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	NCT04244006 (ClinicalTrials.gov)	July 23, 2020	13/1/2020	A Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton Syndrome	A Randomized Double-blinded Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton Syndrome	Netherton Syndrome	Drug: Dupilumab Prefilled Syringe;Other: Placebo Prefilled Syringe	University Hospital, Toulouse	MedSharing	Recruiting	18 Years	N/A	All	24	Phase 2;Phase 3	France
2	EUCTR2019-001220-35-FR (EUCTR)	08/10/2019	23/04/2019	A pilot study of the efficacy and safety of dupilumab versus placebo in patients with Netherton syndrome	A randomized double-blinded pilot study of the efficacy and safety of dupilumab versus placebo in patients with Netherton syndrome - NS-Dupi	Netherton syndrome MedDRA version: 20.0;Level: PT;Classification code 10062909;Term: Netherton's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]		CHU de Toulouse	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	24	Phase 2	France

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

NCT04244006
(ClinicalTrials.gov)

July 23, 2020

13/1/2020

A Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton Syndrome

A Randomized Double-blinded Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton Syndrome

Netherton Syndrome

Drug: Dupilumab Prefilled Syringe;Other: Placebo Prefilled Syringe

University Hospital, Toulouse

MedSharing

Recruiting

18 Years

N/A

All

Phase 2;Phase 3

France

EUCTR2019-001220-35-FR
(EUCTR)

08/10/2019

23/04/2019

A pilot study of the efficacy and safety of dupilumab versus placebo in patients with Netherton syndrome

A randomized double-blinded pilot study of the efficacy and safety of dupilumab versus placebo in patients with Netherton syndrome - NS-Dupi

Netherton syndrome
MedDRA version: 20.0;Level: PT;Classification code 10062909;Term: Netherton's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]

CHU de Toulouse

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

France