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Conditions: Cystic Fibrosis
Interventions: TEZ, IVA, VX-440, Matched Placebo; Drug
Lead sponsor: Vertex Pharmaceuticals Incorporated; Industry
Eligibility: 12 Years and older
Enrollment: 74 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2017
U.S. locations: 22
States / cities: Tucson, Arizona • Palo Alto, California • San Francisco, California + 18 more

Conditions: Healthy Participants, Non-cystic Fibrosis Bronchiectasis
Interventions: Rev-56 for Inhalation, Placebo: Normal saline; Combination Product · Drug
Lead sponsor: Revagenix, Inc.; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 64 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026 – 2027
U.S. locations: 1
States / cities: Miami, Florida

Conditions: Olmsted Syndrome
Interventions: KM-023, Placebo; Drug
Lead sponsor: Orvida Pharma (formerly Kamari Pharma); Network
Eligibility: 18 Years to 55 Years
Enrollment: 64 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2026
U.S. locations: 1
States / cities: Chicago, Illinois

Conditions: Age Related Macular Degeneration (ARMD), AMD - Age-Related Macular Degeneration
Interventions: ABF-101, Placebo; Drug
Lead sponsor: Aptabio Therapeutics, Inc.; Industry
Eligibility: 18 Years to 50 Years
Enrollment: 68 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026 – 2027
U.S. locations: 1
States / cities: Dallas, Texas

Conditions: Acute Respiratory Distress Syndrome (ARDS)
Interventions: ALT-100 mAb, ALT-100 (Placebo); Drug
Lead sponsor: Aqualung Therapeutics Corp.; Industry
Eligibility: 18 Years to 100 Years
Enrollment: 15 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2023 – 2027
U.S. locations: 1
States / cities: Tucson, Arizona

Conditions: Sickle Cell Disease
Interventions: ITU512, Placebo; Drug
Lead sponsor: Novartis Pharmaceuticals; Industry
Eligibility: 12 Years to 55 Years
Enrollment: 161 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2030
U.S. locations: 7
States / cities: Birmingham, Alabama • Miami, Florida • Boston, Massachusetts + 4 more

Conditions: Amyotrophic Lateral Sclerosis (Healthy Volunteers)
Interventions: SAR443820, Placebo; Drug
Lead sponsor: Sanofi; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 84 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2020 – 2021
U.S. locations: 1
States / cities: Saint Paul, Minnesota

Conditions: Healthy Volunteer
Interventions: ABBV-142, Placebo; Drug
Lead sponsor: AbbVie; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 73 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2026
U.S. locations: 1
States / cities: Grayslake, Illinois

Conditions: HIV Infections
Interventions: pGA2/JS7 DNA, Modified vaccinia Ankara/HIV62; Biological
Lead sponsor: National Institute of Allergy and Infectious Diseases (NIAID); NIH
Eligibility: 18 Years to 50 Years
Enrollment: 120 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2006 – 2008
U.S. locations: 6
States / cities: Birmingham, Alabama • Baltimore, Maryland • Boston, Massachusetts + 3 more

Conditions: Healthy Participants
Interventions: AZD4916, Placebo; Drug · Other
Lead sponsor: AstraZeneca; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 160 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2026 – 2027
U.S. locations: 1
States / cities: Glendale, California

Conditions: Ulcerative Colitis
Interventions: UTTR1147A, UTTR1147A Placebo, Vedolizumab, Vedolizumab Placebo; Drug
Lead sponsor: Genentech, Inc.; Industry
Eligibility: 18 Years to 80 Years
Enrollment: 195 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2018 – 2021
U.S. locations: 2
States / cities: Greenville, North Carolina • Salt Lake City, Utah

Conditions: Healthy Volunteers
Interventions: TAK-951 Placebo, TAK-951; Drug
Lead sponsor: Takeda; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 128 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2019 – 2020
U.S. locations: 1
States / cities: Glendale, California

Conditions: Pulmonary Arterial Hypertension (PAH)
Interventions: Allogeneic Human Cardiosphere-Derived Stem Cells, Placebo; Biological
Lead sponsor: Cedars-Sinai Medical Center; Other
Eligibility: 18 Years to 75 Years
Enrollment: 26 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2023
U.S. locations: 1
States / cities: Los Angeles, California

Conditions: Healthy, Late Onset Pompe Disease
Interventions: ABX1100 injection for IV infusion, Placebo injection for IV infusion; Drug
Lead sponsor: Aro Biotherapeutics; Industry
Eligibility: 18 Years and older
Enrollment: 46 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2026
U.S. locations: 3
States / cities: Orange, California • St Louis, Missouri • Fairfax, Virginia

Conditions: SSM, Mastocytosis, Indolent, Mastocytosis, Systemic, Mastocytosis, ISM, BMM, Smoldering Systemic Mastocytosis, Bone Marrow Mastocytosis
Interventions: Bezuclastinib Tablets (Formulation A), Bezuclastinib Tablets (Formulation B), Placebo Tablets; Drug
Lead sponsor: Cogent Biosciences, Inc.; Industry
Eligibility: 18 Years and older
Enrollment: 237 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2022 – 2030
U.S. locations: 24
States / cities: Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 20 more

Conditions: Nonalcoholic Fatty Liver Disease
Interventions: CB4211 Dose 1, CB4211 Dose 2, CB4211 Dose 3, CB4211 Dose 4, CB4211 Dose 5, CB4211 Dose 6, CB4211 Dose TBD, Placebo; Drug
Lead sponsor: CohBar, Inc.; Industry
Eligibility: 18 Years to 60 Years
Enrollment: 88 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2018 – 2021
U.S. locations: 4
States / cities: Chula Vista, California • Dallas, Texas • San Antonio, Texas

Conditions: Infection, Human Immunodeficiency Virus
Interventions: GSK2838232 PIB, GSK2838232 IR1, GSK2838232 IR2, Placebo PIB, Ritonavir; Drug
Lead sponsor: GlaxoSmithKline; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 62 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: Fibrodysplasia Ossificans Progressiva
Interventions: Andecaliximab, Placebo; Drug
Lead sponsor: Ashibio Inc; Industry
Eligibility: 2 Years and older
Enrollment: 92 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024 – 2029
U.S. locations: 3
States / cities: San Francisco, California • Rochester, Minnesota • Philadelphia, Pennsylvania

Conditions: Healthy Volunteers
Interventions: MF-300 SAD, Placebo, MF-300 Food Effect, MF-300 MAD; Drug
Lead sponsor: Epirium Bio Inc.; Industry
Eligibility: 18 Years to 75 Years
Enrollment: 100 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2025
U.S. locations: 2
States / cities: Marlton, New Jersey • Austin, Texas

Conditions: Phosphomannomutase 2 Deficiency, PMM2-CDG
Interventions: GLM101 (Part A, Double-blind), Placebo (Part A, Double-blind), GLM101 (Part B, Open-label); Drug
Lead sponsor: Glycomine, Inc.; Industry
Eligibility: 4 Years and older
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2027
U.S. locations: 4
States / cities: Minneapolis, Minnesota • New York, New York • Philadelphia, Pennsylvania + 1 more

Conditions: Infection, Human Immunodeficiency Virus, HIV Infections
Interventions: GSK2838232 100 mg tablet, GSK2838232 50 mg capsule, Ritonavir 100 mg tablets, Placebo for GSK2838232 tablets; Drug
Lead sponsor: GlaxoSmithKline; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 26 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2017
U.S. locations: 1
States / cities: Baltimore, Maryland

Conditions: HIV Infections
Interventions: VH4004280, VH4011499, VH4004280 Matching Placebo, VH4011499 Matching Placebo, Antiretroviral therapy; Drug
Lead sponsor: ViiV Healthcare; Industry
Eligibility: 18 Years to 65 Years
Enrollment: 44 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2024
U.S. locations: 3
States / cities: Bakersfield, California • DeLand, Florida • Newark, New Jersey

Conditions: Healthy Volunteer
Interventions: BIIB142, BIIB142-Matching Placebo; Drug
Lead sponsor: Biogen; Industry
Eligibility: 18 Years to 55 Years
Enrollment: 78 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2026
U.S. locations: 1
States / cities: Las Vegas, Nevada

Conditions: Kidney Disease
Interventions: GSK4771261, Placebo matching GSK4771261; Drug
Lead sponsor: GlaxoSmithKline; Industry
Eligibility: 25 Years to 65 Years
Enrollment: 86 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2024 – 2027
U.S. locations: 1
States / cities: Rochester, Minnesota

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A Study Evaluating the Safety and Efficacy of VX-440 Combination Therapy in Subjects With Cystic Fibrosis

A Phase 1a/1b Study to Investigate the Safety, Tolerability, Microbiological Response, and Pharmacokinetics of Inhaled Rev-56 in Adult Healthy Volunteers and Adult Non-cystic Fibrosis Bronchiectasis (NCFB) Patients With Chronic P. Aeruginosa

Study of KM-023 in Healthy Volunteers and Patients With Olmsted Syndrome.

A Phase 1 Study of ABF-101 in Single- and Multiple-Ascending Doses

Study of Safety and Efficacy of ALT-100mAb in Participants With Moderate/Severe ARDS

A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease

Single & Multiple Ascending Dose Study of SAR443820 in Healthy Adult Participants

A Study to Assess the Adverse Events and How Subcutaneous (SC) Injections and Intravenous (IV) Infusions of ABBV-142 Move Through the Body of Healthy Adult Participants

Safety of and Immune Response to a DNA HIV Vaccine (pGA2/JS7) Boosted With a Modified Vaccinia HIV Vaccine (MVA/HIV62) in Healthy Adults

A Study to Investigate the Safety and Pharmacokinetics of Single- and Multiple-ascending Doses of AZD4916 in Healthy Volunteers

A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of UTTR1147A Compared With Placebo and With Vedolizumab in Participants With Moderate to Severe Ulcerative Colitis (UC)

A Study of TAK-951 in Healthy Adults

ALlogeneic Cardiosphere-derived Stem Cells (CDCs) for Pulmonary Hypertension therApy

Study to Assess the Safety, Tolerability, PK and PD of ABX1100

(Summit) A Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients With Indolent or Smoldering Systemic Mastocytosis

A Phase 1a/1b Study of CB4211 in Healthy Non-obese Subjects and Subjects With Nonalcoholic Fatty Liver Disease

Single and Repeated Dose Escalation Study of GSK2838232

A Study of Andecaliximab in Participants With Fibrodysplasia Ossificans Progressiva (FOP)

Single and Multiple Ascending Dosing Administration of MF-300 in Healthy Participants

A Study to Assess the Efficacy and Safety of Weekly Doses of GLM101 in Participants With PMM2-CDG

A Two-part Study to Compare a Tablet and Capsule Formulation of GSK2838232 With and Without Food, and to Assess the Safety and Drug Levels of Repeated Once-daily Doses of GSK2838232 Without Ritonavir

Proof of Concept Treatment Study of Orally Administered VH4004280 or VH4011499 in HIV-1 Infected Adults

A Study to Learn About the Safety of BIIB142 and How it is Processed in the Body of Healthy Adult Participants Aged 18 to 55 Years Old

A Study to Evaluate the Safety, Tolerability, and Effects on Blood and Urine Markers of Single Ascending Dose of GSK4771261 in Healthy Participants and Participants With Autosomal Dominant Polycystic Kidney Disease