DDrare: Database of Drug Development for Rare Diseases

325. 遺伝性自己炎症疾患
［臨床試験数：6，薬物数：10（DrugBank：6），標的遺伝子数：2，標的パスウェイ数：33］

Searched query = "Hereditary autoinflammatory syndrome", "NLRC4 abnormality", "Adenosine deaminase 2 deficiency", "ADA2 deficiency", "Aicardi-Goutieres syndrome", "A20 haploinsufficiency"
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 = Abacavir; BARICITINIB; Baricitinib; Emtricitabine; LY3009104; Lamivudine; Reverse transcriptase inhibitors: Zidovudine, Lamivudine, Abacavir; Tenofovir; Tenofovir (TDF) and Emtricitabine (FTC); Zidovudine

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	NCT04517253 (ClinicalTrials.gov)	October 27, 2020	17/8/2020	A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGS	A Phase 2/3, Multicenter, Open-Label Study to Evaluate the Efficacy and Safety of Baricitinib in Adult and Pediatric Japanese Patients With NNS/CANDLE, SAVI, and AGS	Nakajo-Nishimura Syndrome;Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature Syndrome;STING-Associated Vasculopathy With Onset in Infancy;Aicardi Goutieres Syndrome	Drug: Baricitinib	Eli Lilly and Company	NULL	Recruiting	6 Months	N/A	All	5	Phase 2;Phase 3	Japan
2	EUCTR2015-003424-31-GB (EUCTR)		07/12/2015	Treatment of autoinflammatory diseases	Treatment of Conditions Expected to Benefit from JAK 1/2 Inhibition: CANDLE, CANDLE-Related Conditions, SAVI, and Severe Juvenile Dermatomyositis - JAGA	CANDLE, CANDLE-Related Conditions, SAVI, and Severe Juvenile Dermatomyositis, Aicardi-Goutieres Syndrome;Therapeutic area: Diseases [C] - Immune System Diseases [C20]	Product Name: Baricitinib Product Code: LY3009104 INN or Proposed INN: Baricitinib Other descriptive name: BARICITINIB INN or Proposed INN: Baricitinib Other descriptive name: BARICITINIB INN or Proposed INN: baricitinib Other descriptive name: BARICITINIB	Eli Lilly and Company	NULL	NA			Female: yes Male: yes	60	Phase 2	France;United States;United Kingdom

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

NCT04517253
(ClinicalTrials.gov)

October 27, 2020

17/8/2020

A Study of Baricitinib (LY3009104) in Adult and Pediatric Japanese Participants With NNS/CANDLE, SAVI, and AGS

A Phase 2/3, Multicenter, Open-Label Study to Evaluate the Efficacy and Safety of Baricitinib in Adult and Pediatric Japanese Patients With NNS/CANDLE, SAVI, and AGS

Nakajo-Nishimura Syndrome;Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature Syndrome;STING-Associated Vasculopathy With Onset in Infancy;Aicardi Goutieres Syndrome

Drug: Baricitinib

Eli Lilly and Company

NULL

Recruiting

6 Months

N/A

All

Phase 2;Phase 3

Japan

EUCTR2015-003424-31-GB
(EUCTR)

07/12/2015

Treatment of autoinflammatory diseases

Treatment of Conditions Expected to Benefit from JAK 1/2 Inhibition: CANDLE, CANDLE-Related Conditions, SAVI, and Severe Juvenile Dermatomyositis - JAGA

CANDLE, CANDLE-Related Conditions, SAVI, and Severe Juvenile Dermatomyositis, Aicardi-Goutieres Syndrome;Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Product Name: Baricitinib
Product Code: LY3009104
INN or Proposed INN: Baricitinib
Other descriptive name: BARICITINIB
INN or Proposed INN: Baricitinib
Other descriptive name: BARICITINIB
INN or Proposed INN: baricitinib
Other descriptive name: BARICITINIB

Eli Lilly and Company

NULL

Female: yes
Male: yes

Phase 2

France;United States;United Kingdom