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 254. ポルフィリン症 [臨床試験数:34,薬物数:35(DrugBank:20),標的遺伝子数:17,標的パスウェイ数:5] 

Searched query = "Porphyria", "Hereditary coproporphyria", "Erythropoietic protoporphyria", "X-linked dominant protoporphyria"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"    Synonyms (DrugBank) were also searched for.
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
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PhaseCountries
1NCT04056481August 20, 201911 November 2019Expanded Access Protocol of Givosiran for Patients With Acute Hepatic PorphyriaExpanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria (AHP)Acute Hepatic PorphyriaDrug: GivosiranAlnylam PharmaceuticalsNot recruiting12 YearsN/AAllPhase 2United States;France
2NCT03520036July 5, 20184 November 2019Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic ProtoporphyriaA Phase II, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy, Safety, and Tolerability of MT-7117 in Subjects With Erythropoietic ProtoporphyriaErythropoietic Protoporphyria (EPP)Drug: MT-7117 low dose;Drug: MT-7117 high dose;Drug: PlaceboMitsubishi Tanabe Pharma Development America, Inc.Not recruiting18 Years75 YearsAll102Phase 2United States
3NCT03505853April 26, 201825 March 2019A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)A Drug-Drug Interaction Study to Investigate the Effect of Givosiran on the Pharmacokinetics (PK) of Midazolam, Caffeine, Losartan, Omeprazole, and Dextromethorphan in Patients With Acute Intermittent Porphyria (AIP) Who Are Asymptomatic High Excreters (ASHE)Acute Intermittent Porphyria (AIP);Acute Hepatic Porphyria (AHP);Porphyria, Acute Intermittent;Acute PorphyriaDrug: Givosiran;Drug: 5-probe cocktailAlnylam PharmaceuticalsNot recruiting18 Years65 YearsAll10Phase 1Sweden
4EUCTR2017-002432-17-PL12/04/201828 February 2019Study on an Investigational Drug, Givosiran, for the Treatment of Acute Hepatic PorphyriasENVISION: A Phase 3 Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias - ENVISIONAcute Hepatic Porphyrias (AHP)
MedDRA version: 20.0 Level: PT Classification code 10036182 Term: Porphyria acute System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0 Level: LLT Classification code 10036184 Term: Porphyria hepatic System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]
Product Name: Givosiran
Product Code: ALN-AS1
Pharmaceutical Form: Solution for injection
INN or Proposed INN: ALN-60519
CAS Number: 1639325-44-2
Current Sponsor code: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 189-
Pharmaceutical form of the placebo: Solution for injection
Route of administration of the placebo: Subcutaneous use
Alnylam Pharmaceuticals, Inc.Authorised Female: yes
Male: yes
74Phase 3United States;Taiwan;Finland;Spain;Italy;Switzerland;United Kingdom;France;Czech Republic;Mexico;Canada;Poland;Belgium;Brazil;Denmark;Australia;Bulgaria;Netherlands;Germany;Japan;Korea, Republic of;Sweden
5EUCTR2016-002638-54-SE20/12/201720 August 2018Long-term Safety and Clinical Activity of ALN-AS1 in Patients with Acute Intermittent Porphyria (AIP), a rare metabolic disorder.A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients with Acute Intermittent Porphyria who have Completed a Previous Clinical Study with ALN-AS1Acute Intermittent Porphyria (AIP)
MedDRA version: 20.0 Level: LLT Classification code 10000818 Term: Acute intermittent porphyria System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Givosiran
Product Code: ALN-AS1
Pharmaceutical Form: Solution for injection
INN or Proposed INN: ALN-60519
CAS Number: 1639325-44-2
Current Sponsor code: ALN-60519
Other descriptive name: CAS # in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 189-
Alnylam Pharmaceuticals, Inc.AuthorisedFemale: yes
Male: yes
24Phase 1;Phase 2United States;United Kingdom;Sweden
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03118674August 10, 201715 July 2019Harvoni Treatment Porphyria Cutanea TardaNewer Direct-Acting Anti-Viral Agents as Sole Therapy of Porphyria Cutanea Tarda in Subjects With Chronic Hepatitis CPorphyria Cutanea Tarda;Hepatitis CDrug: HarvoniWake Forest University Health SciencesGilead Sciences;National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);National Institutes of Health (NIH)Recruiting18 YearsN/AAll49Phase 2United States
7NCT02979249December 201626 August 2019Oral Iron for Erythropoietic ProtoporphyriasEffect of Oral Iron Therapy on Erythrocyte Protoporphyrin Levels in the Erythropoietic ProtoporphyriasErythropoietic Protoporphyria;EPP;X-linked Protoporphyria;XLPDrug: Oral IronIcahn School of Medicine at Mount SinaiNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)Not recruiting18 YearsN/AAll150N/AUnited States
8NCT02943213November 201616 December 2017Assessment of Intra-subject Variability in the Bioavailability of Chlorpromazine HydrochlorideA Single Center, Single Dose, Open-Label, Two-Period Replicate Pilot Study to Investigate Intra-subject Variability in the Bioavailability of a Formulation Containing Chlorpromazine Hydrochloride (25 mg Sugar Coated Tablets) in at Least 16 Healthy Males and Females Under Fasting ConditionsAnti-Psychotic;Management of Manifestations of Psychotic Disorders;Treatment of Schizophrenia;Control Nausea and Vomiting;Relief of Restlessness and Apprehension Before Surgery;Acute Intermittent Porphyria;Adjunct in the Treatment of Tetanus;Control Manifestations of the Manic Type of Mani-depressive Illness;Relief of Intractable HiccupsDrug: Chlorpromazine HydrochlorideCycle Pharmaceuticals Ltd.ParexelNot recruiting18 Years65 YearsAll20Phase 1South Africa
9NCT02949830October 201611 November 2019A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients With Acute Intermittent Porphyria Who Have Completed a Previous Clinical Study With ALN-AS1Acute Intermittent PorphyriaDrug: givosiran (ALN-AS1)Alnylam PharmaceuticalsNot recruiting18 YearsN/AAll17Phase 1/Phase 2United States;Sweden;United Kingdom
10EUCTR2016-002638-54-GB30/08/201625 June 2018Long-term Safety and Clinical Activity of ALN-AS1 in Patients with Acute Intermittent Porphyria (AIP), a rare metabolic disorder.A Multicenter, Open-label Extension Study to Evaluate the Long-term Safety and Clinical Activity of Subcutaneously Administered ALN-AS1 in Patients with Acute Intermittent Porphyria who have Completed a Previous Clinical Study with ALN-AS1Acute Intermittent Porphyria (AIP)
MedDRA version: 20.0 Level: LLT Classification code 10000818 Term: Acute intermittent porphyria System Organ Class: 100000004850 ;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]
Product Name: Givosiran
Product Code: ALN-AS1
Pharmaceutical Form: Solution for injection
INN or Proposed INN: ALN-60519
CAS Number: 1639325-44-2
Current Sponsor code: ALN-60519
Other descriptive name: CAS# in D.3.9.1 for Sodium Salt. CAS number 1639325-43-1 (Parent Acid)
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 189-
Alnylam Pharmaceuticals, Inc.AuthorisedFemale: yes
Male: yes
24Phase 1;Phase 2United States;United Kingdom;Sweden
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT02922413October 30, 20152 September 2019Panhematin for Prevention of Acute Attacks of PorphyriaSafety and Efficacy of Panhematin™ for Prevention of Acute Attacks of PorphyriaAcute Intermittent Porphyria;Hereditary Coproporphyria;Variegate PorphyriaBiological: Hemin for injection;Other: PlaceboThe University of Texas Medical Branch, GalvestonRecruiting18 YearsN/AAll20Phase 2United States
12NCT02452372May 6, 201525 June 2018A Phase 1 Study of Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)A Phase 1, Single-ascending Dose, Multiple-ascending Dose, and Multi-dose Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN AS1 in Patients With Acute Intermittent Porphyria (AIP)Acute Intermittent PorphyriaDrug: givosiran (ALN-AS1);Drug: Sterile Normal Saline (0.9% NaCl)Alnylam PharmaceuticalsNot recruiting18 Years65 YearsAll40Phase 1United States;Sweden;United Kingdom
13NCT02180412April 28, 201426 August 2019Controlled Trial of Panhematin in Treatment of Acute Attacks of PorphyriaA Double-blind, Randomized, Placebo-controlled, Parallel Group Trial on the Efficacy and Safety of PanhematinTM in the Treatment of Acute Attacks of PorphyriaAcute PorphyriasBiological: Panhematin;Other: GlucoseThe University of Texas Medical Branch, GalvestonNot recruiting18 Years100 YearsAll40Phase 2United States
14NCT02935400April 28, 201426 August 2019Acute Porphyria Biomarkers for Disease ActivityAcute Porphyrias: Biomarkers for Disease Activity and Response to TreatmentAcute Intermittent Porphyria;Hereditary Coproporphyria;Variegate PorphyriaDrug: HeminThe University of Texas Medical Branch, GalvestonNot recruiting18 YearsN/AAll50Phase 3United States
15NCT01605136May 201230 September 2019Phase III Confirmatory Study in Erythropoietic ProtoporphyriaA Phase III, Multicentre, Double-Blind, Randomized, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients With Erythropoietic Protoporphyria (EPP)Erythropoietic ProtoporphyriaDrug: Afamelanotide;Drug: PlaceboClinuvel Pharmaceuticals LimitedNot recruiting18 YearsN/AAll93Phase 3United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT01550705March 201219 October 2017Effect of Isoniazid on Protoporphyrin Levels in Erythropoietic ProtoporphyriaQuantification of the Effects of Isoniazid Treatment on Erythrocyte and Plasma Protoporphyrin IX Concentration and Plasma Aminolevulinic Acid in Patients With Erythropoietic ProtoporphyriaErythropoietic Protoporphyria (EPP);X Linked Erythropoietic ProtoporphyriaDrug: IsoniazidUniversity of UtahNational Institute of Diabetes and Digestive and Kidney Diseases (NIDDK);Icahn School of Medicine at Mount Sinai;University of Alabama at Birmingham;University of California, San Francisco;University of TexasNot recruiting18 YearsN/AAll11N/AUnited States
17NCT01422915May 201116 December 2017Sorbent Therapy of the Cutaneous PorphyriasSorbent Therapy of the Cutaneous PorphyriasErythropoietic ProtoporphyriaDrug: ColestipolBrigham and Women's HospitalNot recruiting22 Years60 YearsAll4Phase 2/Phase 3United States
18NCT01284946January 201119 February 2015Safety and Efficacy of Oral Deferasirox in Patients With Porphyria Cutanea TardaA Phase II, Open Label Clinical Trial Exploring the Safety and the Efficacy of Oral Deferasirox in Patients Newly Diagnosed With Porphyria Cutanea Tarda (PCT) and Non-transfusion Iron OverloadPorphyria Cutanea TardaDrug: ExjadeAssistance Publique - Hôpitaux de ParisAssociation pour l'Etude des Fonctions Digestives (AEFD)Recruiting18 YearsN/ABoth45Phase 2France
19NCT01097044April 201019 February 2015Phase II Confirmatory Study in Erythropoietic Protoporphyria (EPP)A Phase II, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients With Erythropoietic Protoporphyria (EPP)Erythropoietic ProtoporphyriaDrug: Afamelanotide;Drug: PlaceboClinuvel Pharmaceuticals LimitedNot recruiting18 YearsN/ABoth77Phase 2United States
20NCT00979745September 200919 February 2015Phase III Confirmatory Study in Erythropoietic Protoporphyria (EPP)A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients With Erythropoietic Protoporphyria (EPP)Erythropoietic ProtoporphyriaDrug: Afamelanotide;Drug: PlaceboClinuvel Pharmaceuticals LimitedNot recruiting18 Years70 YearsBoth70Phase 3Finland;France;Germany;Ireland;Netherlands;United Kingdom
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2009-011018-51-NL06/08/20093 April 2012A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients with Erythropoietic Protoporphyria (EPP). - Multicentre Phase III EPP Study 2A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients with Erythropoietic Protoporphyria (EPP). - Multicentre Phase III EPP Study 2Erythropoietic Protoporphyria (EPP)
MedDRA version: 9.1 Level: LLT Classification code 10015289 Term: Erythropoietic protoporphyria
Product Name: afamelanotide
Product Code: CUV1647
Pharmaceutical Form: Implant
Pharmaceutical form of the placebo: Implant
Route of administration of the placebo: Subcutaneous use
Clinuvel Pharmaceuticals LimitedNot RecruitingFemale: yes
Male: yes
70Phase 3Finland;United Kingdom;Netherlands;Ireland
22EUCTR2007-000636-13-GB17/06/200812 June 2012A Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP StudyA Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutaneous Bioresorbable CUV1647 Implants in Patients with Erythropoietic Protoporphyria (EPP) - Multicentre Phase III EPP StudyErythropoietic Protoporphyria (EPP)
MedDRA version: 9.1 Level: LLT Classification code 10015289 Term: Erythropoietic protoporphyria
Product Name: CUV1647
Product Code: CUV1647
Pharmaceutical Form: Implant
Pharmaceutical form of the placebo: Implant
Route of administration of the placebo: Subcutaneous use
Clinuvel Pharmaceuticals LimitedAuthorisedFemale: yes
Male: yes
80Phase 3Germany;United Kingdom;Netherlands;France;Italy;Sweden
23NCT00599326January 200819 October 2017Pilot Trial of Deferasirox in the Treatment of Porphyria Cutanea TardaPilot Trial of Deferasirox in the Treatment of Porphyria Cutanea TardaPorphyria Cutanea TardaDrug: DeferasiroxUniversity of Texas Southwestern Medical CenterNovartis PharmaceuticalsNot recruiting18 YearsN/AAll10Phase 3United States
24EUCTR2007-002863-28-GB26/09/200726 November 2018Investigation into the use of colestyramine as a therapy for patients with erythropoietic protoporphyria - EPP001Investigation into the use of colestyramine as a therapy for patients with erythropoietic protoporphyria - EPP001Erythropoietic protoporphyria (EPP)
MedDRA version: 9.1 Level: LLT Classification code 10015289 Term: Erythropoietic protoporphyria
Trade Name: Questran
Product Name: Questran
Pharmaceutical Form: Powder for oral suspension
INN or Proposed INN: COLESTYRAMINE
CAS Number: 11041126
Concentration unit: g gram(s)
Concentration type: equal
Concentration number: 4-
Guy's & St Thomas' NHS Foundation TrustNot RecruitingFemale: yes
Male: yes
21Phase 4United Kingdom
25NCT04053270May 200722 October 2019Multicentre Phase III Erythropoietic Protoporphyria StudyA Phase III, Multicentre, Randomised, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Subcutanenous Bioresorbable CUV1647 Implants in Patients With Erythropoietic Protoporphyria (EPP)Erythropoietic ProtoporphyriaDrug: Afamelanotide;Drug: PlaceboClinuvel Pharmaceuticals LimitedNot recruiting18 Years70 YearsAll100Phase 3
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT01573754March 21, 200626 August 2019Hydroxychloroquine and Phlebotomy for Treating Porphyria Cutanea TardaA Prospective Comparison of Low Dose Hydroxychloroquine and Phlebotomy in the Treatment of Porphyria Cutanea Tarda. IRB 02-435Porphyria Cutanea TardaDrug: Hydroxychloroquine;Procedure: PhlebotomyThe University of Texas Medical Branch, GalvestonRecruiting18 Years100 YearsAll100Phase 2United States
27NCT00418795June 11, 200311 June 2018Porphozym in the Treatment of Acute Attacks in AIPA Multi-centre, Double-blind, Randomized, Placebo-controlled, Parallel Group Trial, Investigating the Efficacy and Safety of Porphozym (Recombinant Human Porphobilinogen Deaminase) in the Treatment of Acute Attacks in AIPAcute Intermittent PorphyriaDrug: recombinant human porphobilinogen deaminase (Porphozym)Zymenex A/SNot recruiting18 YearsN/AAll36Phase 2/Phase 3United States
28NCT00004397January 199819 February 2015Phase I Study of Tin Mesoporphyrin in Patients on Long Term Heme Therapy for Prevention of Acute Attacks of PorphyriaPorphyriaDrug: heme arginate;Drug: tin mesoporphyrinNational Center for Research Resources (NCRR)University of TexasNot recruiting18 YearsN/ABoth20Phase 1
29NCT00004398January 199819 February 2015Phase I Study of Heme Arginate With or Without Tin Mesoporphyrin in Patients With Acute Attacks of PorphyriaPorphyriaDrug: heme arginate;Drug: tin mesoporphyrinNational Center for Research Resources (NCRR)University of TexasNot recruiting18 YearsN/ABoth24Phase 1
30NCT00004396September 199719 February 2015Studies in Porphyria III: Heme and Tin Mesoporphyrin in Acute PorphyriasPorphyriaDrug: heme arginate;Drug: tin mesoporphyrinNational Center for Research Resources (NCRR)University of TexasNot recruiting18 YearsN/ABoth32Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31NCT00004831October 19967 April 2015Study of Cysteine Hydrochloride for Erythropoietic ProtoporphyriaErythropoietic ProtoporphyriaDrug: cysteine hydrochlorideFDA Office of Orphan Products DevelopmentSt. Luke's-Roosevelt Hospital CenterNot recruiting18 Years65 YearsBoth20N/A
32NCT00004940May 19967 April 2015Phase III Study of L-Cysteine in Patients With Erythropoietic ProtoporphyriaErythropoietic ProtoporphyriaDrug: cysteine hydrochlorideBrigham and Women's HospitalNot recruiting18 Years65 YearsBoth50Phase 3
33NCT00004789July 199319 February 2015Phase I/II Study of Heme Arginate and Tin Mesoporphyrin for Acute PorphyriaPorphyriaDrug: heme arginate;Drug: tin mesoporphyrinNational Center for Research Resources (NCRR)University of TexasNot recruiting18 YearsN/ABoth59Phase 1/Phase 2
34NCT00004330March 198719 February 2015Studies in Porphyria IV: Gonadotropin-Releasing Hormone (GnRH) Analogues for Prevention of Cyclic AttacksPorphyriaDrug: luteinizing hormone-releasing factorNational Center for Research Resources (NCRR)University of TexasNot recruiting18 Years55 YearsFemaleN/AUnited States

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