Drugs for Limb-Girdle Muscular Dystrophy (from DrugBank, HMDB, Dgidb, PharmGKB, IUPHAR, NovoSeek, BitterDB):
(show all 32)
# |
|
Name |
Status |
Phase |
Clinical Trials |
Cas Number |
PubChem Id |
1 |
|
(3-Carboxy-2-(R)-Hydroxy-Propyl)-Trimethyl-Ammonium |
Experimental |
Phase 4 |
|
461-06-3 |
|
Synonyms:
CARNITINE
CARNITINE CHLORIDE
|
|
|
2 |
|
Lisinopril |
Approved, Investigational |
Phase 2, Phase 3 |
|
83915-83-7, 76547-98-3 |
5362119 |
Synonyms:
(S)-1-(N(2)-(1-Carboxy-3-phenylpropyl)-L-lysyl)-L-proline
[N2-[(S)-1-CARBOXY-3-phenylpropyl]-L-lysyl-L-proline
Acerbon
Acercomp
Alapril
Carace
Cipral
Cipril
Coric
Inhibril
Inopril
Linopril
Linvas
Lipril
Lisinal
LISINOPRIL
Lisinopril anhydrous
LISINOPRIL DIHYDRATE
LISINOPRIL HYDRATE
Lisinopril maleate (1:1)
Lisinopril sulfate (1:2)
Lisinoprilum
Lisipril
|
Lisoril
Lispril
Loril
LPR
Lysinopril
MK-521
Noperten
Novatec
Presiten
Prinil
Prinivil
Prinivil®|Zestril®
Prinzide
Renacor
Sinopril
Sinopryl
Tensopril
Tensyn
Tersif
Vivatec
Zestoretic
Zestril
|
|
3 |
|
Deflazacort |
Approved, Investigational |
Phase 3 |
|
14484-47-0 |
26709 189821 |
Synonyms:
2-{11-hydroxy-6,9,13-trimethyl-16-oxo-5-oxa-7-azapentacyclo[10.8.0.0,.0,.0,]icosa-6,14,17-trien-8-yl}-2-oxoethyl acetic acid
CALCORT
DEFLAZACORT
EMFLAZA
|
|
|
4 |
|
Ubidecarenone |
Approved, Investigational, Nutraceutical |
Phase 2, Phase 3 |
|
303-98-0 |
5281915 |
Synonyms:
(all-e)-2-(3,7,11,15,19,23,27,31,35,39-Decamethyl-2,6,10,14,18,22,26,30,34,38-tetracontadecaenyl)-5,6-dimethoxy-3-methyl-2,5-cyclohexadiene-1,4-dione
(all-e)-2,3-Dimethoxy-5-methyl-6-(3,7,11,15,19,23,27,31-octamethyl-2,6,10,14,18,22,26,30-dotriacontaoctaenyl)-2,5-cyclohexadiene-1,4-dione
2-((all-e)-3,7,11,15,19,23,27,31,35,39-Decamethyl-2,6,10,14,18,22,26,30,34,38-tetracontadecaenyl)-5,6-dimethoxy-3-methyl-p-benzoquinone
2-(3,7,11,15,19,23,27,31,35,39-Decamethyl-2,6,10,14,18,22,26,30,34,38-tetracontadecaenyl)-5,6-dimethoxy-3-methyl-p-benzoquinone
2,3-Dimethoxy-5-methyl-6-decaprenylbenzoquinone
2-[(2E,6E,10E,14E,18E,22E,26E,30E,34E)-3,7,11,15,19,23,27,31,35,39-Decamethyl-2,6,10,14,18,22,26,30,34,38-tetracontadecaenyl]-5,6-dimethoxy-3-methyl- 2,5-cyclohexadiene-1,4-dione
2-[(2E,6E,10E,14E,18E,22E,26E,30E,34E)-3,7,11,15,19,23,27,31,35,39-Decamethyltetraconta-2,6,10,14,18,22,26,30,34,38-decaen-1-yl]-5,6-dimethoxy-3-methyl-1,4-benzoquinone
4-Ethyl-5-fluoropyrimidine
Adelir
all-trans-Ubiquinone
API 31510
API-31510
Aqua Q 10l10
Aqua Q10
Bio-quinon
Bio-quinone Q10
Coenzyme Q10
Coenzyme Q-10
CO-Enzyme Q10
CoQ
CoQ 10
CoQ10
Ensorb
Kaneka Q10
|
Kudesan
Li-Q-sorb
Liquid-Q
Neuquinon
Neuquinone
NSC-140665
NSC-140865
PureSorb Q 40
Q
Q 10AA
Q 199
Q10
Q-Gel
Q-Gel 100
Q-Ter
Ubidecarenone
UBIDECARENONE FOR SYSTEM SUITABILITY
Ubiquinone
Ubiquinone 10
Ubiquinone 50
Ubiquinone Q10
UBIQUINONE-10
Unbiquinone
Unispheres Q 10
|
|
5 |
|
Cardiotonic Agents |
|
Phase 2, Phase 3 |
|
|
|
6 |
|
Vitamins |
|
Phase 2, Phase 3 |
|
|
|
7 |
|
Trace Elements |
|
Phase 2, Phase 3 |
|
|
|
8 |
|
Antihypertensive Agents |
|
Phase 2, Phase 3 |
|
|
|
9 |
|
Angiotensin-Converting Enzyme Inhibitors |
|
Phase 2, Phase 3 |
|
|
|
10 |
|
Ubiquinone |
|
Phase 2, Phase 3 |
|
|
|
11 |
|
HIV Protease Inhibitors |
|
Phase 2, Phase 3 |
|
|
|
12 |
|
Micronutrients |
|
Phase 2, Phase 3 |
|
|
|
13 |
|
Protective Agents |
|
Phase 2, Phase 3 |
|
|
|
14 |
|
protease inhibitors |
|
Phase 2, Phase 3 |
|
|
|
Synonyms:
|
15 |
|
Anti-Inflammatory Agents |
|
Phase 3 |
|
|
|
16 |
|
Immunosuppressive Agents |
|
Phase 3 |
|
|
|
17 |
|
Immunologic Factors |
|
Phase 3 |
|
|
|
18 |
|
Benzocaine |
Approved, Investigational |
Phase 1, Phase 2 |
|
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
|
|
19 |
|
Tannic acid |
Approved |
Phase 1, Phase 2 |
|
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
|
|
20 |
|
Prednisolone phosphate |
Approved, Vet_approved |
Phase 2 |
|
302-25-0 |
|
Synonyms:
Prednisolone 21-(dihydrogen phosphate)
Prednisolone 21-monophosphate
|
Prednisolone 21-phosphate
|
|
21 |
|
Prednisolone acetate |
Approved, Vet_approved |
Phase 2 |
|
52-21-1 |
|
Synonyms:
ECONOPRED
FLO-PRED
METICORTELONE
NSC-10966
OMNIPRED
PRED FORTE
|
PRED FTE
PRED MILD
PREDNISOLONE 21-ACETATE
PREDNISOLONE ACETATE
STERANE
ULTRACORTENOL
|
|
22 |
|
Prednisolone |
Approved, Vet_approved |
Phase 2 |
|
50-24-8 |
4894 5755 |
Synonyms:
(11b)-11,17,21-Trihydroxypregna-1,4-diene-3,20-dione
(11beta)-11,17,21-Trihydroxypregna-1,4-diene-3,20-dione
(11Î’)-11,17,21-trihydroxypregna-1,4-diene-3,20-dione
1,4-Pregnadiene-11b,17a,21-triol-3,20-dione
1,4-Pregnadiene-11beta,17alpha,21-triol-3,20-dione
1,4-Pregnadiene-11β,17α,21-triol-3,20-dione
1,4-Pregnadiene-3,20-dione-11b,17a,21-triol
1,4-Pregnadiene-3,20-dione-11beta,17alpha,21-triol
1,4-Pregnadiene-3,20-dione-11β,17α,21-triol
3,20-Dioxo-11b,17a,21-trihydroxy-1,4-pregnadiene
3,20-Dioxo-11beta,17alpha,21-trihydroxy-1,4-pregnadiene
3,20-Dioxo-11β,17α,21-trihydroxy-1,4-pregnadiene
Ak-Pred
Ak-Tate
Alphadrol
Articulose-50
Codelcortone
Co-Hydeltra
Cordrol
CORTALONE
Cotogesic
Cotolone
Decaprednil
Decortin H
DEKOTIL
Delcortol
Delta F
Delta(1)-Dehydrocortisol
Delta(1)-Dehydrohydrocortisone
Delta(1)-Hydrocortisone
DELTA.1-CORTISOL
DELTA.1-Cortisol|Orapred®|Pediapred®
Delta-Cortef
Deltacortenol
DELTACORTRIL
Deltacortril Enteric
delta-dehydrocortisol
delta-dehydrohydrocortisone
delta-hydrocortisone
Deltahydrocortisone
DELTALONE
Deltasolone
Delta-Stab
Deltisilone
Depo-Medrol
Derpo Pd
DESOWEN
Dexa-Cortidelt Hostacortin H
Di adreson F
Di-Adreson F
Di-adreson-F
DiAdresonF
Dicortol
DILACORT
Donisolone
Dydeltrone
Eazolin D
Econopred
Econopred Plus
Erbacort
Erbasona
Estilsona
Fernisolone
Fernisolone P
FERNISOLONE-P
Flamasone
Hostacortin H
HYDELTRA
Hydeltrasol
HYDELTRA-TBA
Hydeltrone
Hydrodeltalone
Hydrodeltisone
Hydroretrocortin
Hydroretrocortine
IMS904
Inflamase Forte
Inflamase Mild
I-Pred
Key-Pred
Klismacort
Lentosone
Lite Pred
Medrol
|
Medrol Acetate
Metacortandralone
Methylprednisolone acetate
METICORTELONE
METI-DERM
Metreton
M-Predrol
NEO-DELTA-CORTEF
Nisolone
Nor-Pred T.B.A.
NSC-9120
NSC-9900
Ocu-Pred
Ocu-Pred Forte
Ophtho-Tate
ORAPRED
Panafcortelone
Paracortol
Paracotol
PEDIAPRED
PEVANTI
POLY-PRED
PRDL
Precortalon
Precortancyl
Precortilon
PRECORTISYL
PRECORTISYL FTE
Pred Forte
Pred Mild
Predair
Predair A
Predair Forte
Predalone 50
Predalone T.B.A.
Predate
Predate Tba
Predate-50
Predcor-25
Predcor-50
Predcor-Tba
PRED-G
Predisolone sodium phosphate
PREDL
Predne-Dome
Prednelan
Prednicen
Predni-Dome
Predniliderm
Predniretard
Prednis
Prednisolona
Prednisolona [INN-Spanish]
Prednisolone
Prednisolone acetate
Prednisolone sodium phosphate
Prednisolone tebutate
Prednisolonum
Prednisolonum [INN-Latin]
Predonin
Predonine
PRELONE
Prenolone
Rolisone
SCHERIPROCT
Scherisolon
SERVISONE
Solone
Steran
STERANE
Sterolone
Supercortisol
TRIDESILON
Ulacort
Ultra Pred
Ultracorten H
Ultracortene H
Ultracortene-H
Ultracortene-Hydrogen
VERDESO
Δ(1)-dehydrocortisol
Δ(1)-dehydrohydrocortisone
Δ(1)-hydrocortisone
Δ-cortef
|
|
23 |
|
Prednisone |
Approved, Vet_approved |
Phase 2 |
|
53-03-2 |
5865 |
Synonyms:
1,2-Dehydrocortisone
1,4-Pregnadiene-17a,21-diol-3,11,20-trione
1,4-Pregnadiene-17alpha,21-diol-3,11,20-trione
1,4-Pregnadiene-17α,21-diol-3,11,20-trione
17,21-Dihydroxypregna-1,4-diene-3,11,20-trione
Acis brand OF prednisone
Acsis, prednison
Adasone
Ancortone
Apo-prednisone
Apotex brand OF prednisone
Aventis brand OF prednisone
Betapar
Bicortone
Cartancyl
Colisone
Cortan
Cortancyl
Cortidelt
Cotone
Cutason
Dacorten
Dacortin
Decortancyl
Decortin
Decortisyl
Dehydrocortisone
Dekortin
Delcortin
Dellacort
Dellacort A
Delta Cortelan
Delta E
Delta-cortelan
Deltacortene
delta-Cortisone
Deltacortisone
Deltacortone
Delta-cortone
DELTA-DOME
Deltasone
Deltison
Deltisona
Deltisone
Deltra
Diadreson
Di-Adreson
Diba brand OF prednisone
Econosone
Encorton
Encortone
Enkortolon
Enkorton
Fawns and mcallan brand OF prednisone
FERNISONE
Ferring brand OF prednisone
Fiasone
GALENpharma brand OF prednisone
Halsey drug brand OF prednisone
Hexal brand OF prednisone
Hoechst brand OF prednisone
Hostacortin
ICN brand OF prednisone
Incocortyl
In-Sone
Juvason
Kortancyl
Lichtenstein brand OF prednisone
|
Liquid pred
Lisacort
LODOTRA
Lodotra®|Sterapred®
Me-Korti
Merck brand OF prednisone
Merz brand OF prednisone
Metacortandracin
Meticorten
Mibe brand OF prednisone
Nisona
Nizon
Novoprednisone
NSC-10023
Nurison
Orasone
Origen Prednisone
Panafcort
Panasol
PARACORT
Parmenison
Pehacort
Pharmacia brand OF prednisone
PRD
Precort
Predeltin
PREDN
Predni tablinen
PREDNICEN-M
Prednicorm
Prednicort
Prednicot
Prednidib
Prednilonga
Predniment
Prednison
Prednison acsis
Prednison galen
Prednison hexal
Prednisona
Prednisona [INN-Spanish]
Prednisone
PREDNISONE INTENSOL
PREDNISONE TABLETS
Prednisonum
Prednisonum [INN-Latin]
Prednitone
Prednizon
Prednovister
Presone
Pronison
Pronisone
RAYOS
Rectodelt
Retrocortine
Schering-plough brand OF prednisone
Seatrace brand OF prednisone
SERVISONE
Solvay brand OF prednisone
Sone
Sterapred
Supercortil
Trommsdorff brand OF prednisone
Ultracorten
Ultracortene
Winpred
Wojtab
Zenadrid
|
|
24 |
|
Methylprednisolone hemisuccinate |
Approved |
Phase 2 |
|
2921-57-5 |
1875 |
Synonyms:
Methylprednisolone hemisuccinate
Methylprednisolone hemisuccinic acid
|
Methylprednisolone hydrogen succinate
Methylprednisolone succinate
|
|
25 |
|
Methylprednisolone |
Approved, Vet_approved |
Phase 2 |
|
83-43-2 |
4159 6741 |
Synonyms:
(6a,11b)-11,17,21-Trihydroxy-6-methylpregna-1,4-diene-3,20-dione
(6alpha,11beta)-11,17,21-Trihydroxy-6-methylpregna-1,4-diene-3,20-dione
(6Α,11β)-11,17,21-trihydroxy-6-methylpregna-1,4-diene-3,20-dione
1-Dehydro-6alpha-methylhydrocortisone
1-Dehydro-6a-methylhydrocortisone
1-Dehydro-6α-methylhydrocortisone
6 Methylprednisolone
6alpha-Methyl-11beta,17alpha,21-triol-1,4-pregnadiene-3,20-dione
6alpha-Methylprednisolone
6a-Methyl-11b,17a,21-triol-1,4-pregnadiene-3,20-dione
6a-Methylprednisolone
6-Methylprednisolone
6Α-methyl-11β,17α,21-triol-1,4-pregnadiene-3,20-dione
6Α-methylprednisolone
Artisone-Wyeth
Besonia
Delta(1)-6alpha-Methylhydrocortisone
delta(1)-6a-Methylhydrocortisone
Dopomedrol
Esametone
Firmacort
J3.872E
Lemod
Medesone
Medixon
Medlone 21
Medrate
Medric acid
Medrol
Medrol Adt Pak
Medrol Dosepak
Medrol®|U-67590A
Medrone
Mesopren
|
Metastab
Methyleneprednisolone
Methylprednisolon
Methylprednisolone
Methylprednisolonum
Methylprednisolonum [INN-Latin]
Metilbetasone
Metilprednisolona
Metilprednisolona [INN-Spanish]
Metilprednisolone
Metilprednisolone [Dcit]
Metipred
Metrisone
Metrocort
Metysolon
Moderin
Nirypan
Noretona
NSC-19987
Predni N Tablinen
Promacortine
Reactenol
Sieropresol
Solomet
SOLU-MEDROL
Summicort
Suprametil
U-67590A
Urbason
Urbasone
Wyacort
Δ(1)-6a-methylhydrocortisone
Δ(1)-6α-methylhydrocortisone
|
|
26 |
|
Prednisolone hemisuccinate |
Experimental |
Phase 2 |
|
2920-86-7 |
4897 |
Synonyms:
(+)-prednisolone hemisuccinate
4-(2-{14,17-dihydroxy-2,15-dimethyl-5-oxotetracyclo[8.7.0.0,.0,]heptadeca-3,6-dien-14-yl}-2-oxoethoxy)-4-oxobutanoate
Prednisolonbisuccinat
Prednisolone 21-hemisuccinate
Prednisolone 21-succinate
|
Prednisolone bisuccinate
PREDNISOLONE HEMISUCCINATE
Prednisolone hemisuccinic acid
Prednisolone hydrogen succinate
PREDNISOLONE SUCCINATE
|
|
27 |
|
Antineoplastic Agents, Hormonal |
|
Phase 2 |
|
|
|
28 |
|
Hormones |
|
Phase 2 |
|
|
|
29 |
|
Hormone Antagonists |
|
Phase 2 |
|
|
|
30 |
|
glucocorticoids |
|
Phase 2 |
|
|
|
31 |
|
Methylprednisolone Acetate |
|
Phase 2 |
|
|
584547 |
Synonyms:
Acetyl methylprednisolone
Acetyl-methylprednisolone
DEPMEDALONE
Depo medrol
Depo medrone
Depo-medrol
Depo-medrone
|
MEDROL ACETATE
Methylprednisolone 21 acetate
METHYLPREDNISOLONE ACETATE
Methylprednisolone acetate, (11beta,16alpha)-isomer
Methylprednisolone acetate, (11beta,16beta)-isomer
Methylprednisolone acetic acid
Methylprednisolone-21-acetate
|
|
32 |
|
Pharmaceutical Solutions |
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Interventional clinical trials:
(show all 49)
# |
Name |
Status |
NCT ID |
Phase |
Drugs |
1 |
Identification of Carnitine-responsive Cardiomyopathy and Myopathy in Adult Patients With Dilated and/or Hypertrophic Cardiomyopathy and Limb Girdle Weakness. |
Unknown status |
NCT01904396 |
Phase 4 |
Carnitine |
2 |
PITT0908: Clinical Trial of Coenzyme Q10 and Lisinopril in Muscular Dystrophies |
Completed |
NCT01126697 |
Phase 2, Phase 3 |
Coenzyme Q10 and Lisinopril |
3 |
Deflazacort in Dysferlinopathies (LGMD2B/MM) - a Double Blind, Placebo-controlled Clinical Study |
Completed |
NCT00527228 |
Phase 2, Phase 3 |
deflazacort;placebo |
4 |
A Multicenter Open-Label Study on the Safety and Efficacy of Deflazacort (Emflaza) in Subjects With Limb-Girdle Muscular Dystrophy 2I (LGMD2I) |
Terminated |
NCT03783923 |
Phase 3 |
Deflazacort |
5 |
Phase I/IIA Gene Transfer Clinical Trial for LGMD2D (Alpha-Sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA |
Completed |
NCT01976091 |
Phase 1, Phase 2 |
|
6 |
Study Evaluating MYO-029 in Adult Muscular Dystrophy |
Completed |
NCT00104078 |
Phase 1, Phase 2 |
MYO-029 |
7 |
An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophy |
Completed |
NCT02836418 |
Phase 1, Phase 2 |
ATYR1940 |
8 |
An Open-Label, Intrapatient Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophies |
Completed |
NCT02579239 |
Phase 1, Phase 2 |
|
9 |
A Phase 1-2 Multicenter Study (2-stages) to Evaluate the Safety and Efficacy of Intravenous GNT0006, Adeno-associated Viral Vector Carrying the FKRP Gene, in Patients With FKRP-related Limb-girdle Muscular Dystrophy (LGMDR9, Formerly LGMD2I) |
Recruiting |
NCT05224505 |
Phase 1, Phase 2 |
|
10 |
Open Label Safety and Efficacy of Once Weekly Steroid in Patients With LGMD and Becker Muscular Dystrophy |
Active, not recruiting |
NCT04054375 |
Phase 2 |
Prednisone |
11 |
A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (β-Sarcoglycan Deficiency) |
Active, not recruiting |
NCT03652259 |
Phase 1, Phase 2 |
|
12 |
An Open Label Phase 2 Study of BBP-418 in Patients With Limb Girdle Muscular Dystrophy Type 2I (MLB-01-003) |
Active, not recruiting |
NCT04800874 |
Phase 2 |
BBP-418 |
13 |
A Phase 1/2, Double-blind, Randomized, Placebo-controlled, Dose-escalation Study to Evaluate the Safety of LION-101 Gene Therapy in Adult Subjects (18-65 Years) With Genetic Confirmation of Limb Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) - Part 1 |
Not yet recruiting |
NCT05230459 |
Phase 1, Phase 2 |
|
14 |
Phase 1/2a First-in-human Trial Evaluating Autologous Gene-edited Muscle Stem Cells in Limb Girdle Muscular Dystrophies (GenPHSats-bASKet) |
Not yet recruiting |
NCT05588401 |
Phase 1, Phase 2 |
|
15 |
Safety and Feasibility of Transvenous Limb Perfusion With Normal Saline in Human Muscular Dystrophy |
Completed |
NCT00873782 |
Phase 1 |
|
16 |
Phase I Clinical Study of AAV1-gamma-sarcoglycan Gene Therapy for Limb Girdle Muscular Dystrophy Type 2C |
Completed |
NCT01344798 |
Phase 1 |
|
17 |
Phase I Gene Transfer of rAAV1.tMCK.Human-alpha-sarcoglycan for Limb Girdle Muscular Dystrophy Type 2D (LGMD2D) |
Completed |
NCT00494195 |
Phase 1 |
|
18 |
The Role of Cell Therapy in Modifying the Course of Limb Girdle Muscular Dystrophy- A Longitudinal 5-year Study |
Withdrawn |
NCT02050776 |
Phase 1 |
|
19 |
Intrathecal Autologous Mononuclear Cell Therapy for Limb Girdle Muscular Dystrophy |
Withdrawn |
NCT02245711 |
Phase 1 |
|
20 |
Limb Girdle Muscular Dystrophy Type 2E Recruitment Study |
Unknown status |
NCT03492346 |
|
|
21 |
Evaluation of Limb-Girdle Muscular Dystrophy |
Completed |
NCT00893334 |
|
|
22 |
Fast Troponin as a Biomarker to Assess Exercise-induced Muscle Damage in Muscle Diseases |
Completed |
NCT04349566 |
|
|
23 |
MRI on Persons With Mutations in POMT2 Gene (LGMD2N) |
Completed |
NCT02759302 |
|
|
24 |
Genetic Characterization of Individuals With Limb Girdle Muscular Dystrophy |
Completed |
NCT00457912 |
|
|
25 |
MRI and Muscle Involvement in Patients With Mutations in GMPPB |
Completed |
NCT02635321 |
|
|
26 |
Biomarker Development in LGMD2i |
Completed |
NCT04202627 |
|
|
27 |
PITT0110 - Cardiac Magnetic Resonance: A Parallel Protocol to Cardiac Outcome Measures in Children With Muscular Dystrophy |
Completed |
NCT01081080 |
|
|
28 |
PITT1109: Cardiac Outcome Measures in Children With Muscular Dystrophy |
Completed |
NCT01066455 |
|
|
29 |
Muscle MRI Study of Patients With Becker Muscular Dystrophy and Limb-girdle Muscular Dystrophy Type 2I |
Completed |
NCT02165358 |
|
|
30 |
Longitudinal Assessment and Genetic Understanding of Limb-Girdle Muscular Dystrophy |
Completed |
NCT01783509 |
|
|
31 |
USEFUL: User-centred Assistive SystEm for Arm Functions in neUromuscuLar Subjects |
Completed |
NCT03127241 |
|
|
32 |
Clinical Determinants of Disease Progression in Patients With Limb Girdle Muscular Distrophy Type 2E |
Completed |
NCT04509609 |
|
|
33 |
Natural History of Disease Progression in Individuals With Limb Girdle Muscular Dystrophy Type 2A and Type 2E |
Recruiting |
NCT03488784 |
|
|
34 |
Molecular Analysis of Nucleic Acids Derived From Patients With Neuromuscular Disease and Their Family Members |
Recruiting |
NCT00390104 |
|
|
35 |
Limb-Girdle Video Assessment Concept Elicitation and Feasibility Study |
Recruiting |
NCT05528757 |
|
|
36 |
Global Fukutin-Related Protein Registry |
Recruiting |
NCT04001595 |
|
|
37 |
Journey: A Global, Multicenter, Longitudinal Study of the Natural History of Subjects With Limb Girdle Muscular Dystrophy (LGMD) Type 2E (LGMD2E/R4), Type 2D (LGMD2D/R3), and Type 2C (LGMD2C/R5) |
Recruiting |
NCT04475926 |
|
|
38 |
GRASP-LGMD: Defining Clinical Endpoints in LGMD |
Recruiting |
NCT03981289 |
|
|
39 |
MRI-phenotyping of Patients With Pathogenic Anoctamin 5 Variants |
Recruiting |
NCT05102799 |
|
|
40 |
Schulze Muscular Dystrophy Ability Clinical Study |
Recruiting |
NCT05409079 |
|
|
41 |
Congenital Muscle Disease Patient and Proxy Reported Outcome Study |
Recruiting |
NCT01403402 |
|
|
42 |
Characterization and Identification of Motor Parameters Using Instrumental Assessment in Patients With Limb Girdle Muscular Dystrophy |
Recruiting |
NCT04772027 |
|
|
43 |
Limb-Girdle Muscular Dystrophy Type 2I in Norway - a Cohort Study |
Active, not recruiting |
NCT03930628 |
|
|
44 |
3 Year Follow up on Patients With Pathogenic Anoctamin 5 Variants |
Active, not recruiting |
NCT05206617 |
|
|
45 |
Prospective, Longitudinal Study of the Natural History and Functional Status of Patients With Limb-Girdle Muscular Dystrophy 2I |
Active, not recruiting |
NCT03842878 |
|
|
46 |
International Clinical Outcome Study for Dysferlinopathy |
Active, not recruiting |
NCT01676077 |
|
|
47 |
Evaluation of Clinical, Immunological, Morphological, Molecular and Genetic Characteristics of Patients With Limb-girdle Muscular Dystrophy Type R2 (Type 2B) in the Russian Federation |
Enrolling by invitation |
NCT04824040 |
|
|
48 |
A Multicenter Phenotype-Genotype Analysis of Limb Girdle Muscular Dystrophy Patients in China |
Enrolling by invitation |
NCT04989751 |
|
|
49 |
GRASP-01-003: Trial Readiness and Endpoint Assessment in LGMD R1 |
Not yet recruiting |
NCT05618080 |
|
|
|