A Single-arm Phase III Clinical Study to Evaluate the Efficacy and Safety of Hydroxocobalamin Chloride Injection in Participants With Methylmalonic Acidemia (MMA) With Elevated Homocysteine (Cobalamin C Deficiency)

This study is a Single-Center, Single-Arm, open-label, Phase III clinical study to evaluate the efficacy, safety characteristics of Hydroxocobalamin Chloride Injection (20 mg/mL) for Maintenance Therapy in participants with Methylmalonic Acidemia (MMA) with Elevated Homocysteine (Cobalamin C Deficiency).

开始日期2025-08-31

申办/合作机构

石药集团中奇制药技术（天津）有限公司

NCT05536596

/ Unknown status临床2/3期IIT

Pharmacological Treatment on the Recovery of Neurosensory Disturbance After Bilateral Sagittal Split Osteotomy: a Randomized, Double-blind Trial

The bilateral sagittal split osteotomy (BSSO) of the mandible is one of the most used surgical techniques to achieve a harmonious jaw relation in the context of orthognathic surgery. Nevertheless, one of its main complications is neurosensory damage to the inferior alveolar nerve, which can cause severe impact in the quality of life on patients who suffer from it permanently. The purpose of this randomized clinical trial is to provide rigorous scientific evidence of the pharmacological effect of 1) Melatonin, 2) combination uridine triphosphate (UTP), cytidine monophosphate (CMP), and hydroxycobalamin (UTP/CMP/hydroxycobalamin) and 3) hydroxycobalamin regarding neurosensory disturbances incidence and persistence after BSSO.

开始日期2022-09-30

申办/合作机构

Universitat Internacional de Catalunya

[+1]

IRCT20211207053311N1

/ Recruiting临床3期IIT

Evaluation of response rate to hydroxycobalamin treatment in patients with methylmalonic acidemia

开始日期2022-02-04

申办/合作机构

Mashhad University of Medical Sciences

100 项与羟钴胺相关的临床结果

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100 项与羟钴胺相关的转化医学

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100 项与羟钴胺相关的专利（医药）

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1,526

项与羟钴胺相关的文献（医药）

2025-11-01·JOURNAL OF CRANIO-MAXILLOFACIAL SURGERY

Selegiline as an innovative drug for the treatment of inferior alveolar nerve injury: a randomized clinical trial

Article

作者: Monteiro, João Luiz ; Mesquita, Bruno Silva ; Gomes, Ana Cláudia Amorim ; Lago, Carlos Augusto Pereira ; Andrade, Emanuel Savio Souza ; Vasconcelos, Belmiro Cavalcanti Egito

BACKGROUND:

Inferior alveolar nerve (IAN) injuries are common complications of mandibular orthognathic surgery. Selegiline has demonstrated neuroprotective effects in preclinical studies.

OBJECTIVE:

To evaluate the effect of oral selegiline hydrochloride on neurosensory recovery following bilateral sagittal split osteotomy.

METHODS:

A randomized, double-blind, controlled trial was conducted with 40 patients. Participants received either selegiline (5 mg/day for 30 days) or a combination of uridine 5'-triphosphate (UTP), cytidine-5'-monophosphate (CMP), and hydroxycobalamin (three times daily). Neurosensory assessments included two-point discrimination, directional perception, and 1-point sensitivity, evaluated at six time points. Pain was assessed using a visual analog scale (VAS).

RESULTS:

The selegiline group showed earlier improvement in some parameters, especially in 1-point tactile sensitivity of the lower lip at day 15 (p < 0.05). Differences in two-point discrimination and directional perception were not consistent. Pain scores were lower in the selegiline group at day 15 (p < 0.05). By day 90, both groups had similar recovery levels.

CONCLUSION:

Selegiline hydrochloride may contribute to early neurosensory recovery after IAN injury, particularly in the lower lip. However, these benefits were limited to specific parameters and time points. Further studies are needed to validate its therapeutic potential.

2025-10-01·BRITISH JOURNAL OF ANAESTHESIA

Hydroxocobalamin for vasoplegia in cardiac surgery: a retrospective cohort analysis

Article

作者: Alfirevic, Andrej ; Skubas, Nikolaos J ; Sessler, Daniel I ; Hohlfelder, Benjamin ; Elgharably, Haytham ; Yang, Dongsheng ; Ahuja, Sanchit ; Kelava, Marta ; Khanna, Ashish K ; Shah, Karan ; Zakaria, Luai ; Duncan, Andra E

BACKGROUND:

Vasoplegia during cardiac surgery is common and associated with perioperative mortality. Hydroxocobalamin is a catecholamine-sparing approach for treating vasoplegia. However, there is currently limited evidence to inform its use.

METHODS:

We conducted a single-centre retrospective propensity-matched cohort study of patients who developed vasoplegia after cardiopulmonary bypass. Our primary outcome was vasopressor requirement, expressed as norepinephrine equivalents (NEE), at 1, 4, 24, and 48 h after treatment. Our secondary outcome was a collapsed composite of in-hospital complications comprising stroke, sternal wound infection, mesenteric ischaemia, renal failure, and death.

RESULTS:

We identified 2727 (11%) patients with vasoplegia from 2018 to 2024. The final analysis cohort included 229 matched pairs. Hydroxocobalamin administration was associated with a significant reduction in median NEE infusion rates of -0.04 (95% confidence interval [CI]: -0.07 to -0.02; P=0.008) μg kg^-1 min^-1 at 1 h, -0.05 (95% CI: -0.07 to -0.02; P<0.001) μg kg^-1 min^-1 at 4 h, and -0.04 (95% CI: -0.06 to -0.02; P=0.004) μg kg^-1 min^-1 at 24 h, but not at 48 h, -0.02 (95% CI: -0.05 to 0.0; P=0.220) μg kg^-1 min^-1. Over the initial 24 h, hydroxocobalamin reduced NEE by 14% compared with the reference group. The risk of in-hospital complications was similar in each group (odds ratio: 1.06; 95% CI: 0.74-1.54; P=0.745).

CONCLUSIONS:

Hydroxocobalamin administration was associated with reduced vasopressor requirement over 24 h, but not thereafter. Robust trials are needed to determine whether this relationship is causal, and whether a reduction in vasopressor use improves substantive clinical outcomes.

2025-10-01·Journal of Medical Toxicology

Efficacy of Treating Nifedipine-Induced Shock with Hydroxocobalamin in a Swine Model

Article

作者: Murphy, Christine ; Kerns Ii, William P ; Rozario, Nigel ; Patterson, Charity G ; Gellar, Michael ; Runyon, Michael

INTRODUCTION:

Calcium channel antagonists contribute to many overdose related deaths each year and treatment options are limited. Hydroxocobalamin has shown promise in reversal of multiple shock states, and we evaluated its use in the treatment of nifedipine-induced shock in a swine model.

METHODS:

Twenty-two swine (39 to 50 kg) were anesthetized, instrumented, and acclimatized. Toxicity was induced by administering a nifedipine infusion at 0.0266 mg/kg/min. Once the toxic end point, defined as a 20% decrease from the initial mean arterial pressure, was reached, all animals received a 20 mL/kg bolus of saline and either 60 mL of saline (NP group) or 150 mg/kg of hydroxocobalamin dissolved in 60 mL of saline (NP + HX group). Hemodynamics were analyzed and compared between the NP and NP + HX groups over time using linear mixed models with Bonferroni correction.

RESULTS:

Modeling of the hemodynamic data demonstrated an increase in both systolic blood pressure and change in MAP from the nadir. Mean arterial pressure (MAP) and diastolic blood pressure were increased (p < 0.01) in the NP + HX group at multiple time points. There were no differences detected in the time-to-death between groups.

CONCLUSION:

Improvements in hemodynamics were noted in the group treated with hydroxocobalamin, but there was no evidence for improvement in mortality. Given this, hydroxocobalamin may serve a role in bridging patients to high dose insulin, vasopressors, extracorporeal membrane oxygenation, or transfer to a higher level of care.

项与羟钴胺相关的新闻（医药）

2025-10-18

·GlobeNewswire-Health Care

General Oncology Announces Promising Preliminary Phase 1 Results from SHARON Trial Presented at ESMO 2025

Novel regimen GO-4, combining melphalan, BCNU, hydroxocobalamin, ascorbic acid, and autologous stem cells shows manageable adverse reactions and a strong signal of efficacy in patients with BRCA-related pancreatic cancerBOSTON, Oct. 18, 2025 (GLOBE NEWSWIRE) -- General Oncology, Inc. (“General Oncology” or the “Company”), a clinical-stage biopharmaceutical company dedicated to pioneering first-in-class medicines for metastatic cancers, today announced preliminary results from its ongoing Phase 1 SHARON trial (NCT04150042), evaluating GO-4: the Company’s patented Advanced Redox Modulation regimen consisting of melphalan, BCNU, hydroxocobalamin, ascorbic acid, and autologous stem cell infusion in patients with advanced pancreatic ductal adenocarcinoma (PDAC) or breast cancer harboring an inherited BRCA/PALB2 mutation. The data will be featured in a poster presentation at the European Society for Medical Oncology (ESMO) Congress 2025. “GO-4 is a therapy designed to achieve major log reductions in tumor cell burden. BRCA-mutated cancer cells are hypersensitive to DNA cross-linking agents, but reversion mutations can lead to drug resistance. GO-4 uses Advanced Redox Modulation to impair DNA repair and sensitize resistant cells to melphalan,” said Arnold Glazier, M.D., Chief Scientific Officer of General Oncology. “We are encouraged by the data demonstrating that GO-4 has a manageable safety profile and a strong signal of efficacy, with two stage IV pancreatic cancer patients remaining progression-free, for 4 years and 2 years after two cycles of GO-4 without additional treatment. We are committed to further exploring the full therapeutic potential of this combination therapy with continued research.” Presentation details and key highlights from the preliminary analysis are summarized below: Title: The SHARON Trial: Melphalan, BCNU, Hydroxocobalamin, Ascorbic Acid, and Stem Cells for Pancreatic and Breast Cancer and an Inherited BRCA/PALB2 MutationAbstract Number: 2228PPresenter: Kenneth H. Yu, M.D., Memorial Sloan Kettering Cancer Center, Principal InvestigatorDate and Time: Sunday, October 19 at 12:00–12:45 CESTLocation: Poster Area, Hall 25Key highlights: Eleven patients with metastatic PDAC and one patient with breast cancer received one to two rounds of treatment, six weeks apart; seven patients had progressive disease at enrollment.Safety was the primary endpoint, and no treatment-related mortality or long-term toxicities were reported.In patients with PDAC who had stable or responding disease at enrollment (N = 5), median progression-free survival was 14.2 months, with N = 2 free of disease at 48 and 23 months with no therapy subsequent to two cycles of GO-4. Kenneth H. Yu, M.D., Memorial Sloan Kettering Cancer Center, principal investigator of the SHARON trial, added, “We are always looking to expand treatment options, particularly for patients with advanced diseases who may have limited treatment options. The preliminary data from the SHARON trial demonstrating feasibility and a strong signal of efficacy in patients with BRCA-mutated pancreatic cancer are highly encouraging. The durable responses highlight the need for further clinical evaluation of this approach.” The SHARON trial is ongoing and will be expanding to include patients without a BRCA or PALB2 mutation, and General Oncology plans to continue evaluating outcomes and long-term follow-up in this high-need patient population. About the SHARON TrialThe SHARON trial is a Phase 1 study designed to evaluate the safety, feasibility, and preliminary efficacy of GO-4, the Company’s patented Advanced Redox Modulation regimen consisting of melphalan, BCNU, hydroxocobalamin, ascorbic acid, and autologous stem cell infusion in patients with advanced pancreatic or breast cancer. Patients enrolled to date have had an inherited BRCA/PALB2 mutation. For the remainder of the trial, patients with or without an inherited BRCA/PALB2 mutation will be able to enroll. About General Oncology, Inc.General Oncology is a clinical-stage biopharmaceutical company dedicated to pioneering first-in-class medicines for metastatic cancers and other proliferative diseases. The Company is developing GO-4, an investigational therapy for metastatic cancers, currently completing the Phase 1 SHARON trial (NCT04150042) for metastatic cancers in the setting of a BRCA1/2 or PALB2 mutation. With a commitment to innovation, General Oncology is focused on advancing transformative therapies for cancer patients. For more information about General Oncology, please visit www.generaloncology.com. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995 and applicable securities laws. Forward-looking statements can be identified by words such as “designed to,” “will,” and similar references to future periods. Examples of forward-looking statements include, among others, statements we make regarding the expectations, plans, and prospects generally, and specifically for the SHARON trial, and General Oncology’s drug development programs. Forward-looking statements are neither historical facts nor assurances of future performance. Instead, they are based only on our management’s current beliefs, expectations and assumptions regarding the future of our business, future plans and strategies, projections, anticipated events and trends, the economy and other future conditions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict and many of which are outside of our control. Our actual results and financial condition may differ materially from those indicated in the forward-looking statements. Therefore, you should not rely on any of these forward-looking statements. Important factors that could cause our actual results and financial condition to differ materially from those indicated in the forward-looking statements include, among others, the development and regulatory approval of investigational therapies, patient enrollment, trial execution, safety and efficacy outcomes, and other risks that may be detailed in General Oncology’s filings with the Securities and Exchange Commission. Any forward-looking statements made by us in this press release is based only on information currently available to us and speaks only as of the date on which it is made. General Oncology undertakes no obligation to update or revise any forward-looking statements, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise. Investor Contact:Abid Ansari(617) 468-4646ir@generaloncology.com Media Contact:Madelin Hawtinmhawtins@lifescicomms.com

临床结果临床1期ASCO会议

2025-10-15

·GlobeNewswire-Health Care

General Oncology to Present Preliminary Phase 1 Results from SHARON Trial in Patients with BRCA/PALB2-mutated Pancreatic and Breast Cancer at ESMO 2025

BOSTON, Oct. 13, 2025 -- General Oncology, Inc. (“General Oncology” or the “Company”), a clinical-stage biopharmaceutical company dedicated to pioneering first-in-class medicines for metastatic cancers, today announced that it will deliver a poster presentation featuring preliminary results from its ongoing Phase 1 SHARON trial (NCT04150042) evaluating GO-4: the Company’s patented Advanced Redox Modulation regimen consisting of melphalan, BCNU, hydroxocobalamin, ascorbic acid, and autologous stem cell infusion in patients with advanced pancreatic ductal adenocarcinoma or breast cancer harboring an inherited BRCA/PALB2 mutation at the upcoming European Society of Medical Oncology (ESMO) Congress 2025, taking place October 17-21 in Berlin, Germany. Title: The SHARON Trial: Melphalan, BCNU, Hydroxocobalamin, Ascorbic Acid, and Stem Cells for Pancreatic and Breast Cancer and an Inherited BRCA/PALB2 Mutation Abstract Number: 2228P Presenter: Kenneth H. Yu, M.D., Memorial Sloan Kettering Cancer Center, Principal Investigator Date and Time: Sunday, October 19 at 12:00–12:45 CEST Location: Poster Area, Hall 25 The SHARON trial is a Phase 1 study designed to evaluate the safety, feasibility, and preliminary efficacy of GO-4, the Company’s patented Advanced Redox Modulation regimen consisting of melphalan, BCNU, hydroxocobalamin, ascorbic acid, and autologous stem cell infusion in patients with advanced pancreatic or breast cancer. Patients enrolled to date have had an inherited BRCA/PALB2 mutation. For the remainder of the trial, patients with or without an inherited BRCA/PALB2 mutation will be able to enroll. General Oncology is a clinical-stage biopharmaceutical company dedicated to pioneering first-in-class medicines for metastatic cancers and other proliferative diseases. The Company is developing GO-4, an investigational therapy for metastatic cancers, currently completing the Phase 1 SHARON trial (NCT04150042) for metastatic cancers in the setting of a BRCA1/2 or PALB2 mutation. With a commitment to innovation, General Oncology is focused on advancing transformative therapies for cancer patients. The content above comes from the network. if any infringement, please contact us to modify.

ASCO会议临床结果

2025-07-04

·石药集团

石药集团罕见病药物高浓度盐酸羟钴胺注射液获批临床

7月3日，石药集团（1093.HK）宣布，本集团开发的高浓度盐酸羟钴胺注射液（下称：该产品）已获中华人民共和国国家药品监督管理局批准，可在中国开展临床试验，用于治疗罕见病甲基丙二酸血症（MMA）。MMA是一种常染色体隐性遗传有机酸代谢疾病，最早可在新生儿期发病，并已在中国被收录于国家卫生健康委员会等5部门联合制定的《第一批罕见病目录》中。羟钴胺注射剂是治疗维生素B12反应型MMA的首选推荐药物，需终身使用。目前，国内外尚无针对MMA适应症的羟钴胺注射剂获批上市，对于MMA患者而言，仍存在迫切的未被满足的医疗需求。本次获批的临床适应症为儿童伴有同型半胱氨酸升高的MMA（cb1 C型）。该产品成为国内首款获批开展MMA临床试验的羟钴胺注射剂，其开发有望为MMA患者提供新的治疗选择，具有较高的临床开发价值。【声明】1、本新闻旨在分享公告信息，非广告用途；2、本资料仅供医药专业人士参考，非产品推荐依据，也不应被视为诊疗建议。

申请上市临床1期

100 项与羟钴胺相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D01027	羟钴胺	V03AB33 B03BA03	DB00200

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
氰化物中毒	美国	BTG International, Inc.	2006-12-15
弱视	澳大利亚	Pfizer Australia Pty Ltd.	2006-06-26
Leber氏遗传性视神经萎缩	澳大利亚	Pfizer Australia Pty Ltd.	2006-06-26
巨幼红细胞性贫血	中国	华润双鹤药业股份有限公司	1996-01-01
巨幼红细胞性贫血	中国	上海锦帝九州药业（安阳）有限公司	1996-01-01
恶性贫血	中国	华润双鹤药业股份有限公司	1996-01-01
恶性贫血	中国	上海锦帝九州药业（安阳）有限公司	1996-01-01
神经痛	中国	华润双鹤药业股份有限公司	1996-01-01
神经痛	中国	上海锦帝九州药业（安阳）有限公司	1996-01-01
维生素B12缺乏症	中国	华润双鹤药业股份有限公司	1996-01-01
维生素B12缺乏症	中国	上海锦帝九州药业（安阳）有限公司	1996-01-01
中毒	美国	Merck & Co., Inc.	1975-04-18

未上市

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适应症	最高研发状态	国家/地区	公司	日期
甲基丙二酸血症	临床3期	-	石药集团中奇制药技术（天津）有限公司	2025-08-31
甲基丙二酸血症伴同型半胱氨酸尿症	临床3期	中国	石药集团中奇制药技术（石家庄）有限公司	2025-08-12

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03735316 (CTgov)	临床2期	血管麻痹 \| 低血压	8	Cardiopulmonary Bypass+Hydroxocobalamin (B12a)	醖顧簾鏇膚艱願膚齋艱(網鹽願鑰範襯顧蓋遞簾) = 網簾窪壓顧積獵鹽觸顧觸夢鹹襯襯餘淵夢獵鹹 (鏇鏇鏇膚窪醖淵積繭鑰, 遞鏇膚遞觸網鬱齋鑰鹹 ~ 範醖築遞鹽蓋鹹廠膚膚) 更多	-	2023-05-10
				Cardiopulmonary Bypass (Placebo)	醖顧簾鏇膚艱願膚齋艱(網鹽願鑰範襯顧蓋遞簾) = 餘範襯築艱窪構襯窪製觸夢鹹襯襯餘淵夢獵鹹 (鏇鏇鏇膚窪醖淵積繭鑰, 襯蓋範顧鏇餘願襯蓋繭 ~ 鏇鏇願壓鹽觸憲選構製) 更多
NCT03783091 (IV-HOCSS Trial, Pubmed)	临床2期	脓毒性休克	-	IV-HOC	築獵餘遞鏇簾醖夢顧鬱(鹽膚壓鏇醖廠築鹽艱簾) = 構鹽壓餘願艱醖鹹壓蓋觸鏇構夢鹹襯齋製夢艱 (鹽廠壓衊繭壓淵膚鏇構 ) 更多	积极	2022-09-26
				Placebo	築獵餘遞鏇簾醖夢顧鬱(鹽膚壓鏇醖廠築鹽艱簾) = 獵糧願醖壓願鹹觸鬱觸觸鏇構夢鹹襯齋製夢艱 (鹽廠壓衊繭壓淵膚鏇構 ) 更多