Accepts Healthy Volunteers
Healthy volunteers are participants who do not have a disease or condition, or related conditions or symptoms
An interventional clinical study is where participants are assigned to receive one or more interventions (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes.
An observational clinical study is where participants identified as belonging to study groups are assessed for biomedical or health outcomes.
Searching Both is inclusive of interventional and observational studies.
|Eligible Ages||18 Years - 75 Years|
- - Confirmed clinical diagnosis of IPAH, HPAH, PAH-CTD, PAH-HIV.
- - NYHA Functional Class: II or III.
- - 6MWD > 150 m.
- - Able to maintain O2 saturation at rest ≥ 90% (with or without supplemental O2).
- - The subjects must be on PAH-specific therapies for at least 4 months and on a stable dose for at least 4 weeks prior to enrollment into study.
- - All patients with PAH-HIV must be on a stable and effective HAART combination regimen.
- - Pulmonary capillary wedge pressure (PCWP) or LVEDP < 15 mm Hg.
- - Age: 18 -75 years.
- - Ability to provide informed consent and follow-up with protocol procedures.
- - Diagnosis of PAH other than IPAH, HPAH, PAH-CTD or PAH-HIV.
- - Right atrial pressure > 20 mmHg as measured by right heart catheterization (RHC) on day of pre-infusion.
- - History of clinically-significant coronary artery disease, including myocardial infarction, coronary stent placement or coronary artery bypass surgery within the previous 5 years, LV dysfunction.
- - History or demonstration of significant ventricular tachy-arrhythmias or conduction abnormalities.
- - Significant interstitial lung disease (on imaging and PFTs; FVC: < 60%; - Chronic thromboembolic pulmonary hypertension (CTEPH) - Estimated glomerular filtration rate (GFR) ≤ 50 mL/min.
- - Active uncontrolled infection.
- - Non-pulmonary vascular disease with life expectancy of < 3 years.
- - Hypersensitivity to contrast agents.
- - Active allergic reactions.
- - History of previous stem cell therapy.
- - Participation in an on-going protocol studying an experimental drug or device.
- - Current alcohol or drug abuse because of anticipated difficulty in complying with protocol-related procedures.
- - Pregnant/nursing women as well as men and women of child-bearing potential without use of active and highly reliable contraception.
- - Known history of viral hepatitis.
- - Abnormal liver function (transaminases > 3 times the upper reference range; total bilirubin > 2 times the upper reference range without a reversible, identifiable cause.
- - Evidence of tumor on screening of chest/abdominal/pelvic (body) CT scan.
- - History of malignancy within the last 5 years, except for resected skin basal cell or squamous cell carcinoma, treated cervical dysplasia or treated in-situ cervical cancer grade 1.
- - Any prior organ transplant.
- - Being actively listed for, or under active consideration for, an organ transplant of any kind, including lung transplantation.
- - Known hypersensitivity to bovine products.
- - Known hypersensitivity to dimethyl sulfoxide (DMSO) - Any condition or treatment which (in the opinion of investigator), places the patient at an unacceptable risk if enrolled.
- - Patients with PAH-HIV will be excluded with any of the following clinical conditions: - CD4 T-cell count < 200 /mm3 within 90 days prior to screening.
- - A detectable viral load within 90 days prior to screening.
- - Active opportunistic infections within 90 days prior to screening.
- - Changes in antiretroviral regimen within 90 days prior to screening.
- - Significant anemia or a falling Hb would make patient ineligible.
This trial id was obtained from ClinicalTrials.gov, a service of the U.S. National Institutes of Health, providing information on publicly and privately supported clinical studies of human participants with locations in all 50 States and in 196 countries.
Phase 1: Studies that emphasize safety and how the drug is metabolized and excreted in humans.
Phase 2: Studies that gather preliminary data on effectiveness (whether the drug works in people who have a certain disease or condition) and additional safety data.
Phase 3: Studies that gather more information about safety and effectiveness by studying different populations and different dosages and by using the drug in combination with other drugs.
Phase 4: Studies occurring after FDA has approved a drug for marketing, efficacy, or optimal use.
The sponsor is the organization or person who oversees the clinical study and is responsible for analyzing the study data.
|Cedars-Sinai Medical Center|
The person who is responsible for the scientific and technical direction of the entire clinical study.
|Michael I Lewis, MD|
|Principal Investigator Affiliation||Cedars-Sinai Medical Center|
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
The disease, disorder, syndrome, illness, or injury that is being studied.
|Pulmonary Arterial Hypertension (PAH)|
Patients with IPAH, HPAH, PAH-CTD and PAH-HIV meeting all inclusion and no exclusion criteria will be enrolled. An open label phase 1a study (evaluating dosage and safety) will be conducted. This will followed by a randomized double blind placebo controlled Phase 1b study after Data Safety and Monitoring Board (DSMB) review of the one-month safety data for all the Phase 1a subjects. All patients must have documented PAH diagnosed within the last 5 years and all need to be on stable background PAH specific agents for at least 4 months. The 4 different etiologies of Pulmonary Arterial Hypertension (PAH) included in this (IND) (IPAH, HPAH, PAH-CTD, PAH-HIV) will be diagnosed based on the following: i) clinical features and tests to support a diagnosis of PAH: the diagnosis of PAH requires right heart catheterization (RHC) to confirm a hemodynamic profile compatible with PAH. This includes a mean pulmonary artery pressure (PAP) ≥ than 25 mmHg at rest, with a pulmonary capillary wedge pressure < 15 mmHg. (If slightly elevated, will confirm with LVEDP measure as is our usual standard of care) and pulmonary vascular resistance (PVR) of > 3 Wood units. In addition, there should be no features to suggest other associations for PAH (also included in Group 1) or evidence to suggest PAH owing to left heart disease (Group 2), PH due to lung diseases (Group 3), Chronic thromboembolic pulmonary hypertension (Group 4) or miscellaneous disorders of unclear mechanism ii) clinical features and tests to support a specific designation of each subset of PAH:
- - Idiopathic PAH (IPAH): This is a diagnosis of exclusion in which a firm diagnosis of PAH is made and there are no other etiologies or associations determined that fall into Group 1.
- - Heritable PAH (HPAH): This diagnosis is based on a family history of PAH with or without a documented genetic mutation associated with PAH (such as BMPR2 mutations that are present in up to 75% of HPAH patients).
- - PAH - Connective Tissue Disease (PAH-CTD): These patients have a confirmed diagnosis of PAH as well as firm evidence to support a diagnosis of a connective tissue disease.
- - PAH- Human Immunodeficiency Virus (HIV): Patients will have a firm diagnosis with positive HIV testing (i.e. positive 4th generation immunoassay and positive confirmatory testing such as Western Blot or HIV-1/HIV-2 antibody differentiation immunoassay) and managed by an infectious disease/HIV specialist.
Experimental: Biological: Allogeneic Human Cardiosphere-Derived Cells (CDCs)
The Phase 1a portion (N=6 subjects) consists of an open-label, single-arm, study design - dose escalation. The potentially conducted Phase 1b portion of the study (N=20 subjects) consists of a double-blind, randomized, placebo-controlled study design.
Placebo Comparator: Placebo
The placebo study arm only applies to the Phase Ib portion of the study design. The Phase Ia portion (N=6 subjects) consists of an open-label, single-arm, study design. The potentially conducted Phase Ib portion of the study (N=20 subjects) consists of a double-blind, randomized, placebo-controlled study design with a 1:1 ratio.
Biological: - Allogeneic Human Cardiosphere-Derived Stem Cells
Human Allogeneic Cardiosphere-Derived Cells is a biologic product consisting largely of cells grown from donated human heart muscle tissue
Biological: - Placebo
For use in Phase 1b - Double-blind randomized control portion of the study
Contact a Trial Team
If you are interested in learning more about this trial, find the trial site nearest to your location and contact the site coordinator via email or phone. We also strongly recommend that you consult with your healthcare provider about the trials that may interest you and refer to our terms of service below.