Patients With Thalassemia Intermedia,GDF 15NCT01201135
GDF 15 in Sickle Cell Disease and Hereditary Spherocytosis
An observational study of Patients With Thalassemia Intermedia, and Congenital Dyserythropoietic Anemia Type I, run by Wolfson Medical Center.
Trial summary
Patients with thalassemia intermedia, congenital dyserythropoietic anemia type I , and sideroblastic anemia were found to express very high levels of serum GDF15, and this contributed to the inappropriate suppression of hepcidin with subsequent secondary iron overload.The aim of our present study is to asses the… Read more about this study
- Who is it for?
- Ages 5 years and older
- Conditions the study is for
- Patients With Thalassemia Intermedia,, Congenital Dyserythropoietic Anemia Type I
- How many people
- 80 participants
- Where
- No sites listed yet
- Study dates
- Sep 2010to Sep 2011
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 5 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: The study will contain 40 patients with Sickle cell disease and 40 patients with hereditary spherocytosis. After ICF (Informed Consent Form) has been signed by the patients the following laboratory tests will be taken once during the study: 1. GDF 15( 3ml of serum) (at the laboratory of hematology at Wolfson Medical Center/Israel) 2. Hepcidine (3ml of serum) (at the laboratory of Prof. T. Ganz, USA). The blood samples should be taken at least one week apart from blood transfusion. In case of infection or acute inflammation , blood samples should be taken only one week after resolution of these conditions.
Who may be able to join
- non
Who cannot join
- non
Trial locations and contacts
No sites are listed yet.
About this study
Official title: The Impact of Growth Differentiating Factor (GDF) 15 in Sickle Cell Disease and Hereditary Spherocytosis
Patients with thalassemia intermedia, congenital dyserythropoietic anemia type I , and sideroblastic anemia were found to express very high levels of serum GDF15, and this contributed to the inappropriate suppression of hepcidin with subsequent secondary iron overload.The aim of our present study is to asses the levels of GDF15 and hepcidin in patients with Sickle cell disease and hereditary spherocytosis
Read the detailed description
The identification of the ferroportin/hepcidin axis has allowed the effect of erythroid activity on iron balance to be studied and has created the basis for better defining the erythroid regulators.
In iron-loading anemias, ineffective erythropoiesis suppresses hepcidin production, which result in dysregulating iron homeostasis. Miller and co-workers showed that release of cytokines like growth differentiation factor 15 (GDF15) during the process of ineffective erythropoiesis inhibits hepcidin production, thus defining a molecular link between ineffective erythropoiesis, suppression of hepcidin production and parenchymal iron loading.
- Medical subject headings
- Anemia, Dyserythropoietic, Congenital
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Sickle cell disease | Not described | |
| hereditary spherocytosis. | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
GDF 15
Measured: year
Secondary outcomes (1)
Hepcidine
Measured: year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2010 (estimated)
- Study startSep 2010
- Primary completionSep 2011 (estimated)
- Study completionSep 2011 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Patients With Thalassemia Intermedia,Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Time perspective
- Prospective
- Enrolment
- 80 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Wolfson Medical Center
- Sponsor type
- Other government
- Investigators
- GHOTI HOSSAM, doctor, Principal investigator, HEMATOLOGY DEPARTMENT ON WOLFSSON MEDICAL CENTER
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01201135
- Sponsor's protocol ID
- GDF-15CTIL
- Acronym
- GDF 15
- Status last verified
- Sep 2010
- First submitted
- 12 Sep 2010
- Last updated
- 14 Sep 2010
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Sep 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- The Impact of Growth Differentiating Factor (GDF) 15 in Sickle Cell Disease and Hereditary Spherocytosis
- ClinicalTrials.gov ID
- NCT01201135
- Acronym / sponsor ID
- GDF 15 / GDF-15CTIL
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 80 participants (estimated)
- Lead sponsor
- Wolfson Medical CenterOther government
- Study start
- Sep 2010
- Primary completion
- Sep 2011 (estimated)
- Study completion
- Sep 2011 (estimated)
- First posted
- 14 Sep 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Patients with thalassemia intermedia, congenital dyserythropoietic anemia type I , and sideroblastic anemia were found to express very high levels of serum GDF15, and this contributed to the inappropriate suppression of hepcidin with subsequent secondary iron overload.The aim of our present study is to asses the levels of GDF15 and hepcidin in patients with Sickle cell disease and hereditary spherocytosis
Detailed description
The identification of the ferroportin/hepcidin axis has allowed the effect of erythroid activity on iron balance to be studied and has created the basis for better defining the erythroid regulators.
In iron-loading anemias, ineffective erythropoiesis suppresses hepcidin production, which result in dysregulating iron homeostasis. Miller and co-workers showed that release of cytokines like growth differentiation factor 15 (GDF15) during the process of ineffective erythropoiesis inhibits hepcidin production, thus defining a molecular link between ineffective erythropoiesis, suppression of hepcidin production and parenchymal iron loading.
Study design
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Sickle cell disease | – | – |
| hereditary spherocytosis. | – | – |
Who may qualify
- Ages
- 5 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- The study will contain 40 patients with Sickle cell disease and 40 patients with hereditary spherocytosis. After ICF (Informed Consent Form) has been signed by the patients the following laboratory tests will be taken once during the study: 1. GDF 15( 3ml of serum) (at the laboratory of hematology at Wolfson Medical Center/Israel) 2. Hepcidine (3ml of serum) (at the laboratory of Prof. T. Ganz, USA). The blood samples should be taken at least one week apart from blood transfusion. In case of infection or acute inflammation , blood samples should be taken only one week after resolution of these conditions.
Key inclusion criteria (1 listed)
- non
Key exclusion criteria (1 listed)
- non
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
GDF 15
Time frame: year
1 secondary outcome
Hepcidine
Time frame: year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Sep 2010.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
GHOTI HOSSAM, doctor principal investigator
HEMATOLOGY DEPARTMENT ON WOLFSSON MEDICAL CENTER
Questions about this trial
What is this clinical trial studying?
An observational study of Patients With Thalassemia Intermedia, and Congenital Dyserythropoietic Anemia Type I, run by Wolfson Medical Center.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 14 Sep 2010. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Ghoti Hossam, doctor (035028110, drghoti123@yahoo.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
GDF 15 (time frame: year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Sep 2010.