and Part 3 (Week 1 and Week 4): Area Under the Serum Concentration-Time Curve From Time 0 to 168 Hours (h) (AUC0-168h) PostdoseDay 1 (predose) to Day 8 (168 hours post-dose) for parts 1, 2, and 3 (Week 1) and Day 22 (predose) to Day 29 (168 hours post-dose) for part 3 (Week 4)
Part 1 and Part 2: Area Under the Serum Concentration-Time Curve up to the Last Measurable Concentration (AUClast)Day 1 to Day 169 for part 1 and part 2
Part 1 and Part 2: AUC From Time 0 to Infinity (AUCinf)Day 1 to Day 169 for part 1 and part 2
Part 1: Total Body Clearance (CL) Of RO7121932Day 1 to Day 169
Part 2 and Part 3 (Week 4): Apparent Clearance (CL/F) of RO7121932Day 1 to Day 169 for part 2; Day 22 to Day 29 for part 3
Part 1, Part 2 and Part 3: Terminal Rate Constant of RO7121932Day 1 to Day 169 for part 1 and part 2; Day 22 to Day 197 for part 3
Part 1, Part 2 and Part 3: Apparent Terminal Half-Life (T1/2) of RO7121932Day 1 to Day 169 for part 1 and part 2; Day 22 to Day 197 for part 3
Part 1 and Part 3: Cerebrospinal Fluid (CSF) Concentration of RO7121932Screening, Day 2 to Day 169 for part 1; Screening, Day 2 to Day 197 for part 3
Part 1, Part 2 and Part 3: Percentage of Participants With Anti-RO7121932 AntibodiesPredose on Day 1, and on Days 8, 22, 29, 57, 85, 169 for part 1; Predose on Day 1, and on Days 8, 22, 57, 85, 169 for part 2 Predose on Days 1, 8, 15, 22, and on Days 29, 57, 85, 120, 197 for part 3
Part 1, Part 2 and Part 3: Time Course of B-cell Frequencies in BloodScreening, Days 1, 2, 8, 22, 57, 85, 169 for part 1; Screening, Days 1, 2, 8, 22, 57, 85, 169 for part 2; Screening, Days 1, 2, 8, 15, 22, 57, 85, 120, 197 for part 3
Part 1 and Part 3: Time Course of B-cell Frequencies in CSFScreening, Day 2 to 169 for part 1; Screening, Day 2 to 197 for part 3
Part 1, Part 2 and Part 3: Change From Baseline in B-cell Frequencies in BloodScreening, Days 1, 2, 8, 22, 57, 85, 169 for part 1; Screening, Days 1, 2, 8, 22, 57, 85, 169 for part 2; Screening, Days 1, 2, 8, 15, 22, 57, 85, 120, 197 for part 3
Part 1 and Part 3: Change From Baseline in B-cell Frequencies in CSFScreening, Day 2 to 169 for part 1; Screening, Day 2 to 197 for part 3

Trial Locations

Locations (32)

Hospital Universitari Vall dHebron (CEMCAT)

🇪🇸

Barcelona, Spain

Stanford University Medical Center

🇺🇸

Stanford, California, United States

Yale University Multiple Sclerosis Center

🇺🇸

New Haven, Connecticut, United States

University of South Florida

🇺🇸

Tampa, Florida, United States

University of Massachusetts Medical School

🇺🇸

Worcester, Massachusetts, United States

UC Health, LLC.

🇺🇸

Cincinnati, Ohio, United States

Cliniques Universitaires St-Luc

🇧🇪

Bruxelles, Belgium

UZ Gent

🇧🇪

Gent, Belgium

Montreal Neurological Institute and Hospital

🇨🇦

Montreal, Quebec, Canada

Universitätsklinikum "Carl Gustav Carus"

🇩🇪

Dresden, Germany

Universitätsmedizin Göttingen Georg-August-Universität

🇩🇪

Göttingen, Germany

Klinikum rechts der Isar der TU Muenchen

🇩🇪

München, Germany

Universitätsklinikum Münster Klinik u. Poliklinik f. Neurologie

🇩🇪

Münster, Germany

Universitätsklinikum Tübingen, Zentrum für Neurologie

🇩🇪

Tübingen, Germany

Universitätsklinikum Ulm

🇩🇪

Ulm, Germany

Hadassah University Hospital - Ein Kerem

🇮🇱

Jerusalem, Israel

Tel Aviv Sourasky Medical Center

🇮🇱

Tel Aviv, Israel

IRCCS Ospedale San Raffaele

🇮🇹

Milano, Lombardia, Italy

Fond. Istituto Neurologico C.Besta

🇮🇹

Milano, Lombardia, Italy

ARENSIA Exploratory Medicine Phase I, PMSI Republican Clinical Hospital

🇲🇩

Chisinau, Moldova, Republic of

Uniwersyteckie Centrum Kliniczne

🇵🇱

Gda?sk, Poland

Regionalny Szpital Specjalistyczny im. W. Bieganskiego

🇵🇱

Grudzi?dz, Poland

MedPolonia

🇵🇱

Poznan, Poland

Osrodek Badan Klinicznych Euromedis

🇵🇱

Szczecin, Poland

Instytut Psychiatrii i Neurologii II Klinika Neurologiczna

🇵🇱

Warszawa, Poland

SPSK nr 1

🇵🇱

Zabrze, Poland

Hospital de Braga

🇵🇹

Braga, Portugal

Hospital Santo Antonio dos Capuchos

🇵🇹

Lisboa, Portugal

Centro Hospitalar Entre o Douro e Vouga E.P.E. - Hospital de São Sebastião

🇵🇹

Santa Maria Da Feira, Portugal

ARENSIA Exploratory Medicine SRL - Bucharest (Monza Medical Center)

🇷🇴

Bucharest, Romania

ARENSIA Exploratory Medicine, County Emergency Hospital

🇷🇴

Cluj Napoca, Romania

University Clinical Center of Serbia

🇷🇸

Belgrade, Serbia

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