246. Methylmalonic acidemia Clinical trials / Disease details
Clinical trials : 23 / Drugs : 31 - (DrugBank : 8) / Drug target genes : 17 - Drug target pathways : 23
Showing 1 to 10 of 23 diseases
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | ChiCTR2100054613 | 2022-08-01 | 2021-12-21 | Effects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with Combined methylmalonic acidemia and homocystinuria Effects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with ... | Effects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with Combined methylmalonic acidemia and homocystinuria Effects of early application of PEG-rhGH on neurodevelopment and cognitive function in children with ... | Methylmalonic acidemia | Experimental group:PEG-rhGH;Control Group:None; | Zhengzhou Children's Hospital | NULL | Pending | 0 | 3 | Both | Experimental group:30;Control Group:30; | Phase 4 | China |
| 2 | NCT05506254 (ClinicalTrials.gov) | July 20, 2022 | 16/8/2022 | Long-term Follow-up Study of Patients Who Received hLB-001 Gene Therapy | A Long-term Follow-up Study of Patients in LogicBio Clinical Trials for Methylmalonic Acidemia Characterized by MMUT Mutations Who Received hLB-001 A Long-term Follow-up Study of Patients in LogicBio Clinical Trials for Methylmalonic AcidemiaCharac ... | Methylmalonic Acidemia | Drug: hLB-001 | LogicBio Therapeutics, Inc | Alexion | Active, not recruiting | N/A | N/A | All | 8 | United States | |
| 3 | NCT05040178 (ClinicalTrials.gov) | June 30, 2022 | 16/6/2021 | An Observational Study of Carbaglu® for the Treatment of MMA and PA in Adults and Pediatrics | A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidemia (MMA) and Propionic Acidemia (PA) in Adult and Pediatric Patient Populations A Non-Interventional Post-Authorization Safety Study (PASS) of Carbaglu® for the Treatment of Hypera ... | Hyperammonemia;Methylmalonic Acidemia;Propionic Acidemia | Drug: Carglumic Acid | Recordati Rare Diseases | Target PharmaSolutions, Inc. | Recruiting | N/A | N/A | All | 20 | United States | |
| 4 | NCT05295433 (ClinicalTrials.gov) | March 8, 2022 | 14/2/2022 | An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in the mRNA-3705-P101 Study An Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participan ... | A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activity of mRNA-3705 in Participants Previously Enrolled in the mRNA-3705-P101 Study A Phase 1/2, Global, Open-Label, Extension Study to Evaluate the Long-Term Safety and Clinical Activ ... | Methylmalonic Acidemia | Drug: mRNA-3705 | ModernaTX, Inc. | NULL | Recruiting | 1 Year | N/A | All | 33 | Phase 1/Phase 2 | Canada;United Kingdom |
| 5 | JPRN-jRCT2041210065 | 04/09/2021 | 04/09/2021 | A Phase I/II Study of Bucladesine in Patients with Severe Methylmalonic Acidemia | A Phase I/II Multi-Institutional Study of Bucladesine in Patients with Severe Methylmalonic Acidemia - McAMP A Phase I/II Multi-Institutional Study of Bucladesine in Patients with Severe Methylmalonic Acidemia ... | methylmalonic acidemia;C537358 | Bucladesine will be administered intravenously over 60 minutes once a week. Starting dose is 0.3mg/kg. Dosage will be incerased to 0.55mg/kg, then 1.65mg/kg with monitoring PK and safety data. Bucladesinewill be administered intravenously over 60 minutes once a week. Starting dose is 0.3mg/kg ... | Ito Tetsuya | NULL | Recruiting | >= 3month old | Not applicable | Both | 6 | Phase 1-2 | Japan |
| 6 | NCT04899310 (ClinicalTrials.gov) | August 6, 2021 | 19/5/2021 | A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia A Study to Assess Safety, Pharmacokinetics, and Pharmacodynamics of mRNA-3705 in Participants With I ... | A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants With Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Tolerability, Pharm ... | Methylmalonic Acidemia | Biological: mRNA-3705 | ModernaTX, Inc. | NULL | Recruiting | 1 Year | N/A | All | 33 | Phase 1/Phase 2 | United States;Canada;Netherlands;United Kingdom |
| 7 | NCT04581785 (ClinicalTrials.gov) | May 29, 2021 | 2/10/2020 | Gene Therapy With hLB-001 in Pediatric Patients With Severe Methylmalonic Acidemia | A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalonic Acidemia Characterized by MMUT Mutations A Phase 1/2 Open-label Clinical Study of hLB-001 Gene Therapy in Pediatric Patients With Methylmalon ... | Methylmalonic Acidemia | Biological: hLB-001 | LogicBio Therapeutics, Inc | Alexion | Active, not recruiting | 6 Months | 12 Years | All | 8 | Phase 1/Phase 2 | United States;Saudi Arabia |
| 8 | NCT04836494 (ClinicalTrials.gov) | March 25, 2021 | 29/3/2021 | A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Health ... | A First-in-human, Randomized, Placebo-controlled, Single and Multiple Ascending Dose Escalation to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of BBP-671 in Healthy Subjects and In Patients With Propionic Acidemia or Methylmalonic Acidemia A First-in-human, Randomized, Placebo-controlled, Single and Multiple Ascending Dose Escalation to E ... | Healthy Volunteers;Propionic Acidemia;Methylmalonic Acidemia;Organic Acidemia | Drug: BBP-671;Drug: Placebo | CoA Therapeutics, Inc., a BridgeBio company | NULL | Recruiting | 15 Years | 55 Years | All | 128 | Phase 1 | United States |
| 9 | NCT04732429 (ClinicalTrials.gov) | March 15, 2021 | 21/1/2021 | Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia | A Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia Followed by a Randomized, Double-blind, Placebo-controlled, 2-period Crossover Study and an Open-label, Long-term Extension Study A Phase 2 Open-label, Dose Escalation Study of HST5040 in Subjects With Propionic or Methylmalonic A ... | Methylmalonic Acidemia;Propionic Acidemia | Drug: HST5040;Drug: Placebo | HemoShear Therapeutics | NULL | Recruiting | 2 Years | N/A | All | 12 | Phase 2 | United States;Australia |
| 10 | NCT04284917 (ClinicalTrials.gov) | November 28, 2019 | 24/2/2020 | Long-term Efficacy of Carglumic Acid in Organic Acidemia. | Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients With Propionic Acidemia (PA) or Methylmalonic Acidemia (MMA). Evaluate the Long Term Effectiveness & Safety of the Use of Carglumic Acid (Carbaglu®) in Patients W ... | Propionic Acidemia (PA) , Methylmalonic Acidemia (MMA) | Drug: Carglumic Acid (Carbaglu®) | National Taiwan University Hospital | NULL | Enrolling by invitation | 18 Years | N/A | All | 5 | N/A | Taiwan |