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Evaluation of the Efficacy in Decreasing Iron Absorption in Patients With Congenital Dyserythropoietic Anemia Type I by Treatment With LOSEC

NCT01795794 · Status: UNKNOWN · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2013-02-27

No results posted yet for this study

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.

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.

Conditions

  • CDA Type I

Interventions

DRUG

omeprazole

Sponsors & Collaborators

  • Soroka University Medical Center

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2013-04-30
Primary Completion
2013-12-31
Completion
2013-12-31

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01795794 on ClinicalTrials.gov