160. 先天性魚鱗癬 Congenital ichthyosis Clinical trials / Disease details


臨床試験数 : 42 薬物数 : 71 - (DrugBank : 21) / 標的遺伝子数 : 18 - 標的パスウェイ数 : 112

  
17 trials found
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PhaseCountries
1EUCTR2021-003210-39-AT
(EUCTR)
01/02/202208/09/2021A phase I/II, multicenter, randomized, double-blind, placebo within-patient controlled study to evaluate the safety and efficacy SXR1096 skin cream in patients with Netherton syndrome (NS)A phase I/II, multicenter, randomized, double-blind, placebo within-patient controlled, first-time-in-man (FTIM) Proof of Concept (PoC) study to evaluate the safety and efficacy of topically applied SXR1096 skin cream in patients with Netherton syndrome (NS) 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]
Product Code: SXR1096
INN or Proposed INN: SXR1096
Other descriptive name: SXR1096
Sixera PharmaNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 1;Phase 2Finland;Austria
2EUCTR2021-003210-39-FI
(EUCTR)
23/11/202120/08/2021A phase I/II, multicenter, randomized, double-blind, placebo within-patient controlled study to evaluate the safety and efficacy of SXR1096 skin cream in patients with Netherton syndrome (NS)A phase I/II, multicenter, randomized, double-blind, placebo within-patient controlled, first-time-in-man (FTIM) Proof of Concept (PoC) study to evaluate the safety and efficacy of topically applied SXR1096 skin cream in patients with Netherton syndrome (NS) 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]
Product Code: SXR1096Sixera PharmaNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 1;Phase 2Finland
3NCT04996485
(ClinicalTrials.gov)
March 1, 202121/7/2021Scientific Substantiation and Assessment of the Effectiveness of Pathogenetic Methods of Therapy for Congenital Ichthyosis in ChildrenScientific Substantiation and Assessment of the Effectiveness of Pathogenetic Methods of Therapy for Congenital Ichthyosis in ChildrenCongenital IchthyosisDrug: Secukinumab Injection;Drug: Ustekinumab Injection;Drug: Dupilumab Injection;Other: Symptomatic therapyNational Medical Research Center for Children's Health, Russian FederationNULLRecruiting6 Months18 YearsAll50Phase 4Russian Federation
4NCT04244006
(ClinicalTrials.gov)
July 23, 202013/1/2020A Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton SyndromeA Randomized Double-blinded Pilot Study of the Efficacy and Safety of Dupilumab Versus Placebo in Patients With Netherton SyndromeNetherton SyndromeDrug: Dupilumab Prefilled Syringe;Other: Placebo Prefilled SyringeUniversity Hospital, ToulouseMedSharingRecruiting18 YearsN/AAll24Phase 2/Phase 3France
5EUCTR2019-001220-35-FR
(EUCTR)
08/10/201923/04/2019A pilot study of the efficacy and safety of dupilumab versus placebo in patients with Netherton syndromeA 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]
Trade Name: Dupixent
Product Name: DUPILUMAB
Product Code: SAR231893
CHU de ToulouseNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
24 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance
6NCT02864082
(ClinicalTrials.gov)
March 8, 20173/8/2016A Safety and Tolerability Study of Topical PAT-001 in Congenital IchthyosisA Randomized, Bilateral Comparison, Vehicle-Controlled, Safety and Tolerability Study of Topical PAT-001 for the Treatment of Congenital IchthyosisCongenital IchthyosisDrug: PAT-001, 0.1%;Drug: PAT-001, 0.2%;Drug: VehiclePatagonia Pharmaceuticals, LLCNULLCompleted12 YearsN/AAll19Phase 2United States
7NCT03041038
(ClinicalTrials.gov)
December 201617/1/2017The Efficacy and Safety of Secukinumab in Patients With IchthyosesA Multicenter Study With a Randomized, Double-Blind, Placebo-Controlled Period, Followed by an Open-Label Maintenance Dosing Period to Evaluate the Efficacy and Safety of Secukinumab in Patients With IchthyosesIchthyosis;Autosomal Recessive Congenital Ichthyosis;Lamellar Ichthyosis;Congenital Ichthyosiform Erythroderma;Epidermolytic Ichthyosis;Netherton SyndromeDrug: Secukinumab;Drug: PlaceboNorthwestern UniversityIcahn School of Medicine at Mount SinaiCompleted18 YearsN/AAll21Phase 2United States
8JPRN-jRCTs031180409
25/12/201322/03/2019Treatment 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 AkiharuNULLCompleteNot applicableNot applicableBoth158Phase 2Japan
9EUCTR2011-000917-38-NL
(EUCTR)
13/09/201309/07/2013CBPR277X2101A first–in-human study to evaluate safety and tolerability of repeated topical administrations of BPR277 ointment in healthy volunteers, and safety, tolerability, and preliminary efficacy of multiple topical administrations of BPR277 in patients with atopic dermatitis and Netherton syndrome Part 2: Atopic Dermatitis (AD)Part 3: Netherton Syndrome (NS)
MedDRA version: 14.1;Level: LLT;Classification code 10003639;Term: Atopic dermatitis;System Organ Class: 100000004858
MedDRA version: 14.1;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]
Product Code: BPR277
INN or Proposed INN: Not available
Other descriptive name: BPR277
Novartis Pharma Services AGNULLNot RecruitingFemale: yes
Male: yes
85Germany;Netherlands;France;United States
10EUCTR2011-003212-22-GB
(EUCTR)
03/06/201308/03/2013A clinical trial to study the effects of genetically modified patients' skin stem cellsPhase I study of ex-vivo lentiviral gene therapy for the inherited skin disease Netherton Syndrome - Gene Therapy for Netherton Syndrome Netherton Syndrome (NS)
MedDRA version: 14.1;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]
Product Name: Epithelial sheet generated from transduced autologous keratinocytes using pCCL-INVO-SP
INN or Proposed INN: epithelial sheet generated from transduced autologous keratinocytes using pCCL-INVO-SP
GOSH/ICH Joint Research & Development OfficeNULLNot Recruiting Female: yes
Male: yes
5 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom
11EUCTR2011-000917-38-DE
(EUCTR)
14/09/201118/07/2011CBPR277X2101A first–in-human study to evaluate safety and tolerability of repeated topical administrations of BPR277 ointment in healthy volunteers, and safety, tolerability, and preliminary efficacy of multiple topical administrations of BPR277 in patients with atopic dermatitis and Netherton syndrome Part 2: Atopic Dermatitis (AD)Part 3: Netherton Syndrome (NS)
MedDRA version: 14.1;Level: LLT;Classification code 10003639;Term: Atopic dermatitis;System Organ Class: 100000004858
MedDRA version: 14.1;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]
Product Code: BPR277
INN or Proposed INN: not available
Other descriptive name: BPR277
Product Code: BPR277
INN or Proposed INN: not available
Other descriptive name: BPR277
Novartis Pharma Services AGNULLNot RecruitingFemale: yes
Male: yes
85France;United States;Netherlands;Germany
12EUCTR2011-001205-27-NL
(EUCTR)
04/08/201125/07/2011The 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
NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: no
Netherlands
13NCT01527318
(ClinicalTrials.gov)
August 201116/1/2012The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM)The Effect of Fibrate Therapy in Two Patients With Neutral Lipid Storage Disease With Myopathy (NLSDM)Neutral Lipid Storage DiseaseDrug: Fibrate treatmentMaastricht University Medical CenterNULLCompleted18 Years70 YearsBoth6Phase 4Netherlands
14NCT01428297
(ClinicalTrials.gov)
May 201119/8/2011A Study Evaluating the Safety and Efficacy of Topical BPR277 for the Treatment of Atopic Dermatitis and Netherton SyndromeA First-In-human Study to Evaluate Safety and Tolerability of Topical BPR277 in Healthy Volunteers, and Proof of Concept (PoC) Studies to Evaluate the Safety, Tolerability, and Efficacy of Topical BPR277 in Patients With Atopic Dermatitis and Netherton SyndromeHealthy Volunteers;Atopic Dermatitis;Netherton SyndromeDrug: BPR277 ointment (controlled application);Drug: Placebo (Vehicle);Drug: BPR277 ointment;Drug: BPR277Novartis PharmaceuticalsNULLCompleted18 Years65 YearsAll12Phase 1United States;Netherlands;France;Germany
15EUCTR2009-015895-87-NL
(EUCTR)
23/12/200901/10/2009Application of zileuton (Zyflo CR) in patients with Sjögren Larsson Syndrome - Zileuton in Sjögren Larsson SyndromeApplication of zileuton (Zyflo CR) in patients with Sjögren Larsson Syndrome - Zileuton in Sjögren Larsson Syndrome Sjögren Larsson syndrome (SLS) is an autosomal recessive inherited neurometabolic disorder which is characterized by a clinical triad of congenital ichthyosis, spastic di- or tetraplegia and mental retardation. A disturbance of lipid metabolism due to deficiency of the microsomal fatty aldehyde dehydrogenase (FALDH) underlies SLS.Trade Name: Zyflo CR
Product Name: zileuton
INN or Proposed INN: ZILEUTON
Other descriptive name: lipoxygenase inhibitor
UMC St RadboudNULLNot RecruitingFemale: yes
Male: yes
20Phase 3Netherlands
16NCT00004690
(ClinicalTrials.gov)
September 199624/2/2000Phase III Study of Monolaurin Cream Therapy for Patients With Congenital IchthyosisIchthyosisDrug: monolaurin creamCellegy PharmaceuticalsNULLCompleted2 YearsN/ABoth90Phase 3NULL
17EUCTR2021-003210-39-DE
(EUCTR)
25/02/2022A phase I/II, multicenter, randomized, double-blind, placebo within-patient controlled study to evaluate the safety and efficacy SXR1096 cream in patients with Netherton syndrome (NS)A phase I/II, multicenter, randomized, double-blind, placebo within-patient controlled, first-in-human (FIH) Proof of Concept (PoC) study to evaluate the safety and efficacy of topically applied SXR1096 cream in patients with Netherton syndrome (NS) 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]
Product Code: SXR1096
INN or Proposed INN: SXR1096
Other descriptive name: SXR1096
Sixera PharmaNULLNAFemale: yes
Male: yes
20Phase 1;Phase 2Finland;Austria;Germany