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 the Kirklin Clinic
Birmingham, Alabama, 35294, 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. Gilbert, Arizona
  3. Gilbert, Arizona
  4. Gilbert, Arizona
  5. Tucson, Arizona
  6. Tucson, Arizona
  7. Tucson, Arizona
  8. Los Angeles, California
  9. Palo Alto, California
  10. Stanford, California
  11. Washington, District of Columbia
  12. Tampa, Florida
  13. Atlanta, Georgia
  14. Columbus, Georgia
  15. Boston, Massachusetts
  16. Midland, Michigan
  17. Newark, New Jersey
  18. Newark, New Jersey
  19. Newark, New Jersey
  20. New York, New York
  21. Cleveland, Ohio
  22. Portland, Oregon
  23. Philadelphia, Pennsylvania
  24. Charleston, South Carolina
  25. Dallas, Texas
  26. Murray, Utah
  27. Seattle, Washington
  28. Buenos Aires, Ciudad Autónoma DE Buenosaires
  29. Rosario, Santa FE
  30. Buenos Aires,
  31. Santa Fe,
  32. Aalst, Oost-vlaanderen
  33. Leuven,
  34. Vancouver, British Columbia
  35. Halifax, Nova Scotia
  36. Ottawa, Ontario
  37. Peterborough, Ontario
  38. Montreal, Quebec
  39. Sherbrooke, Quebec
  40. Roma, Rome
  41. Perugia, Umbria
  42. Perugia, Umbria
  43. Bari,
  44. Brescia,
  45. Firenze,
  46. Pavia,
  47. Pavia,
  48. San Benedetto del Tronto,
  49. Trieste,
  50. Trieste,
  51. Ciudad de México, Distrito Federal
  52. Monterrey, Nuevo LEÓN
  53. Amsterdam, Noord-holland
  54. A Coruña, A Coruna
  55. Palma de Mallorca, Baleares
  56. Majadahonda, Madrid
  57. El Palmar, Murcia
  58. Vigo, Pontevedra
  59. Barcelona,
  60. Barcelona,
  61. Córdoba,
  62. Madrid,
  63. Glasgow, Glasgow CITY
  64. London,
  65. London,
  66. London,
  67. Gilbert, Arizona
  68. Tucson, Arizona
  69. Aurora, Colorado
  70. Clearwater, Florida
  71. Boise, Idaho
  72. Saint Louis, Missouri
  73. Newark, New Jersey
  74. New York, New York
  75. New York, New York
  76. Columbus, Ohio
  77. Philadelphia, Pennsylvania
  78. Germantown, Tennessee
  79. Tullahoma, Tennessee
  80. Houston, Texas
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 CT.gov Call Center1-800-718-1021[email protected]
Listed Location Countries  ICMJE Argentina,   Belgium,   Canada,   Italy,   Mexico,   Netherlands,   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 CT.gov Call CenterPfizer
PRS Account Pfizer
Verification Date August 2020

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