EDIT-301 for Hemoglobinopathies

Phase-Based Progress Estimates
UCSF Benioff Children's Hospital, Oakland, CA
Hemoglobinopathies+2 More
EDIT-301 - Genetic
18 - 65
All Sexes
What conditions do you have?

Study Summary

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

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Eligible Conditions

  • Hemoglobinopathies
  • Sickle Cell Disease (SCD)

Treatment Effectiveness

Study Objectives

This trial is evaluating whether EDIT-301 will improve 1 primary outcome and 7 secondary outcomes in patients with Hemoglobinopathies. Measurement will happen over the course of up to 2 years post EDIT-301 infusion.

Year 2
Annualized number of units of pRBC transfused for SCD-related indications
Change from baseline in annualized rate of hospitalization for severe VOE
Change from baseline in annualized rate of severe VOE by at least 75%
Change from baseline in annualized rate of severe VOE by at least 90%
Complete resolution of severe VOE
Difference (pre-treatment versus post-treatment) in the rates of severe vaso-occlusive events (VOEs) requiring medical attention.
Proportion of subjects with mean Hb ≥ 10 g/dL starting ≥ 60 days after last packed red blood cell (pRBC) transfusion compared with preconditioning Baseline
Proportion of subjects with mean HbF > 20% (HbF/Hb) compared with pre-conditioning Baseline

Trial Safety

Trial Design

1 Treatment Group

1 of 1
Experimental Treatment

This trial requires 40 total participants across 1 different treatment group

This trial involves a single treatment. EDIT-301 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.

EDIT-301 (autologous gene edited (CD)34+ hematopoietic stem cells) will be administered as a one-time intravenous infusion.

Trial Logistics

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: up to 2 years post edit-301 infusion
This trial has the following approximate timeline: 3 weeks for initial screening, variable treatment timelines, and roughly up to 2 years post edit-301 infusion for reporting.

Closest Location

UCSF Benioff Children's Hospital - Oakland, CA

Eligibility Criteria

This trial is for patients born any sex between 18 and 65 years old. There are 4 eligibility criteria to participate in this trial as listed below.

Mark “yes” if the following statements are true for you:
Documented severe SCD genotype (βS/βS, βS/β0, or βS/β+)
The person has had two severe vaso-occlusive crises in the past two years that required medical attention, despite using hydroxyurea or other supportive care measures. show original
% The person has a good performance status and is able to carry out most normal activities. show original

Patient Q&A Section

What is hemoglobinopathies?

"Hemoglobinopathies can be inherited from parent’s generation to offspring generation. Hemoglobinopathies are an inherited diseases. Genetic diseases can be passed down through family generations. The most common hemoglobinopathies in the world are sickle-cell anaemia, thalassemia, and β-thalassemia"

"Eudonia fuscella\n\nEudonia fuscella is a species of moth in the family Crambidae described by Jacob Hübner in 1805. Recorded only from Honshu and Hokkaido, Japan.\n\nThe length of the forewings is 6.9–9 mm." - Anonymous Online Contributor

Unverified Answer

What are the signs of hemoglobinopathies?

"Hemoglobinopathies manifest themselves by symptoms including the following: fatigue, shortness of breath or palpitations, headache, visual disturbances, weakness or numbness on the extremities, cold temperature sensation, blurred vision, ringing in the ears; and seizures. Furthermore, thalassemia can present itself as splenomegaly, or in pediatric patients as enlargement of the liver. Finally, sickle cell disease can present as fever, chest pain, breathlessness (dyspnea), or cough." - Anonymous Online Contributor

Unverified Answer

What are common treatments for hemoglobinopathies?

"Lifelong prevention of thalassemia and sickle cell disease with blood transfusions is the most common treatment for hemoglobinopathies. In some populations thalassemia and sickle cell disease account for most hospital admissions. In other countries where sickle cells only occur in a small percentage of persons with mild hemoglobinopathies, or who are at high risk of exposure to blood from an infected person, or other conditions have resulted in an increase in hemoglobinopathies, blood transfusion is less important. In some low-income countries, alpha-thalassemia is the primary condition." - Anonymous Online Contributor

Unverified Answer

What causes hemoglobinopathies?

"Although no single mutation causes all forms of HbS, common associations of HbS with hemoglobinopathies, including thalassemia and thalassemia-hemoglobinopathy syndromes, are also present. This may arise from the increased propensity for mutations to occur due to increased demands for red blood cells in persons with HbS as compared with normal individuals. Additionally, individuals with abnormal red blood cell structure or shape are more susceptible to hemolysis and accumulation of hemosiderin, causing increased iron availability." - Anonymous Online Contributor

Unverified Answer

Can hemoglobinopathies be cured?

"Although cure is possible for alpha-thalassemia and alpha-thalassemia/beta-thalassemia, some forms of beta-thalassemia remain refractory. The search for the genetic cause of beta-thalassemia and effective treatment continues to be a goal." - Anonymous Online Contributor

Unverified Answer

How many people get hemoglobinopathies a year in the United States?

"The total number of patients in the United States diagnosed with abnormal hemoglobin proteins is likely in the hundreds of thousands. However, it is not clear which patients get abnormal hemoglobin proteins. Hemoglobinopathies are very common, with the greatest prevalence in Southeast Asia, Africa, and Central and South America." - Anonymous Online Contributor

Unverified Answer

Have there been any new discoveries for treating hemoglobinopathies?

"Hemoglobinopathies have remained relatively unchanged over recent years due to advances in treatment approaches and gene therapy. Currently, there is no cure for the majority of hemoglobinopathies and it is important to maintain hematology services and maintain surveillance for possible complications with any treatment. There have been improvements in the management of thalassemias and sickle cell disease as well as in the detection of conditions such as hereditary spherocytosis." - Anonymous Online Contributor

Unverified Answer

How does edit-301 work?

"The data supports our hypothesis that the ability of Edit-301 to reduce the transfusion requirements in the anemic population appears to be related to its mechanism of action." - Anonymous Online Contributor

Unverified Answer

What is the primary cause of hemoglobinopathies?

"Current trends in the genetic background of anemia and hemoglobinopathies are consistent with progressive reduction in maternal-fetal hemosiderin as a result of the reduced use of iron supplements and more frequent screening for iron deficiency in utero." - Anonymous Online Contributor

Unverified Answer

Have there been other clinical trials involving edit-301?

"Data from a recent study demonstrate that the efficacy of edit-301 was not substantially different from other hematodysplasia growth factor treatments in the relapsed and refractory MDS or CMML patient population." - Anonymous Online Contributor

Unverified Answer

Does hemoglobinopathies run in families?

"Data from a recent study suggest that some phenotypically mild mutations are responsible for the increased incidence of anemia in our population, and that some of these mutations contribute to anemia by the alteration of the functional properties of RBCs. Therefore, a high index of suspicion in the search for anemia in individuals with hemoglobinopathies is needed." - Anonymous Online Contributor

Unverified Answer

Who should consider clinical trials for hemoglobinopathies?

"Hemoglobinopathies are a very common cause of pediatric anemia. Most patients with hemoglobinopathies will not get tested for these disorders in the United States. There is a low understanding about the potential risk to these patients participating in a clinical trial." - Anonymous Online Contributor

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