A Study of ARRY-371797 in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation

NCT03439514

Last updated date
Study Location
Azienda Sanitaria Universitaria lntegrata di Trieste
Groton, Connecticut, 06340, United States
Contact
1-800-718-1021

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Eligibility Criteria
condition
The disease, disorder, syndrome, illness, or injury that is being studied.
Dilated Cardiomyopathy, Lamin A/C Gene Mutation
Sex
Females and Males
Age
Pediatric Trials: 0-17 Years
Adult Trials: 18+ Years
18 + years
Inclusion Criteria
The factors, or reasons, that allow a person to participate in a clinical study.
Show details

- Patients with symptomatic lamin A/C protein (LMNA)-related cardiomyopathy Class II/III/ or Class IV defined as:

- Gene positive for a deleterious mutation in the LMNA gene as determined by the study central laboratory or by initial laboratory testing (central confirmation of initial laboratory results is required prior to randomization and study treatment).

- Evidence of cardiac impairment in EF

- Patient will have an implantable cardioverter defibrillator/cardiac resynchronization therapy defibrillator (ICD/CRT-D). ICD implanted at least 4 weeks prior to initiation of study treatment or CRT-D initiated at least 6 months prior to initiation of study treatment

- Class II/III patients must have objective functional impairment evidenced by a reduction in 6-minute walk test (6MWT);

- Stable medical and/or device therapy consistent with American Heart Association (AHA) / American College of Cardiology (ACC) or European Society of Cardiology (ESC) guidelines

- Patients must meet acceptable hematology, hepatic and renal laboratory values as specified

Selected Key

Exclusion Criteria
The factors, or reasons, that prevent a person from participating in a clinical study.
Show details


- Presence of other form(s) of cardiomyopathy contributing to HF (e.g., inflammatory or
infiltrative cardiomyopathy) or clinically significant cardiac anatomic abnormality
(e.g., LV aneurysm).


- Clinically significant coronary artery disease (e.g., coronary revascularization,
exercise-induced angina) per Investigator judgment.


- Uncorrected, hemodynamically significant (i.e., moderate-severe) primary structural
valvular disease not due to HF.


- Currently receiving or deemed at high risk of requiring chronic renal replacement
therapy (e.g., hemodialysis or peritoneal dialysis) within 6 months.


- Treatment with any investigational agent(s) for HF within 28 days prior to Day 1. Any
treatment with an investigational agent(s) requires approval from the Medical Monitor.


- Malignancy that is active or has been diagnosed within 3 years prior to screening,
except surgically curatively resected in situ malignancies or surgically cured early
breast cancer, prostate cancer, skin cancer (basal cell carcinoma, squamous cell
carcinoma) or cervical cancer.


- Non-cardiac condition that limits lifespan to < 1 year.


- Serum positive for hepatitis B surface antigen, viremic hepatitis C, or human
immunodeficiency virus (HIV) at screening.

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Dilated Cardiomyopathy, Lamin A/C Gene MutationA Study of ARRY-371797 in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
NCT03439514
  1. Groton, Connecticut
  2. Trieste,
  3. Birmingham, Alabama
  4. Birmingham, Alabama
  5. Birmingham, Alabama
  6. Birmingham, Alabama
  7. Chandler, Arizona
  8. Chandler, Arizona
  9. Gilbert, Arizona
  10. Gilbert, Arizona
  11. Gilbert, Arizona
  12. Gilbert, Arizona
  13. Tucson, Arizona
  14. Tucson, Arizona
  15. Tucson, Arizona
  16. Tucson, Arizona
  17. Tucson, Arizona
  18. Tucson, Arizona
  19. Los Angeles, California
  20. Los Angeles, California
  21. Palo Alto, California
  22. Stanford, California
  23. Aurora, Colorado
  24. Aurora, Colorado
  25. Washington, District of Columbia
  26. Washington, District of Columbia
  27. Clearwater, Florida
  28. Tampa, Florida
  29. Tampa, Florida
  30. Atlanta, Georgia
  31. Atlanta, Georgia
  32. Columbus, Georgia
  33. Boise, Idaho
  34. Boise, Idaho
  35. Meridian, Idaho
  36. Meridian, Idaho
  37. Boston, Massachusetts
  38. Midland, Michigan
  39. Saint Louis, Missouri
  40. Saint Louis, Missouri
  41. Saint Louis, Missouri
  42. Newark, New Jersey
  43. Newark, New Jersey
  44. Newark, New Jersey
  45. Newark, New Jersey
  46. New York, New York
  47. New York, New York
  48. New York, New York
  49. New York, New York
  50. New York, New York
  51. Cleveland, Ohio
  52. Columbus, Ohio
  53. Columbus, Ohio
  54. Columbus, Ohio
  55. Portland, Oregon
  56. Philadelphia, Pennsylvania
  57. Charleston, South Carolina
  58. Charleston, South Carolina
  59. Germantown, Tennessee
  60. Tullahoma, Tennessee
  61. Dallas, Texas
  62. Dallas, Texas
  63. Dallas, Texas
  64. Houston, Texas
  65. Murray, Utah
  66. Murray, Utah
  67. Seattle, Washington
  68. Buenos Aires, Ciudad Autónoma DE Buenos Aires
  69. Rosario, Santa FE
  70. Rosario, Santa FE
  71. Buenos Aires,
  72. Santa Fe,
  73. Aalst, Oost-vlaanderen
  74. Leuven,
  75. Vancouver, British Columbia
  76. Halifax, Nova Scotia
  77. Ottawa, Ontario
  78. Peterborough, Ontario
  79. Montreal, Quebec
  80. Sherbrooke, Quebec
  81. Trieste, Friuli-venezia Giulia
  82. Roma, Lazio
  83. Perugia - Località S. Andrea Delle Fratte, Perugia
  84. Pavia, PV
  85. Firenze, Toscana
  86. Perugia, Umbria
  87. Bari,
  88. Bari,
  89. Brescia,
  90. Firenze,
  91. Pavia,
  92. San Benedetto del Tronto,
  93. Trieste,
  94. Trieste,
  95. Ciudad de Mexico, Distrito Federal
  96. Monterrey, Nuevo LEÓN
  97. Monterrey, Nuevo LEÓN
  98. Ciudad de México,
  99. Amsterdam, Noord-holland
  100. Hamar,
  101. Oslo,
  102. Santiago de Compostela, A Coruña
  103. Majadahonda, Madrid
  104. El Palmar, Murcia
  105. Vigo, Pontevedra
  106. A Coruna,
  107. Barcelona,
  108. Barcelona,
  109. Cordoba,
  110. Córdoba,
  111. Madrid,
  112. Majadahonda,
  113. Palma de Mallorca,
  114. Glasgow, Glasgow CITY
  115. London,
  116. London,
  117. London,
ALL GENDERS
18 Years+
years
MULTIPLE SITES
Advanced Information
Descriptive Information
Brief Title  ICMJE A Study of ARRY-371797 in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
Official Title  ICMJE A Phase 3, Multinational, Randomized, Placebo-controlled Study of ARRY-371797 in Patients With Symptomatic Dilated Cardiomyopathy Due to a Lamin A/C Gene Mutation
Brief Summary This is a randomized, double-blind, placebo-controlled study in patients with dilated cardiomyopathy (DCM) due to a gene encoding the lamin A/C protein (LMNA) mutation. The study will further evaluate a dose level of ARRY-371797 that has shown preliminary efficacy and safety in this patient population. After the primary analysis has been performed, eligible patients may receive open-label treatment with ARRY-371797.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description:
The study will be conducted in 2 parts: a randomized, double-blind treatment period for at least 24 weeks, followed by an ARRY-371797 open-label treatment period.
Masking: Double (Participant, Investigator)
Masking Description:
During the randomized, double-blind period, patients, Investigators, site personnel and the sponsor personnel directly involved with the conduct of the study will remain blinded to assigned treatment, except for regulatory reporting requirements.
Primary Purpose: Treatment
Condition  ICMJE
  • Dilated Cardiomyopathy
  • Lamin A/C Gene Mutation
Intervention  ICMJE
  • Drug: ARRY-371797
    400 mg twice daily (BID)
  • Other: Placebo
    BID
Study Arms  ICMJE
  • Experimental: Part 1 Double-blind Treatment
    ARRY-371797 tablet orally OR matching placebo tablet orally
    Interventions:
    • Drug: ARRY-371797
    • Other: Placebo
  • Experimental: Part 2 Open-label Treatment
    ARRY-371797 tablet orally
    Intervention: Drug: ARRY-371797
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: February 13, 2018)
160
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE December 12, 2024
Estimated Primary Completion Date May 31, 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Selected Key Inclusion Criteria:

  • Patients with symptomatic lamin A/C protein (LMNA)-related cardiomyopathy Class II/III/ or Class IV defined as:

    • Gene positive for a deleterious mutation in the LMNA gene as determined by the study central laboratory or by initial laboratory testing (central confirmation of initial laboratory results is required prior to randomization and study treatment).
    • Evidence of cardiac impairment in EF
  • Patient will have an implantable cardioverter defibrillator/cardiac resynchronization therapy defibrillator (ICD/CRT-D). ICD implanted at least 4 weeks prior to initiation of study treatment or CRT-D initiated at least 6 months prior to initiation of study treatment
  • Class II/III patients must have objective functional impairment evidenced by a reduction in 6-minute walk test (6MWT);
  • Stable medical and/or device therapy consistent with American Heart Association (AHA) / American College of Cardiology (ACC) or European Society of Cardiology (ESC) guidelines
  • Patients must meet acceptable hematology, hepatic and renal laboratory values as specified

Selected Key Exclusion Criteria:

  • Presence of other form(s) of cardiomyopathy contributing to HF (e.g., inflammatory or infiltrative cardiomyopathy) or clinically significant cardiac anatomic abnormality (e.g., LV aneurysm).
  • Clinically significant coronary artery disease (e.g., coronary revascularization, exercise-induced angina) per Investigator judgment.
  • Uncorrected, hemodynamically significant (i.e., moderate-severe) primary structural valvular disease not due to HF.
  • Currently receiving or deemed at high risk of requiring chronic renal replacement therapy (e.g., hemodialysis or peritoneal dialysis) within 6 months.
  • Treatment with any investigational agent(s) for HF within 28 days prior to Day 1. Any treatment with an investigational agent(s) requires approval from the Medical Monitor.
  • Malignancy that is active or has been diagnosed within 3 years prior to screening, except surgically curatively resected in situ malignancies or surgically cured early breast cancer, prostate cancer, skin cancer (basal cell carcinoma, squamous cell carcinoma) or cervical cancer.
  • Non-cardiac condition that limits lifespan to < 1 year.
  • Serum positive for hepatitis B surface antigen, viremic hepatitis C, or human immunodeficiency virus (HIV) at screening.
Sex/Gender  ICMJE
Sexes Eligible for Study:All
Ages  ICMJE 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Pfizer Pfizer CT.gov Call Center1-800-718-1021[email protected]
Listed Location Countries  ICMJE Argentina,   Belgium,   Canada,   Italy,   Mexico,   Netherlands,   Norway,   Spain,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT03439514
Other Study ID Numbers  ICMJE ARRAY-797-301
C4411002 ( Other Identifier: Alias Study Number )
2017-004310-25 ( EudraCT Number )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE
Plan to Share IPD:Yes
Plan Description:Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/d….
URL:https://www.pfizer.com/science/clinical_trials/trial_data_and_results/d…
Responsible Party Pfizer
Study Sponsor  ICMJE Pfizer
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Study Director:Pfizer Pfizer CT.gov Call CenterPfizer
PRS Account Pfizer
Verification Date March 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP