Pulmonary Arterial Hypertension or PAH is a progressive condition for which there is no cure.
Even with substantial pharmacologic advances in the modern treatment era, survival still
remains unacceptably poor, as reported in large PAH registries. Preclinical studies suggest
that the administration of allogeneic CDCs have the potential to reduce adverse arteriolar
remodeling in PAH which was the basis for the approved investigational new drug (IND). The
use of CDCs as an adjunctive therapy in patients comprising 4 sub-groups of patients with PAH
in which inflammation and immune dysfunction are key pathophysiologic drivers of PAH.
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.
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.
The person who is responsible for the scientific and technical direction of the entire clinical study.
Category of organization(s) involved as sponsor (and collaborator) supporting the trial.
The disease, disorder, syndrome, illness, or injury that is being studied.
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:
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
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.
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Maria Thottam, BS