246. メチルマロン酸血症 Methylmalonic acidemia Clinical trials / Disease details

臨床試験数 : 19 / 薬物数 : 26 - (DrugBank : 3) / 標的遺伝子数 : 17 - 標的パスウェイ数 : 23

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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	NCT05040178 (ClinicalTrials.gov)	March 2022	16/6/2021	A Surveillance Protocol of Carbaglu® for the Treatment of Hyperammonemia	A Surveillance Protocol of Carbaglu® for the Treatment of Hyperammonemia Due to Methylmalonic Acidem ...	Hyperammonemia	Drug: Carglumic Acid	Recordati Rare Diseases	NULL	Not yet recruiting	N/A	N/A	All	10		United States
2	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 ...	methylmalonic acidemia;C537358	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
3	NCT04899310 (ClinicalTrials.gov)	August 6, 2021	19/5/2021	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, Pharm ...	Methylmalonic Acidemia	Biological: mRNA-3705	ModernaTX, Inc.	NULL	Recruiting	1 Year	N/A	All	33	Phase 1/Phase 2	Canada;United Kingdom
4	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 Methylmalon ...	Methylmalonic Acidemia	Biological: hLB-001	LogicBio Therapeutics, Inc.	NULL	Recruiting	6 Months	12 Years	All	8	Phase 1/Phase 2	United States;Saudi Arabia
5	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 Health ...	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	18 Years	55 Years	All	128	Phase 1	United States
6	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 A ...	Methylmalonic Acidemia;Propionic Acidemia	Drug: HST5040;Drug: Placebo	HemoShear Therapeutics	NULL	Recruiting	2 Years	N/A	All	12	Phase 2	United States
7	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 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
8	EUCTR2019-001061-32-GB (EUCTR)	25/10/2019	30/05/2019	A clinical study to evaluate mRNA-3704 in Patients with MMA	A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and ...	Isolated Methylmalonic AcidemiaDue to Methylmalonyl-CoA Mutase Deficiency;Therapeutic area: Body pro ...	Product Name: mRNA-3704 INN or Proposed INN: NA Other descriptive name: NA	ModernaTX, Inc.	NULL	Not Recruiting			Female: yes Male: yes	34	Phase 1;Phase 2	United States;United Kingdom
9	NCT03810690 (ClinicalTrials.gov)	May 28, 2019	14/1/2019	Open Label Study of mRNA-3704 in Patients With Isolated Methylmalonic Acidemia	A Global, Phase 1/2, Open Label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and ...	Methylmalonic Acidemia (MMA);Metabolism, Inborn Errors	Biological: mRNA-3704	ModernaTX, Inc.	NULL	Withdrawn	1 Year	N/A	All	0	Phase 1/Phase 2	United States
10	NCT04176523 (ClinicalTrials.gov)	January 15, 2019	20/11/2019	Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ...	Understanding the Long-Term Management of Organic Acidemia Patients With CARBAGLU®: A Mixed Methods ...	Methylmalonic Acidemia;Propionic Acidemia	Drug: Carglumic Acid	Recordati Rare Diseases	NULL	Recruiting	6 Months	99 Years	All	75		France;Italy;Norway;Spain;United Kingdom

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