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Trial record 3 of 4 for:    regn2477

Safety and Efficacy of REGN2477+REGN1033 in Patients With Sporadic Inclusion Body Myositis

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ClinicalTrials.gov Identifier: NCT03710941
Recruitment Status : Not yet recruiting
First Posted : October 18, 2018
Last Update Posted : November 12, 2018
Sponsor:
Information provided by (Responsible Party):
Regeneron Pharmaceuticals

Brief Summary:

The primary objective of the study is to evaluate the efficacy of REGN2477+REGN1033 in combination on total lean mass, as measured by Dual-energy X-ray absorptiometry (DXA) in patients with sporadic inclusion body myositis (sIBM).

The secondary objectives of the study are:

  • To evaluate the efficacy of REGN2477+REGN1033 on the IBM-Functional Rating Scale (IBM-FRS)
  • To evaluate the efficacy of REGN2477+REGN1033 on the sIBM Physical Functioning Assessment (sIFA)
  • To evaluate the safety and tolerability of REGN2477+REGN1033
  • To evaluate the effects of REGN2477+REGN1033 on body composition by DXA, including appendicular lean mass and total fat mass
  • To evaluate the efficacy of REGN2477+REGN1033 on measures of muscle performance and physical function
  • To evaluate the efficacy of REGN2477+REGN1033 on patient reported outcome measures including the fear of falling, falls and near falls, and health-related quality of life
  • To evaluate the pharmacokinetic(s) (PK) profile of REGN2477+REGN1033, including functional REGN2477 and functional REGN1033 concentrations in serum over time
  • To evaluate the immunogenicity of REGN2477+REGN1033

Condition or disease Intervention/treatment Phase
Sporadic Inclusion Body Myositis Drug: REGN2477+REGN1033 Drug: Matching placebo Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 120 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study of the Safety and Efficacy of Intravenously Administered REGN2477+REGN1033 in Patients With Sporadic Inclusion Body Myositis
Estimated Study Start Date : December 4, 2018
Estimated Primary Completion Date : June 26, 2020
Estimated Study Completion Date : September 4, 2020


Arm Intervention/treatment
Experimental: REGN2477+REGN1033
Single, sequential, repeat-dose IV or matching placebo
Drug: REGN2477+REGN1033
Single, sequential, repeat-dose Intravenous (IV) or matching placebo

Experimental: Placebo
Single, sequential, repeat-dose IV
Drug: Matching placebo
Single, sequential, repeat-dose Intravenous (IV)




Primary Outcome Measures :
  1. Percent change in total lean mass as measured by dual-energy X-ray absorptiometry (DXA) [ Time Frame: Up to Week 20 ]

Secondary Outcome Measures :
  1. Change in the Inclusion Body Myositis-Functional Rating Scale (IBM-FRS) [ Time Frame: Up to Week 26 ]
  2. Change in the Sporadic inclusion body myositis (sIBM) Physical Functioning Assessment (sIFA) [ Time Frame: Up to Week 26 ]
  3. Incidence and severity of treatment-emergent adverse events (TEAEs) [ Time Frame: Up to Week 30 ]
  4. Percent change in total and regional body composition (including lean mass and fat mass) as measured by DXA [ Time Frame: Up to Week 30 ]
  5. Absolute change in total and regional body composition (including lean mass and fat mass) as measured by DXA [ Time Frame: Up to Week 30 ]
  6. Change in the 1-repetition maximum (1-RM) chest press strength [ Time Frame: Up to Week 26 ]
  7. Change in hand-grip strength as measured by dynamometry [ Time Frame: Up to Week 26 ]
  8. Change in distance walked in the 6-minute walk test (6MWT) [ Time Frame: Up to Week 26 ]
  9. Change in time to complete the 10-meter walk test (10MWT) [ Time Frame: Up to Week 26 ]
  10. Change in instrumented stair climb power [ Time Frame: Up to Week 26 ]
  11. Change in instrumented, sensor-based sit-to-stand test time [ Time Frame: Up to Week 26 ]
  12. Change in fear of falling as measured via the Falls Efficacy Scale-International (FES-I) [ Time Frame: Up to Week 30 ]
  13. Change in Patient Global Impression of Severity assessments (PGIS/PGIC) [ Time Frame: Up to Week 30 ]
  14. Change in Patient Global Impression of Change Items assessments (PGIS/PGIC) [ Time Frame: Up to Week 30 ]
  15. Change in Clinician Global Impression of Severity assessments (CGIS/CGIC) [ Time Frame: Up to Week 30 ]
  16. Change in Clinician Global Impression of Change Items assessments (CGIS/CGIC) [ Time Frame: Up to Week 30 ]
  17. Change in 36-item Short Form Health Survey (SF-36), including the Physical Function Items (PF-10) and the Vitality Scale of the SF-36 [ Time Frame: Up to Week 30 ]
  18. Change in Thigh muscle volume as measured by MRI (sub-study) [ Time Frame: Up to Week 30 ]
  19. Frequency of falls and near-falls as measured by the Hopkins Falls Grading Scale [ Time Frame: Up to Week 30 ]
  20. Pharmacokinetics (PK) profile of REGN2477 [ Time Frame: Up to Week 30 ]
    Assessed via serum concentration of REGN2477 over time

  21. Pharmacokinetics (PK) profile of REGN1033 [ Time Frame: Up to Week 30 ]
    Assessed via serum concentration of REGN1033 over time

  22. Immunogenicity of REGN2477+REGN1033 [ Time Frame: Up to Week 30 ]
    As determined by the presence or absence of anti-drug antibodies (ADA)



Information from the National Library of Medicine

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Ages Eligible for Study:   45 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  • Men and postmenopausal* women
  • Diagnosed with sIBM based on the European Neuromuscular Centre (ENMC) IBM Research Diagnostic Criteria
  • Ability to walk ≥150 meters, with or without a walking aid such as cane or walker, in 6 minutes.
  • Ability to climb 4 steps of stairs unassisted (may use handrails)
  • Willing and able to comply with clinic visits and study-related procedures

Key Exclusion Criteria:

  • Other neurological conditions (eg, hemiplegia post stroke, Parkinson's) or musculo-skeletal conditions (eg, severe osteoarthritis) causing mobility impairment
  • Mini-Mental State Examination (MMSE) score <24
  • Ongoing, chronic, high-dose (>20 mg prednisone equivalent per day), systemic corticosteroid therapy within 6 weeks prior to screening.
  • Any condition that precludes adequate intake of energy and protein; malnutrition; presence of an eating disorder.
  • Unintentional weight loss of ≥10% in the past 6 months (patient-reported)
  • Hospitalization for heart failure in last year or New York Heart Association Class 4
  • History of hypertrophic cardiomyopathy
  • Any drugs known to influence muscle mass and performance such as anabolic steroids or growth hormone within 6 weeks prior to screening
  • Unable to fit on the site's DXA scanner table, within borders for scanning of total lean mass

Note: Other protocol Inclusion/Exclusion criteria apply


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03710941


Contacts
Contact: Clinical Trials Administrator 844-734-6643 clinicaltrials@regeneron.com

Sponsors and Collaborators
Regeneron Pharmaceuticals
Investigators
Study Director: Clinical Trial Management Regeneron Pharmaceuticals

Responsible Party: Regeneron Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03710941     History of Changes
Other Study ID Numbers: R2477-1033-sIBM-1828
2018-002853-31 ( EudraCT Number )
First Posted: October 18, 2018    Key Record Dates
Last Update Posted: November 12, 2018
Last Verified: November 2018

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Myositis
Myositis, Inclusion Body
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Diseases
Nervous System Diseases