33 completed, 8 unknown, 6 recruiting, 2 active not recruiting. 14 Not applicable, 9 Phase 2, 6 Phase 1, 6 Phase 3, 4 Phase 4.
Follow Hyperhomocysteinemia: new trials by email Literature Review of Hyperhomocysteinemia trials Research Report on Hyperhomocysteinemia
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Homocysteine and Early Diastolic Dysfunction in Newly Diagnosed Hypertension Homocysteine Measurement and Echocardiography - Hypertension (HTN), Hyperhomocysteinemia | Recruiting | Necmettin Erbakan University | 500 | 2025-06-02 | 2026-03-24 | |
| Health Related Quality of Life (HrQoL) in Classical Homocystinuria (CBS Deficiency) Interview - Homocystinuria | Recruiting | University Children's Hospital, Zurich | 80 | 2025-01-15 | 2025-04-10 | |
| The Effect of Dietary Management and Cysteine Supplementation on Growth Parameters and Biochemical Control for Pediatric Qatari Patients Affected with Classical B6 Non-responsive Homocystinuria. Cysteine - Classical Homocystinuria | Not applicable | Unknown | Hamad Medical Corporation | 50 | 2024-12-01 | 2024-10-02 |
| Proof of Concept Creatine Supplementation for Homocystinuria Study Creatine, Dietary methionine tracer and protein intake - Homocystinuria | Not applicable | Active not recruiting | University of British Columbia | 9 | 2024-08-19 | 2026-02-12 |
| A Long-term Extension Study to Assess the Long-term Safety and Efficacy of Pegtibatinase Treatment in Participants ≥5 to ≤65 Years of Age With Classical Homocystinuria (HCU) (ENSEMBLE) Pegtibatinase - Homocystinuria | Phase 3 | Enrolling by invitation | Travere Therapeutics, Inc. | 100 | 2024-04-30 | 2026-05-28 |
| Incidence and Risk Factors of Ocular Complications Among Patients With Homocystinuria Lensectomy and vitrectomy - Complications | Unknown | King Fahad Armed Forces Hospital | 6 | 2024-01-01 | 2024-08-13 | |
| Pegtibatinase as a Treatment for Participants With Classical Homocystinuria (HCU) Receiving Standard of Care Treatment Pegtibatinase, Placebo - Homocystinuria | Phase 3 | Recruiting | Travere Therapeutics, Inc. | 70 | 2023-12-28 | 2026-10-08 |
| Evaluation of a Genetically Determined Personalized Approach in Prescribing Biologically Active Substances in Patients With Elevated Blood Homocysteine Levels. B-TMG supplementation, B-SAM supplementation, B-TMG placebo - Hyperhomocysteinemia | Not applicable | Recruiting | S.LAB (SOLOWAYS) | 111 | 2023-10-23 | 2025-06-08 |
| Selective Screening of Children for Hereditary Metabolic Diseases by Tandem Mass Spectrometry in Kazakhstan Obtaining Dry Blood Spots From Healthy Newborns (Aged 1-7 Days), Obtaining Dry Blood Spots From Healthy Children Aged 8 Days - 7 Years, Obtaining Dry Blood Spots From Healthy Children Aged 8-18 Years - Propionic/Methylmalonic Acidemias, Maple Syrup Urine Disease | Unknown | West Kazakhstan Medical University | 2250 | 2022-10-03 | 2023-06-18 | |
| Baby Detect : Genomic Newborn Screening Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1 | Completed | Centre Hospitalier Universitaire de Liege | 6824 | 2022-09-01 | 2025-08-12 | |
| Safety, Tolerability and Pharmacodynamics of SYNB1353 in Healthy Adult Volunteers SYNB1353 - Homocystinuria | Phase 1 | Completed | Synlogic | 31 | 2022-07-07 | 2023-03-17 |
| Evaluating the Impact of B Vitamin Supplementation (Soloways™) on Homocysteine and LDL-C Levels in Patients With MTHFR, MTR, and MTRR Polymorphisms. Vitamin B, Placebo - Hyperhomocysteinemia, Hyperlipoproteinemias | Not applicable | Completed | S.LAB (SOLOWAYS) | 54 | 2022-03-02 | 2025-03-27 |
| Effect of Acetaminophen and N-Acetylcysteine on Liver Metabolism on Homocystinuria Acetaminophen, N-acetylcysteine - CBS Deficiency | Phase 1 / Phase 2 | Suspended | Hospital de Clinicas de Porto Alegre | 10 | 2022-02-11 | 2021-06-30 |
| Evaluation of the Express Plus Range PKU express plus - Phenylketonurias, PKU | Not applicable | Completed | Vitaflo International, Ltd | 28 | 2021-06-14 | 2026-05-18 |
| A Multiple Ascending Dose Study of ACN00177 (Pegtarviliase) in Subjects With CBS Deficiency Pegtarviliase IV, Pegtarviliase SC - Homocystinuria Due to Cystathionine Beta-Synthase Deficiency | Phase 1 / Phase 2 | Terminated | Aeglea Biotherapeutics | 13 | 2021-05-13 | 2023-07-27 |
| Metabolic Disorders and Vertigo diagnostic blood test, neuropsychological test - Benign Paroxysmal Positional Vertigo (BPPV), Vestibular Migraine | Unknown | I.M. Sechenov First Moscow State Medical University | 100 | 2019-12-10 | 2021-06-23 | |
| Methylenetetrahydrofolate Reductase Deficiency in Ischemic Stroke Methylenetetrahydrofolate reductase (MTHFR) deficiency - Hyperhomocysteinemia, Thrombotic, Cbs-Related, Ischemic Stroke | Not applicable | Completed | Istanbul Medeniyet University | 32 | 2019-10-31 | 2020-02-27 |
| Effects of Exercise on Metabolic Parameters in Classical Homocystinuria Homocystinuria | Completed | University of Lausanne | 12 | 2019-07-01 | 2021-03-30 | |
| Effects of Supplementation of Compound Nutrients on Plasma Homocysteine in Chinese Adults With Hyperhomocysteinemia supplementation of compound nutrients, placebo control - Hyperhomocysteinemia | Not applicable | Unknown | Huilian Zhu | 100 | 2019-05-20 | 2019-05-14 |
| Pegtibatinase as a Treatment for Patients With Classical Homocystinuria (HCU) (Also Known as the COMPOSE Study) Pegtibatinase, Placebo - Homocystinuria | Phase 1 / Phase 2 | Recruiting | Travere Therapeutics, Inc. | 39 | 2019-01-22 | 2026-08-10 |
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Homocystinuria (20) · Cardiovascular Diseases (7) · Hypertension (7) · Vitamin B 12 Deficiency (5) · Glutaric Acidemia I (4) · Metabolism, Inborn Errors (4) · Tyrosinemias (4) · 3-Hydroxy-3-Methylglutaryl-CoA Lyase Deficiency (3) · Acidemia, isovaleric (3) · Argininosuccinic Aciduria (3) · Beta ketothiolase deficiency (3) · Biotinidase Deficiency (3) · Carbamoyl-Phosphate Synthase I Deficiency Disease (3) · Carnitine palmitoyl transferase 1A deficiency (3)
Amino Acid Metabolism, Inborn Errors (53) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (53) · Genetic Diseases, Inborn (53) · Nutritional and Metabolic Diseases (53) · Metabolic Diseases (52) · Metabolism, Inborn Errors (49) · Avitaminosis (35) · Deficiency Diseases (35) · Malnutrition (35) · Nutrition Disorders (35) · Vitamin B Deficiency (34) · Malabsorption Syndromes (33) · Nervous System Diseases (27) · Brain Diseases (26) · Central Nervous System Diseases (26)
Folic Acid (6) · Vitamin B 12 (3) · Acetylcysteine (2) · Betaine (2) · Enalapril (2) · 5-methyltetrahydrofolate (1) · Acetaminophen (1) · Aging (1) · Amlodipine (1) · Blood Specimen Collection (1) · Contraceptives, Oral (1) · Creatine (1) · Cysteine (1) · Echocardiography (1) · Hydrochlorothiazide (1)
Travere Therapeutics, Inc. (4) · S.LAB (SOLOWAYS) (2) · Shenzhen Ausa Pharmed Co.,Ltd (2) · Aeglea Biotherapeutics (1) · Asociación Científica Latina A.C. (1) · Assistance Publique - Hôpitaux de Paris (1) · Assiut University (1) · Bangkok Metropolitan Administration Medical College and Vajira Hospital (1) · Baylor Research Institute (1) · Center for Health Sciences, Serbia (1) · Centre Hospitalier Universitaire de Liege (1) · Clionsky Neuro Systems Inc. (1) · Dr. Loges & Co. GmbH (1) · Fuling Central Hospital of Chongqing City (1) · Georgetown University (1)
United States (12) · China (6) · United Kingdom (6) · Qatar (5) · France (4) · Turkey (Türkiye) (4) · Australia (3) · Belgium (3) · Brazil (3) · Germany (3) · Ireland (3) · Italy (3) · Russia (3) · Saudi Arabia (3) · Canada (2)
Michael Imperiale, MD (4) · Altair J Mocelin, MD PHD (1) · Amalia A Tsiami, PhD (1) · Andrew Prentice, PhD (1) · Anita MacDonald (1) · Anna Bedenko, PhD student (1) · Birgit Goyvaerts, Dr. (1) · Bruce Barshop (1) · Charles P Venditti, M.D. (1) · Christopher S. Wilcox (1) · Cortney Caudill (1) · Curt Scharfe, MD, PhD (1) · David C Chappell, PhD (1)
PMID 19829124, PMID 21048060, PMID 21791335, PMID 22614770, PMID 23898205, PMID 25190203, PMID 25391194, PMID 25567501, PMID 25985870, PMID 26672496, PMID 26790480, PMID 27756315, PMID 28153005, PMID 31315600, PMID 31408467, PMID 32527283, PMID 35142618, PMID 36376887, PMID 36717385, PMID 37243446, PMID 38283151, PMID 38892484, PMID 40382700, PMID 42861767
2018 (1), 2019 (1), R01HL045267 (1), R01HL052630 (1), R01HL054711 (1)