DDrare: Database of Drug Development for Rare Diseases

Valproate, levocarnitine (DrugBank: Valproate, Levocarnitine)

10 diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
3	脊髄性筋萎縮症	1
5	進行性核上性麻痺	0
13	多発性硬化症／視神経脊髄炎	0
53	シェーグレン症候群	0
58	肥大型心筋症	0
96	クローン病	0
97	潰瘍性大腸炎	0
265	脂肪萎縮症	0
298	遺伝性膵炎	0
316	カルニチン回路異常症	0

3. 脊髄性筋萎縮症　［臨床試験数：179，薬物数：102（DrugBank：26），標的遺伝子数：52，標的パスウェイ数：78］

Searched query = "Spinal muscular atrophy", "Myelopathic muscular atrophy", "SMA I", "Werdnig-Hoffman disease", "SMA II", "Dubowitz disease", "SMA III", "Kugelberg-Welander disease", "SMA IV"
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 / 179 trial found

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT01671384 (ClinicalTrials.gov)	August 2013	13/8/2012	Valproate and Levocarnitine in Children With Spinal Muscular Atrophy	Randomized Placebo Controlled Trial of Valproate and Levocarnitine in Children With Spinal Muscular Atrophy Aged 2-15 Years	Spinal Muscular Atrophy	Drug: Valproate, Levocarnitine;Drug: Placebo	All India Institute of Medical Sciences, New Delhi	NULL	Recruiting	2 Years	15 Years	Both	60	Phase 3	India

5. 進行性核上性麻痺　［臨床試験数：82，薬物数：107（DrugBank：36），標的遺伝子数：60，標的パスウェイ数：90］

Searched query = "Progressive supranuclear palsy", "PSP"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 82 trial found

13. 多発性硬化症／視神経脊髄炎　［臨床試験数：3,050，薬物数：2,147（DrugBank：348），標的遺伝子数：244，標的パスウェイ数：228］

Searched query = "Multiple sclerosis/Neuromyelitis optica", "Multiple sclerosis", "Neuromyelitis optica", "MS", "NMOSD", "Devic disease", "Balo concentric sclerosis", "Baló concentric sclerosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 3,050 trial found

53. シェーグレン症候群　［臨床試験数：234，薬物数：270（DrugBank：85），標的遺伝子数：49，標的パスウェイ数：174］

Searched query = "Sjogren syndrome", "Sjögren syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 234 trial found

58. 肥大型心筋症　［臨床試験数：92，薬物数：126（DrugBank：35），標的遺伝子数：40，標的パスウェイ数：141］

Searched query = "Hypertrophic cardiomyopathy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 92 trial found

96. クローン病　［臨床試験数：2,209，薬物数：1,276（DrugBank：240），標的遺伝子数：166，標的パスウェイ数：210］

Searched query = "Crohn disease", "Terminal ileitis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 2,209 trial found

97. 潰瘍性大腸炎　［臨床試験数：2,269，薬物数：1,331（DrugBank：241），標的遺伝子数：114，標的パスウェイ数：181］

Searched query = "Ulcerative colitis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 2,269 trial found

265. 脂肪萎縮症　［臨床試験数：109，薬物数：164（DrugBank：59），標的遺伝子数：26，標的パスウェイ数：94］

Searched query = "Lipodystrophy", "Berardinelli-Seip syndrome", "Lawrence syndrome", "Barraquer-Simons syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 109 trial found

298. 遺伝性膵炎　［臨床試験数：93，薬物数：168（DrugBank：51），標的遺伝子数：48，標的パスウェイ数：134］

Searched query = "Hereditary pancreatitis", "Chronic pancreatitis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 93 trial found

316. カルニチン回路異常症　［臨床試験数：4，薬物数：10（DrugBank：4），標的遺伝子数：2，標的パスウェイ数：9］

Searched query = "Carnitine cycle disorder", "Carnitine palmitoyltransferase I deficiency", "CPT1 deficiency", "Carnitine palmitoyltransferase II deficiency", "CPT2 deficiency", "Carnitine-acylcarnitine translocase deficiency", "CACT deficiency", "Carnitine transporter deficiency", "OCTN-2 deficiency"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: . Trials are sorted by Date_enrollment from most recent to oldest in the table.

0 / 4 trial found