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Trial record 3 of 1145 for:    chelation

An Algorithm to Start Iron Chelation in Minimally Transfused Young Beta-thalassemia Major Patients

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ClinicalTrials.gov Identifier: NCT02173951
Recruitment Status : Unknown
Verified January 2015 by Mohsen Saleh Elalfy, Ain Shams University.
Recruitment status was:  Not yet recruiting
First Posted : June 25, 2014
Last Update Posted : January 14, 2015
Sponsor:
Information provided by (Responsible Party):
Mohsen Saleh Elalfy, Ain Shams University

Brief Summary:

A prospective randomized study on Safety, Tolerability and Efficacy of oral Low dose DFP (50 mg/kg/day) in minimally transfused B-TM after 5 transfusions when SF reaches 500 ng/m and with either appearance of LPI > 0.2 or TSAT reaches 50% compared with non treatment arm.

So the aim of this study:

  1. To determine the time as well as amount of transfused iron ( calculated in mg iron/kg ) which lead to Serum ferritin reaches 500 ng /ml and LPI appearance >0.2 as well as TSAT reaches 50 % .
  2. Tolerability and safety of early low dose DFP 50mg/kg and effectiveness to postpone or prevent SF from reaching 1000 ng/ml or LPI >0.6 or TSAT >70% in comparison to patients not starting chelation therapy
  3. Determine adverse events whether drug or non drug related

Condition or disease Intervention/treatment Phase
Beta Thalassemia Major Drug: Deferiprone Phase 2 Phase 3

  Show Detailed Description

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 64 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Decisional Algorithm to Start Iron Chelation in Minimally Transfused Young Beta-thalassemia Major Patients Naive to Iron Chelation Therapy. A Comparative Randomized Prospective Study
Study Start Date : July 2014
Estimated Primary Completion Date : July 2015
Estimated Study Completion Date : July 2015


Arm Intervention/treatment
Active Comparator: arm 1 iron chelation
Active Comparator arm : iron chelation Included 32 thalassemia major patients with low serum ferritin (≥500) . They will receive low dose Deferiprone( DFP )on 50 mg/kg/d.
Drug: Deferiprone

in arm 1 ( active comparator) will receive a starting dose of Deferiprone (DFP) 50mg⁄ kg ⁄ d, administered orally 3 times daily. Routine dose adjustments will be made according to serum ferritin trends and safety.

Patients reaching serum ferritin ≥1000 will be subjected to dose escalation of DFP to 75 mg/kg/d.

Patients in Placebo Comparator arm when reaching end point elevation of SF to around 1000 ng/ml or more or Tsat > 90 % and or LPI > 0.6 will start deferiprone 75 mg/kg/d

Other Name: ferriprox
Placebo Comparator: arm 2 blood transfusion
Placebo Comparator arm: blood transfusion only Included 32 thalassemia patients with low serum ferritin (≥500). They receive blood transfusion with no chelation. Patients will start deferiprone 75 mg/kg/d when reaching Primary end point which is elevation of SF to around 1000 ng/ml or more or Tsat > 90 % and or LPI > 0.6
Drug: Deferiprone

in arm 1 ( active comparator) will receive a starting dose of Deferiprone (DFP) 50mg⁄ kg ⁄ d, administered orally 3 times daily. Routine dose adjustments will be made according to serum ferritin trends and safety.

Patients reaching serum ferritin ≥1000 will be subjected to dose escalation of DFP to 75 mg/kg/d.

Patients in Placebo Comparator arm when reaching end point elevation of SF to around 1000 ng/ml or more or Tsat > 90 % and or LPI > 0.6 will start deferiprone 75 mg/kg/d

Other Name: ferriprox



Primary Outcome Measures :
  1. determine the time and number of transfusion units as well as amount of infused iron that will lead to appearance of LPI >0.2 or TSAT>50 % , serum ferritin ≥ 500 ng/ml in the studied thalassemic patients which warrant start of iron chelation [ Time Frame: 12 months ]
    To determine the time as well as amount of transfused iron ( calculated in mg iron/kg ) at which there is LPI appearance of >0.2 as well as TSAT reaching 70 %, a serum ferritin ≥ 500 in order to start Iron chelation therapy


Secondary Outcome Measures :
  1. Evaluation of safety of early use of iron chelation Therapy (ICT) in terms of drug related AEs or SAEs [ Time Frame: 12 months ]
    To determine the Tolerability and safety of early low dose DFP 50mg/kg and effectiveness to postpone or prevent SF from reaching 1000 ng/ml or LPI >0.6 or TSAT >70% in comparison to patients not starting chelation therapy



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Ages Eligible for Study:   6 Months to 36 Months   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Young beta thalassemia major patients (diagnosed by HPLC, CBC) who started transfusion therapy who received 5-7transfusions or less, aged more than 6 months.
  2. Pre-transfusional Hb should be >9 g/dL.
  3. Serum ferritin should be ≤ 500ng/ml, transferrin saturation ≤ 50%.

Exclusion Criteria:

  • 1. Beta thalassemia intermedia patients, patients with other transfusion dependent anemias (myelodysplasia, other chronic hemolytic anemias , pure red cell aplasia , aplastic anemia ) 2. Patients with levels of ALT >5 the upper limit of normal (ULN), serum creatinine > ULN on 2 measurements.

    3. Patients with history of agranulocytosis [absolute neutrophil count (ANC) <0.5×109/L].

    4. Non complaint patients acknowledged by reviewing the patient's records.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02173951


Contacts
Contact: Amira AM Adly, Asst. prof 0105245837 amiradiabetes@yahoo.com

Locations
Egypt
Pediatric Hematology clinic, Ain Shams University Not yet recruiting
Cairo, Egypt
Sponsors and Collaborators
Ain Shams University
Investigators
Principal Investigator: Mohsen S Elalfy, professour Ain Shams University

Responsible Party: Mohsen Saleh Elalfy, Professor of pediatric, Ain Shams University
ClinicalTrials.gov Identifier: NCT02173951     History of Changes
Other Study ID Numbers: start of ICT in young BTM
First Posted: June 25, 2014    Key Record Dates
Last Update Posted: January 14, 2015
Last Verified: January 2015

Keywords provided by Mohsen Saleh Elalfy, Ain Shams University:
young minimally transfused beta thalassemia major
labile plasma iron (LPI)
Transferrin saturation (TSAT)
Deferiprone
Transfusional iron loading rate
Transfusion units

Additional relevant MeSH terms:
Iron Chelating Agents
Chelating Agents
Thalassemia
beta-Thalassemia
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Hemoglobinopathies
Genetic Diseases, Inborn
Iron
Deferiprone
Trace Elements
Micronutrients
Growth Substances
Physiological Effects of Drugs
Sequestering Agents
Molecular Mechanisms of Pharmacological Action