Myelodysplastic SyndromePhase 2NCT01356875
Treatment of Myelodysplastic Syndromes Comparing Hydralazine/Ac. Valproic and Supportive Care in Patients Not Candidates, Refractory and/or Intolerant to Intensive Chemotherapy
A Phase 2 study testing BSC and HIDRA/VPA in people with Myelodysplastic Syndrome, run by Basque Health Service at 1 site in Spain.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myelodysplastic Syndrome
- What is being tested?
- BSC and HIDRA/VPA
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 42 participants
- Where
- 1 siteSpain
- Study dates
- Sep 2011to Jan 2015
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Spain1 site
Txagorritxu Hospital
Vitoria-Gasteiz, Alava, 01009
Breno Moreno de Gusmao, MD, +34 945 007000, breno.morenodegusmao@osakidetza.net
About this study
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
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.
- Conditions
- Myelodysplastic Syndrome
- Being tested
- BSC, HIDRA/VPA
- Keywords
- myelodysplastic syndrome, Transfusion, Hydralazine, valproic acid
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BSCDrug
The support group will be receive transfusional BSC, erythropoietin and / or G-CSF as determined by the physician.
HIDRA/VPADrug
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.
| Study group | Role | What they receive |
|---|---|---|
| HIDRA/VPA | Experimental | 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. Receives: Drug: HIDRA/VPA |
| best supportive care (BSC) | Active comparator | The support group will be receive transfusional BSC, erythropoietin and / or G-CSF as determined by the physician. Receives: Drug: BSC |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 8 weeks
Secondary outcomes (1)
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.
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 May 2011 (estimated)
- Study startSep 2011
- Primary completionSep 2014 (estimated)
- Study completionJan 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Basque Health Service
- Sponsor type
- Other government
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01356875
- Sponsor's protocol ID
- SMD-TXAGO
- Status last verified
- Apr 2011
- First submitted
- 18 May 2011
- Last updated
- 20 May 2011
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 20 May 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT01356875
- Acronym / sponsor ID
- SMD-TXAGO
- Conditions
- Myelodysplastic Syndrome
- Keywords
- myelodysplastic syndrome, Transfusion, Hydralazine, valproic acid
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- Basque Health ServiceOther government
- Study start
- Sep 2011
- Primary completion
- Sep 2014 (estimated)
- Study completion
- Jan 2015 (estimated)
- First posted
- 20 May 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| HIDRA/VPA | Experimental | Drug: HIDRA/VPAIn 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. |
| best supportive care (BSC) | Active comparator | Drug: BSCThe support group will be receive transfusional BSC, erythropoietin and / or G-CSF as determined by the physician. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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).
Key exclusion criteria (6 listed)
- Patients who have any of these exclusion criteria may not be included in the trial:
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BSCThe support group will be receive transfusional BSC, erythropoietin and / or G-CSF as determined by the physician. | Drug | best supportive care (BSC) |
| HIDRA/VPAIn 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. | Drug | HIDRA/VPA |
Primary outcome
change in hemoglobin from baseline in patients who express an erythroid response.
Time frame: 8 weeks
Independence RBC transfusion requirement for ≥ 8 weeks (56 days) followed by treatment at any time.
1 secondary outcome
Erythroid response according to the criteria of the international working group of SMDs at 24 weeks
Time frame: 24 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 May 2011.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BSC and HIDRA/VPA in people with Myelodysplastic Syndrome, run by Basque Health Service at 1 site in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 20 May 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Breno Moreno de Gusmao, MD (+34-945 007000, breno.morenodegusmao@osakidetza.net), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
change in hemoglobin from baseline in patients who express an erythroid response. (time frame: 8 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 May 2011.