View clinical trials related to Acidosis.
Filter by:The JUMP (Journey to Understand MMA and PA) Study is being conducted by HemoShear Therapeutics and AllStripes, a rare disease online research platform. JUMP is designed to accelerate understanding of the natural course of methylmalonic acidemia (MMA) and propionic acidemia (PA) disease and treatment for families, researchers, clinicians and industry. The study will collect and provide patient medical record information from multiple institutions for families to access in one place at no cost. AllStripes will remove identifying information like name and address from these medical records and aggregate this data for the HemoShear team to better understand the medical experience and progression of MMA and PA over time. In addition, academic researchers, healthcare practitioners and patient advocacy groups can apply to use the collective patient community data to answer specific research questions at no cost. HemoShear is collecting natural history data on MMA and PA because the company needs insight into the real-world experience of many patients to better understand the disease and be able to scientifically demonstrate whether the potential new treatment they are developing is effective in improving outcomes. This natural history study will include retrospective and prospective components. The retrospective component will consist of data abstracted from primarily electronic health records (eHR) and some paper records. The prospective component will include ongoing collection of medical records from enrolled participants, and participants may opt in to complete health-related questionnaires and an optional genetic testing sub-study. After signing informed consent, participants or their legal guardians will grant permission to AllStripes to collect their health records for data abstraction. Participants may opt into an optional no cost genetic testing sub-study. The JUMP (Journey to Understand MMA and PA) sub-study will help assess whether the genetic variant of the affected person may relate to disease severity and treatment response. Getting genetic testing will enable participants to understand the genetic mutation that causes their type of methylmalonic acidemia (MMA) or propionic acidemia (PA). Knowing the genetic mutations (whether from the MMUT, MMAA or MMAB gene or PCCA or PCCB) can help the impacted person, their caregivers and healthcare professionals understand the potential course of disease and select approaches to better manage the disease. The additional information will enable HemoShear and AllStripes to understand whether different genetic variants impact the disease journey and outcomes. A separate informed consent will be obtained for participating in the sub-study.
The purpose of this study is to assess the safety, tolerability, PK and PD of BBP-671 in healthy volunteers and patients with Propionic Acidemia or Methylmalonic Acidemia.
This is an interventional study to assess the safety, PK, and efficacy of HST5040 in 12 subjects - 6 with Methylmalonic Acidemia (MMA) and 6 with Propionic Acidemia (PA). The study consists of 3 parts: - Part A: Open-label, within-subject, dose escalation study in PA and MMA subjects ≥ 2 years old to identify a safe and pharmacologically active (optimal) dose of HST5040 for use in Part B. Subjects will continue in a Part A open-label extension until all subjects complete Part A and the optimal dose of HST5040 is identified for use in Part B. - Part B: 6-month, randomized, double-blind, placebo-controlled, 2-period crossover in the same subjects from Part A to evaluate safety and efficacy of the optimal dose of HST5040 in addition to standard of care (SoC). - Part C: open-label long-term extension study in PA and MMA subjects ≥ 2 years old (N = approximately 12, 6 each) to evaluate the long-term safety and efficacy of the optimal dose of HST5040. This study will determine whether HST5040 can improve levels of disease-associated toxins that accumulate in patients with PA and MMA.
The main objective of this study is to evaluate the efficacy of SZC as compared to placebo in maintaining normal sK+ in patients with hyperkalemia and metabolic acidosis associated with CKD
The SUNRISE trial is a first-in-human (FIH), open-label, Phase 1/2 clinical trial designed to assess the safety, tolerability and preliminary efficacy of a single intravenous infusion of hLB-001 in pediatric patients with MMA characterized by methylmalonyl-CoA mutase gene (MMUT) mutations. hLB-001 is a liver-targeted, recombinant engineered adeno-associated viral (rAAV) vector utilizing the LK03 capsid (rAAV-LK03), designed to non-disruptively integrate the human methylmalonyl-CoA mutase gene at the albumin locus. The trial is expected to enroll pediatric patients with ages ranging from 6 months to 12 years, initially starting with 3 to 12 year-old patients and then adding patients aged 6 months to 2 years.
Early Basal Insulin Administration in Adult Diabetic Ketoacidosis Management
This is a single-arm study to evaluate safety and tolerability of oral IW-6463 in adults diagnosed with MELAS.
The purpose of this study is to evaluate the effect of TRC101 on the progression of chronic kidney disease (CKD) and to evaluate the safety profile of TRC101 in CKD patients with metabolic acidosis.
This is a phase 3, prospective, multicenter, randomized, double-blinded, placebo-controlled withdrawal study comparing the efficacy of ADV7103 versus placebo in preventing the development of metabolic acidosis defined by serum bicarbonate levels in pediatric (6 months to < 18 years of age) and adult (18 to 65 years of age) subjects with primary Distal Renal Tubular Acidosis (dRTA).
Objectives: Intravenous (IV) fluid administration is a fundamental component of diabetic ketoacidosis (DKA) treatment. Normal saline (NS), the most common IV fluid used in DKA management, contains more chloride than human blood. Excessive amounts of chloride have been shown to cause a detrimental metabolic acidosis. Other IV fluids have more physiologic chloride levels, such as lactated ringers (LR). This study will compare the rates of hyperchloremic metabolic acidosis in children treated with NS to those treated with LR to determine the effect on overall length of acidosis and length of stay in the hospital or intensive care unit. Design: Single-center, double blinded, randomized controlled trial. Subjects: Children aged 0 to 18 years who present with diabetic ketoacidosis and require pediatric intensive care unit admission. Patients with evidence of shock, multi-organ failure or clinically significant cerebral edema will be excluded. The projected study population will be 104 patients, 52 in each arm. Interventions: Patients will be enrolled within 1 hour of presentation to the emergency room or pediatric intensive care unit if transferred directly from another facility. They will be randomized to receive intravenous fluids containing 0.9% saline or lactated ringers. All patients will be treated using the institutional DKA protocol with the content of the intravenous fluids being the only difference in treatment between arms. Study intervention lasts until the end of the acute management of DKA. Planned measurements and study outcomes: The primary study outcome will be duration of metabolic acidosis. Resolution of metabolic acidosis will be defined in three ways: 1. Normalization of the ketosis; 2. Normalization of the serum pH; 3. Normalization of the serum bicarbonate level. Secondary outcomes will include length of stay in the pediatric intensive care unit and length of stay in the hospital. All outcomes will be correlated with the overall chloride load given via intravenous fluids during DKA management. Regression modelling will control for any baseline differences between the groups in regards to severity of DKA, and if newly diagnosed or poorly controlled diabetes mellitus.