U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

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Showing 701 - 710 of 13311 results

Status:
Investigational
Source:
NCT03098797: Phase 2/Phase 3 Interventional Completed Barth Syndrome
(2017)
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Elamipretide or SS-31 (D-Arg-2', 6'-dimethyltyrosine-Lys-Phe-NH2) is a mitochondria-targeted antioxidant. Elamipretide, is a peptide compound that readily penetrates cell membranes, and targets the inner mitochondrial membrane where it binds reversibly to cardiolipin. In preclinical or clinical studies, elamipretide increases mitochondrial respiration, improves the electron transport chain function and ATP production and reduces formation of pathogenic ROS levels. This elamipretide-cardiolipin association has been shown to normalize the structure of the inner mitochondrial membrane, thereby improving mitochondrial function. Functional benefit is achieved through improvement of ATP production and interruption and potential reversal of damaging oxidative stress. Stealth BioTherapeutics is investigating elamipretide in late stage clinical studies in three primary mitochondrial diseases—primary mitochondrial myopathy, Barth syndrome and Leber’s hereditary optic neuropathy – as well as an earlier stage clinical study in dry age-related macular degeneration.
Status:
Investigational
Source:
NCT03202303: Phase 2 Interventional Recruiting Autism Spectrum Disorder
(2019)
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Cannabidivarin is a homolog of cannabidiol, with a well-established antiepileptiform profile in preclinical studies, both in vitro and in vivo animal models of epilepsy. The oral bioavailability of cannabidivarin is very low (about 6%) due to erratic absorption and first pass metabolism. After oral administration, the maximum plasma concentration of Cannabidivarin is rising in about three hours and the drug has a large volume of distribution, because of his link to protein plasma, being highly liposoluble, so CBDV can penetrate well to the brain. Cannabidivarin is also metabolized in the liver to 7-COOH and 6-OH metabolites, but the mechanism is also unknown. There is an ongoing phase II double-blind, placebo-controlled trial that is assessing the efficacy and safety of cannabidivarin in Children With Autism Spectrum Disorder (ASD).
Status:
Investigational
Source:
NCT02381288: Phase 2 Interventional Terminated Low Testosterone
(2015)
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

TAK-448 is an investigational oligopeptide analog of kisspeptin and a potent agonist of the GPR54 receptor. In animals, acute TAK-448 administration stimulates luteinizing hormone (LH)/follicle-stimulating hormone release, whereas continuous subcutaneous exposure rapidly down-regulates the pituitary-gonadal axis, with rapid reduction of testosterone levels in a dose-dependent manner. TAK-448 has exhibited potent antitumor activity in rat androgen-dependent prostate cancer models. In accordance with the T reductions, TAK-448 treatment showed also more rapid reduction in plasma prostate-specific antigen(PSA) levels. TAK-448 had been in phase II clinical trials for the treatment of prostate cancer. However, this research has been discontinued.
Status:
Investigational
Source:
INN:elacridar [INN]
Source URL:

Class (Stereo):
CHEMICAL (ACHIRAL)


Conditions:

Elacridar is an oral bioenhancer that targets multiple drug resistance in tumors. Elacridar is a strong and relatively specific inhibitor of P-gp and BCRP, two main efflux transporters. Development of elacridar is assumed to have been discontinued.
Status:
Investigational
Source:
NCT02445976: Phase 2 Interventional Completed Prostate Cancer
(2015)
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)



Seviteronel (VT-464) is a 17,20-lyase selective inhibitor of CYP17A1, which plays key roles in adrenal and intratumoral de novo biosynthesis of androgens. The inhibition of 17,20-lyase activity by seviteronel (VT-464) is enough to reduce androgen levels, and its preserving of 17alpha-hydroxylase activity largely avoids interference with the production of other steroidal hormones. Seviteronel (VT-464) also has shown AR-antagonist activity independent of CYP17 enzyme inhibition. It is currently in phase 2 clinical trials as a therapeutic for castration-resistant prostate cancer patients.
Status:
Investigational
Source:
NCT04688164: Phase 3 Interventional Completed Major Depressive Disorder
(2021)
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Investigational
Source:
NCT03731026: Phase 3 Interventional Unknown status Breast Cancer
(2018)
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Investigational
Source:
NCT02536781: Phase 2/Phase 3 Interventional Completed Inflammation of Mouth
(2015)
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Investigational
Source:
NCT03761979: Not Applicable Interventional Completed Low Bone Density
(2017)
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Investigational
Source:
NCT03870776: Phase 2 Interventional Active, not recruiting Depression
(2019)
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Mapreg is developing pregnenolone methyl ether (MAP4343), an injectable neurosteroid stimulator of the tubulin polymerisation and neurite growth stimulator. The synthetic pregnenolone-derivative MAP4343 (3β-methoxy-preg-nenolone) binds in vitro to microtubule-associated-protein 2 (MAP2), stimulates the polymerization of tubulin, enhances the extension of neurites and protects neurons against neurotoxic agents. MAP4343 has been selected after screening of a large library ofnatural and synthetic steroids. MAP4343 has similar in vitro activity as pregnenolone; it cannot be converted into metabolites with hormonal activities, and has been shown to have in vivo beneficial effects in rat models of spinal cord injury. MAP4343 has an interesting pharmacological profile because no in vitro affinity for any CNS neurotransmitter receptor was found. MAP4343 has been shown to have antidepressant efficacy in rats. In the rat isolation-rearing model of depression, administration of MAP4343 showed persistent efficacy in recovering recognition memory deficit, stronger and more rapid anxiolytic activity, and more rapid rescue of passive coping behavior compared with FLX. The behavioral effects of MAP4343 correlated with changes in α-tubulin isoforms in the hippocampus, amygdala, and pre-frontal cortex (PFC). Its efficacy was also assessed in vivo with the most commonly used thoracic spinal cord compression/contusion models in rats. In the three models used, the post-traumatic subcutaneous injection of MAP4343 significantly improved the recovery of locomotor function after spinal cord injury, as shown by an earlier and more complete recovery compared to vehicle-treated rats. The results obtained in three different rat models of spinal cord injury demonstrate the beneficial effects of this therapeutic strategy and identify MAP4343 as a potential treatment for acute spinal cord injury. MAP4343 received EU Orphan Drug designation for spinal cord injury. MAP4343 is in phase II clinical trials by Mapreg for the treatment of depression and in phase I clinical trials for the treatment of spinal cord injury and traumatic brain injury.