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


臨床試験数 : 36 薬物数 : 72 - (DrugBank : 21) / 標的遺伝子数 : 18 - 標的パスウェイ数 : 111

  
No.TrialIDDate_
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Public_titleScientific_titleConditionInterventionPrimary_
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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: SXR1096Sixera 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
4ChiCTR2000037278
2020-09-012020-08-27Effects and Safety of Simvastatin Ointment in the Treatment of Congenital IchthyosisEffects and Safety of Simvastatin Ointment in the Treatment of Congenital Ichthyosis inherited ichthyosisARCI group:Concentration of simvastatin cream;XLI group:Concentration of simvastatin cream;Xinhua Hospital Affiliated to Shanghai Jiao Tong University School of MedicineNULLRecruitingBothARCI group:45;XLI group:10;China
5NCT04244006
(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
6NCT04154293
(ClinicalTrials.gov)
December 3, 20191/11/2019A Vehicle Controlled Study to Evaluate Safety and Efficacy of Topical TMB-001 for Treatment of Congenital IchthyosisA Randomized, Parallel, Double-Blind, Vehicle Controlled Study to Evaluate the Safety and Efficacy of Two Concentrations of Topical TMB-001 for the Treatment of Congenital IchthyosisCongenital IchthyosisDrug: Isotretinoin;Other: VehicleTimber Pharmceuticals LLCNULLCompleted9 YearsN/AAll34Phase 2United States;Australia
7EUCTR2019-001220-35-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
8NCT04047732
(ClinicalTrials.gov)
August 27, 201929/7/2019Topical 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 IchthyosisBiological: KB105Krystal Biotech, Inc.NULLActive, not recruiting18 YearsN/AAll6Phase 1/Phase 2United States
9NCT03445650
(ClinicalTrials.gov)
July 18, 201814/2/2018RESET Trial - Part 1 (Followed by Reset Trial - Part 2) - A Phase 3 Trial in Subjects With Sjögren-Larsson Syndrome (SLS)A Phase 3 Randomized, Double-Blind, Vehicle-Controlled, Parallel Group Trial to Evaluate the Safety and Efficacy of ADX-102 1% Topical Dermal Cream (Reproxalap) in Subjects With Sjögren-Larsson Syndrome (SLS)Sjogren-Larsson SyndromeDrug: ADX-102 1% Topical Dermal Cream (reproxalap);Drug: Vehicle of ADX-102 Topical Dermal CreamAldeyra Therapeutics, Inc.NULLCompleted3 YearsN/AAll11Phase 3United States
10EUCTR2016-001948-19-IT
(EUCTR)
05/09/201725/05/2021A Study to Assess the Efficacy and Safety of Abatacept in Adults with Active Primary Sj¿grens SyndromeA Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Subcutaneous Abatacept in Adults with Active Primary Sj¿grens Syndrome - - Adults with moderately to severely Active Primary Sjogrens Syndrome
MedDRA version: 20.1;Level: PT;Classification code 10048676;Term: Sjogren-Larsson syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 21.0;Level: LLT;Classification code 10040766;Term: Sjogren's disease;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
MedDRA version: 21.0;Classification code 10040767;Term: Sjogren's syndrome;Classification code 10042846;Term: Syndrome Sjogren's;Classification code 10040765;Term: Sjogren's;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: Orencia
INN or Proposed INN: 00305800
BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATIONNULLNot RecruitingFemale: yes
Male: yes
288Phase 3Czechia;Italy;France;Czech Republic;Mexico;Canada;Argentina;Brazil;Australia;Denmark;Germany;Norway;Japan;Korea, Republic of;Sweden
11EUCTR2016-000101-37-GR
(EUCTR)
07/04/201715/03/2017Proof of Concept Study to Evaluate the Efficacy and Safety of BMS-931699 (lulizumab) or BMS-986142 in Primary Sjögren's SyndromeA Phase II, Randomized, Multi-Center, Double-Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of BMS-931699 (lulizumab) or BMS-986142 in Subjects with Moderate to Severe Primary Sjögren's Syndrome - POC H2H study subjects with moderate to severe Sjögren's syndrome
MedDRA version: 19.1;Level: LLT;Classification code 10040766;Term: Sjogren's disease;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
MedDRA version: 19.1;Level: PT;Classification code 10040767;Term: Sjogren's syndrome;Classification code 10042846;Term: Syndrome Sjogren's;Classification code 10040765;Term: Sjogren's;Classification code 10048676;Term: Sjogren-Larsson syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: BTK Inhibitor
Product Code: BMS-986142
INN or Proposed INN: BTK Inhibitor
Other descriptive name: BMS986142
Product Name: BTK Inhibitor
Product Code: BMS-986142
INN or Proposed INN: BTK Inhibitor
Other descriptive name: BMS986142
Product Name: Anti-CD28dab
Product Code: BMS-931699
INN or Proposed INN: Anti-CD28dAb
Other descriptive name: Anti-CD28dAb
Bristol-Myers Squibb International CorporationNULLNot RecruitingFemale: yes
Male: yes
160Phase 2Poland;Belgium;Romania;Peru;Denmark;South Africa;Norway;Netherlands;United States;Greece;Russian Federation;Chile;Colombia;United Kingdom;Italy;France;Hungary;Mexico
12EUCTR2016-000101-37-IT
(EUCTR)
29/03/201727/02/2018Proof of Concept Study to Evaluate the Efficacy and Safety of BMS-931699 (lulizumab) or BMS-986142 in Primary Sjögren's SyndromeA Phase II, Randomized, Multi-Center, Double-Blind, Placebo Controlled Study to Evaluate the Efficacy and Safety of BMS-931699 (lulizumab) or BMS-986142 in Subjects with Moderate to Severe Primary Sjögren's Syndrome - POC H2H study subjects with moderate to severe Sjögren's syndrome
MedDRA version: 20.0;Level: LLT;Classification code 10040766;Term: Sjogren's disease;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
MedDRA version: 20.1;Level: PT;Classification code 10048676;Term: Sjogren-Larsson syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10040767;Term: Sjogren's syndrome;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
MedDRA version: 20.0;Classification code 10042846;Term: Syndrome Sjogren's;Classification code 10040765;Term: Sjogren's;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Product Name: BTK Inhibitor
Product Code: BMS-986142
INN or Proposed INN: BTK Inhibitor
Other descriptive name: BMS986142
Product Name: BTK Inhibitor
Product Code: 1643368-58-4
INN or Proposed INN: BTK Inhibitor
Other descriptive name: BMS986142
Product Name: Anti-CD28dab
Product Code: BMS-931699
INN or Proposed INN: Anti-CD28dAb
Other descriptive name: Anti-CD28dAb
BRISTOL-MYERS SQUIBB INTERNATIONAL CORPORATIONNULLNot RecruitingFemale: yes
Male: yes
120Phase 2United States;Greece;Russian Federation;Chile;Colombia;Italy;United Kingdom;France;Mexico;Poland;Belgium;Romania;Peru;Denmark;South Africa;Netherlands;Norway
13NCT02864082
(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
14EUCTR2016-001948-19-SE
(EUCTR)
02/02/201719/10/2016A Study to Assess the Efficacy and Safety of Abatacept in Adults with Active Primary Sjögrens SyndromeA Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Subcutaneous Abatacept in Adults with Active Primary Sjögrens Syndrome Adults with moderately to severely Active Primary Sjögrens Syndrome
MedDRA version: 20.0;Level: LLT;Classification code 10040766;Term: Sjogren's disease;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
MedDRA version: 20.0;Level: PT;Classification code 10040767;Term: Sjogren's syndrome;Classification code 10042846;Term: Syndrome Sjogren's;Classification code 10040765;Term: Sjogren's;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
MedDRA version: 20.1;Classification code 10048676;Term: Sjogren-Larsson syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: Orencia
INN or Proposed INN: ABATACEPT
Bristol-Myers Squibb International CorporationNULLNot RecruitingFemale: yes
Male: yes
288Phase 3Italy;France;Czech Republic;Mexico;Canada;Argentina;Brazil;Australia;Denmark;Norway;Germany;Japan;Sweden;Korea, Republic of
15EUCTR2016-001948-19-CZ
(EUCTR)
28/12/201627/10/2016A Study to Assess the Efficacy and Safety of Abatacept in Adults with Active Primary Sjögrens SyndromeA Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Subcutaneous Abatacept in Adults with Active Primary Sjögrens Syndrome Adults with moderately to severely Active Primary Sjögrens Syndrome
MedDRA version: 20.0;Level: LLT;Classification code 10040766;Term: Sjogren's disease;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
MedDRA version: 20.0;Level: PT;Classification code 10040767;Term: Sjogren's syndrome;Classification code 10042846;Term: Syndrome Sjogren's;Classification code 10040765;Term: Sjogren's;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
MedDRA version: 20.1;Classification code 10048676;Term: Sjogren-Larsson syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: Orencia
INN or Proposed INN: ABATACEPT
Bristol-Myers Squibb International CorporationNULLNot Recruiting Female: yes
Male: yes
288 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noItaly;France;Czech Republic;Mexico;Canada;Argentina;Brazil;Australia;Denmark;Norway;Germany;Japan;Sweden;Korea, Republic of
16EUCTR2016-001948-19-FR
(EUCTR)
02/12/201607/11/2016A Study to Assess the Efficacy and Safety of Abatacept in Adults with Active Primary Sjögrens SyndromeA Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Subcutaneous Abatacept in Adults with Active Primary Sjögrens Syndrome Adults with moderately to severely Active Primary Sjögrens Syndrome
MedDRA version: 19.0;Level: LLT;Classification code 10040766;Term: Sjogren's disease;System Organ Class: 10028395 - Musculoskeletal and connective tissue disorders
MedDRA version: 19.0;Level: PT;Classification code 10040767;Term: Sjogren's syndrome;Classification code 10042846;Term: Syndrome Sjogren's;Classification code 10040765;Term: Sjogren's;Classification code 10048676;Term: Sjogren-Larsson syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Immune System Diseases [C20]
Trade Name: Orencia
INN or Proposed INN: ABATACEPT
Bristol-Myers Squibb International CorporationNULLNot RecruitingFemale: yes
Male: yes
288Phase 3Italy;France;Czech Republic;Mexico;Canada;Argentina;Brazil;Australia;Denmark;Germany;Norway;Japan;Sweden;Korea, Republic of
17NCT03041038
(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
18NCT02830763
(ClinicalTrials.gov)
September 5, 20166/7/2016Clinical Study on the Safety of CNT-02 for TGCV and NLSD-MClinical Study on the Safety and Efficacy of Medium-chain Fatty Acid Capsules (CNT-02) for Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV) and Neutral Lipid Storage Disease With Myopathy (NLSD-M)Primary Triglyceride Deposit Cardiomyovasculopathy (TGCV);Neutral Lipid Storage Disease With Myopathy (NLSD-M)Dietary Supplement: CNT-02Translational Research Center for Medical Innovation, Kobe, Hyogo, JapanNULLTerminated20 YearsN/AAll2N/AItaly;Japan
19NCT02402309
(ClinicalTrials.gov)
March 201517/3/2015A Study of Topical NS2 Cream to Treat Ichthyosis in Sjögren-Larsson Syndrome (SLS)Phase II Study of the Safety, Pharmacokinetics, and Exploratory Activity of Once Daily (QD) Topical Application of NS2 Cream to Treat Ichthyosis in Subjects With Sjögren-Larsson Syndrome (SLS)Sjögren-Larsson SyndromeDrug: Active topical NS2 1% dermatologic cream;Drug: Vehicle placebo 0.0% NS2 dermatologic creamAldeyra Therapeutics, Inc.NULLCompleted6 YearsN/AAll12Phase 2United States
20NCT02383316
(ClinicalTrials.gov)
January 201523/12/2014Study of Metabolic Modifications in Children With Noonan SyndromeStudy of Metabolic Modifications in Children With Noonan SyndromeChild SyndromeOther: Oral Glucose tolerance testUniversity Hospital, ToulouseNULLCompleted7 Years18 YearsAll20N/AFrance
21NCT02113904
(ClinicalTrials.gov)
January 27, 20142/4/2014Clinical Trial Using Humira in Netherton SyndromePhase II Clinical Trial Using Humira in Netherton SyndromeNetherton SyndromeDrug: AdalimumabAssistance Publique - Hôpitaux de ParisNULLCompleted4 YearsN/AAll11Phase 2France
22JPRN-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 AkiharuNULLNot RecruitingNot applicableNot applicableBoth158Phase 2Japan
23EUCTR2013-002205-54-FR
(EUCTR)
17/09/201319/06/2015N/AN/A - ANTI-TNF-SN
MedDRA version: 18.0;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: Humira
Product Name: Humira
Product Code: L04AB04
ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
France
24EUCTR2011-000917-38-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
25EUCTR2011-003212-22-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
26NCT03333200
(ClinicalTrials.gov)
January 11, 201224/4/2017Longitudinal Study of Neurodegenerative DisordersLongitudinal Study of Neurodegenerative DisordersMLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency DiseaseOther: Palliative Care;Biological: Hematopoetic Stem Cell TransplantationUniversity of PittsburghNULLRecruitingN/AN/AAll1500United States
27EUCTR2011-000917-38-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
28EUCTR2011-001205-27-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
29NCT01527318
(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
30NCT01110642
(ClinicalTrials.gov)
July 201122/4/2010Novel Treatment for Syndromic IchthyosesOpen-label, Pilot Study to Assess Cholesterol-Lovastatin Solution in the Treatment of Syndromic IchthyosesSyndromic Ichthyoses;CHILD Syndrome;Smith Lemli Opitz Syndrome;Conradi SyndromeDrug: LovastatinNorthwestern UniversityNULLWithdrawn1 YearN/ABoth0Phase 2United States
31NCT01428297
(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
32NCT01222000
(ClinicalTrials.gov)
October 201012/10/2010Treatment of the Recessive Nonbullous Congenital Ichthyosis by the Epigallocatechine CutaneousTREATMENT OF THE RECESSIVE NONBULLOUS CONGENITAL ICHTHYOSIS BY THE EPIGALLOCATECHINE CUTANEOUSLamellar IchthyosisDrug: apply VEREGEN ® 10 % on a randomized area and the moisturizing cream of the other sideCentre Hospitalier Universitaire de NiceNULLNot yet recruiting8 YearsN/ABoth8Phase 3France
33EUCTR2009-015895-87-NL
(EUCTR)
23/12/200901/10/2009Application of zileuton (Zyflo CR) in patients with Sjögren Larsson Syndrome - Zileuton in Sjögren Larsson Syndrome Application 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
34EUCTR2009-013656-77-FR
(EUCTR)
15/10/200906/08/2009Traitment of recessive nonbullous congenital ichthyosis with topic Epigallocatechin.Traitment of recessive nonbullous congenital ichthyosis with topic Epigallocatechin. Recessive nonbullous congenital ichthyosis
MedDRA version: 12.0;Level: HLT;Classification code 10021197;Term: Ichthyoses
Trade Name: VEREGEN(R)
Product Name: VEREGEN(R)
Nice University HospitalNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
0France
35NCT00208026
(ClinicalTrials.gov)
September 200513/9/2005Safety Study of Elidel (Pimecrolimus) 1% Cream to Treat Netherton SyndromeExploratory Safety and Systemic Absorption of Elidel (Pimecrolimus) 1% Cream for the Treatment of Netherton SyndromeNetherton SyndromeDrug: Pimecrolimus 1% CreamChildren's Hospital of PhiladelphiaNovartis PharmaceuticalsCompleted2 Years18 YearsAll3Phase 1/Phase 2United States
36NCT00004690
(ClinicalTrials.gov)
September 199624/2/2000Phase III Study of Monolaurin Cream Therapy for Patients With Congenital IchthyosisIchthyosisDrug: monolaurin creamCellegy PharmaceuticalsNULLCompleted2 YearsN/ABoth90Phase 3NULL