DDrare: Database of Drug Development for Rare Diseases

告示番号	疾患名（ページ内リンク）	臨床試験数
284	ダイアモンド・ブラックファン貧血	1

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	EUCTR2010-019645-25-GB (EUCTR)	19/07/2010	16/06/2010	A 24 week trial to study the safety and tolerability of SSP-004184 in the treatment of patients with longterm iron overload who need iron chelation therapy, with the option of 72 weeks further dosing.	A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, and Pharmacodynamics of SSP-004184 in the Treatment of Chronic Iron Overload Requiring Chelation Therapy, with a 72 Week Dosing Extension. - SPD602-201	Patients with transfusional iron overload due to hereditary anemias such as sickle cell disease, ß-thalassemia and Diamond-Blackfan anemia; acquired anemias such as Myelodysplastic Syndrome and other forms of bone marrow failure. MedDRA version: 14.1;Level: LLT;Classification code 10065974;Term: Chronic iron overload;System Organ Class: 100000004861;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]	Product Name: SSP-004184 50 mg Capsule Product Code: SSP-004184 50 mg INN or Proposed INN: Not assigned Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide Product Name: SSP-004184 100 mg Capsule Product Code: SSP-004184 100 mg INN or Proposed INN: Not assigned Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide Product Name: SSP-004184 250 mg Capsule Product Code: SSP-004184 250 mg INN or Proposed INN: Not assigned Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide Product Name: SSP-004184 375 mg Capsule Product Code: SSP-004184 375 mg INN or Proposed INN: Not assigned Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide Product Name: SSP-004184 500 mg Capsule Product Code: SSP-004184 500 mg INN or Proposed INN: Not assigned Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazo	Shire Development LLC	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 2	United States;Thailand;Turkey;Italy;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

EUCTR2010-019645-25-GB
(EUCTR)

19/07/2010

16/06/2010

A 24 week trial to study the safety and tolerability of SSP-004184 in the treatment of patients with longterm iron overload who need iron chelation therapy, with the option of 72 weeks further dosing.

A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, and Pharmacodynamics of SSP-004184 in the Treatment of Chronic Iron Overload Requiring Chelation Therapy, with a 72 Week Dosing Extension. - SPD602-201

Patients with transfusional iron overload due to hereditary anemias such as sickle cell disease, ß-thalassemia and Diamond-Blackfan anemia; acquired anemias such as Myelodysplastic Syndrome and other forms of bone marrow failure.
MedDRA version: 14.1;Level: LLT;Classification code 10065974;Term: Chronic iron overload;System Organ Class: 100000004861;Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Product Name: SSP-004184 50 mg Capsule
Product Code: SSP-004184 50 mg
INN or Proposed INN: Not assigned
Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
Product Name: SSP-004184 100 mg Capsule
Product Code: SSP-004184 100 mg
INN or Proposed INN: Not assigned
Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
Product Name: SSP-004184 250 mg Capsule
Product Code: SSP-004184 250 mg
INN or Proposed INN: Not assigned
Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
Product Name: SSP-004184 375 mg Capsule
Product Code: SSP-004184 375 mg
INN or Proposed INN: Not assigned
Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide
Product Name: SSP-004184 500 mg Capsule
Product Code: SSP-004184 500 mg
INN or Proposed INN: Not assigned
Other descriptive name: (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazo

Shire Development LLC

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United States;Thailand;Turkey;Italy;United Kingdom