Back to results

Treatment of Myelodysplastic Syndromes Comparing Hydralazine/Ac. Valproic and Supportive Care in Patients Not Candidates, Refractory and/or Intolerant to Intensive Chemotherapy

Primary Purpose

Myelodysplastic Syndrome

Status

Unknown status

Phase

Phase 2

Locations

Spain

Study Type

Interventional

Intervention

BSC

HIDRA/VPA

About this trial

This is an interventional treatment trial for Myelodysplastic Syndrome focused on measuring myelodysplastic syndrome, Transfusion, Hydralazine, valproic acid

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Ability to understand and voluntarily sign the consent form.
Age ≥ 18 years at the time of signing the informed consent form.
Ability and willingness to meet the schedule of study visits and other protocol requirements.
Documented diagnosis of SMDs according to the criteria of disease risk IPSS intermediate-2, high risk or any risk with transfusion requirements.
Transfusion-dependent anemia erythrocytes defined as the absence of a period of 56 consecutive days without transfusion of red blood cells for at least 112 days prior (16 weeks).
Engage both women and men to use highly effective contraception.
Patients are not candidates for treatment with azacitidine or chemotherapy

Exclusion Criteria:

Patients who have any of these exclusion criteria may not be included in the trial:
1. Pregnant or breastfeeding.
2. After hematopoietic stem cell transplantation.
3. Patients with vitamin B12 deficiency, Folic Acid and Iron
4. Any severe psychiatric illness or condition that prevents the patient sign the consent form for the patient or involves an unacceptable risk in case of participating in the study.
5. Hypersensitivity to hydralazine and / or AC. Valproic

Sites / Locations

Txagorritxu Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

HIDRA/VPA

best supportive care (BSC)

Arm Description

In each cycle (30 days), Hydralazine 50mg tablets every 12 hours and Valproic 500mg tablets every 8 hours will be administrated orally to HYDRA / VPA group.Each patient will receive 6 cycles of hydralazine and valproic acid.

The support group will be receive transfusional BSC, erythropoietin and / or G-CSF as determined by the physician.

Outcomes

Primary Outcome Measures

change in hemoglobin from baseline in patients who express an erythroid response.

Independence RBC transfusion requirement for ≥ 8 weeks (56 days) followed by treatment at any time.

Secondary Outcome Measures

Erythroid response according to the criteria of the international working group of SMDs at 24 weeks

Number and type of administered blood transfusions, time between the last transfusion prior to inclusion in the study and the first transfusion after the 8 weeks of treatment.

Full Information

NCT ID

NCT01356875

First Posted

May 18, 2011

Last Updated

May 19, 2011

Sponsor

Basque Health Service

1. Study Identification

Unique Protocol Identification Number

NCT01356875

Brief Title

Treatment of Myelodysplastic Syndromes Comparing Hydralazine/Ac. Valproic and Supportive Care in Patients Not Candidates, Refractory and/or Intolerant to Intensive Chemotherapy

Official Title

Phase II Clinical Trial for Treatment of Myelodysplastic Syndromes Comparing Hydralazine / Ac.Valproico and Supportive Care in Patients Not Candidates, Refractory and / or Intolerant to Intensive Chemotherapy

Study Type

Interventional

2. Study Status

Record Verification Date

April 2011

Overall Recruitment Status

Unknown status

Study Start Date

September 2011 (undefined)

Primary Completion Date

September 2014 (Anticipated)

Study Completion Date

January 2015 (Anticipated)

3. Sponsor/Collaborators

Name of the Sponsor

Basque Health Service

4. Oversight

Data Monitoring Committee

5. Study Description

Brief Summary

Transfusional dependence has been associated closely and independently with low survival in patients with myelodysplastic syndrome (MDS), especially in patients at low risk according to IPSS. Treatment of patients with hydralazine + valproic acid as an alternative to treatment with 5-azacytidine has lower cost and possibly as effective with fewer side effects. The objective of this phase II study is to determine the effectiveness of combination therapy with hydralazine + Ac. Valproic compared with best supportive care. The investigators will select 42 patients per group, and after 14 weeks of treatment the investigators will study in both groups the hematological response (transfusion-dependent, hemoglobin, cytogenetics and morphology) and treatment safety (adverse reactions and vital signs) to 1 year after starting treatment. The concentration of hemoglobin, the number of transfusions, platelets, neutrophils and other continuous variables in both groups will be compared by Student t or Mann-Whitney, as appropriate. For comparison of cytogenetic and morphological response and other categorical variables between groups Chi square will be used. And within each group the investigators will compare each of these variables before and after treatment by t-test for paired data or Wilcoxon test.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Myelodysplastic Syndrome

Keywords

myelodysplastic syndrome, Transfusion, Hydralazine, valproic acid

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

42 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

HIDRA/VPA

Arm Type

Experimental

Arm Description

Arm Title

best supportive care (BSC)

Arm Type

Active Comparator

Arm Description

The support group will be receive transfusional BSC, erythropoietin and / or G-CSF as determined by the physician.

Intervention Type

Drug

Intervention Name(s)

BSC

Intervention Description

The support group will be receive transfusional BSC, erythropoietin and / or G-CSF as determined by the physician.

Intervention Type

Drug

Intervention Name(s)

HIDRA/VPA

Intervention Description

In each cycle (30 days), Hydralazine 50mg tablets every 12 hours and Valproic 500mg tablets every 8 hours will be administrated orally to HYDRA / VPA group.Each patient will receive 6 cycle.

Primary Outcome Measure Information:

Title

change in hemoglobin from baseline in patients who express an erythroid response.

Description

Independence RBC transfusion requirement for ≥ 8 weeks (56 days) followed by treatment at any time.

Time Frame

8 weeks

Secondary Outcome Measure Information:

Title

Erythroid response according to the criteria of the international working group of SMDs at 24 weeks

Description

Number and type of administered blood transfusions, time between the last transfusion prior to inclusion in the study and the first transfusion after the 8 weeks of treatment.

Time Frame

24 weeks

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Ability to understand and voluntarily sign the consent form. Age ≥ 18 years at the time of signing the informed consent form. Ability and willingness to meet the schedule of study visits and other protocol requirements. Documented diagnosis of SMDs according to the criteria of disease risk IPSS intermediate-2, high risk or any risk with transfusion requirements. Transfusion-dependent anemia erythrocytes defined as the absence of a period of 56 consecutive days without transfusion of red blood cells for at least 112 days prior (16 weeks). Engage both women and men to use highly effective contraception. Patients are not candidates for treatment with azacitidine or chemotherapy Exclusion Criteria: Patients who have any of these exclusion criteria may not be included in the trial: Pregnant or breastfeeding. After hematopoietic stem cell transplantation. Patients with vitamin B12 deficiency, Folic Acid and Iron Any severe psychiatric illness or condition that prevents the patient sign the consent form for the patient or involves an unacceptable risk in case of participating in the study. Hypersensitivity to hydralazine and / or AC. Valproic

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Breno Moreno de Gusmao, MD

Phone

+34-945 007000

breno.morenodegusmao@osakidetza.net

Facility Information:

Facility Name

Txagorritxu Hospital

City

Vitoria-Gasteiz

State/Province

Alava

ZIP/Postal Code

01009

Country

Spain

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Breno Moreno de Gusmao, MD

Phone

+34 945 007000

breno.morenodegusmao@osakidetza.net

12. IPD Sharing Statement

Learn more about this trial

Treatment of Myelodysplastic Syndromes Comparing Hydralazine/Ac. Valproic and Supportive Care in Patients Not Candidates, Refractory and/or Intolerant to Intensive Chemotherapy

We'll reach out to this number within 24 hrs