Idiopathic Dilated CardiomyopathyPhase 2NCT02033278
Infusion Intracoronary of Mononuclear Autologous Adult no Expanded Stem Cells of Bone Marrow on Functional Recovery in Patients With Idiopathic Dilated Cardiomyopathy and Heart Failure.
A Phase 2 study testing Infusion of autologous mononuclear bone marrow cells in people with Idiopathic Dilated Cardiomyopathy, run by Andalusian Initiative for Advanced Therapies - Fundación Pública Andaluza Progreso y Salud at 9 sites in Spain.
Trial summary
Clinical trial phase IIb, double-blind, randomized, controlled with placebo. There is sufficient preliminary evidence to consider intracoronary injection of bone marrow progenitor cells as a viable, safe and beneficial treatment in patients with dilated cardiomyopathy, although the biological mechanism of action of… Read more about this study
- Who is it for?
- Ages 18 to 70 years
- Conditions the study is for
- Idiopathic Dilated Cardiomyopathy
- What is being tested?
- Infusion of autologous mononuclear bone marrow cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 27 participants
- Where
- 9 sitesSpain
- Study dates
- 6 Jan 2014to 21 Apr 2020
- 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 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients of both sexes and ages between 18 and 70 years.
- Patients diagnosed with dilated cardiomyopathy established by echocardiography.
- Minimum evolution since diagnosis of 6 months.
- Absence of coronary injury tested with multislice CT and/or hemodynamic study performed after study entry, or within the previous 36 months (or before in specific low risk clinical profiles) if no angina symptomatology is present.
- Patients receiving optimized medical therapy for at least 6 months prior to enrollment (individually adjusted according to functional status).
- Ejection fraction of the left ventricle <40% or ejection fraction of the left ventricle 40% -50% if left ventricular tele-diastolic volume is > 110 ml/m2.
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- Presence of sinus rhythm.
- Writen informed consent for participation in the trial.
- Normal laboratory parameters, defined by: Leukocytes ≥ 3000; Neutrophils ≥ 1500; Platelets ≥ 100,000; Aspartate aminotransferase / Alanine aminotransferase ≤ 2.5 standard range institution; Creatinine ≤ 2.5 mg / dL; Haemoglobin > 9 g/dL
- Women of childbearing potential must have negative results on a pregnancy test and agree to use medically approved methods of contraception thoughout follow up.
Who cannot join
- Secondary Dilated cardiomyopathy.
- Recent history of myocarditis (< 6 months prior to study entry).
- Patients amenable to receive cardiac resynchronization therapy
- Patients in active waiting list for heart transplantation.
- Coexistence of other serious systemic diseases.
- Coexistence of any type of blood disease
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- Pregnant or breastfeeding women; or women of childbearing potential not comminting to use effective contraception.
- Patients who are currently participating, or have completed their participation in a clinical trial within the last 3 months. Patients who have participated in any advanced therapies clinical trial any time previously.
- Patients with malignant or pre-malignant tumors.
- Positive serology for hepatitis B virus, hepatitis C virus or human immunodeficiency virus.
- Use of any protocolo prohibited medication. A wash-out period of 2 months can be considered for inclusion in the trial.
Trial locations and contacts
9 sites in 1 country.
9 sites in 1 country
Spain9 sites
Hospital Universitario Puerta del Mar
Cadiz
Hopistal Universitario Reina Sofía
Córdoba
Hospital Universitario Virgen de las Nieves
Granada
Hospital Universitario Juan Ramón Jimenez
Huelva
Hospital de Jerez
Jerez de la Frontera
Hospital Regional U. Carlos Haya
Málaga, 29014
Hospital Costa del Sol
Marbella
Hospital Universitario Virgen del Rocío
Seville
Hospital Universitario Virgen Macarena
Seville
About this study
Official title: Multicenter Phase IIb Clinical Trial, Double-blind, Randomized, Controlled Placebo for to Assess the Efficacy of Intracoronary Infusion of Autologous Adult Stem Cells Mononuclear Marrow Unexpanded on Functional Recovery in Patients With Idiopathic Dilated Cardiomyopathy and Heart Failure.
Clinical trial phase IIb, double-blind, randomized, controlled with placebo. There is sufficient preliminary evidence to consider intracoronary injection of bone marrow progenitor cells as a viable, safe and beneficial treatment in patients with dilated cardiomyopathy, although the biological mechanism of action of bone marrow cells in the myocardium is not known. In this project we propose to investigate comparatively and from a biological and clinical point of view the applicability of regenerative therapy with autologous bone marrow cells in patients with dilated cardiomyopathy.
Read the detailed description
The study population correspond to male and female patients with idiopathic dilated cardiomyopathy.
51 patients diagnosed with this disease are included. After inclusion, will proceed to the random allocation to study group or control group in a 2:1 ratio, 34 patients in the treatment group and 17 in the control group.
The total duration is expected to be 48 months: The inclusion period is 24 months and each patient assigned to the experimental group will be followed for 24 months, whereas that one ramdomized to the control group, will have a folow-up of 12 months. Upon completion there of, the patients will be followed in routine clinical practice.
This is a double blind study, in which all patients will perform the bone marrow harvesting.
All patients will receive the best medical treatment individualized (ACEIs or Angiotensin II receptor blocker, beta-blockers, diuretics and eplerenone) for at least 6 months prior to their participation in the clinical trial, so that the situation is stable and pharmacological basal condition is the same for everyone.
The bone marrow cells of patients assigned to placebo group will be cryopreserved, and once the trial is completed, the blind will be opened and all the patients who had been randomized to the control group, may be processed by the route of compassionate use with their own mononuclear bone marrow cells previously frozen.
The patients who are randomized to experimental group will be treated by the conventional treatment + infusion of autologous mononuclear bone marrow cells not expanded whereas the patients who are randomized to control group will be treated by the conventional treatment + infusion of placebo.
The main objective is to assess comparative the efficacy of intracoronary injection of bone marrow stem cells autologous to improve ventricular function in patients with idiopathic dilated cardiomyopathy who receive conventional medical treatment, compared with a control group who receive a infusion of placebo and conventional medical treatment. The improvement in ventricular function assessed by changes in angiographically determined ejection fraction.
Secondary objectives of the study are:
- To analyze the predictors of good clinical response, functional and biological treatment with adult stem cells autologous mononuclear bone marrow not expanded in terms of functional recovery.
The following parameters were evaluated: Functional class (NYHA), Natriuretic peptide B, Stress test (exercise time), Echocardiographic parameters of ventricular function, for example LVEF (%), TDV (ml), TSV (ml) and TAPSE (ms) and Biological parameters of cellular functionality, for example CD133 +, CD34 +, CD34 +/CD177 + and CD34 +/CD38- (in %).
- To determine, in the light of the obtained results, the application protocol suitable cell therapy for the treatment of dilated cardiomyopathy.
- Conditions
- Idiopathic Dilated Cardiomyopathy
- Being tested
- Infusion of autologous mononuclear bone marrow cells, Placebo infusion
- Medical subject headings
- Cardiomyopathy, Dilated
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Infusion of autologous mononuclear bone marrow cellsDrug
Infusion of autologous mononuclear bone marrow cells plus conventional medical treatment (as indicated by clinician)
Placebo infusionDrug
Placebo infusion plus conventional medical treatment (as indicated by clinician)
| Study group | Role | What they receive |
|---|---|---|
| Infusion of autologous mononuclear bone marrow cells | Experimental | Infusion of autologous mononuclear bone marrow cells plus conventional medical treatment (as indicated by clinician) Receives: Drug: Infusion of autologous mononuclear bone marrow cells |
| Placebo infusion | Placebo comparator | Placebo infusion plus conventional medical treatment (as indicated by clinician) Receives: Drug: Placebo infusion |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in ventricular function measured angiographically.
Measured: 24 months
Secondary outcomes (5)
Degree of clinical improvement based on the absence of major cardiac events (MACE) during follow-up.
Measured: 24 months
Clinical and analytical progress (NYHA grade & BNP)
Measured: 24 months
Time of evolution since diagnosis of idiopathic dilated cardiomyopathy prior to study entry.
Measured: 24 months
Functional recovery as measured with ergometry
Measured: 24 months
Echocardiography and Electrocardiography variables
Measured: 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2014 (estimated)
- Study start6 Jan 2014
- Primary completion19 Oct 2018
- Study completion21 Apr 2020
Why it stopped: Low recruitment rate, bibliographic evidence of lack of efficacy
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Idiopathic Dilated CardiomyopathyStudies 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
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 27 participants (actual)
Sponsor and investigators
- Lead sponsor
- Andalusian Initiative for Advanced Therapies - Fundación Pública Andaluza Progreso y Salud
- Sponsor type
- University, hospital or other
- Collaborators
- Iniciativa Andaluza en Terapias Avanzadas
- Responsible party
- Sponsor
- Investigators
- Miguel Romero Moreno, MD, PhD, Principal investigator, Hospital General Universitario Reina Sofía de MurciaManuel Sobrino Marquez, MD, Principal investigator, Hospitales Universitarios Virgen del RocíoLuis A Iñigo García, MD, Principal investigator, Hospital Costa del SolRicardo Zayas Rueda, MD, Principal investigator, Hospital Universitario Puerta del MarManuel De Mora Martín, MD, PhD, Principal investigator, Hospital Regional U. Carlos HayaSilvia López Fernández, MD, Principal investigator, University Hospital Virgen de las NievesJosé Francisco Díaz Fernández, MD, Principal investigator, Hospital Universitario Juan Ramón JimenezJesús Oneto Otero, MD, Principal investigator, Hospital de Jerez
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02033278
- Sponsor's protocol ID
- CMMo/MD/2013
- Status last verified
- Sep 2020
- First submitted
- 9 Jan 2014
- Last updated
- 18 Sep 2020
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Romero M, Suarez-de-Lezo J, Herrera C, Pan M, Lopez-Aguilera J, Suarez-de-Lezo J Jr, Baeza-Garzon F, Hidalgo-Lesmes FJ, Fernandez-Lopez O, Martinez-Atienza J, Cebrian E, Martin-Palanco V, Jimenez-Moreno R, Gutierrez-Fernandez R, Nogueras S, Carmona MD, Ojeda S, Cuende N, Mata R. Randomised, double-blind, placebo-controlled clinical trial for evaluating the efficacy of intracoronary injection of autologous bone marrow mononuclear cells in the improvement of the ventricular function in patients with idiopathic dilated myocardiopathy: a study protocol. BMC Cardiovasc Disord. 2019 Aug 22;19(1):203. doi: 10.1186/s12872-019-1182-4.
PubMed 31438858
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multicenter Phase IIb Clinical Trial, Double-blind, Randomized, Controlled Placebo for to Assess the Efficacy of Intracoronary Infusion of Autologous Adult Stem Cells Mononuclear Marrow Unexpanded on Functional Recovery in Patients With Idiopathic Dilated Cardiomyopathy and Heart Failure.
- ClinicalTrials.gov ID
- NCT02033278
- Acronym / sponsor ID
- CMMo/MD/2013
- Conditions
- Idiopathic Dilated Cardiomyopathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Low recruitment rate, bibliographic evidence of lack of efficacy
- Enrolment
- 27 participants (actual)
- Lead sponsor
- Andalusian Initiative for Advanced Therapies - Fundación Pública Andaluza Progreso y SaludUniversity, hospital or other
- Collaborators
- Iniciativa Andaluza en Terapias Avanzadas
- Study start
- 6 Jan 2014 (actual)
- Primary completion
- 19 Oct 2018 (actual)
- Study completion
- 21 Apr 2020 (actual)
- First posted
- 10 Jan 2014 (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.
Clinical trial phase IIb, double-blind, randomized, controlled with placebo. There is sufficient preliminary evidence to consider intracoronary injection of bone marrow progenitor cells as a viable, safe and beneficial treatment in patients with dilated cardiomyopathy, although the biological mechanism of action of bone marrow cells in the myocardium is not known. In this project we propose to investigate comparatively and from a biological and clinical point of view the applicability of regenerative therapy with autologous bone marrow cells in patients with dilated cardiomyopathy.
Detailed description
The study population correspond to male and female patients with idiopathic dilated cardiomyopathy.
51 patients diagnosed with this disease are included. After inclusion, will proceed to the random allocation to study group or control group in a 2:1 ratio, 34 patients in the treatment group and 17 in the control group.
The total duration is expected to be 48 months: The inclusion period is 24 months and each patient assigned to the experimental group will be followed for 24 months, whereas that one ramdomized to the control group, will have a folow-up of 12 months. Upon completion there of, the patients will be followed in routine clinical practice.
This is a double blind study, in which all patients will perform the bone marrow harvesting.
All patients will receive the best medical treatment individualized (ACEIs or Angiotensin II receptor blocker, beta-blockers, diuretics and eplerenone) for at least 6 months prior to their participation in the clinical trial, so that the situation is stable and pharmacological basal condition is the same for everyone.
The bone marrow cells of patients assigned to placebo group will be cryopreserved, and once the trial is completed, the blind will be opened and all the patients who had been randomized to the control group, may be processed by the route of compassionate use with their own mononuclear bone marrow cells previously frozen.
The patients who are randomized to experimental group will be treated by the conventional treatment + infusion of autologous mononuclear bone marrow cells not expanded whereas the patients who are randomized to control group will be treated by the conventional treatment + infusion of placebo.
The main objective is to assess comparative the efficacy of intracoronary injection of bone marrow stem cells autologous to improve ventricular function in patients with idiopathic dilated cardiomyopathy who receive conventional medical treatment, compared with a control group who receive a infusion of placebo and conventional medical treatment. The improvement in ventricular function assessed by changes in angiographically determined ejection fraction.
Secondary objectives of the study are:
- To analyze the predictors of good clinical response, functional and biological treatment with adult stem cells autologous mononuclear bone marrow not expanded in terms of functional recovery.
The following parameters were evaluated: Functional class (NYHA), Natriuretic peptide B, Stress test (exercise time), Echocardiographic parameters of ventricular function, for example LVEF (%), TDV (ml), TSV (ml) and TAPSE (ms) and Biological parameters of cellular functionality, for example CD133 +, CD34 +, CD34 +/CD177 + and CD34 +/CD38- (in %).
- To determine, in the light of the obtained results, the application protocol suitable cell therapy for the treatment of dilated cardiomyopathy.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Infusion of autologous mononuclear bone marrow cells | Experimental | Drug: Infusion of autologous mononuclear bone marrow cellsInfusion of autologous mononuclear bone marrow cells plus conventional medical treatment (as indicated by clinician) |
| Placebo infusion | Placebo comparator | Drug: Placebo infusionPlacebo infusion plus conventional medical treatment (as indicated by clinician) |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- Patients of both sexes and ages between 18 and 70 years.
- Patients diagnosed with dilated cardiomyopathy established by echocardiography.
- Minimum evolution since diagnosis of 6 months.
- Absence of coronary injury tested with multislice CT and/or hemodynamic study performed after study entry, or within the previous 36 months (or before in specific low risk clinical profiles) if no angina symptomatology is present.
- Patients receiving optimized medical therapy for at least 6 months prior to enrollment (individually adjusted according to functional status).
Key exclusion criteria (11 listed)
- Secondary Dilated cardiomyopathy.
- Recent history of myocarditis (< 6 months prior to study entry).
- Patients amenable to receive cardiac resynchronization therapy
- Patients in active waiting list for heart transplantation.
- Coexistence of other serious systemic diseases.
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 |
|---|---|---|
| Infusion of autologous mononuclear bone marrow cellsInfusion of autologous mononuclear bone marrow cells plus conventional medical treatment (as indicated by clinician) | Drug | Infusion of autologous mononuclear bone marrow cells |
| Placebo infusionPlacebo infusion plus conventional medical treatment (as indicated by clinician) | Drug | Placebo infusion |
Primary outcome
Changes in ventricular function measured angiographically.
Time frame: 24 months
5 secondary outcomes
Degree of clinical improvement based on the absence of major cardiac events (MACE) during follow-up.
Time frame: 24 months
Clinical and analytical progress (NYHA grade & BNP)
Time frame: 24 months
Time of evolution since diagnosis of idiopathic dilated cardiomyopathy prior to study entry.
Time frame: 24 months
Functional recovery as measured with ergometry
Time frame: 24 months
Echocardiography and Electrocardiography variables
Time frame: 24 months
Results and safety
The study was stopped early (reason given: Low recruitment rate, bibliographic evidence of lack of efficacy). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Sep 2020.
- Derived from this study: PubMed 31438858Romero M, Suarez-de-Lezo J, Herrera C, Pan M, Lopez-Aguilera J, Suarez-de-Lezo J Jr, Baeza-Garzon F, Hidalgo-Lesmes FJ, Fernandez-Lopez O, Martinez-Atienza J, Cebrian E, Martin-Palanco V, Jimenez-Moreno R, Gutierrez-Fernandez R, Nogueras S, Carmona MD, Ojeda S, Cuende N, Mata R. Randomised, double-blind, placebo-controlled clinical trial for evaluating the efficacy of intracoronary injection of autologous bone marrow mononuclear cells in the improvement of the ventricular function in patients with idiopathic dilated myocardiopathy: a study protocol. BMC Cardiovasc Disord. 2019 Aug 22;19(1):203. doi: 10.1186/s12872-019-1182-4.
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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.
Miguel Romero Moreno, MD, PhD principal investigator
Hospital General Universitario Reina Sofía de Murcia
Manuel Sobrino Marquez, MD principal investigator
Hospitales Universitarios Virgen del Rocío
Luis A Iñigo García, MD principal investigator
Hospital Costa del Sol
9 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Infusion of autologous mononuclear bone marrow cells in people with Idiopathic Dilated Cardiomyopathy, run by Andalusian Initiative for Advanced Therapies - Fundación Pública Andaluza Progreso y Salud at 9 sites 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 10 inclusion and 11 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: stopped early, as of the sponsor's last update on 18 Sep 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites 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. Some participants in this study may receive a placebo. 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?
The study was stopped early (reason given: Low recruitment rate, bibliographic evidence of lack of efficacy). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in ventricular function measured angiographically. (time frame: 24 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02033278, which also lists 1 linked publication.
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 18 Sep 2020.