Sickle Cell Disease study: CRISPR_SCD001
Official title Transplantation of Clustered Regularly Interspaced Short Palindromic Repeats Modified Hematopoietic Progenitor Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease
ClinicalTrials.gov ID: NCT04774536
What this study is testing
What is CRISPR_SCD001?
This is an investigational treatment being studied as a potential therapy for severe Sickle Cell Disease. It is designed to use gene editing technology to modify blood-forming stem cells before they are infused into the body.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study aims to see if a one-time infusion of gene-edited stem cells is safe and how well it works for people with severe sickle cell disease.
- Who it's for
- The study is looking for people aged 12 to 35 who have severe sickle cell disease.
- What's involved
- Participants will receive a single infusion of stem cells that have been modified using a gene-editing tool called CRISPR.
- Time commitment
- The study involves a one-time treatment and follow-up visits at one of two research centers.
- Phase 2: a mid-size study of how well it works
- Which group you join is decided by chance.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 to 35
You may be able to join if
- History of two or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care...
- History of at least 4 severe vaso-occlusive pain events in the 2-year period preceding enrollment despite the institution of supportive care measures...
- 2 or more episodes of splenic sequestration, defined as an acute decrease in hemoglobin by at least 2 g/dL accompanied by splenomegaly in the 2 years...
- Recurrent priapism (episodes lasting at least 4 hours at least twice in the last 12 months or 3 times in the last 24 months before enrollment)...
- Any episode of hepatic sequestration defined as right upper quadrant pain, hepatomegaly, and rapidly decreasing hemoglobin level with hepatic...
You likely can't join if
- Participants with uncontrolled bacterial, viral or fungal infection in the 6 weeks before enrollment (currently taking medication with evidence of...
- Participants with evidence of HIV infection or seropositivity for HIV or active hepatitis B or C.
- Participants who have received a Hematopoietic Cell Transplant (HCT)
- Participants who have received a solid organ transplant.
- Participants who have participated in another clinical trial in which the participant received an investigational or off-label use of a drug or...
- Females who are pregnant or breast feeding.
See the full eligibility criteria
- History of two or more episodes of acute chest syndrome (ACS) in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. asthma therapy and/or hydroxyurea)
- History of at least 4 severe vaso-occlusive pain events in the 2-year period preceding enrollment despite the institution of supportive care measures (i.e. a pain management plan and/or treatment with hydroxyurea)
- 2 or more episodes of splenic sequestration, defined as an acute decrease in hemoglobin by at least 2 g/dL accompanied by splenomegaly in the 2 years before enrollment.
- Recurrent priapism (episodes lasting at least 4 hours at least twice in the last 12 months or 3 times in the last 24 months before enrollment) recalcitrant to medical treatment
- Any episode of hepatic sequestration defined as right upper quadrant pain, hepatomegaly, and rapidly decreasing hemoglobin level with hepatic dysfunction within the 2 years before enrollment.
- Leg ulcer recalcitrant to treatment within 2 years prior to enrollment.
- Karnofsky performance score ≥60.
- Cardiac function: Left ventricular ejection fraction (LVEF) \>40%; or LV shortening fraction \> 26% by cardiac echocardiogram or by (multiple-gated acquisition) MUGA scan.
- Pulmonary function: Pulse oximetry with a baseline O2 saturation of ≥85% and diffusion capacity of lung for carbon monoxide(DLCO) \> 40% (corrected for hemoglobin).
- Renal function: Serum creatinine ≤ 1.5 x upper limit of normal for age and estimated or measured creatinine clearance ≥ 70 mL/min/1.73 m2.
- Hepatic function: i. Serum conjugated (direct) bilirubin \ 12 and negative elastography imaging are eligible if a clinically indicated liver biopsy shows no significant fibrosis.
- Baseline prothrombin time or partial thromboplastin time \<1.5 ULN except if receiving a prophylactic anticoagulant which causes an elevated prothrombin or partial thromboplastin time.
- Written informed consent or assent obtained from subject or subject's legal representative and ability for subject to comply with the requirements of the study.
- Participants with uncontrolled bacterial, viral or fungal infection in the 6 weeks before enrollment (currently taking medication with evidence of progression of clinical symptoms or radiologic findings).
- Participants with evidence of HIV infection or seropositivity for HIV or active hepatitis B or C.
- Participants who have received a Hematopoietic Cell Transplant (HCT)
- Participants who have received a solid organ transplant.
- Participants who have participated in another clinical trial in which the participant received an investigational or off-label use of a drug or device within 3 months prior to enrollment.
- Females who are pregnant or breast feeding.
- Females of child bearing potential (to include all female participants \> 10 years of age, unless postmenopausal for a minimum of 1 year before the time of consent or surgically sterilized) who do not agree to practice...
- Males (even if surgically sterilized) who do not agree to practice effective barrier contraception, or who do not agree to practice true abstinence from the time of signing informed consent through 12 months post-stem...
- Participants who have had a stroke OR who are receiving red blood cell (RBC) transfusions to prevent primary stroke or silent cerebral infarction.
- Patients who have a suitable human leukocyte antigen identical (HLA-ID) sibling donor willing and able to donate bone marrow.
- Presence of a condition or abnormality that in the opinion of the Investigator would compromise the safety of the patient or the quality of the data.
- Any non-homozygous sickle hemoglobin (HbSS) genotype of SCD
- diagnosis of myelodysplastic syndrome (MDS) based on morphology and/or cytogenetics (based on WHO definitions) OR
- any evidence of a pathogenic clonal variant in any candidate gene detected by a standard, licensed next-generation sequencing clinical assay for gene mutations associated with hematological malignancies.
- The participant has an identified pathogenic mutation associated with myeloid malignancy as identified by RHP or a variant of unknown significance (VUS) judged to be pathogenic for myeloid malignancy as determined by...
The study team makes the final eligibility decision.
Where it is enrolling
- Davis, California, United States
- Los Angeles, California, United States
- Oakland, California, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 12 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Davis, California, United States; Los Angeles, California, United States; Oakland, California, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.