DDrare: Database of Drug Development for Rare Diseases

Searched query = "Diamond-Blackfan anemia"
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 = (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazo; (S)-4,5-dihydro-2-[2-hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide; (S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-m; (S)-4,5-dihydro-2[hydroxy-3-(3,6,9-trioxadecyloxy)phenyl]-4-methyl-4-thiazolecarboxylate magnesium hydroxide; Abatacept; Alefacept; Alemtuzumab; Alpha beta depletion; Anti-thymocyte globulin; Antithymocyte globulin; BPX-501 T cells; Busulfan; Busulfan, Cyclophosphamide, ATG, GCSF; Busulfan, Fludarabine, ATG, TLI; CD3/CD19 depleted leukocytes; CD34 Stem Cell Selection Therapy; CD45RA depleted leukocytes; Campath, Fludarabine, Cyclophosphamide; Cyclophosphamide; Cyclophosphamide Dose Level 1; Cyclophosphamide Dose Level 2; Cyclophosphamide Dose Level 3; Cyclophosphamide Dose Level 4; Cyclosporine; DFO; Daclizumab; Deferasirox; Deferitazole; Deferoxamine; Desferal; EXJADE; Eltrombopag; Enriched Hematopoetic Stem Cell Infusion; Exjade; FBS0701; FBS0701 CAPSULE; FBS0701 Capsule; FBS0701/SSP004184; Filgrastim; Fludarabine; Fludarabine monophosphate; Fludarabine phosphate; G-CSF; Human Placental Derived Stem Cell; Hydroxide; Hydroxyurea; ICL670; IRON CHELATING AGENTS; Iron; Iron chelating agents; L-leucine; Lenalidomide; Leucine; Magnesium; Magnesium hydroxide; Melphalan; Mesenchymal Stromal Cells; Methotrexate; Methylprednisolone; Mycophenolate; Mycophenolate mofetil; Myeloablative Preparative Regimen; Other: laboratory biomarker analysis; Phosphate; Prednisone; Procedure: Bone marrow transplantation; Procedure: Leukapheresis; Procedure: Stem cell infusion; Procedure: Stem cell transplant; Procedure: Total lymphoid irradiation; Procedure: allogeneic bone marrow transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: peripheral blood stem cell transplantation; Procedure: radiation therapy; Procedure: umbilical cord blood transplantation; Radiation: Total Body Irradiation; Radiation: radiation therapy; Radiation: total-body irradiation; Reduced Intensity Preparative Regimen; Reduced Toxicity Ablative Regimen; Rimiducid; Rituximab; Rivogenlecleucel; SSP-004184 100 mg; SSP-004184 100 mg Capsule; SSP-004184 250 mg; SSP-004184 250 mg Capsule; SSP-004184 375 mg; SSP-004184 375 mg Capsule; SSP-004184 50 mg; SSP-004184 50 mg Capsule; SSP-004184 500 mg; SSP-004184 500 mg Capsule; SSP-004184AQ 100 mg; SSP-004184AQ 100 mg Capsule; SSP-004184AQ 200 mg; SSP-004184AQ 200 mg Capsule; SSP-004184AQ 250 mg; SSP-004184AQ 250 mg Capsule; SSP-004184AQ 375 mg; SSP-004184AQ 375 mg Capsule; SSP-004184AQ 50 mg; SSP-004184AQ 50 mg Capsule; Sotatercept; Sotatercept with prednisone boost; Tacrolimus; Thiotepa; Thiotepa--escalated dose; Thiotepa--single daily dose; Trifluoperazine

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	EUCTR2011-006322-25-IT (EUCTR)	20/08/2012	03/09/2012	Extension to the treatment of iron overload with chelation therapy	A PHASE 3, OPEN-LABEL, MILTICENTRE, EXTENSION SAFETY AND TOLERABILITY STUDY FOR TRANSFUSIONALLY IRON OVERLOADED CHILDRE, ADOLISCENTS AND ADULTS USING FBS0701 (SSP-004184) - SPD602-301 (FBS0701 - CTP - 15)	ADULT PATIENTS SUFFERING FROM IRON OVERLOAD OF TRANSFUSION-DEPENDENT DOCUMENTED, IN WHICH THE FOLLOWING PRIMARY DIAGNOSIS: ANEMIA HEREDITARY (EG sickle cell anemia), thalassemia and Diamond-Blackfan anemia MedDRA version: 15.0;Level: LLT;Classification code 10065974;Term: Chronic iron overload;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: FBS0701 CAPSULE Product Code: FBS0701 /SSP004184 INN or Proposed INN: iron chelating agents Product Name: FBS0701 CAPSULE Product Code: FBS0701 /SSP004184 INN or Proposed INN: IRON CHELATING AGENTS Product Name: FBS0701 CAPSULE Product Code: FBS0701 /SSP004184 INN or Proposed INN: IRON CHELATING AGENTS Product Name: FBS0701 CAPSULE Product Code: FBS0701 /SSP004184 INN or Proposed INN: IRON CHELATING AGENTS Product Name: FBS0701 CAPSULE Product Code: FBS0701 INN or Proposed INN: IRON CHELATING AGENTS Product Name: FBS0701 CAPSULE Product Code: FBS0701 /SSP004184 INN or Proposed INN: IRON CHELATING AGENTS	FERROKIN BIOSCIENCES INC.	NULL	Not Recruiting			Female: yes Male: yes	1500	Phase 3	United States;United Kingdom;Italy
2	EUCTR2011-005675-16-IT (EUCTR)	15/05/2012	02/08/2012	Treatment of chronic iron overload with a chelation therapy (FBS0701)	A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two FBS0701 Doses in the Treatment of Chronic Iron Overload Requiring Chelation Therapy - FBS0701 in the treatment of chronic iron overload	Patients with transfusional iron overload, with the following primary diagnosis:hereditary anemia (such as sickle cell disease),ß-thalassemia and Diamond Blackfan anemia;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]	Product Name: FBS0701 CAPSULE Product Code: FBS0701 INN or Proposed INN: iron chelating agents Product Name: FBS0701 CAPSULE Product Code: FBS0701 INN or Proposed INN: Iron chelating agents Product Name: FBS0701 CAPSULE Product Code: FBS0701 INN or Proposed INN: Iron chelating agents Product Name: FBS0701 CAPSULE Product Code: FBS0701 INN or Proposed INN: iron chelating agents Product Name: FBS0701 CAPSULE Product Code: FBS0701 INN or Proposed INN: iron chelating agents	FERROKIN BIOSCIENCES INC.	NULL	Not Recruiting			Female: yes Male: yes	50	Phase 2	United States;Canada;Lebanon;Turkey;Italy

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

EUCTR2011-006322-25-IT
(EUCTR)

20/08/2012

03/09/2012

Extension to the treatment of iron overload with chelation therapy

A PHASE 3, OPEN-LABEL, MILTICENTRE, EXTENSION SAFETY AND TOLERABILITY STUDY FOR TRANSFUSIONALLY IRON OVERLOADED CHILDRE, ADOLISCENTS AND ADULTS USING FBS0701 (SSP-004184) - SPD602-301 (FBS0701 - CTP - 15)

ADULT PATIENTS SUFFERING FROM IRON OVERLOAD OF TRANSFUSION-DEPENDENT DOCUMENTED, IN WHICH THE FOLLOWING PRIMARY DIAGNOSIS: ANEMIA HEREDITARY (EG sickle cell anemia), thalassemia and Diamond-Blackfan anemia
MedDRA version: 15.0;Level: LLT;Classification code 10065974;Term: Chronic iron overload;System Organ Class: 10027433 - Metabolism and nutrition disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Name: FBS0701 CAPSULE
Product Code: FBS0701 /SSP004184
INN or Proposed INN: iron chelating agents
Product Name: FBS0701 CAPSULE
Product Code: FBS0701 /SSP004184
INN or Proposed INN: IRON CHELATING AGENTS
Product Name: FBS0701 CAPSULE
Product Code: FBS0701 /SSP004184
INN or Proposed INN: IRON CHELATING AGENTS
Product Name: FBS0701 CAPSULE
Product Code: FBS0701 /SSP004184
INN or Proposed INN: IRON CHELATING AGENTS
Product Name: FBS0701 CAPSULE
Product Code: FBS0701
INN or Proposed INN: IRON CHELATING AGENTS
Product Name: FBS0701 CAPSULE
Product Code: FBS0701 /SSP004184
INN or Proposed INN: IRON CHELATING AGENTS

FERROKIN BIOSCIENCES INC.

NULL

Not Recruiting

Female: yes
Male: yes

1500

Phase 3

United States;United Kingdom;Italy

EUCTR2011-005675-16-IT
(EUCTR)

15/05/2012

02/08/2012

Treatment of chronic iron overload with a chelation therapy (FBS0701)

A Phase 2, 24 Week, Randomized, Open Label, Multi-Center Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two FBS0701 Doses in the Treatment of Chronic Iron Overload Requiring Chelation Therapy - FBS0701 in the treatment of chronic iron overload

Patients with transfusional iron overload, with the following primary diagnosis:hereditary anemia (such as sickle cell disease),ß-thalassemia and Diamond Blackfan anemia;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Product Name: FBS0701 CAPSULE
Product Code: FBS0701
INN or Proposed INN: iron chelating agents
Product Name: FBS0701 CAPSULE
Product Code: FBS0701
INN or Proposed INN: Iron chelating agents
Product Name: FBS0701 CAPSULE
Product Code: FBS0701
INN or Proposed INN: Iron chelating agents
Product Name: FBS0701 CAPSULE
Product Code: FBS0701
INN or Proposed INN: iron chelating agents
Product Name: FBS0701 CAPSULE
Product Code: FBS0701
INN or Proposed INN: iron chelating agents

FERROKIN BIOSCIENCES INC.

NULL

Not Recruiting

Female: yes
Male: yes

Phase 2

United States;Canada;Lebanon;Turkey;Italy