Trial record 2 of 2 for:    "Congenital dyserythropoietic anemia type 1"

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

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified February 2013 by Soroka University Medical Center
Sponsor:
Information provided by (Responsible Party):
Soroka University Medical Center
ClinicalTrials.gov Identifier:
NCT01795794
First received: February 11, 2013
Last updated: February 26, 2013
Last verified: February 2013
  Purpose

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.


Condition Intervention Phase
CDA Type I
Drug: omeprazole
Phase 4

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Resource links provided by NLM:


Further study details as provided by Soroka University Medical Center:

Primary Outcome Measures:
  • evaluate number of adverse effects per patient treated with LOSEC + levels of iron, ferritin,complete blood count and chemistry panel. [ Time Frame: 1 year ] [ Designated as safety issue: No ]

Estimated Enrollment: 12
Study Start Date: April 2013
Estimated Study Completion Date: December 2013
Estimated Primary Completion Date: December 2013 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 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.
Drug: omeprazole
Other Name: losec

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.

  Eligibility

Ages Eligible for Study:   12 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

No Contacts or Locations Provided
  More Information

No publications provided

Responsible Party: Soroka University Medical Center
ClinicalTrials.gov Identifier: NCT01795794     History of Changes
Other Study ID Numbers: sor034612ctil
Study First Received: February 11, 2013
Last Updated: February 26, 2013
Health Authority: Israel: Ministry of Health

Additional relevant MeSH terms:
Anemia, Dyserythropoietic, Congenital
Anemia, Hemolytic, Congenital
Anemia, Hemolytic
Anemia
Hematologic Diseases
Genetic Diseases, Inborn
Omeprazole
Anti-Ulcer Agents
Gastrointestinal Agents
Therapeutic Uses
Pharmacologic Actions
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action

ClinicalTrials.gov processed this record on July 22, 2014