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RECRUITING Phase 1

Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease

NCT04819841 · View on ClinicalTrials.gov ↗

Study Summary

This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynamic data.

Conditions Studied

Interventions

  • GENETIC nula-cel Drug Product

Study Locations (4)

California

  • Children's Hospital Los Angeles — Los Angeles
  • Lucile Packard Children's Hospital — Palo Alto

Missouri

  • Washington University — St Louis

Ohio

  • Nationwide Children's Hospital — Columbus

Trial Details

FieldValue
Enrollment Target 15 participants
Start Date 2021-11-15
Est. Completion 2028-12-31
Phase Phase 1

Sponsor

Kamau Therapeutics

32 total trials

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Full Details on ClinicalTrials.gov ↗

What the Registry Record Tells You About NCT04819841

The ClinicalTrials.gov registry entry for NCT04819841 describes a study currently listed as recruiting. It is categorized as Phase 1, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 15 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Kamau Therapeutics, which has 32 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.

The record links to 1 condition, with Sickle Cell Disease appearing as the primary indexed condition, and to 1 intervention — of which nula-cel Drug Product is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.

Geographic footprint matters for practical reasons: NCT04819841 reports 4 study locations spanning 3 distinct geographic areas — top geographies include California, Missouri, Ohio. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.

Frequently Asked Questions

What is clinical trial NCT04819841 about?

NCT04819841 is a clinical study titled "Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease". This study is a first-in-human, single-arm, open-label Phase I/II study of nula-cel in approximately 15 participants, diagnosed with severe Sickle Cell Disease. The primary objective is to evaluate safety of the treatment in this patient population, as well as preliminary efficacy and pharmacodynami...

What is the current status of trial NCT04819841?

This trial is currently recruiting. It is a Phase 1 study. The enrollment target is 15 participants. The study started on 2021-11-15. Estimated completion is 2028-12-31.

What conditions does trial NCT04819841 study?

This clinical trial studies the following conditions: Sickle Cell Disease. These conditions were identified from the trial registry and reflect the primary focus areas of the research.

What interventions are being tested in trial NCT04819841?

The interventions under investigation include: nula-cel Drug Product (GENETIC). Each intervention is being evaluated for safety and efficacy as part of this clinical study.

Who is sponsoring clinical trial NCT04819841?

This trial is sponsored by Kamau Therapeutics, which has 32 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.

Where is trial NCT04819841 being conducted?

This trial has 4 study locations across California, Missouri, Ohio. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.

Related

Data sourced from official U.S. government datasets. See our methodology for details. Retrieved and formatted by PlainTrial Editorial