Not medical advice. This website is not medical advice, a diagnosis, a match to a trial, or a recommendation to join a study. Listings are plain-English rewrites of public ClinicalTrials.gov records. Whether a site is still enrolling, and whether you qualify, can only be confirmed by the study team and your clinician. Source registry: ClinicalTrials.gov. Editorial rules: methodology.
PlainTrials
RecruitingPhase 1 / Phase 2interventionalRules rewrite

Phase 1 / Phase 2: testing CRISPR_SCD001 for Sickle Cell Disease

This is an open label, non-randomly assigned, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD). The study will measure the hematopoietic stem cell transplantation (HSCT) using CRISPR/Cas9 edited red blood cells (known as CRISPR\_SCD001 Drug Product).

Official registry: https://clinicaltrials.gov/study/NCT04774536 · Mark Walters, MD · enrollment listed as 9

What this phase means

Phase 2: researchers already know a roughly safe dose (from phase 1). This stage asks whether the treatment actually helps the condition, and watches side effects more closely.

Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

What the registry says happens

If you join, the listed intervention(s) are CRISPR_SCD001. The registry lists allocation as na. This is an open label, non-randomly assigned, 2-center, phase 1/2 trial of a single infusion of sickle allele modified cluster of differentiation (CD34+) hematopoietic stem progenitor cells (HSPCs) in subjects with in subjects ≥12 years old to 35 years old severe Sickle Cell Disease (SCD).

They plan to enroll about 9 people at 3 sites. Taking part typically means taking a study drug (or dummy pill) plus check-ins.

Official title (unrewritten)

Transplantation of CRISPRCas9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease

Interventions: CRISPR_SCD001

Primary outcome (registry wording): Incidence of adverse events and grade 3 or higher serious adverse events, using the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE)

Who it is for (from inclusion criteria)

  • Ages 12 Years to 35 Years
  • Male or female 12.00 - 34.99 years of age (at time of consent) who have one or more of the following:
  • 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
  • Participants must have adequate physical function as measured by all of the following:
  • Karnofsky performance score ≥60

Public study contact (from the registry)

Mark Walters, MD

(510) 428-3374

Mark.Walters@ucsf.edu

Published on ClinicalTrials.gov. Contact them as a possible participant. They decide eligibility.

Sites listed on ClinicalTrials.gov

3 listed

Related recruiting trials in this snapshot

RecruitingNot a traditional drug phaseWashington D.C., District of Columbia
Natural History of Sickle Cell Disease

This study is not a treatment protocol and no experimental treatments are involved. Study participants may be seen as needed for clinical, translational and basic research studies, or as medically indicated.

NCT00081523 · Official ClinicalTrials.gov record · National Heart, Lung, and Blood Institute (NHLBI)

RecruitingNot a traditional drug phaseBethesda, Maryland
Blood Sampling for Research Related to Sickle Cell Disease

This study will collect representative blood samples from healthy children and adults and from children and adults who have unique red blood cell features that are related to sickle cell disease. Sickle cell disease is a blood disease that limits the ability of red blood cells to carry oxygen throughout the body.

NCT00542230 · Official ClinicalTrials.gov record · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)

FAQ about this NCT record

Is NCT04774536 still recruiting?

The sponsor posted status “Recruiting” on ClinicalTrials.gov at last ingest. Sites can pause independently. Confirm with the official record and the listed contact.

What phase is NCT04774536?

Phase 1 / Phase 2. Phase labels are FDA/ICH categories applied to the registry Phase field. They are not a finding that this study succeeded.

Can PlainTrials tell me if I qualify for NCT04774536?

No. The inclusion and exclusion lists on this page are readable extracts of the public eligibility block. Only the study team can screen you.

How to read a ClinicalTrials.gov record · Questions to ask the coordinator · Glossary