GPH101 Drug Product for Anemia, Sickle Cell

Phase-Based Estimates
1
Effectiveness
1
Safety
University of Alabama at Birmingham, Birmingham, AL
Anemia, Sickle Cell+1 More
GPH101 Drug Product - Genetic
Eligibility
< 65
All Sexes
Eligible conditions
Anemia, Sickle Cell

Study Summary

This study is evaluating whether a drug may help reduce pain for individuals with sickle cell disease.

See full description

Eligible Conditions

  • Anemia, Sickle Cell
  • Sickle Cell Disease (SCD)

Treatment Effectiveness

Effectiveness Estimate

1 of 3

Compared to trials

Study Objectives

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.

Month 24
Evaluation of globin chain expression
12 months post-infusion
Incidence rate of treatment-related mortality
Transplant-related mortality
24 months post-infusion
Bone marrow HSPC or peripheral myeloid gene correction marking
Change from baseline in SCD-indicated lab parameters, including but not limited to bilirubin, reticulocyte counts, erythropoietin, LDH, eGFR, haptoglobin, CRP
Change from baseline in transcranial Doppler (TCD) velocity in applicable participants with available TCD assessments
Frequency and severity of AEs/SAEs
Overall survival
Total HbA without disease-indicated transfusion support for ≥3 continuous months
Total HbS without disease-indicated transfusion support for ≥3 continuous months
Total Hgb without disease-indicated transfusion support for ≥3 continuous months
42 days post-infusion
Proportion of patients who reach neutrophil engraftment
Success of HSC engraftment
Month 18
Proportion of participants with complete resolution of severe vaso-occlusive crises (sVOCs)
Month 24
Incidence rate of any sVOCs
Month 24
Assessment of vaso-occlusive crises (VOC), Acute Chest Syndrome (ACS) episodes, and other SCD-related events compared to the 2 years prior to enrollment as documented by a visit to a medical facility
Change in annualized packed red blood cell (pRBC) transfusion requirements (volume and frequency) for SCD indications
Evaluation of HbA as a fraction of total Hb
Evaluation of HbS as a fraction of total Hb
Evaluation of HbS as a percentage of total Hgb
Evaluation of adult Hgb as a percentage of total Hgb
Evaluation of gene correction levels in peripheral myeloid cells
Evaluation of globin chain expression compared to baseline
Evaluation of peripheral myeloid gene correction levels
Proportion of participants achieving HbS <50% for at least 3 months
Time to neutrophil engraftment
Time to platelet engraftment
Total Hgb without disease-indicated transfusion support

Trial Safety

Safety Estimate

1 of 3

Compared to trials

Trial Design

2 Treatment Groups

Control
GPH101 Drug Product

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.

GPH101 Drug Product
Genetic
GPH101 Drug Product is a human autologous CRISPR-Cas9 edited and sickle mutation-corrected HSPC product.
ControlNo treatment in the control group

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 1, 2, 3, 6, 9, 12, 15, 18, 21, and 24 months post-infusion
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly 1, 2, 3, 6, 9, 12, 15, 18, 21, and 24 months post-infusion for reporting.

Closest Location

University of Alabama at Birmingham - Birmingham, AL

Eligibility Criteria

This trial is for patients born any sex aged 65 and younger. You must have received 1 prior treatment for Anemia, Sickle Cell or the other condition listed above. There are 4 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Patient age 12 to 40 years. show original
You have had 4 or more episodes of severe VOC in the preceding 2 years. show original
ACS (≥ 2 episodes in the prior 2 years with at least one episode in the past year)
Lansky/Karnofsky performance status of ≥ 80

Patient Q&A Section

Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the average age someone gets anemia, sickle cell?

Add answer

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.

Unverified Answer

What is anemia, sickle cell?

Add answer

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.

Unverified Answer

What are the signs of anemia, sickle cell?

Add answer

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.

Unverified Answer

What causes anemia, sickle cell?

Add answer

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.

Unverified Answer

How many people get anemia, sickle cell a year in the United States?

Add answer

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.

Unverified Answer

What are common treatments for anemia, sickle cell?

Add answer

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.

Unverified Answer

Can anemia, sickle cell be cured?

Add answer

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.

Unverified Answer

Is gph101 drug product safe for people?

Add answer

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.

Unverified Answer

Is gph101 drug product typically used in combination with any other treatments?

Add answer

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.

Unverified Answer

How serious can anemia, sickle cell be?

Add answer

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.

Unverified Answer

What are the common side effects of gph101 drug product?

Add answer

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.

Unverified Answer

Have there been other clinical trials involving gph101 drug product?

Add answer

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.

Unverified Answer
See if you qualify for this trial
Get access to this novel treatment for Anemia, Sickle Cell by sharing your contact details with the study coordinator.