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Found 904 clinical trials
A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia

This is a study of mRNA-3705 in participants with isolated elevated methylmalonic acid (MMA) due to methylmalonyl-coenzyme A (CoA) mutase (MUT) deficiency. The main goal of the study is to

  • 0 views
  • 10 Jul, 2022
  • 4 locations
  • 0 views
  • 27 Oct, 2022
  • 2 locations
Apotransferrin in Atransferrinemia

Atransferrinemia is a very rare disorder, which is caused by a deficiency of the protein transferrin. No regular treatment is available for these patients. The objective of this study is to

  • 9 views
  • 04 Oct, 2022
  • 3 locations
Gene Therapy Clinical Study in Adult PKU (pheNIX)

This is a Phase 1/2, open-label, randomized, concurrently-controlled, dose escalation study to evaluate the safety and efficacy of HMI-102 in adult PKU subjects with PAH deficiency. Participants

gene therapy
pah gene
deficiency
  • 178 views
  • 09 Feb, 2022
  • 12 locations
Congenital Adrenal Hyperplasia Once Daily Hydrocortisone Treatment (CareOnTIME)

OH deficiency.

21-ohd
deficiency
metabolic syndrome
adrenal insufficiency
glucocorticoid therapy
  • 56 views
  • 14 Apr, 2022
  • 1 location
Cardiorespiratory Exercise & Chinese Medicine for Rehabilitation of Discharged Coronavirus Disease (COVID-19) Patients

metabolites regulate immune function and possibly autoimmune deficiency in the rehabilitation process.

  • 0 views
  • 03 Jul, 2021
  • 1 location
Coronary Artery Disease in Patients With Friedreich's Ataxia

clusters, molecules that are essential for energy production in our cells as well as repair of our genetic code embedded in DNA molecules. Friedreich's ataxia (FRDA) and deficiency of FXN results in a

cardiomyopathy
iron
deficiency
  • 1 views
  • 23 Mar, 2022
  • 1 location
Effect of Postprandial Insulin Administration of Faster-acting Insulin Analogue Versus Pre-prandial Administration of Acting-insulin Analogue in Cystic Fibrosis Related Diabetes (MIRE)

Cystic fibrosis related diabetes (CFRD) is a major factor of morbidity and mortality at all disease stages. Insulin deficiency has serious clinical consequences by increasing malnutrition, since

  • 0 views
  • 12 May, 2022
  • 1 location
Fuzuloparib Arsenic Trioxide Platinum Resistance Relapsed Ovarian Cancer

, the population that can benefit from PARPi is mainly focusing on BRCAm, then homologous-recombination deficiency patients. Limited data revealed the ORR was only 3-4% in homologous recombination

  • 0 views
  • 25 Mar, 2022
  • 1 location
Glutathione, Oxidative Stress and Mitochondrial Function in COVID-19

, diabetic patients and immunocompromised patients (such as HIV), and the investigators have studied these 3 populations over the past 20 years and have found that they all have deficiency of the endogenous

  • 1 views
  • 25 Mar, 2022
  • 1 location