DDrare: Database of Drug Development for Rare Diseases

160. 先天性魚鱗癬
［臨床試験数：32，薬物数：62（DrugBank：18），標的遺伝子数：15，標的パスウェイ数：107］

Searched query = "Congenital ichthyosis", "Keratinopathic ichthyosis", "Epidermolytic ichthyosis", "Harlequin ichthyosis", "Congenital Ichthyosiform Erythroderma", "Foliate ichthyosis", "Ichthyosis syndrome", "Netherton syndrome", "Sjogren-Larsson syndrome", "Sjögren-Larsson syndrome", "Keratitis-ichtyosis-deafness syndrome", "Dorfman-Chanarin syndrome", "Neutral lipid storage disease", "NLSD", "Multiple sulfatase deficiency", "Austin disease", "Recessive X-linked ichthyosis", "RXLI", "X-linked recessive ichthyosis", "Ichthyosis, brittle hair, impaired intelligence, decreased fertility and short stature", "IBID", "Trichothiodystrophy", "Follicular ichthyosis", "Congenital hemidysplasia, ichthyosiform erythroderma or nevus, and limb defects syndrome", "CHILD syndrome", "Conradi-Hunermann-Happle syndrome", "Conradi-Hünermann-Happle syndrome", "CHHS"
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.

Drug = 1643368-58-4; ABATACEPT; ADX-102 1% Topical Dermal Cream (reproxalap); Abatacept; Active topical NS2 1% dermatologic cream; Adalimumab; Anti-CD28dAb; Anti-CD28dab; Apply VEREGEN ® 10 % on a randomized area and the moisturizing cream of the other side; Atorvastatin; BEZAFIBRATE; BMS-931699; BMS-986142; BMS986142; BPR277; BPR277 ointment; BPR277 ointment (controlled application); BTK Inhibitor; Bezafibrate; Bezalip; Bezalip Retard; Bezalip retard; CNT-02; Cholesterol; DUPILUMAB; Dupilumab; Dupilumab Prefilled Syringe; Dupixent; Epithelial sheet generated from transduced autologous keratinocytes using pCCL-INVO-SP; Fibrate treatment; Glucose; Hematopoetic Stem Cell Transplantation; Humira; Isotretinoin; KB105; L04AB04; Lipoxygenase inhibitor; Lovastatin; Monolaurin cream; None; Orencia; Other: Oral Glucose tolerance test; Other: Palliative Care; Other: Vehicle; Oxiconazole; PAT; PAT-001, 0.1%; PAT-001, 0.2%; Pimecrolimus; Pimecrolimus 1% Cream; RVG 18388; SAR231893; Secukinumab; Simvastatin; VEREGEN(R); Vehicle; Vehicle of ADX-102 Topical Dermal Cream; Veregen; ZILEUTON; Zileuton; Zyflo; Zyflo CR

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	JPRN-jRCTs031180409	25/12/2013	22/03/2019	Treatment of the genetic defect of cholesterol biosynthetic pathway.	Treatment of the genetic defect of cholesterol biosynthetic pathway by the topical application of statin and cholesterol.	CHILD syndrome, Conradi syndrome, porokeratosis, atopic dermatitis, psoriasis, seborrheic dermatitis Dermatitis and eczema	To patients, 1% atorvastatin/ 2% cholesterol lotion and/or 1% atorvastatin/ 2% cholesterol ointment, and/or 1% oxiconazole cream are topically applied on the skin. To healthy subjects, administration of atorvastatin or topical application of 1% atorvastatin/ 2% cholesterol is performed once to investigate drug metabolism.	Kubo Akiharu	NULL	Not Recruiting	Not applicable	Not applicable	Both	158	Phase 2	Japan
2	NCT01110642 (ClinicalTrials.gov)	July 2011	22/4/2010	Novel Treatment for Syndromic Ichthyoses	Open-label, Pilot Study to Assess Cholesterol-Lovastatin Solution in the Treatment of Syndromic Ichthyoses	Syndromic Ichthyoses;CHILD Syndrome;Smith Lemli Opitz Syndrome;Conradi Syndrome	Drug: Lovastatin	Northwestern University	NULL	Withdrawn	1 Year	N/A	Both	0	Phase 2	United States

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

JPRN-jRCTs031180409

25/12/2013

22/03/2019

Treatment of the genetic defect of cholesterol biosynthetic pathway.

Treatment of the genetic defect of cholesterol biosynthetic pathway by the topical application of statin and cholesterol.

CHILD syndrome, Conradi syndrome, porokeratosis, atopic dermatitis, psoriasis, seborrheic dermatitis
Dermatitis and eczema

To patients, 1% atorvastatin/ 2% cholesterol lotion and/or 1% atorvastatin/ 2% cholesterol ointment, and/or 1% oxiconazole cream are topically applied on the skin. To healthy subjects, administration of atorvastatin or topical application of 1% atorvastatin/ 2% cholesterol is performed once to investigate drug metabolism.

Kubo Akiharu

NULL

Not Recruiting

Not applicable

Both

158

Phase 2

Japan

NCT01110642
(ClinicalTrials.gov)

July 2011

22/4/2010

Novel Treatment for Syndromic Ichthyoses

Open-label, Pilot Study to Assess Cholesterol-Lovastatin Solution in the Treatment of Syndromic Ichthyoses

Syndromic Ichthyoses;CHILD Syndrome;Smith Lemli Opitz Syndrome;Conradi Syndrome

Drug: Lovastatin

Northwestern University

NULL

Withdrawn

1 Year

N/A

Both

Phase 2

United States