A Phase I Study of AC220 for Children With Relapsed or Refractory Acute Lymphoblastic Leukemia or Acute Myelogenous Leukemia
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|ClinicalTrials.gov Identifier: NCT01411267|
Recruitment Status : Completed
First Posted : August 8, 2011
Last Update Posted : May 21, 2013
|Condition or disease||Intervention/treatment||Phase|
|Lymphoblastic Leukemia, Acute, Childhood Myelogenous Leukemia, Acute, Childhood||Drug: AC220 Drug: Cytarabine Drug: Etoposide Drug: Methotrexate||Phase 1|
This is a study for patients with relapsed acute lymphoblastic leukemia (ALL) or acute myelogenous leukemia (AML). Some people diagnosed with leukemia have changes in a receptor located on the surface of white blood cells called FLT3. This is known as a FLT3 mutation. FLT3 plays an important role in the way cells grow and divide. In normal cells, the FLT3 receptor is switched off most of the time and only switches on when it gets a chemical signal from outside. But cells with the FLT3 mutation have the grow signal permanently switched on. This means leukemia cells with the FLT3 mutation are growing and dividing all the time. Doctors have found that people with leukemia cells that carry FLT3 mutations are less likely to go into remission with chemotherapy and have a higher risk of the leukemia coming back after treatment.
This is a study of an investigational drug called AC220. AC220 is considered investigational because it has not been approved in the United States by the Food and Drug Administration (FDA). AC220 is a drug which is able to "turn off" the FLT3 grow signal. AC220 will be given with cytarabine and etoposide to treat the relapsed leukemia. This is a phase I study, which means that the study is being done to find the highest dose of AC220 that can be given safely with the drugs cytarabine and etoposide to children and young adults.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||24 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Phase I Study of AC220 for Children With Relapsed or Refractory ALL or AML|
|Study Start Date :||August 2011|
|Actual Primary Completion Date :||April 2013|
|Actual Study Completion Date :||May 2013|
- Drug: AC220
Dose assigned at study entry. AC220 will be given orally once daily on days 7-28.
- Drug: Cytarabine
1000 mg/m2/day IV given every 12 hours on days 1 through 5.
IT cytarabine given intrathecally to patients with AML on day "0" of course 1 and 2. Dose defined by age
- 30 mg for patients age 1-1.99 years of age
- 50 mg for patients age 2-2.99 years of age
- 70 mg for patients >3 years of age
- Cytosine Arabinoside
- Drug: Etoposide
150 mg/m2/day IV on days 1 through 5.Other Names:
- Drug: Methotrexate
IT methotrexate given intrathecally to patients with ALL on day "0" of course 1 and 2. Dose defined by age
- 8 mg for patients age 1-1.99
- 10 mg for patients age 2-2.99
- 12 mg for patients 3-8.99 years of age
- 15 mg for patients >9 years of age
- The dose of AC220 that can be given safely with etoposide and cytarabine. [ Time Frame: 6 weeks ]The incidence of dose limiting toxicity (DLT) will be measured. The maximum tolerated dose will be the highest study dose at which 1 or fewer of six patients experience DLT during cycle 1 of therapy.
- The response rate after treatment. [ Time Frame: 10 weeks ]
- Composite pharmacokinetics of AC220. [ Time Frame: 3 years ]Measure the levels of AC220 in the blood during treatment.
- FLT-3 mutation in cancer cells before and after treatment. [ Time Frame: 3 years ]
- Inhibition of FLT3 phosphorylation [ Time Frame: 3 years ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01411267
Show 28 Study Locations
|Study Chair:||Todd Cooper, MD||Children's Healthcare of Atlanta, Emory University|