CDA Type I Clinical Trial
Verified date | February 2013 |
Source | Soroka University Medical Center |
Contact | n/a |
Is FDA regulated | No |
Health authority | Israel: Ministry of Health |
Study type | Interventional |
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.
Status | Not yet recruiting |
Enrollment | 12 |
Est. completion date | December 2013 |
Est. primary completion date | December 2013 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 12 Years and older |
Eligibility |
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 |
Endpoint Classification: Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
n/a |
Lead Sponsor | Collaborator |
---|---|
Soroka University Medical Center |
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | evaluate number of adverse effects per patient treated with LOSEC + levels of iron, ferritin,complete blood count and chemistry panel. | 1 year | No |