Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Arginine · 1 trial · 1 indication
Total time plasma arginine levels are maintained above the half-saturating concentration (Km) of cationic amino acid transporter protein-1 (CAT-1), which is 150 µM (normal range of extracellular plasma arginine concentration). pK samples will be collected at 6 time-points within 8 hours: prior to arginine treatment (time 0), and at 60, 90, 120 minutes, 4 and 8 hours after the initiation of arginine therapy, and then every 24 hours up to 7 days.
The formation of NO metabolites will be measured by determination of its stable end products in serum; nitrite (NO2-) and nitrate (NO3-). Change in nitric oxide metabolites will be calculated as the difference in metabolites from the time prior to arginine treatment (baseline) to the end of the intervention period.
| Arm | Type | Description |
|---|---|---|
| Standard dose | EXPERIMENTAL | Subjects with sickle cell disease (SCD) and vaso-occlusive painful events (VOE) will be randomized to receive an intravenous (IV) infusion of a standard dose of arginine (100 mg/kg) three times a day for seven days or until discharged from the hospital, whichever occurs first |
| Loading dose + standard dose | EXPERIMENTAL | Subjects with sickle cell disease and vaso-occlusive painful events (VOE) will be randomized to receive an intravenous (IV) infusion of an initial loading dose of arginine (200 mg/kg) given over 30 minutes and then receive an intravenous (IV) infusion of a standard dose of arginine (100 mg/kg) three times a day for seven days or until discharged from the hospital, whichever occurs first |
| Loading dose + continuous infusion | EXPERIMENTAL | Subjects with sickle cell disease and vaso-occlusive painful events (VOE) will be randomized to receive an intravenous (IV) infusion of an initial loading dose of arginine (200 mg/kg) given over 30 minutes and then receive a continuous intravenous (IV) infusion of 300 mg/kg/24hr for 7 days or until discharged from the hospital, whichever occurs first |
| Non-Randomized Loading dose 500 mg/kg + standard dose | EXPERIMENTAL | Arginine will be dispensed intravenously (in the vein) as an initial bolus (loading) arginine dose at 500 mg/kg once, followed by a standard dose of 100mg/kg every 8 hours until discharge or for a total of 21 doses of arginine, whichever comes first. |
| Non-Randomized Loading dose 300 mg/kg + standard dose | EXPERIMENTAL | Arginine will be dispensed intravenously (in the vein) as an initial bolus (loading) arginine dose at 300 mg/kg once, followed by a standard dose of 100mg/kg every 8 hours until discharge or for a total of 21 doses of arginine, whichever comes first. |
| Non-Randomized Loading dose 400mg/kg + standard dose | EXPERIMENTAL | Arginine will be dispensed intravenously (in the vein) as an initial bolus (loading) arginine dose at 400 mg/kg once, followed by a standard dose of 100mg/kg every 8 hours until discharge or for a total of 21 doses of arginine, whichever comes first. |
| Name | Type | Description |
|---|---|---|
| Arginine | DRUG | Arginine will be dispensed intravenously (in the vein) in the standard dose of arginine as 100 mg/kg three times a day for seven days or until discharge. * Loading dose: 200 mg/kg once * Continuous IV: 300 mg/kg/24 hours |
| Arginine (Loading) | DRUG | Arginine will be dispensed intravenously (in the vein) as an initial bolus (loading) at each specified group dose once, followed by a standard dose of 100mg/kg every 8 hours until discharge or for a total of 21 doses of arginine, whichever comes first. |
| Arginine (Continuous) | DRUG | Arginine will be dispensed intravenously (in the vein) as a continuous IV infusion of 300 mg/kg/24hr |
Inclusion Criteria: * Established diagnosis of sickle cell disease--Hemoglobin SS (Hb-SS) or Sβᴼ-thalassemia * 7-21 years of age * Weight \>= 25kg (55lbs) * Pain requiring medical care in an acute care setting (emergency department (ED), hospital ward, day hospital, clinic) requiring parenteral opi...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Novo Nordisk A/S Sponsored ADR Class B | NVO | 5 | PHASE3 | Etavopivat Low dose |
| Novartis AG Sponsored ADR | NVS | 4 | PHASE3 | Crizanlizumab |
| Sanofi SA Sponsored ADR | SNY | 2 | PHASE3 | PCV21, 20vPCV |
| Vertex Pharmaceuticals Incorporated | VRTX | 3 | PHASE3 | CTX001 |
| Agios Pharmaceuticals, Inc. | AGIO | 2 | PHASE2 | Mitapivat |
| Pfizer Inc. | PFE | 1 | PHASE2 | Osivelotor |
| Bristol-Myers Squibb Company | BMY | 1 | PHASE1 | BMS-986470, Famotidine, Pantoprazole |
| Fulcrum Therapeutics, Inc. | FULC | 1 | PHASE2 | Pociredir |
| Beam Therapeutics, Inc. | BEAM | 2 | PHASE1 | BEAM-101 |
| Editas Medicine, Inc. | EDIT | 2 | PHASE1 | EDIT-301 |
| Disc Medicine, Inc. | IRON | 1 | PHASE1 | DISC-3405 |
| Illumina, Inc. | ILMN | 1 | - | Undisclosed |
Arginine is an investigational small molecule being studied for the treatment of pain in children with Sickle Cell Disease. It is currently in Phase 1 clinical development and has not been approved by the FDA. The drug is being evaluated in a clinical trial to assess its safety and efficacy for this indication.
Arginine is being developed by National Healthcare Corp. (NHC), a biopharmaceutical company. The company is conducting a Phase 1 clinical trial to evaluate the drug for the treatment of pain in children with Sickle Cell Disease. National Healthcare Corp. is listed on the stock exchange under the ticker symbol NHC.
Arginine is currently in Phase 1 clinical development. It is an investigational drug being studied for the treatment of pain in children with Sickle Cell Disease. The drug has not yet been approved by the FDA and is still undergoing clinical trials to evaluate its safety and efficacy.
Arginine is being studied in a Phase 1 clinical trial with the identifier NCT02447874, titled "Arginine Therapy for the Treatment of Pain in Children With Sickle Cell Disease." This trial is currently recruiting participants in the United States and has an enrollment target of 21 patients aged 7 years and older.
Arginine is the drug name used in the clinical trial for this investigational therapy. The trial is evaluating arginine as a treatment for pain in children with Sickle Cell Disease. The drug is being developed by National Healthcare Corp. and is currently in Phase 1 clinical development.