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
University of Alabama at Birmingham Hospital
Birmingham, Alabama, 35233, 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. Birmingham, Alabama
  2. Birmingham, Alabama
  3. Birmingham, Alabama
  4. Gilbert, Arizona
  5. Atlanta, Georgia
  6. Boise, Idaho
  7. New York, New York
  8. Portland, Oregon
  9. Murray, Utah
  10. Halifax, Nova Scotia
  11. Sherbrooke, Quebec
  12. Trieste, Friuli-venezia Giulia
  13. Firenze, Toscana
  14. Bari,
  15. Amsterdam, Noord-holland
  16. Santiago de Compostela, A Coruña
  17. Vigo, Pontevedra
  18. A Coruna,
  19. Palma de Mallorca,
  20. Birmingham, Alabama
  21. Chandler, Arizona
  22. Chandler, Arizona
  23. Gilbert, Arizona
  24. Gilbert, Arizona
  25. Gilbert, Arizona
  26. Gilbert, Arizona
  27. Tucson, Arizona
  28. Tucson, Arizona
  29. Tucson, Arizona
  30. Tucson, Arizona
  31. Tucson, Arizona
  32. Tucson, Arizona
  33. Los Angeles, California
  34. Los Angeles, California
  35. Palo Alto, California
  36. Stanford, California
  37. Stanford, California
  38. Stanford, California
  39. Aurora, Colorado
  40. Aurora, Colorado
  41. Aurora, Colorado
  42. Washington, District of Columbia
  43. Washington, District of Columbia
  44. Clearwater, Florida
  45. Clearwater, Florida
  46. Largo, Florida
  47. Tampa, Florida
  48. Tampa, Florida
  49. Tampa, Florida
  50. Atlanta, Georgia
  51. Columbus, Georgia
  52. Columbus, Georgia
  53. Boise, Idaho
  54. Boise, Idaho
  55. Boise, Idaho
  56. Boise, Idaho
  57. Meridian, Idaho
  58. Meridian, Idaho
  59. Boston, Massachusetts
  60. Midland, Michigan
  61. Saint Louis, Missouri
  62. Saint Louis, Missouri
  63. Saint Louis, Missouri
  64. Newark, New Jersey
  65. Newark, New Jersey
  66. Newark, New Jersey
  67. Newark, New Jersey
  68. New York, New York
  69. New York, New York
  70. New York, New York
  71. New York, New York
  72. New York, New York
  73. Cleveland, Ohio
  74. Columbus, Ohio
  75. Columbus, Ohio
  76. Columbus, Ohio
  77. Philadelphia, Pennsylvania
  78. Philadelphia, Pennsylvania
  79. Charleston, South Carolina
  80. Charleston, South Carolina
  81. Germantown, Tennessee
  82. Tullahoma, Tennessee
  83. Dallas, Texas
  84. Dallas, Texas
  85. Dallas, Texas
  86. Dallas, Texas
  87. Houston, Texas
  88. Temple, Texas
  89. Murray, Utah
  90. Seattle, Washington
  91. Buenos Aires, Ciudad Autónoma DE Buenos Aires
  92. Rosario, Santa FE
  93. Rosario, Santa FE
  94. Buenos Aires,
  95. Santa Fe,
  96. Aalst, Oost-vlaanderen
  97. Leuven,
  98. Vancouver, British Columbia
  99. Vancouver, British Columbia
  100. Halifax, Nova Scotia
  101. Halifax, Nova Scotia
  102. Ottawa, Ontario
  103. Peterborough, Ontario
  104. Montreal, Quebec
  105. Roma, Lazio
  106. Perugia - Località S. Andrea Delle Fratte, Perugia
  107. Pavia, PV
  108. Perugia, Umbria
  109. Bari,
  110. Brescia,
  111. Firenze,
  112. Pavia,
  113. San Benedetto del Tronto,
  114. Trieste,
  115. Trieste,
  116. Trieste,
  117. Ciudad de Mexico, Distrito Federal
  118. Monterrey, Nuevo LEÓN
  119. Monterrey, Nuevo LEÓN
  120. Ciudad de México,
  121. Hamar,
  122. Oslo,
  123. Majadahonda, Madrid
  124. El Palmar, Murcia
  125. Barcelona,
  126. Barcelona,
  127. Cordoba,
  128. Córdoba,
  129. Madrid,
  130. Glasgow, Glasgow CITY
  131. London,
  132. London,
  133. 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 December 2020

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