Will you participate? ARIKACE Clinical Trail Now Enrolling, Designated Orphan Drug for NTM Treatment

Recruitment is now underway at 17 sites around the country for a multi-center clinical trial of ARIKACE – inhaled liposomal Amikacin – to treat pulmonary NTM disease. As of March 2013, ARIKACE has been designated by the US Food & Drug Administration (FDA) as an "orphan drug" for the treatment of NTM lung disease.

“Arikace is potentially the most significant development in recent years for the fight against pulmonary NTM, and its designation of Orphan Drug status is an exciting development for all NTM patients, as this designation is based on real data and confers tax incentives to continue work to bring this potentially important drug to market,” said Philip Leitman, President of NTM Info & Research.

“Arikace has several advantages over Amikacin, including a longer half life, and possibly a greater ability to penetrate both biofilms and macrophages, which may make the drug more effective. Although these expectations need to be verified in the clinical trial, I am truly excited by what I have been told,” he added.

The participating sites recruiting patients for the Phase 2 clinical trial, called TARGET-NTM (Treatment with ARIKACE to Realize Greater Efficacy Trial), are listed below with contact information.

ALABAMA: University of Alabama at Birmingham; contact Valerie Eubanks Tarn at vetarn@peds.uab.edu or 205-558-2940.

CALIFORNIA: Stanford University Medical Center; contact Susan Jacobs at ssjpulm@stanford.edu or 650-725-8082.

COLORADO: National Jewish Health; contact Kim McPeak at mcpeakk@njhealth.org or 303-398-1708.

D.C.: Georgetown University; contact D. Michele Cooney at cooneyd@georgetown.edu or 202-444-4982.

FLORIDA (Gainesville): University of Florida, Pulmonary Division; contact Christina Eagan at Christina.Eagan@medicine.ufl.edu or 352-273-8990.

FLORIDA (Miami): University of Miami; contact Eliana Mendes at emendes@med.miami.edu or 305-243-2568.

FLORIDA (Tampa): Tampa General Hospital; contact Suzanne Roberson at sroberson@tgh.org or 813-844-7179.

KANSAS (Kansas City): University of Kansas Medical Center; contact Adam Schooley at aschooley@kumc.edu or 913-588-4022.

MARYLAND: NIH/NIAID; contact Charles Fiorentino at fiorentinoc@niaid.nih.gov or 301-443-5447.

NEW YORK: NYU Medical Center; contact Stephanie Lau at stephanie.lau@nyumc.org or 212-263-7951.

NORTH CAROLINA: UNC Chapel Hill; contact Sharikia Burt at Sharikia_Burt@med.unc.edu or 919-966-2531.

OHIO (Cleveland): Case Medical Center's Rainbow Babies & Children's Hospital; contact David Weaver at david.weaver@uhhospitals.org or 216-844-1841, or Colette Bucur at colette.bucur@uhhospitals.org or 216-844-1902.

OREGON: Oregon Health & Science University; contact Jennifer Ku at kuj@ohsu.edu or 503-494-1384.

PENNSYLVANIA: University of Pennsylvania Medical Center; contact Lisa Gardo at Lisa.Gardo@uphs.upenn.edu or 215-615-0276, or Victoria Fleck at fleckv@uphs.upenn.edu or 215-662-3115.

SOUTH CAROLINA: Medical University of South Carolina; contact Ashley Jones at jonesash@musc.edu or 843-792-4349.

TEXAS: University of Texas/Tyler; contact Jan Hoeft, RN at janice.hoeft@uthct.edu or 903-877-5518, or Mike Lay at Michael.Lay@uthct.edu.

WISCONSIN: Medical College of Wisconsin & Froedtert Hospital; contact Rachel Harris at racharris@mcw.edu or 414-955-7030.

If information on other participating centers becomes publicly available, that information will be added to this page on our website.

 

Below is a list of Key Inclusion and Exclusion Criteria for participation in the study.

Key Inclusion Criteria:

  • Diagnosis of pulmonary NTM disease in accordance with ATS/IDSA criteria with evidence of nodular bronchiectasis and/or cavitary disease by CT scan.
  • History of infection with either Mycobacterium avium complex (MAC) or Mycobacterium abscessus (or both), with at least 2 documented positive sputum cultures in the prior 2 years – at least one positive culture within 6 months prior to screening for clinical trial participation.
  • Positive sputum culture obtained at screening visit (either MAC or abscessus, or both).
  • Receiving ATS/IDSA guidelines-based treatment regimen for at least 6 months prior to screening with persistently positive sputum cultures.
  • Ability to produce at least 3mL of sputum or willingness to undergo an induction that produces at least 3 mL of sputum for clinical evaluation.
  • If a female of childbearing potential, patient must agree to practice an acceptable method of birth control.
  • Male or female between 18 and 85 years of age.

Key Exclusion Criteria:

  • Forced expiratory volume in one second less than 30% of predicted at screening.
  • Active pulmonary malignancy, or any malignancy requiring radiation or chemotherapy within one year prior to screening, or anticipated during the study period.
  • Pulmonary tuberculosis requiring treatment or treated within 2 years.
  • History of lung transplantation.
  • Clinically significant cardiac disease.
  • Hemoptysis of 60 mL or more in a 24-hour period within 4 weeks prior to screening.
  • Active allergic bronchopulmonary mycosis or any other condition requiring systemic steroids at a dose equivalent to at least 10 mg/day of prednisone within 3 months prior to screening, or anticipated during the study period.
  • Hypersensitivity to aminoglycosides.
  • Any change in chronic multi-drug regimen for NTM within 28 days prior to Study Day 1.
  • Evidence of biliary cirrhosis with portal hypertension.
  • History of daily, continuous oxygen supplementation.
  • Smoking tobacco or any substance within 6 months prior to screening, or anticipated inability to refrain from smoking throughout the study


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