Search clinical trials for: PK Deficiency

Total 13005 results

First Affiliated Hospital Bengbu Medical College

Recruiting

Safety and Efficacy Study of NGGT002 in PKU Adult Subjects

Phenylketonurias

China
ADARx Pharmaceuticals, Inc.

Recruiting

Safety, Tolerability, PK, PD of ADX-324 in Healthy Volunteers and Hereditary Angioedema Patients

Hereditary Angioedema

Australia
ViiV Healthcare

Recruiting

A Study to Investigate Safety, Tolerability, and Pharmacokinetics (PK) of VH4524184 and the Potential for Changes in Cytochrome P450 3A (CYP3A) Activity

HIV Infections

United States
KalVista Pharmaceuticals, Ltd.

Recruiting

PK Subtrial in Adolescent Patients With HAE Type I or II Participating in the KVD900-302 Trial

Hereditary Angioedema

United States, Bulgaria, France, Greece, Israel, Netherlands, Spain, United Kingdom, Australia, Germany, South Africa, Slovakia, Austria, New Zealand, Romania, Canada, Japan
Ionis Pharmaceuticals, Inc.

Recruiting

A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, Puerto Rico, Belgium, Bulgaria, Canada, France, Germany, Israel, Italy, Netherlands, Poland, Spain, Turkey, United Kingdom
University of California, Berkeley
National Institute of Mental Health (NIMH); Ministry of Health, Tanzania; Management... and other collaborators

Recruiting

Rudi Kundini, Pamoja Kundini (RKPK: Phase II)

HIV (Human Immunodeficiency Virus)

Tanzania
Agios Pharmaceuticals, Inc.

Recruiting

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Not Regularly Transfused, Followed by a 5-Year Extension Period (ACTIVATE-Kids)

Pediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic Anemia

United States, Spain, Germany, Netherlands, Canada, France, Israel, Switzerland, Italy
The Aurum Institute NPC
Johns Hopkins University; Weill Medical College of Cornell University; University...

Recruiting

Safety and Tolerability of 1 Month Daily (1HP) and 3 Months Weekly (3HP) Isoniazid and Rifapentine With Pharmacokinetics of Dolutegravir (DTG) in Pregnant People With HIV (DOLPHIN Moms)

Pregnancy | HIV Seropositivity | Tuberculosis Infection

South Africa
Makerere University

Recruiting

Optimizing Malaria Treatment for HIV-Malaria Co-infected Individuals (OPTIMAL)

Malaria | HIV Coinfection

Uganda
University of Florida
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Recruiting

Adequacy of the New Pediatric Isoniazid/Rifampin/Pyrazinamide (HRZ) Tablet

Tuberculosis | Human Immunodeficiency Virus | Coinfection

Ghana
University of Florida
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Recruiting

Pharmacokinetics of Intracellular TFV-DP and FTC-TP in HIV-infected Adolescents

Tuberculosis | Coinfection | Human Immunodeficiency Virus (HIV)

Ghana
Agios Pharmaceuticals, Inc.

Recruiting

Pyruvate Kinase Deficiency Global Longitudinal Registry (PEAK Registry)

Pyruvate Kinase Deficiency

United States, Germany, Spain, Italy, United Kingdom, Czechia, France, Canada, Korea, Republic of, Netherlands, Turkey, Portugal, Denmark, Ireland, Switzerland, Thailand
University of Pittsburgh

Recruiting

Longitudinal Study of Neurodegenerative Disorders

Lysosomal Storage Diseases | Leukodystrophy | Niemann-Pick Diseases | Batten Disease | Gaucher Disease | Krabbe Disease | Alpha-Mannosidosis | Osteopetrosis | Tay-Sachs Disease | Pelizaeus-Merzbacher Disease | Sandhoff Disease | GM1 Gangliosidoses | MPS I | MPS II | Vanishing White Matter Disease | Multiple Sulfatase Deficiency... and other conditions

United States
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study of AG-348 in Adult Participants With Pyruvate Kinase (PK) Deficiency

Pyruvate Kinase Deficiency

United States, Canada, Netherlands, United Kingdom, France, Italy
Agios Pharmaceuticals, Inc.

Active, not recruiting

A Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Participants With Pyruvate Kinase Deficiency (PKD) Who Are Regularly Transfused, Followed by a 5-Year Extension Period (ACTIVATE-KidsT)

Pediatric Pyruvate Kinase Deficiency | Pediatric Hemolytic Anemia

United States, Spain, Denmark, Turkey, Netherlands, Czechia, United Kingdom, Germany, Canada, Switzerland
Ionis Pharmaceuticals, Inc.

Available

Donidalorsen Expanded Access Program for Patients With Hereditary Angioedema

Hereditary Angioedema
Hektoen Institute for Medical Research
National Institute of Mental Health (NIMH)

Completed

Transgender Youth and PrEP: PK, Safety, Uptake & Adherence - Demonstration Project

HIV/AIDS | Gender | Behavior and Behavior Mechanisms

United States
Hektoen Institute for Medical Research
National Institute of Mental Health (NIMH)

Completed

Transgender Youth and PrEP: PK, Safety, Uptake & Adherence - Intervention Development

HIV/AIDS | Gender | Behavior and Behavior Mechanisms

United States
Hektoen Institute for Medical Research
National Institute of Mental Health (NIMH)

Completed

Transgender Youth and PrEP: PK, Safety, Uptake & Adherence - PK Study

HIV/AIDS | Gender | Behavior and Behavior Mechanisms

United States
RTI International
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Enrolling by invitation

Early Check: Expanded Screening in Newborns

Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditions

United States
Ionis Pharmaceuticals, Inc.

Active, not recruiting

An Extension Study of Donidalorsen (IONIS-PKK-LRx) in Participants With Hereditary Angioedema

Hereditary Angioedema

Netherlands, United States
Rocket Pharmaceuticals Inc.

Active, not recruiting

Gene Therapy for Pyruvate Kinase Deficiency (PKD)

Pyruvate Kinase Deficiency

Spain, United States
Ionis Pharmaceuticals, Inc.

Completed

OASIS-HAE: A Study to Evaluate the Safety and Efficacy of Donidalorsen (ISIS 721744 or IONIS-PKK-LRx) in Participants With Hereditary Angioedema (HAE)

Hereditary Angioedema

United States, France, Netherlands, Spain, United Kingdom, Belgium, Bulgaria, Canada, Denmark, Germany, Israel, Italy, Poland, Turkey
ViiV Healthcare

Completed

Study to Evaluate Pharmacokinetic (PK), Safety and Tolerability of Cabotegravir (CAB) 400 Milligrams Per Milliliter (mg/mL) Formulation in Healthy Adult Participants

HIV Infections

United States, New Zealand
Boehringer Ingelheim

Completed

Dual Boosted - Protease Inhibitor (PI) Pharmacokinetics (PK) Trial (Tipranavir / Ritonavir) in Highly Treatment-experienced HIV-1 Infected Patients

HIV Infections

Netherlands, Germany, Canada, United States, France, United Kingdom, Switzerland, Denmark, Australia, Greece, Portugal, Belgium, Italy
University of California, San Francisco
University of Colorado, Denver; National Heart, Lung, and Blood Institute (NHLBI) and other collaborators

Active, not recruiting

Options for Delivering Isoniazid-Rifapentine (3HP) for TB Prevention (3HP Options Implementation Trial)

HIV/AIDS | Latent Tuberculosis | Tuberculosis

Uganda
Agios Pharmaceuticals, Inc.

Completed

Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008

Pyruvate Kinase Deficiency

United States, Italy, Netherlands
Homology Medicines, Inc

Terminated

Safety and Efficacy of HMI-103 in Participants With Classical PKU Due to PAH Deficiency

Phenylketonuria | Phenylketonurias | PAH Deficiency

United States
Lumos Pharma

Active, not recruiting

PK and PD Study of LUM-201 in Children With Idiopathic Growth Hormone Deficiency: (OraGrowtH212) (OraGrowtH212)

Growth Hormone Deficiency

Chile
Agios Pharmaceuticals, Inc.

Terminated

Pyruvate Kinase (PK) Deficiency Global Longitudinal Registry: Patient-Reported Outcomes (PRO) (PKD)

Anemia | Anemia, Hemolytic | Pyruvate Kinase Deficiency | Pyruvate Kinase Deficiency Anemia

United States, United Kingdom, Italy, Spain, Netherlands
Daniel Benjamin
Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators

Completed

Pharmacokinetics of Understudied Drugs Administered to Children Per Standard of Care (PTN_POPS)

Heart Failure | Epilepsy | Pain | Pneumonia | Sepsis | Inflammation | Hypertension | Chronic Kidney Diseases | Hemophilia | Schizophrenia | Central Nervous System Infections | Nosocomial Pneumonia | Insomnia | Anxiety | Bipolar Disorder | Neutropenia | Staphylococcal Infections | Bradycardia | Seizures | Urinary Tract Infections | Pulmonary... and other conditions

United States, Canada, United Kingdom, Israel, Singapore
ViiV Healthcare

Completed

First-Time-in-Human (FTIH) Study to Evaluate the Safety, Tolerability and Pharmacokinetics (PK) of VH4004280 in Healthy Participants

HIV Infections

United States
Homology Medicines, Inc

Terminated

Gene Therapy Clinical Study in Adult PKU (pheNIX)

Phenylketonurias | PAH Deficiency

United States
ViiV Healthcare

Completed

First-Time-in-Human (FTIH) Study to Evaluate the Safety, Tolerability and Pharmacokinetics (PK) of VH4011499 in Healthy Participants

HIV Infections

United States
Spanish Society of Thrombosis and Haemostasis
Takeda; Instituto de Investigación Hospital Universitario La Paz

Completed

Pharmacokinetic Characterization of the Hemophilia A Population in Spain Using myPKFiT®

Hemophilia A | Hemophilia | Factor VIII Deficiency

Spain
Ionis Pharmaceuticals, Inc.

Completed

A Study to Assess the Clinical Efficacy of Donidalorsen (Also Known as IONIS-PKK-LRx and ISIS 721744) in Participants With Hereditary Angioedema

Hereditary Angioedema

United States, Netherlands
BioMarin Pharmaceutical

Completed

A Prospective Clinical Study of Phenylketonuria (PKU)

Phenylketonurias

United States
American Regent, Inc.

Withdrawn

Safety, Tolerability, PK and PD of Intravenous Ferric Carboxymaltose in Infants With Iron Deficiency Anemia

Iron Deficiency, Anaemia

United States
Inhibrx, Inc.

Completed

Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency (rhAAT-Fc)

Alpha-1 Antitrypsin Deficiency | AATD

United States, New Zealand, United Kingdom
Hospital Clinic of Barcelona
University of Barcelona

Completed

Pharmacokinetic Model of Fibrinogen Plasma Levels After Fibrinogen Administration (PKMF)

Fibrinogen; Deficiency, Acquired | PHARMACOKINETICS

Spain
American Regent, Inc.

Completed

A Study to Characterize the PK and PD Profile of IV FCM in Pediatric Subjects 1-17 Years Old With IDA

Iron Deficiency Anemia (IDA)

Poland, Russian Federation
Homology Medicines, Inc

Enrolling by invitation

Long-Term Follow Up Study of Subjects Previously Administered HMI 102

Phenylketonuria | PAH Deficiency

United States
Agios Pharmaceuticals, Inc.

Completed

A Study to Evaluate Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Anemia, Hemolytic | Pyruvate Kinase Deficiency

United States, Spain, Switzerland, Turkey, Germany, France, United Kingdom, Japan, Netherlands, Brazil, Canada, Czechia, Denmark, Italy, Korea, Republic of, Thailand
University of Southern California

Terminated

PK/PD of Vitamin D3 in Adults With CF

Vitamin D Deficiency | Cystic Fibrosis

United States
OPKO Health, Inc.

Active, not recruiting

Safety, Efficacy, PK and PD of CTAP101 (Calcifediol) ER Capsules for SHPT in HD Patients VDI

Chronic Kidney Diseases | Vitamin D Deficiency | Stage 5 Chronic Kidney Disease | Secondary Hyperparathyroidism Due to Renal Causes

United States
BioMarin Pharmaceutical

Completed

Observational Study on the Long Term Safety of Kuvan® Treatment in Patients With Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or BH4 Deficiency (KAMPER)

Hyperphenylalaninemia (HPA) Due to Phenylketonuria (PKU) or Tetrahydrobiopterin (BH4) Deficiency

Sweden, France, Italy, Spain, Germany, Netherlands, Austria, Slovakia, Portugal
Genzyme, a Sanofi Company

Completed

Safety, Tolerability, PK, and Efficacy Evaluation of Repeat Ascending Doses of Olipudase Alfa in Pediatric Patients <18 Years of Age With Acid Sphingomyelinase Deficiency (ASCEND-Peds)

Sphingomyelin Lipidosis

Brazil, France, Italy, United States, Germany, United Kingdom
University of North Carolina, Chapel Hill
Eunice Kennedy Shriver National Institute of Child Health and Human Development...

Completed

Prospective Cohort Evaluating Pregnancy Rates, PK Interactions Among HIV+ Women on EFV Initiating LNG Implant or DMPA (FP-ART)

HIV Infections

Malawi
Agios Pharmaceuticals, Inc.

Completed

A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Anemia, Hemolytic | Pyruvate Kinase Deficiency

United States, United Kingdom, Netherlands, Canada, Denmark, France, Italy, Ireland, Thailand
KalVista Pharmaceuticals, Ltd.

Completed

Phase 1 Crossover Study in Healthy Subjects to Evaluate the PK Profile of KVD824 Following Single and Multiple Doses of Modified Release (MR) Formulations

Hereditary Angioedema

United Kingdom

Search clinical trials for: PK Deficiency

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