Drugs for Congenital Hemolytic Anemia (from DrugBank, HMDB, Dgidb, PharmGKB, IUPHAR, NovoSeek, BitterDB):
(show all 33)
# |
|
Name |
Status |
Phase |
Clinical Trials |
Cas Number |
PubChem Id |
1 |
|
Benzocaine |
Approved, Investigational |
Phase 3 |
|
1994-09-7, 94-09-7 |
2337 |
Synonyms:
(P-(Ethoxycarbonyl)phenylamine
4-(Ethoxycarbonyl)aniline
4-(Ethoxycarbonyl)phenylamine
4-Aminobenzoate
4-Aminobenzoate ethyl ester
4-Aminobenzoic acid
4-Aminobenzoic acid ethyl ester
4-Carbethoxyaniline
Acetate, benzocaine
Aethoform
AEZODENT
Amben ethyl ester
Americaine
Anaesthan-syngala
Anaesthesin
Anaesthin
Anestezin
Anesthesin
Anesthesine
Anesthone
ANTHISAN PLUS
AR01
AR-01
Baby anbesol
Bensokain
Benzoak
Benzocaina
BENZOCAINE
Benzocaine acetate
Benzocaine formate
Benzocaine hydrobromide
Benzocaine hydrochloride
Benzocaine methanesulfonate
Benzocainum
BURNEZE
CHILDREN'S CHLORASEPTIC
DEQUACAINE
Dermoplast
Diet ayds
Ethoform
Ethyl 4-aminobenzoate
|
Ethyl 4-aminobenzoic acid
Ethyl aminobenzoate
Ethyl aminobenzoic acid
Ethyl P-aminobenzenecarboxylate
Ethyl p-aminobenzoate
Ethyl p-aminobenzoic acid
Ethyl p-aminophenylcarboxylate
Ethyl p-aminophenylcarboxylic acid
Ethylester kyseliny P-aminobenzoove
Formate, benzocaine
Hurricaine
Hydrobromide, benzocaine
Hydrochloride, benzocaine
Identhesin
Keloform
LANACANE
MEDILAVE
Methanesulfonate, benzocaine
NESTOSYL
Norcain
Norcaine
NSC-41531
NSC-4688
Orabase-b
Ora-jel
ORAJEL
Orthesin
p-(Ethoxycarbonyl)aniline
P-Aminobenzoate
P-Aminobenzoic acid
P-Aminobenzoic acid ethyl ester
Parathesin
Parathesine
p-Carbethoxyaniline
p-Ethoxycarboxylic aniline
Slim mint gum
Solarcaine
Solu H
Topcaine
TYROSOLVEN
TYROZETS
|
|
2 |
|
Tannic acid |
Approved |
Phase 3 |
|
1401-55-4 |
16129878 16129778 |
Synonyms:
Acid, tannic
ácido tánico
Acids, tannic
E181
FEMA NO. 3042
Gallotannic acid
|
Gallotannin
NSC-656273
TANNIC ACID
Tannic acids
Tannins
|
|
3 |
|
Pyruvate |
|
Phase 3 |
|
|
|
4 |
|
Alemtuzumab |
Approved, Investigational |
Phase 1, Phase 2 |
|
216503-57-0 |
|
Synonyms:
ALEMTUZUMAB
ALEMTUZUMAB (GENETICAL RECOMBINATION)
CAMPATH
campath 1H|Campath®|campath-1H|Lemtrada®
CAMPATH MABCAMPATH
CAMPATH MABCAMPATH, LEMTRADA
|
CAMPATH-1H
GZ402673
GZ-402673
LDP-03
LEMTRADA
MABCAMPATH
|
|
5 |
|
Sirolimus |
Approved, Investigational |
Phase 1, Phase 2 |
|
53123-88-9 |
5284616 6436030 |
Synonyms:
(-)-Rapamycin
(1R,9S,12S,15R,16E,18R,19R,21R,23S,24E,26E,28E,30S,32S,35R)-1,18-Dihydroxy-12-{(2S)-1-[(1S,3R,4R)-4-hydroxy-3-methoxycyclohexyl]propan-2-yl}-19,30-dimethoxy-15,17,21,23,29,35-hexamethyl-11,36-dioxa-4-azatricyclo[30.3.1.0(4,9)]hexatriaconta-16,24,26,28-tetraene-2,3,10,14,20-pentone
Antibiotic ay 22989
AY-22989
AY-22989|Fyarro® (sirolimus albumin-bound particles)|Rapamune®|rapamycin|WY-090217
L04AA10
NSC-226080
Perceiva
RAPA
Rapammune
|
Rapamune
Rapamycin
Rapamysin
RPM
Sirolimus
Sirolimús
Sirolimusum
SM-88 COMPONENT SIROLIMUS
WY-090217
|
|
6 |
|
Miconazole |
Approved, Investigational, Vet_approved |
Phase 1, Phase 2 |
|
22916-47-8 |
4189 |
Synonyms:
1-(2,4-Dichloro-beta-((2,4-dichlorobenzyl)oxy)phenethyl)imidazole
1-[2-(2,4-Dichloro-benzyloxy)-2-(2,4-dichloro-phenyl)-ethyl]-1H-imidazole
Brentan
CONOFITE
Dactarin
DERMAZOLE
DUMICOAT
Esteve brand OF miconazole
florid(nitrate)|Monistat®|R-14889
Janssen cilag brand OF miconazole
Janssen-cilag brand OF miconazole
Johnston and johnston brand OF miconazole nitrate
LORAMYC
MCZ
MICATIN
|
Miconasil nitrate
Miconazol
Miconazole
Miconazole esteve brand
Miconazole janssen-cilag brand
Miconazole nitrate
Miconazolum
Monistat
MONISTAT-3
MONISTAT-DERM
Nitrate, miconasil
Nitrate, miconazole
NSC-170986
Oravig
VUSION
|
|
7 |
|
Clotrimazole |
Approved, Vet_approved |
Phase 1, Phase 2 |
|
23593-75-1 |
2812 |
Synonyms:
(Chlorotrityl)imidazole
1-((2-Chlorophenyl)diphenylmethyl)-1H-imidazole
1-(a-(2-Chlorophenyl)benzhydryl)imidazole
1-(alpha-(2-Chlorophenyl)benzhydryl)imidazole
1-(O-Chloro-a,a-diphenylbenzyl)imidazole
1-(O-Chloro-alpha,alpha-diphenylbenzyl)imidazole
1-(O-Chlorotrityl)imidazole
1-(O-Chloro-α,α-diphenylbenzyl)imidazole
1-(Α-(2-chlorophenyl)benzhydryl)imidazole
ABTRIM
ACTAVALL
Bay b 5097
BAY-5097
Bayer brand 1 OF clotrimazole
Bayer brand 2 OF clotrimazole
CANDIDEN
Canesten
Canesten 1-day cream combi-pak
Canesten 1-day therapy
Canesten 3-day therapy
Canesten 6-day therapy
CANESTEN AF
Canesten combi-pak 1-day therapy
Canesten combi-pak 3-day therapy
Canesten cream
CANESTEN CRM COMBI
CANESTEN INTERNAL
Canesten solution
CANESTEN VC
Canesten®|Lotrimin®
Canestine
Chlotrimazole
Clotrimaderm
Clotrimaderm cream
Clotrimazol
CLOTRIMAZOLE
Clotrimazole schering brand
Clotrimazolum
Desamix F
Empecid
FB b 5097
Fem care
FemCare
FUNGEDERM
GNF-PF-3499
Gyne lotrimin
Gyne-lotrimin
Gyne-lotrimin 3
Gyne-lotrimin 3 combination pack
Gyne-lotrimin combination pack
Gyne-lotrimin3
Gyne-lotrimin3 combination pack
|
Gynix
Kanesten
Klotrimazole
Lopac-C-6019
Lotrimax
Lotrimin
LOTRIMIN (TN)
Lotrimin af
Lotrimin af cream
Lotrimin af jock-itch cream
Lotrimin af lotion
Lotrimin af solution
Lotrimin cream
Lotrimin lotion
Lotrimin solution
Lotrisone
MASNODERM
mono-Baycuten
Monobaycuten
Mycelax
Mycelex
MYCELEX (TN)
Mycelex 7
Mycelex cream
Mycelex g
Mycelex otc
Mycelex solution
Mycelex troches
Mycelex twin pack
Mycelex: mycosporinrimazole
Mycelex-7
Mycelex-7 combination pack
Mycelex-g
MYCIL GOLD
Myclo cream
Myclo solution
Myclo spray solution
Myclo-gyne
Mycosporin
Mykosporin
Neo-zol cream
NSC-257473
Otomax
Pedisafe
Prestwick_120
PRIVACOM
Rimazole
Schering brand OF clotrimazole
Tibatin
Trimysten
Trivagizole 3
Veltrim
|
|
8 |
|
Triethylenetetramine |
Approved, Investigational |
Phase 2 |
|
112-24-3 |
5565 |
Synonyms:
2-AMINOETHYL-[2-(2-AMINOETHYLAMINO)ETHYL]AMINE
Merck brand OF trientine hydrochloride
N,N'-bis(2-aminoethyl)-1,2-ethanediamine
N'-[2-(2-AMINOETHYLAMINO)ETHYL]ETHANE-1,2-DIAMINE
N'-[2-(2-AZANYLETHYLAMINO)ETHYL]ETHANE-1,2-DIAMINE
NSC-443
Syprine
TETA
|
Trien
Trientine
Trientine dihydrochloride
Trientine hydrochloride
TRIETHYLENE TETRAMINE
Triethylenetetraamine
Triethylenetetramine
|
|
9 |
|
Cholecalciferol |
Approved, Nutraceutical, Vet_approved |
Phase 2 |
|
67-97-0, 1406-16-2 |
5280795 10883523 |
Synonyms:
(+)-Vitamin D3
(1S,3Z)-3-[(2E)-2-[(1R,3AR,7as)-7a-methyl-1-[(2R)-6-methylheptan-2-yl]-2,3,3a,5,6,7-hexahydro-1H-inden-4-ylidene]ethylidene]-4-methylidene-cyclohexan-1-ol
(1S,3Z)-3-[(2E)-2-[(1R,3AS,7AR)-1-[(1R)-1,5-DIMETHYLHEXYL]-7A-METHYL-2,3,3A,5,6,7-HEXAHYDRO-1H-INDEN-4-YLIDENE]ETHYLIDENE]-4-METHYLENE-1-CYCLOHEXANOL
(1S,3Z)-3-[(2E)-2-[(1R,3AS,7AR)-1-[(1R)-1,5-DIMETHYLHEXYL]-7A-METHYL-2,3,3A,5,6,7-HEXAHYDRO-1H-INDEN-4-YLIDENE]ETHYLIDENE]-4-METHYLENE-CYCLOHEXAN-1-OL
(1S,3Z)-3-[(2E)-2-[(1R,3AS,7AR)-7A-METHYL-1-[(2R)-6-METHYLHEPTAN-2-YL]-2,3,3A,5,6,7-HEXAHYDRO-1H-INDEN-4-YLIDENE]ETHYLIDENE]-4-METHYLIDENECYCLOHEXAN-1-OL
(1S,3Z)-3-[(2E)-2-[(1R,3AS,7AR)-7A-METHYL-1-[(2R)-6-METHYLHEPTAN-2-YL]-2,3,3A,5,6,7-HEXAHYDRO-1H-INDEN-4-YLIDENE]ETHYLIDENE]-4-METHYLIDENE-CYCLOHEXAN-1-OL
(1S,3Z)-3-{2-[(1R,4E,7AR)-7A-METHYL-1-[(2R)-6-METHYLHEPTAN-2-YL]-OCTAHYDRO-1H-INDEN-4-YLIDENE]ETHYLIDENE}-4-METHYLIDENECYCLOHEXAN-1-OL
(3 beta,5Z,7E)-9,10-Secocholesta-5,7,10(19)-trien-3-ol
(3b,5Z,7E)-9,10-Secocholesta-5,7,10(19)-trien-3-ol
(3beta,5Z,7E)-9,10-Secocholesta-5,7,10(19)-trien-3-ol
(3BETA,Z,7E)-9,10-SECOCHOLESTA-5,7,10(19)-TRIEN-3-OL
(3Î’,5Z,7E)-9,10-secocholesta-5,7,10(19)-trien-3-ol
(5E,7E)-9,10-SECOCHOLESTA-5,7,10-TRIEN-3-OL
(5Z,7E)-(3S)-9,10-Secocholesta-5,7,10(19)-trien-3-ol
3-BETA,Z,7E-9,10-SECOCHOLESTR-5,7,10(19)-TRIEN-3-OL
7-DEHYDROCHOLESTEROL
9,10-SECOCHOLESTA-5,7,10(19)-TRIEN-3BETA-OL
9,10-SECOCHOLESTA-5,7,10(19)-TRIEN-3-BETA-OL
9,10-SECOCHOLESTA-5,7,10(19)-TRIEN-3-OL
9,10-SECOCHOLESTA-5,7,10-TRIEN-3-OL
ACCRETE D3
ACIFEROL D3
ACTIVATED
Activated 7-dehydrocholesterol
ADCAL-D3
ARACHITOL
AVITICOL
BIOLIFE VITAMIN D3
BIO-VITAMIN D3
CACIT D3
CALCEOS
CALCICHEW D3
CALCICHEW D3 FTE
CALCICHEW D3 ONCE DAILY
CALCI-D
Calciferol
Calciol
CALDRINK D3
CALFOVIT D3
CC
Cholecalciferol
CHOLECALCIFEROL D3
Cholecalciferols
Cholecalciferolum
COLECAL D3
Colecalciferol
Colecalciferolum
COLEKAL-D3
COLEVIT D3
CUBICOLE D3
D3 LEMON MELTS
D3-50
D3-VIGANTOL
DEKRISTOL
DELSTEROL
Delta-D
DEPARAL
DESUNIN
DEVARON
Dihydrocholesterol
DLUX 400
D-MAX
D-TRACETTEN
|
D-VIT3
EBIVIT
E-D3
EVACAL D3
FERACOL
FULTIUM DAILY D3
FULTIUM-D3
GRANUVIT D3
HUX D3
INVITA D3
ISO D3
KORA LIQUID
MICRO-DEE
NATECAL D3
NATURE'S REMEDY VITAMIN D3
NEO-D
NPHD3
NSC-375571
Oleovitamin D3
OSTEOCAPS D3
PLENACHOL
PRO D3
PRO D3 FOLIC
PRO D3 FORTE
PROVITINA
QUINTOX
RICKETON
ROYALVIT D3
STEXEROL-D3
STIVIT-D3
SUNVIT-D3 1,000
SUNVIT-D3 10,000
SUNVIT-D3 2,000
SUNVIT-D3 20,000
SUNVIT-D3 3,000
SUNVIT-D3 400
SUNVIT-D3 5,000
SUNVIT-D3 50,000
SUNVIT-D3 800
SYNERVIT-D3
THEICAL D-3
THORENS
TRIVITAN
VIDDE-3-HYDROSOL
VI-DE3
VIDEKHOL
VIGANTOL
VIGANTOLETTEN
VIGANTOLETTEN 500
VIGORSAN
VIT D3 STREULI
VITA-D3
VITAMIN D
Vitamin D 3
VITAMIN D ASSAY SYSTEM SUITABILITY
Vitamin D NOS
Vitamin D, unspecified form
Vitamin D3
Vitamin D-3
VITINC DAN-DEE-3
ZYMAD
δ-D
|
|
10 |
|
Ergocalciferol |
Approved, Nutraceutical |
Phase 2 |
|
50-14-6 |
5280793 |
Synonyms:
(+)-Vitamin D2
(1S,3Z)-3-[(2E)-2-[(1R,3AS,7AR)-1-[(E,2R,5R)-5,6-DIMETHYLHEPT-3-EN-2-YL]-7A-METHYL-2,3,3A,5,6,7-HEXAHYDRO-1H-INDEN-4-YLIDENE]ETHYLIDENE]-4-METHYLIDENECYCLOHEXAN-1-OL
(1S,3Z)-3-[(2E)-2-[(1R,3AS,7AR)-1-[(E,2R,5R)-5,6-DIMETHYLHEPT-3-EN-2-YL]-7A-METHYL-2,3,3A,5,6,7-HEXAHYDRO-1H-INDEN-4-YLIDENE]ETHYLIDENE]-4-METHYLIDENE-CYCLOHEXAN-1-OL
(1S,3Z)-3-[(2E)-2-[(1R,3AS,7AR)-7A-METHYL-1-[(E,1R,4R)-1,4,5-TRIMETHYLHEX-2-ENYL]-2,3,3A,5,6,7-HEXAHYDRO-1H-INDEN-4-YLIDENE]ETHYLIDENE]-4-METHYLENE-1-CYCLOHEXANOL
(1S,3Z)-3-[(2E)-2-[(1R,3AS,7AR)-7A-METHYL-1-[(E,1R,4R)-1,4,5-TRIMETHYLHEX-2-ENYL]-2,3,3A,5,6,7-HEXAHYDRO-1H-INDEN-4-YLIDENE]ETHYLIDENE]-4-METHYLENE-CYCLOHEXAN-1-OL
(1S,3Z)-3-{2-[(1R,3AS,4E,7AR)-1-[(2R,3E,5R)-5,6-DIMETHYLHEPT-3-EN-2-YL]-7A-METHYL-OCTAHYDRO-1H-INDEN-4-YLIDENE]ETHYLIDENE}-4-METHYLIDENECYCLOHEXAN-1-OL
(3b,5Z,7E,22E)-9,10-Secoergosta-5,7,10(19),22-tetraen-3-ol
(3beta,5Z,7E,22E)-9,10-Secoergosta-5,7,10(19),22-tetraen-3-ol
(3Î’,5Z,7E,22E)-9,10-secoergosta-5,7,10(19),22-tetraen-3-ol
(5E,7E,22E)-9,10-Secoergosta-5,7,10,22-tetraen-3-ol
(5Z,7E,22E)-(3S)-9,10-Seco-5,7,10(19),22-ergostatetraen-3-ol
(5Z,7E,22E)-(3S)-9,10-Secoergosta-5,7,10(19),22-tetraen-3-ol
22-Tetraen 3beta 9,10,secoergosta-5,7,10(19)-ol
4-Methylene-3-[2-[tetrahydro-7a-methyl-1-(1,4,5-trimethyl-2-hexenyl)-4(3ah)-indanylidene]ethylidene]-cyclohexanol
9,10-Secoergosta-5,7,10(19),22-tetraen-3b-ol
Activated ergosterol
beta-Ol
Buco-D
Calciferol
CALCIFEROL IN ARACH OIL
Calciferols
Calciferon 2
CALTRATE
Condacaps
Condocaps
Condol
Crtron
Crystallina
D2, Vitamin
Daral
D-Arthin
Davitamon D
Davitin
Decaps
Dee-osterol
Dee-ron
Dee-ronal
Dee-roual
delta-Arthin
Deltalin
delta-Tracetten
De-rat concentrate
Deratol
Detalup
D-FORTE
Diactol
Divit urto
Doral
Drisdol
D-Tracetten
ECIFEROL D2
Ercalciol
|
Ergocalciferol
ERGOCALCIFÉROL
Ergocalciferol oil
Ergocalciferols
Ergocalciferolum
ERGO-D2
ERGORAL D2
Ergorone
Ergosterol activated
Ergosterol irradiated
Ertron
Fortodyl
Geltabs
Hi-deratol
Infron
Irradiated ergosta-5,7,22-trien-3beta-ol
LANES
Metadee
Mina D2
Mulsiferol
Mykostin
Novovitamin-D
NSC-62792
Oleovitamin D
OLEOVITAMIN D, SYNTHETIC
Oleovitamin D2
Osteil
Ostelin
OSTO-D2
Radiostol
Radstein
Radsterin
Rodine C
Shock-ferol
Shock-ferol sterogyl
Sterogyl
STEROGYL 15H
STEROGYL-15
Synthetic vitamin D
Uvesterol D
Uvesterol-D
Vio D
Vio-D
Viostdrol
Viosterol
Viosterol in oil
VITAMIN D
Vitamin D 2
Vitamin D2
Vitamina D2
Vitavel-D
|
|
11 |
|
Anti-Bacterial Agents |
|
Phase 1, Phase 2 |
|
|
|
12 |
|
Anti-Infective Agents |
|
Phase 1, Phase 2 |
|
|
|
13 |
|
Antifungal Agents |
|
Phase 1, Phase 2 |
|
|
|
14 |
|
Antibiotics, Antitubercular |
|
Phase 1, Phase 2 |
|
|
|
15 |
|
Immunosuppressive Agents |
|
Phase 1, Phase 2 |
|
|
|
16 |
|
Antineoplastic Agents, Immunological |
|
Phase 1, Phase 2 |
|
|
|
17 |
|
Vitamins |
|
Phase 2 |
|
|
|
18 |
|
Vitamin D2 |
|
Phase 2 |
|
|
3249 |
Synonyms:
Calciferols
D2, Vitamin
Ergocalciferol
|
Ergocalciferols
Vitamin D 2
Vitamin D2
|
|
19 |
|
Hormones |
|
Phase 2 |
|
|
|
20 |
|
Trace Elements |
|
Phase 2 |
|
|
|
21 |
|
Calciferol |
|
Phase 2 |
|
|
|
22 |
|
Ergocalciferols |
|
Phase 2 |
|
|
|
23 |
|
Calcium, Dietary |
|
Phase 2 |
|
|
|
24 |
|
Micronutrients |
|
Phase 2 |
|
|
|
25 |
|
Chelating Agents |
|
Phase 2 |
|
|
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26 |
|
Chrysarobin |
|
Phase 2 |
|
|
|
27 |
|
Calcium |
Nutraceutical |
Phase 2 |
|
7440-70-2 |
271 |
Synonyms:
Ca
Ca(2+)
Ca2+
Calcio
CALCIUM
Calcium element
|
CALCIUM ion
Calcium(2+)
Calcium, doubly charged positive ion
Calcium, elemental
Elemental calcium
Kalzium
|
|
28 |
|
Immunologic Factors |
|
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29 |
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Immunoglobulins, Intravenous |
|
|
|
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30 |
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Immunoglobulins |
|
|
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|
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31 |
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Antibodies |
|
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32 |
|
gamma-Globulins |
|
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33 |
|
Rho(D) Immune Globulin |
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Interventional clinical trials:
(show all 18)
# |
Name |
Status |
NCT ID |
Phase |
Drugs |
1 |
An Open-Label Study To Evaluate the Efficacy and Safety of AG-348 in Regularly Transfused Adult Subjects With Pyruvate Kinase (PK) Deficiency |
Completed |
NCT03559699 |
Phase 3 |
AG-348 |
2 |
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of AG-348 in Not Regularly Transfused Adult Subjects With Pyruvate Kinase Deficiency |
Completed |
NCT03548220 |
Phase 3 |
Placebo;AG-348 |
3 |
A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension Period |
Recruiting |
NCT05144256 |
Phase 3 |
Mitapivat;Mitapivat-matching placebo |
4 |
A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase Deficiency Who Are Not Regularly Transfused, Followed by a 5-Year Open-label Extension Period |
Recruiting |
NCT05175105 |
Phase 3 |
Mitapivat;Mitapivat-matching placebo |
5 |
An Open-Label, Multicenter, Extension Study of AG-348 in Adult Subjects With Pyruvate Kinase Deficiency Previously Enrolled in AG-348 Studies |
Active, not recruiting |
NCT03853798 |
Phase 3 |
AG-348 |
6 |
Nonmyeloablative Allogeneic Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation For Severe Congenital Anemias Including Sickle Cell Disease (SCD) and Beta-Thalassemia |
Recruiting |
NCT00061568 |
Phase 1, Phase 2 |
Alemtuzumab;Sirolimus |
7 |
An Explananatory, Proof-of-concept Study of Senicapoc in Patients With Familial Dehydrated Stomatocytosis Caused by the V282M Mutation in the Gardos (KCNN4) Channel |
Recruiting |
NCT04372498 |
Phase 1, Phase 2 |
Senicapoc (synonyms: ICA-17043; 2,2-bis-(4-fluorophenyl)-2-phenylacetamide) |
8 |
Daily Vitamin D for Sickle-cell Respiratory Complications |
Active, not recruiting |
NCT04170348 |
Phase 2 |
Daily oral vitamin D3, 3,333 IU;Bolus oral vitamin D3, 100,000 IU;Placebo oral tablet |
9 |
A Phase 2, Open Label, Randomized, Dose Ranging, Safety, Efficacy, Pharmacokinetic and Pharmacodynamic Study of AG-348 in Adult Patients With Pyruvate Kinase Deficiency |
Active, not recruiting |
NCT02476916 |
Phase 2 |
AG-348 |
10 |
Gene Therapy for Pyruvate Kinase Deficiency (PKD): A Phase I Clinical Trial to Evaluate the Safety of the Infusion of Autologous CD34+ Cells Transduced With a Lentiviral Vector Carrying the Codon Optimized Red Cell Pyruvate Kinase (coRPK) Gene in Adult and Pediatric Subjects With PKD |
Recruiting |
NCT04105166 |
Phase 1 |
|
11 |
Pyruvate Kinase Deficiency Epidemiological Study. An International, Multicentre, Epidemiological Observational Study |
Unknown status |
NCT03866590 |
|
|
12 |
Pyruvate Kinase Deficiency (PKD) Natural History Study |
Completed |
NCT02053480 |
|
|
13 |
National Exhaustive Cohort of Hereditary Stomatocytoses and Other Channelopathies Affecting the Red Blood Cell |
Recruiting |
NCT04778657 |
|
|
14 |
Pyruvate Kinase Deficiency Global Longitudinal Registry Substudy of Protocol AG348-C-008: Cognition in Participants With Pyruvate Kinase Deficiency |
Recruiting |
NCT04995315 |
|
|
15 |
Pyruvate Kinase Deficiency Global Longitudinal Registry: Patient-Reported Outcomes |
Recruiting |
NCT04964323 |
|
|
16 |
Pyruvate Kinase Deficiency Global Longitudinal Registry |
Recruiting |
NCT03481738 |
|
|
17 |
Acute Human Parvovirus B19 Infection Triggers Immune-Mediated Transient Bone Marrow Failure Syndrome, Extreme Direct Hyperbilirubinemia and Acute Hepatitis in Patients With Hereditary Hemolytic Anemias: Multicenter Pathophysiological Study |
Active, not recruiting |
NCT04822090 |
|
|
18 |
Characterization Of Acquired Pyruvate Kinase Deficiency In Clonal Myeloid Neoplasms |
Not yet recruiting |
NCT04902833 |
|
|
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