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Sterile normal saline (0.9% nacl)    (DrugBank: -)

3 diseases
告示番号疾患名（ページ内リンク）臨床試験数
62発作性夜間ヘモグロビン尿症1
234ペルオキシソーム病（副腎白質ジストロフィーを除く。）2
254ポルフィリン症1

62. 発作性夜間ヘモグロビン尿症　［臨床試験数：202，薬物数：120（DrugBank：25），標的遺伝子数：15，標的パスウェイ数：90］
Searched query = "Paroxysmal nocturnal hemoglobinuria", "PNH"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 202 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT02352493
(ClinicalTrials.gov)		January 201523/1/2015A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNHA Phase 1/2 Single-ascending and Multiple-ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-CC5 in Healthy Adult Volunteers and Patients With Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal Hemoglobinuria (PNH)Drug: ALN-CC5;Drug: Sterile Normal Saline (0.9% NaCl)Alnylam PharmaceuticalsNULLCompleted18 Years45 YearsAll62Phase 1;Phase 2Spain;United Kingdom

234. ペルオキシソーム病（副腎白質ジストロフィーを除く。）　［臨床試験数：38，薬物数：37（DrugBank：12），標的遺伝子数：12，標的パスウェイ数：42］
Searched query = "Peroxisomal disease (except Adrenoleukodystrophy)", "Peroxisomal disease", "Peroxisomal disorder", "Peroxisome biogenesis disorder", "PEX gene disorder", "Zellweger syndrome", "Neonatal adrenoleukodystrophy", "Rhizomelic chondrodysplasia punctata type 1", "RCDP type 1", "RCDP1", "Peroxisomal beta-oxidation enzyme deficiency", "Acyl-CoA oxidase deficiency", "AOX deficiency", "D-Bifunctional protein deficiency", "DBP deficiency", "Sterol carrier protein X deficiency", "SCPx deficiency", "2-methylacyl-CoA racemase deficiency", "Alpha-methylacyl-CoA racemase deficiency", "AMACR deficiency", "Refsum disease", "Plasmalogen biosynthesis enzyme deficiency", "Rhizomelic chondrodysplasia punctata type 2", "RCDP type 2", "RCDP2", "Rhizomelic chondrodysplasia punctata type 3", "RCDP type 3", "RCDP3", "Primary hyperoxaluria type 1", "PH1", "Acatalasemia", "Acatalasia", "Contiguous ABCD1/DXS1357E deletion syndrome", "CADDS"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
2 / 38 trials found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT03847909
(ClinicalTrials.gov)		October 28, 201915/2/2019A Study to Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (Subcutaneous Use) in Patients With Primary HyperoxaluriaPrimary Hyperoxaluria Type 1 (PH1);Primary Hyperoxaluria Type 2 (PH2);Kidney Diseases;Urologic Diseases;Genetic DiseaseDrug: DCR-PHXC;Drug: Sterile Normal Saline (0.9% NaCl)Dicerna Pharmaceuticals, Inc.NULLRecruiting6 YearsN/AAll36Phase 2United States;Australia;Canada;France;Germany;Israel;Italy;Japan;Lebanon;Netherlands;New Zealand;Poland;Romania;Spain;United Kingdom
2NCT03681184
(ClinicalTrials.gov)		November 27, 201819/9/2018A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1ILLUMINATE-A: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study With an Extended Dosing Period to Evaluate the Efficacy and Safety of Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1Primary Hyperoxaluria Type 1 (PH1)Drug: Lumasiran;Drug: Sterile Normal Saline (0.9% NaCl)Alnylam PharmaceuticalsNULLActive, not recruiting6 YearsN/AAll39Phase 3United States;France;Germany;Israel;Netherlands;Switzerland;United Arab Emirates;United Kingdom

254. ポルフィリン症　［臨床試験数：59，薬物数：52（DrugBank：17），標的遺伝子数：17，標的パスウェイ数：33］
Searched query = "Porphyria", "CEP", "Erythropoietic protoporphyria", "EPP", "X-linked dominant protoporphyria", "XLDP", "PCT", "HEP", "AIP", "ADP", "VP", "Hereditary coproporphyria", "HCP"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
1 / 59 trial found
No.TrialIDDate_
enrollment		Date_
registration		Public_titleScientific_titleConditionInterventionPrimary_
sponsor		Secondary_
sponsor		Recruitment_
Status		Inclusion_
agemin		Inclusion_
agemax		Inclusion_
gender		Target_
size		PhaseCountries
1NCT02452372
(ClinicalTrials.gov)		May 6, 201519/5/2015A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)A Phase 1, Single-ascending Dose, Multiple-ascending Dose, and Multi-dose Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN AS1 in Patients With Acute Intermittent Porphyria (AIP)Acute Intermittent PorphyriaDrug: givosiran (ALN-AS1);Drug: Sterile Normal Saline (0.9% NaCl)Alnylam PharmaceuticalsNULLCompleted18 Years65 YearsAll40Phase 1United States;Sweden;United Kingdom