ONGOING RESEARCH

We continue to be active in Environmental Research and routinely present our research annually at National and International meetings.

In addition, we publish our research regularly in multiple peer reviewed journals.

Currently Open:

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis
o   Sponsor: Corbus Pharmaceuticals
o   Short title: Corbus 2b
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2GACnux&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=HYuMg1k5uKkuUv6Me05ttxGTYntUUmPE6RmEo40G-eY&e=

A Multi-Center, Randomized Placebo-Controlled, Phase 1, Two-Part Study Designed to Assess the Safety, Tolerability, Pharmacokinetics, Food Effect, and Drug-Drug Interactions of PTI-801 in Healthy Volunteers, and Safety, Tolerability, and Pharmacokinetics of PTI-801 in Subjects with Cystic Fibrosis
o   Sponsor: Proteostasis Therapeutics, Inc.
o   Short title: PTI-801
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2GtaFOX&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=S50P8y_Zr7btAeAi2yuEk8XdJlB8pFlsK0lZgH5Jyk4&e=

A Phase 1 Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of PTI-808 in Healthy Adult Subjects and in Adults with Cystic Fibrosis
o   Sponsor: Proteostasis Therapeutics, Inc.
o   Short title: PTI-808
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2GBP20m&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=RpfBwfsv-bu9Z9jP0-XG-A-Lr9M9MRQUHIQmBEqNkxA&e=

A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety, Tolerability, Pharmacokinetics, and Effect of PTI-428 in Subjects with Cystic Fibrosis
o   Sponsor: Proteostasis Therapeutics, Inc.
o   Short title: PTI-808
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly2KSlzmQ&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=FvAV-tI-j-OlWmBXDoSqTP8w6VxK3POyFZ_0g6kdhCA&e=

Engineering Evaluation of the Helix Ventilator
o   Sponsor: Respironics
o   Short title: Helix

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects with Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation (F/MF)
o   Sponsor: Vertex Pharmaceuticals Incorporated
o   Short title: Vx-445-102
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2veEduF&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=gay_OuyZxwA9tabXl9ISuYX4IpS7OzWOTUcd_WjfcRc&e=

A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-445 Combination Therapy in Subjects with Cystic Fibrosis Who Are Homozygous for the F508del Mutation (F/F)
o   Sponsor: Vertex Pharmaceuticals Incorporated
o   Short title: Vx-445-103
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2IOMVrx&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=1uEUQFLo6rxo3qMfB9RC-pPwN7d1G5yUAG_3YAQM5wM&e=

A Post-approval Observational Study to Evaluate the Long-term Effectiveness and Safety of Orkambi in US Patients Who Completed Study VX12-809-105 Part A
o   Vertex Pharmaceuticals Incorporated
o   Vx-809-105
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2XvYE2f&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=4upJidlvEqLZ1RUmIBxrw6I0IUf7SjkcfMyiFsK1LBc&e=

A Multicentre, Randomized, Double-Blind, Placebo Controlled, Parallel Group, Phase 3 Study to Evaluate the Efficacy and Safety of Tezepelumab in Adults and Adolescents with Severe Uncontrolled Asthma
o   Sponsor: AstraZeneca
o   Short title: Navigator
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2UMhQvH&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=2thgtzWff6LWhZc7YeDgaV9L7g3EVbftdLhrj8XKHPo&e=

Randomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of Two Treatment Regimens of Aztreonam 75mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas Aeruginosa (PA) Infection/Colonization
o   Sponsor: Gilead Sciences
o   Short title: Aztreonam Lysine for Pseudomonas Infection Eradication / ALPINE 2
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2IKHz0g&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=uyh0E11_4atC76uKUcd8Oian3BaBRAn5aL2B9zz_w6c&e=

Recorded Home Oximetry to Optimize Weaning in Premature Infants
o   Sponsor: Boston Children’s Hospital
o   Short title: RHO
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2ZskQfu&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=hoRnLmmZ-BXlJ77GVJoePcdKAR92UdsKZOS3To16CuI&e=

Resistant Airflow Obstruction in Children with Asthma
o   Sponsor: NYMC
o   Short title: ROCAS

Characterizing CFTR Modulated Changes in Sweat Chloride and their Association with Clinical Outcomes
o   Sponsor: Cystic Fibrosis Foundation Therapeutics
o   Short title: CHEC
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2DCPpG7&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=t109l91hmjm8EYt41AqkQZXmishAXRa7_bhGegB5uV0&e=

Standardized Treatment of Pulmonary Exacerbations II
o   Sponsor: Cystic Fibrosis Foundation Therapeutics
o   Short title: STOP2
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2Zq2Jqu&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=wJsX81N2V9YRBzswpS0W8HZFvJU7wHqDxDXD3Jpm_7s&e=

Longitudinal Assessment of Risk Factors For and Impact of Pseudomonas Aeruginosa Acquisition and Early Anti-Pseudomonal Treatment in Children with CF
o   Sponsor: Cystic Fibrosis Foundation Therapeutics
o   Short title: EPIC-002
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2GzArSM&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=FfblgDM16iBfvN2mEihLdZCHS6sqd8KpLor-PVk0bsM&e=

A Long-Term Prospective Observational Safety Study of the Incidence of and Risk Factors for Fibrosis Colonopathy in US Patients with Cystic Fibrosis Treated with Pancreatic Enzyme Replacement Therapy: A Harmonized Protocol Across Sponsors
o   Sponsor: Cystic Fibrosis Foundation Therapeutics / Seattle Children’s Hospital
o   Short Title: CFFC-OB-11
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2GCqswl&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=aHuU5kcVYnwREMqXR5tdYv4IpN04s7iwOOiWbhxUYsA&e=

Childhood Pulmonary Complications in a Birth Cohort after a Randomized Trial of Antenatal Corticosteroids: the ALPS Follow-Up Study
o   Sponsor: NHLBI / Eunice Kennedy Shriver National Institute of Child Health and Human Development
o   Short title: ALPS Follow-up
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2PoJxVe&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=n5k1bCrBVbGwKwOD6Wq2KNz2RV_Uwc83ljLSv0Kn_Cw&e=

CF Patient Registry
o   Sponsor: Cystic Fibrosis Foundation
o   Short title: Port CF

An Open-Label Ascending Dose Cohort Study to Assess the Safety, Pharmacokinetics, and Preliminary Efficacy of neoGAA (GZ402666) in Patients with Infantile-onset Pompe Disease Treated with Alglucosidase Alfa who Demonstrate Clinical Decline or Sub-optimal Response
o   Sponsor: Sanofi/Genzyme
o   Short title: MINI-Comet
o   Clinicaltrials.gov: https://urldefense.proofpoint.com/v2/url?u=https-3A__bit.ly_2USfDPh&d=DwIF-g&c=A51OX6aSaU1ywwq_3bUC2Q&r=6wU1n-WBPw93Cq6Cej4WZV3hXsywTgFMxfcNVwxWJh0&m=ShctijXstlNs7LgW8mp15E0IZyuO3UgJ8IY9YXnzvxA&s=fq83z8ThRneR0bS_jg42Fl1om181PYbew7NOPipQwl4&e=

ADVOCACY PROJECTS:

Quality Improvement Work Assessing for the Use of AAR (Assess, Advise, Refer) with Regards to Smoking Cessation Counseling for Children with Environmental Tobacco Smoke Exposure

The Hudson Valley Carbon Monoxide Detector Advocacy Project