DDrare: Database of Drug Development for Rare Diseases

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	EUCTR2011-001205-27-NL (EUCTR)	04/08/2011	25/07/2011	The effect of lipid lowering medication on lipid accumulation in patients with neutral lipid storage disease with muscle weakness.	The effect of fibrate therapy in two patients with neutral lipid storage disease with myopathy (NLSDM). - Fibrate Trail	Neutral lipid storage disease with myopathy MedDRA version: 13.1;Level: PT;Classification code 10007636;Term: Cardiomyopathy;System Organ Class: 10007541 - Cardiac disorders MedDRA version: 13.1;Level: HLGT;Classification code 10013317;Term: Lipid metabolism disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders MedDRA version: 13.1;Classification code 10028641;Term: Myopathy;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]	Trade Name: Bezalip Retard Product Name: Bezalip Retard Product Code: RVG 18388 INN or Proposed INN: BEZAFIBRATE Other descriptive name: none		NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: no			Netherlands

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

EUCTR2011-001205-27-NL
(EUCTR)

04/08/2011

25/07/2011

The effect of lipid lowering medication on lipid accumulation in patients with neutral lipid storage disease with muscle weakness.

The effect of fibrate therapy in two patients with neutral lipid storage disease with myopathy (NLSDM). - Fibrate Trail

Neutral lipid storage disease with myopathy
MedDRA version: 13.1;Level: PT;Classification code 10007636;Term: Cardiomyopathy;System Organ Class: 10007541 - Cardiac disorders
MedDRA version: 13.1;Level: HLGT;Classification code 10013317;Term: Lipid metabolism disorders;System Organ Class: 10027433 - Metabolism and nutrition disorders
MedDRA version: 13.1;Classification code 10028641;Term: Myopathy;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]

Trade Name: Bezalip Retard
Product Name: Bezalip Retard
Product Code: RVG 18388
INN or Proposed INN: BEZAFIBRATE
Other descriptive name: none

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: no

Netherlands