This trial is evaluating whether GPH101 Drug Product will improve 6 primary outcomes and 22 secondary outcomes in patients with Anemia, Sickle Cell. Measurement will happen over the course of 42 days post-infusion.
This trial requires 15 total participants across 2 different treatment groups
This trial involves 2 different treatments. GPH101 Drug Product is the primary treatment being studied. Participants will all receive the same treatment. There is no placebo group. The treatments being tested are in Phase 1 & 2 and have already been tested with other people.
Sickle cell anemia affects a growing number of individuals in the US. The average age when individuals get sickle cell anemia is approximately twenty-one years.
There is growing evidence, both animal and human, that sickle cell disease (SCD) is a risk factor for retinal necrosis. In the USA between 5 and 10% of individuals with SCD are classified as having retinopathy. The etiology of retinopathy in SCD patients is not well documented. There are several plausible mechanisms for retinal infarction, including, but not limited to, vaso-occluding sickle cell trait, hyperviscosity due to globin gene polymorphism and microvascular alterations associated with sickle cell trait, hyperviscosity due to globin gene polymorphism and sickle crisis.
Many of the symptoms felt by patients are similar to those of anemia and sickle cell, so the main sign that identifies anemia is pallor. Anemia and sickle cell are both highly prevalent diseases in the USA. The most significant symptom of anemia is pallor, and is a common sign of anemia and sickle cell disease.
Sickle-cell anemia has multiple causes including environmental factors, as well as hereditary or genetic factors. It is an uncommon cause in people outside Africa. A complete blood count showing anemia and low red blood cell levels is always indicated. There is also a strong association between sickle cell and stroke and the presence of anemia is an important consideration in these patients with sickle cell anemia if it is found that anemia has contributed to their stroke.
Approximately one half million US children have anemia, SCD, or both, which is a public health concern given the high incidence and mortality of these conditions. The rate of anemia is higher for black children <5 years of age than for white children and both black and white children <5 years of age have higher rates of SCD than do white children and adults. In a recent study, nearly 8% of all US children had evidence of anemia. Anemia among the sickle cell trait, a much more common variant of alpha thalassemia, is more prevalent in the US black population.
The most preferred treatments are ferrous iron or ferrofolic supplement, erythropoietin and deferoxamine injections. The most preferred therapies for sickle cell anemia include ferrous iron and iron chelators.\n\nThis list is based on the most commonly used treatments for common diseases. It cannot be taken as a list of the most appropriate treatments, as the most appropriate treatment depends on the patient's specific needs. Many patients may not be fully covered by this list. Patients should consult a medical professional for advice on specific diseases.\n\nThe list below has been modified from the "International Classification of Diseases for Oncology" for ICD-O-3.
Patients with SCD should be offered iron supplementation, as it has been shown to improve outcomes when used after surgery, and this may be justified by its cost-efficient nature.
In summary, the safety of G-HP1 will be further assessed in this group of patients in order to expand the use of this effective and safe drug in human beings.
These data suggest that Gph101 alone may have an effect on hematopoietic cells. No data on its potential to function alone or in combination with other anti-cancer agents were found.
Data from a recent study highlights that the effect of anemia in children admitted to a paediatric PICU is substantial. We also show that in our case the use of recombinant human erythropoietin for anemia is a valuable treatment for sickle cell disease.
Side effects of the Gph101 drug product and other DMSO-based gene therapy approaches can be minimized by optimizing dose and dosing frequency. Also, by selecting drug-specific bioassay, preclinical and clinical results obtained to date appear more predictive of the corresponding clinical trial findings.
There have been many publications from researchers who worked with gph101. However, as the studies are predominantly short term, limited, and do not have placebo controls and they are observational rather than randomized trials, they do not meet the standard required by the editorials guidelines governing the conduct of clinical trials as required by ICMJE. These limitations preclude the editorials from giving them full endorsement, and are consistent with the editors own conclusion in the same publication that all future research should be done using placebo-controlled, randomized studies. In fact, there is some reason to believe that the trials of gph101 published so far are underpowered; they were generally too small, with only one group being as large as 25 participants in each treatment arm.