Study of NRX 195183 Therapy for Patients With Relapsed or Refractory Acute Promyelocytic Leukemia

Study Overview
=================
Brief Summary
-----------------
The purpose of this study is to determine whether NRX 195183 is effective in the treatment of relapsed or refractory Acute Promyelocytic Leukemia

Detailed Description
-----------------
A common current therapeutic approach to APL uses oral ATRA and chemotherapy in induction and consolidation. This approach has significantly improved survival in newly diagnosed APL patients. However, approximately 30% of patients relapse. Recently, an approach involving the combination of oral ATRA and arsenic trioxide has been tested. The prognosis for relapsed patients is very poor. This study seeks to investigate NRX 195183 monotherapy in patients who have failed or are resistant to or are intolerant of any prior therapy.

Official Title
-----------------
Phase II Study of NRX 195183 Therapy for Patients With Relapsed or Refractory Acute Promyelocytic Leukemia

Conditions
-----------------
Acute Promyelocytic Leukemia

Intervention / Treatment
-----------------
* Drug: NRX 195183 Soft Gelatin Capsule


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: Clinical diagnosis of APL morphology or FAB M3 variant confirmed by RT-PCR assay or chromosome analysis/FISH showing t(15:17) translocation. Patients must also have relapse from, resistance to or intolerance of any one or more of the following therapies: ATRA Cytotoxic chemotherapy Arsenic trioxide Patients must be 18 or older. Bilirubin equal or less than 1.5 times the upper limit of normal. Creatinine equal or less than 1.5 times the upper limit of normal. Patients entered into this study should be non-pregnant and non-nursing and should not plan on becoming pregnant while on treatment. Treatment under this protocol would expose an unborn child to significant risks. Women and men of reproductive potential should agree to use an effective means of birth control. There is an extremely high risk that a severely deformed infant will result if NRX 195183 is administered during pregnancy. Exclusion Criteria: Non-APL, AML patients should be excluded from the study. Other serious illnesses which would limit survival to 6 months. Psychiatric conditions which would prevent compliance with treatment or informed consent. Uncontrolled or severe cardiovascular disease. This would include history of a recent acute myocardial infarction, uncontrolled congestive heart failure, or active angina. AIDS or HIV positive patients, although HIV test is not required for accrual.

Ages Eligible for Study
-----------------
Minimum Age: 18 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 1<br> | Drug: NRX 195183 Soft Gelatin Capsule<br>* Daily oral NRX 195183 administration for 90 days to assess CR rate. Patients with CR may continue maintenance therapy after a 2-week drug holiday. Maintenance therapy will consist of two 3-month daily oral NRX 195183 administration separated by a 2-week drug holiday.<br>|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Complete Remission |  | 90 Days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Molecular Complete Remission |  | 9 months | 

 Terms related to the study
=================
Keywords Provided by Centre Hospitalier Valida
-----------------
Acute Promyelocytic Leukemia, Cancer, Leukemia, APL, ATRA, Tretinoid, Vesanoid, Retinoic Acid Receptor Alpha, All Trans Retinoic Acid, Arsenic Trioxide, Trisenox, Retinoid, Relapsed or Refractory Acute Promyelocytic Leukemia