CDA Type IPhase 4NCT01795794
Evaluation of the Efficacy in Decreasing Iron Absorption in Patients With Congenital Dyserythropoietic Anemia Type I by Treatment With LOSEC
A Phase 4 study testing omeprazole in people with CDA Type I, run by Soroka University Medical Center.
Trial summary
Congenital Dyserythropoietic Anemia Type I (CDAI) is a recessive autosomal disease caused by ineffective erythropoiesis that causes Anemia \& accumulation of iron due to increased absorption of iron in the intestine. The iron is being accumulated in the body \& causes damage of the liver, heart \& endocrine glands. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- CDA Type I
- What is being tested?
- omeprazole
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- No sites listed yet
- Study dates
- Apr 2013to Dec 2013
- Phase
- Phase 4
- 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 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- patients with CDAI
- over 30 kg
- can swallow tablets
- ferritin levels higher than the normal range but still don't require treatment.
Who cannot join
- N.A
About this study
Congenital Dyserythropoietic Anemia Type I (CDAI) is a recessive autosomal disease caused by ineffective erythropoiesis that causes Anemia \& accumulation of iron due to increased absorption of iron in the intestine.
The iron is being accumulated in the body \& causes damage of the liver, heart \& endocrine glands.
Read more
The standard follow up of iron levels is done by ferritin blood test \& although the test is not accurate it is the most available.
Medical treatment to removal of iron excess from the body is given in ferritin levels of 500-1000.
3 drugs are approved in the market:
- Deferoxamine, given subcutaneous during the night, 5-7 nights/week and therefore is less used today.
- Deferiprone-given 3 times a day, is a weaker chelator, although it seems like it is good to give it in combination with one of the 2 other drugs because it removes the iron from the heart's cells better.
A rare but severe adverse effect is Agranulocytosis. This drug is usually not given in childbirths.
-The 3rd drug which is mostly in use today is Deferasirox, given once daily, but has also adverse effects, among them- damage of liver \& kidney function, damage to the digestive system, hearing and seeing.
Iron is being absorbed in acidic area, \& thus drugs which decrease the acidity like hydrogen pump's inhibitors, can inhibit the absorption of iron, and indeed there are reports that these drugs decrease the absorption of iron and were used as treatment to hemochromatosis as well.
Those hydrogen pump's inhibitors have also adverse effects. The rarest but most severe is intestine infection by Clostridium.
In children, reports of adverse effects are minimal- mostly headaches \& abdominal pains and nosocomial infections. In adults, there are reports of fractures, magnesium deficiency \& vitamin B12 deficiency. All in all, the adverse effects are rare \& uncommon.
Read the detailed description
The aim of this study is to check whether treatment with drug which inhibits the hydrogen pump and therefore decreases acidity, will reduce the iron's absorption in patients with CDA TYPE I and whether it will be possible to use it for a period instead of giving drugs which have much more adverse effects, for removal of iron excess from the body.
Therefore, we would like to enroll 10-12 patients over 30 kg who can swallow tablets (age over 12) and whose ferritin levels are higher from the normal range, but still don't require medical treatment (ferritin levels between 400-700).
These patients will be given LOSEC 20 mg X 1/day for 6 months. That group will be her own control group in the 6 months later. Drug will be dispensed to the patients.
In the beginning of the study, after signing Informed Consent Form, blood tests will be taken for Complete Blood Count, iron levels, iron saturation, ferritin, complete chemistry panel, including calcium \& magnesium.
Same tests will be taken after 3 and 6 months and also 3 and 6 months after end of treatment.
In every event of fever, headaches, abdominal pain or diarrhea, patients need to apply the doctor and report.
- Conditions
- CDA Type I
- Being tested
- omeprazole
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
omeprazoleDrug
Also called losec
| Study group | Role | What they receive |
|---|---|---|
| LOSEC | Experimental | LOSEC will be given 20 mg X 1/day for 6 months and then for the next 6 months the same group will be the "control" group of herself. Receives: Drug: omeprazole |
What is being measured
The primary outcome is the main question the study is designed to answer.
evaluate number of adverse effects per patient treated with LOSEC + levels of iron, ferritin,complete blood count and chemistry panel.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2013 (estimated)
- Study startApr 2013
- Primary completionDec 2013 (estimated)
- Study completionDec 2013 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CDA Type IStudies 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Soroka University Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01795794
- Sponsor's protocol ID
- sor034612ctil
- Status last verified
- Feb 2013
- First submitted
- 11 Feb 2013
- Last updated
- 27 Feb 2013
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Evaluation of the Efficacy in Decreasing Iron Absorption in Patients With Congenital Dyserythropoietic Anemia Type I by Treatment With LOSEC
- ClinicalTrials.gov ID
- NCT01795794
- Acronym / sponsor ID
- sor034612ctil
- Conditions
- CDA Type I
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Soroka University Medical CenterUniversity, hospital or other
- Study start
- Apr 2013
- Primary completion
- Dec 2013 (estimated)
- Study completion
- Dec 2013 (estimated)
- First posted
- 21 Feb 2013 (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.
Congenital Dyserythropoietic Anemia Type I (CDAI) is a recessive autosomal disease caused by ineffective erythropoiesis that causes Anemia \& accumulation of iron due to increased absorption of iron in the intestine.
The iron is being accumulated in the body \& causes damage of the liver, heart \& endocrine glands.
The standard follow up of iron levels is done by ferritin blood test \& although the test is not accurate it is the most available.
Medical treatment to removal of iron excess from the body is given in ferritin levels of 500-1000.
3 drugs are approved in the market:
- Deferoxamine, given subcutaneous during the night, 5-7 nights/week and therefore is less used today.
- Deferiprone-given 3 times a day, is a weaker chelator, although it seems like it is good to give it in combination with one of the 2 other drugs because it removes the iron from the heart's cells better.
A rare but severe adverse effect is Agranulocytosis. This drug is usually not given in childbirths.
-The 3rd drug which is mostly in use today is Deferasirox, given once daily, but has also adverse effects, among them- damage of liver \& kidney function, damage to the digestive system, hearing and seeing.
Iron is being absorbed in acidic area, \& thus drugs which decrease the acidity like hydrogen pump's inhibitors, can inhibit the absorption of iron, and indeed there are reports that these drugs decrease the absorption of iron and were used as treatment to hemochromatosis as well.
Those hydrogen pump's inhibitors have also adverse effects. The rarest but most severe is intestine infection by Clostridium.
In children, reports of adverse effects are minimal- mostly headaches \& abdominal pains and nosocomial infections. In adults, there are reports of fractures, magnesium deficiency \& vitamin B12 deficiency. All in all, the adverse effects are rare \& uncommon.
Detailed description
The aim of this study is to check whether treatment with drug which inhibits the hydrogen pump and therefore decreases acidity, will reduce the iron's absorption in patients with CDA TYPE I and whether it will be possible to use it for a period instead of giving drugs which have much more adverse effects, for removal of iron excess from the body.
Therefore, we would like to enroll 10-12 patients over 30 kg who can swallow tablets (age over 12) and whose ferritin levels are higher from the normal range, but still don't require medical treatment (ferritin levels between 400-700).
These patients will be given LOSEC 20 mg X 1/day for 6 months. That group will be her own control group in the 6 months later. Drug will be dispensed to the patients.
In the beginning of the study, after signing Informed Consent Form, blood tests will be taken for Complete Blood Count, iron levels, iron saturation, ferritin, complete chemistry panel, including calcium \& magnesium.
Same tests will be taken after 3 and 6 months and also 3 and 6 months after end of treatment.
In every event of fever, headaches, abdominal pain or diarrhea, patients need to apply the doctor and report.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| LOSEC | Experimental | Drug: omeprazoleLOSEC will be given 20 mg X 1/day for 6 months and then for the next 6 months the same group will be the "control" group of herself. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- patients with CDAI
- over 30 kg
- can swallow tablets
- ferritin levels higher than the normal range but still don't require treatment.
Key exclusion criteria (1 listed)
- N.A
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| omeprazoleAlso: losec | Drug | LOSEC |
Primary outcome
evaluate number of adverse effects per patient treated with LOSEC + levels of iron, ferritin,complete blood count and chemistry panel.
Time frame: 1 year
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2013.
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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing omeprazole in people with CDA Type I, run by Soroka University 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 4 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 27 Feb 2013. 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
evaluate number of adverse effects per patient treated with LOSEC + levels of iron, ferritin,complete blood count and chemistry panel. (time frame: 1 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 27 Feb 2013.