Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease

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About the Study

This study seeks to understand the safety of a gene editing therapy called CRISPR_SCD001 and if this treatment can help people with severe sickle cell disease get better. 

Study doctors will use a ‘gene changing’ tool called CRISPR. The therapy changes the sickle gene mutation in a patient’s stem cells to a non-sickle gene that makes healthy hemoglobin. These gene-edited stem cells will be given back to the patient. 

Patients will undergo high-dose chemotherapy to make room for the gene-edited stem cells to grow. They are then transplanted back into the patient. 

This therapy has the potential to improve the health of individuals with sickle cell disease.

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Participation information

Countries

2 United States of America sites

Age

12-35 years

Gender

All

Genotypes

SCD type SS

Study Information

Study type

Clinical

Interventions

CRISPR_SCD001

Phase

1/2

Compensation

A monetary stipend will be provided per visit to help offset housing and lodging costs. Please see “About the Study” details for information on Patient Support Resources.

Am I Eligible?

Requirements

You may be eligible to participate in this study if you:
  1. Have sickle cell disease (HbSS).
  2. Are between the ages of 12 and 35 years old.
  3. Have experienced severe sickle cell symptoms, such as:
    1. At least two episodes of acute chest syndrome (ACS) in the last two years.
    2. At least four severe vaso-occlusive pain events (VOCs) in the last two years that required hospital or emergency room visits
  4. Can care for yourself (you have the ability to perform daily activities)
  5. Have good heart, lung, kidney, and liver function.
  6. Do not have major liver damage from excess iron.
  7. Have written consent from a guardian (if under 18 years of age).

Other inclusions may apply. Please visit this site and complete the eligibility survey to see if you are a good fit: https://ucsf.co1.qualtrics.com/jfe/form/SV_3JFOMmGHAjv6dJY

Restrictions

You can't participate in this study if you:
  1. Have had a stroke or are getting regular blood transfusions to prevent a stroke or other brain issues.
  2. Have or had a serious infection (bacterial, viral, or fungal) in the last 6 weeks leading up to enrolling in the study, especially if you were taking medication but the infection was getting worse.
  3. Have evidence of HIV infection or active hepatitis B or C (liver infections).
  4. Have received a bone marrow transplant (also known as a Hematopoietic Cell Transplant (HCT)).
  5. Have received a solid organ transplant (like a kidney or liver transplant).
  6. Have participated in another clinical trial with an investigational drug or off-label use of a drug or device in the last 3 months leading up to enrolling in the study.
  7. Are pregnant or breastfeeding.
  8. Do not agree to practice effective methods of contraception or practice abstinence from the time of signing through 12 months post-stem cell infusion.
  9. Have a sickle cell disease genotype other than HbSS.
  10. Have a brother or sister who is a perfect match for a bone marrow transplant.
  11. Have any other medical condition that, in the doctor’s opinion, would make it unsafe for you to participate or could affect the study results.

Other exclusions may apply. Please visit this site and complete the eligibility survey to see if you are a good fit: https://ucsf.co1.qualtrics.com/jfe/form/SV_3JFOMmGHAjv6dJY

Study point of contact

Marci Moriarity, RN
510-428-3885 ext. 5396
[email protected]
Cyrus Bascon
510-428-3885 ext. 6953
[email protected]

Study Sites

  • Los Angeles, California, United States of America, University of California, Los Angeles
  • Oakland, California, United States of America, UCSF Benioff Children’s Hospital
Enroll in this Study
Last updated October 25, 2024