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	NCT04047732 (ClinicalTrials.gov)	August 27, 2019	29/7/2019	Topical KB105 Gene Therapy for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)	A Phase I/II Clinical Trial of Topical KB105, a Replication-incompetent, Non-integrating HSV-1 Vector Expressing Human Transglutaminase 1 (TGM1) for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)	TGM-1 Related Autosomal Recessive Congenital Ichthyosis	Biological: KB105	Krystal Biotech, Inc.	NULL	Recruiting	18 Years	N/A	All	6	Phase 1;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

NCT04047732
(ClinicalTrials.gov)

August 27, 2019

29/7/2019

Topical KB105 Gene Therapy for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)

A Phase I/II Clinical Trial of Topical KB105, a Replication-incompetent, Non-integrating HSV-1 Vector Expressing Human Transglutaminase 1 (TGM1) for the Treatment of TGM1-deficient Autosomal Recessive Congenital Ichthyosis (ARCI)

TGM-1 Related Autosomal Recessive Congenital Ichthyosis

Biological: KB105

Krystal Biotech, Inc.

NULL

Recruiting

18 Years

N/A

All

Phase 1;Phase 2

United States