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

NCT ID: NCT01795794

Last Updated: 2013-02-27

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

UNKNOWN

Clinical Phase

PHASE4

Total Enrollment

12 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-04-30

Study Completion Date

2013-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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

See the medical conditions and disease areas that this research is targeting or investigating.

CDA Type I

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

LOSEC

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.

Group Type EXPERIMENTAL

omeprazole

Intervention Type DRUG

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

omeprazole

Intervention Type DRUG

Other Intervention Names

Discover alternative or legacy names that may be used to describe the listed interventions across different sources.

losec

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

* patients with CDAI
* over 30 kg
* can swallow tablets
* ferritin levels higher than the normal range but still don't require treatment.

Exclusion Criteria

* N.A
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Soroka University Medical Center

OTHER

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

sor034612ctil

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.