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A Clinical Study to Evaluate DNTH103 in Adults With Multifocal Motor Neuropathy

Phase 2
Recruiting
Conditions
Multifocal Motor Neuropathy
Interventions
Drug: Placebo
Registration Number
NCT06537999
Lead Sponsor
Dianthus Therapeutics
Brief Summary

The purpose of this Phase 2 study is to evaluate the safety, tolerability, pharmacometrics, and efficacy of DNTH103 in participants with multifocal motor neuropathy (MMN).

Detailed Description

Not available

Recruitment & Eligibility

Status
RECRUITING
Sex
All
Target Recruitment
36
Inclusion Criteria
  1. Must have given written informed consent before any study-related activities are carried out

  2. Adult males and females, 18 to 75 years of age (inclusive)

  3. Weight range between 40 to 120 kg

  4. Confirmed diagnosis of definite or probable MMN

  5. Evidence of:

    1. Responsiveness to Ig treatment; and
    2. Receiving a stable Ig regimen
  6. Documented vaccinations against encapsulated bacteria in accordance with local requirements and vaccine availability

  7. Female participants must be of nonchildbearing potential or if of childbearing potential, must agree not to donate ova, not to attempt to become pregnant and, if engaging in sexual intercourse with a male partner, must agree to use a highly effective method of contraception

  8. Male participants must be surgically sterile for at least 90 days prior to Screening or agree not to donate sperm and, if engaging in sexual intercourse with a female partner who could become pregnant, must agree to use an acceptable method of contraception

Exclusion Criteria
  1. History or presence of significant medical/surgical condition including any acute illness or major surgery considered to be clinically significant or that could impact efficacy assessments
  2. Any coexisting conditions which may interfere with outcome assessments (eg, severe diabetic neuropathy)
  3. Concurrent or previous use of rituximab, cyclophosphamide, mycophenolate mofetil, azathioprine, or cyclosporine. If a participant has previously used these medications, the last dose must be at least 6 months prior to randomization
  4. Currently or previously on complement inhibitors including in a clinical trial setting
  5. Prior history (at any time) of N. meningitidis infection
  6. Diagnosis of an autoimmune disorder other than MMN
  7. Positive test results for active human immunodeficiency virus (HIV-1 or HIV-2), hepatitis B surface antigen (HBsAg), or hepatitis C virus (HCV) antibodies during Screening
  8. History of active malignancy within 5 years prior to Screening, except basal cell carcinoma of the skin, curatively resected squamous cell carcinoma of the skin, cervical carcinoma in situ curatively treated or low-grade prostate adenocarcinoma for which appropriate management is observation alone
  9. Participation in another clinical study of an investigational drug within 90 days or 5 half-lives of the investigational agent (whichever is longer) prior to randomization (Day 1)
  10. Any other overlapping condition for which the condition or treatment of the condition may affect the study assessments or outcomes
  11. Any other condition, including mental illness or prior therapy, that in the opinion of the Investigator would make the participant unsuitable for this study, including inability to cooperate fully with the requirements of the study protocol or likelihood of noncompliance with any study requirements

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
PlaceboPlacebo-
DNTH103 low dose Q2WDNTH103-
DNTH103 high dose Q2WDNTH103-
Primary Outcome Measures
NameTimeMethod
Incidence of treatment-emergent adverse events (TEAEs) and treatment emergent serious adverse events (SAEs)Baseline to Week 17

Incidence of treatment-emergent adverse events (TEAEs) and treatment emergent serious adverse events (SAEs)

Secondary Outcome Measures
NameTimeMethod
Area under curve (AUC) of the change from baseline in grip strengthBaseline to Week 17

Grip strength measured in kPa using a vigorimeter

Time to first retreatment with immunoglobulin (Ig) since the final Ig treatment before randomizationBaseline to Week 17

Time between the participant's last dose of Ig and when they require the next dose of Ig after entering the randomized, controlled treatment period of the study

Time between first retreatment with Ig and second retreatment with IgBaseline to Week 17

The amount of time between a participant receiving one dose of Ig and then requiring a second dose of Ig during the study for worsening of symptoms

AUC of the change from baseline in Medical Research Council (MRC)-10 sum scoreBaseline to Week 17

MRC-10 evaluates motor strength/weakness from a predetermined set of 10 muscle pairs (upper and lower limbs) on a scale of 0 to 5

Mean value and mean change from baseline in MRC-10 sum scoreBaseline to Week 17

MRC-10 evaluates motor strength/weakness from a predetermined set of 10 muscle pairs (upper and lower limbs) on a scale of 0 to 5

Mean change from baseline in adjusted Inflammatory Neuropathy Cause and Treatment (INCAT) Disability scoreBaseline to Week 17

INCAT comprises 2 parts, the arm score and the leg score. Each part is scored between 0 and 5 points, resulting in an INCAT total score between 0 and 10. Adjusted INCAT disability score excludes changes in upper limb function from 0 (normal) to 1 (minor symptoms)

Time to first clinical deterioration (CD)Baseline to Week 17

The time it takes a participant to meet defined criteria for worsening of symptoms in the study

Mean value, mean change, and percentage change from baseline in grip strengthBaseline to Week 17

Grip strength measured in kPa using a vigorimeter

Mean value and mean change from baseline in Multifocal Motor Neuropathy Rasch-Built Overall Disability Scale (MMN-RODS) scoreBaseline to Week 17

MMN-RODS consists of 25 items scored on a 3-point scale

Mean value and mean change from baseline in average time to complete the 9-Hole Peg Test (9-HPT)Baseline to Week 17

9-HPT is a quantitative measure of upper extremity (arm and hand) function and dexterity

Mean change from baseline in Euro-Quality of Life 5 Dimensions 5 Levels (EQ-5D-5L) scaleBaseline to Week 17

EQ-5D-5L is comprised of 5 dimensions: mobility, selfcare, usual activities, pain/discomfort, and anxiety/depression

Mean change from baseline in EQ-5D-5L visual analog scale (VAS)Baseline to Week 17

A vertical VAS is included in the EQ 5D 5L. Participants mark their health status from 0 to 100

Count and proportion of participants with Patient Global Impression of Change (PGIC) score of improved or betterBaseline to Week 17

PGIC is a 7-point scale depicting a participant's rating of overall improvement

Mean change from baseline in Health-Related Productivity Questionnaire (HRPQ) outcomesBaseline to Week 17

HRPQ is a participant diary tool designed to provide data on health-related impacts to labor force participation

Effectiveness, side effects, convenience, and overall satisfaction scores as assessed by Treatment Satisfaction Questionnaire for Medications (TSQM)-14Baseline to Week 17

TSQM-14 is a 14-item treatment satisfaction questionnaire that evaluates the following domains: effectiveness, side effects, convenience, and global satisfaction

Incidence of treatment-emergent adverse events (TEAEs) and treatment emergent serious adverse events (SAEs)Up to Week 52 of OLE

Incidence of treatment-emergent adverse events (TEAEs) and treatment emergent serious adverse events (SAEs)

Serum concentrations of DNTH103Baseline to Week 17

Blood samples will be collected for measurement of serum concentrations of DNTH103 at various timepoints both pre- and post-dose

Incidence and titer of antidrug antibody (ADA) levels against DNTH103Baseline to Week 17

Blood samples will be collected to measure ADA against DNTH103 at various timepoints

Mean value and mean change from baseline in MRC-14 sum scoreBaseline to Week 17

MRC-14 evaluates motor strength/weakness from a predetermined set of 14 muscle pairs (upper and lower limbs) on a scale of 0 to 5

Mean change from baseline in Fatigue Severity Scale (FSS) scoreBaseline to Week 17

FSS assesses disabling fatigue in participants with chronic illness

Trial Locations

Locations (1)

Clinical Study Site

🇵🇱

Katowice, Poland

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