Ireland
--:--IST
Latest
Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide The €100K Reality Check: Why a Six-Figure Pharma Salary in Ireland Feels Different Than in Switzerland or Germany Ireland's €93.8 Billion Non-EU Pharma Export Engine: 2025 Eurostat Trade Data and Economic Impact Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide The €100K Reality Check: Why a Six-Figure Pharma Salary in Ireland Feels Different Than in Switzerland or Germany Ireland's €93.8 Billion Non-EU Pharma Export Engine: 2025 Eurostat Trade Data and Economic Impact
Clinical Trials in the USA / NCT04819841
Recruiting Phase 1/2

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

NCT04819841 · tracked via the Priya Life Science USA tracker
Sponsor
Kamau Therapeutics
Phase
Phase 1/2
Started
2021-11-15
Last updated
2026-07-02

Condition(s) studied

Sickle Cell Disease

Investigational drug(s) / intervention(s)

nula-cel Drug Product

nula-cel Drug Product: nula-cel is administered via IV infusion following a myeloablative conditioning regimen

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.

Eligibility

Sex
ALL
Min age
12 Years
Max age
50 Years
Healthy volunteers
No
Inclusion Criteria: * ≥12 to ≤ 40 years * Severe disease, as defined by having experienced at least one of the following SCD-related events despite appropriate supportive care measures: * recurrent severe VOC (≥ 4 episodes in the preceding 2 years) * ACS (≥ 2 episodes in the prior 2 years with at least one episode in the past year) * Lansky/Karnofsky performance status of ≥ 80 Exclusion Criteria: * Available 10/10 HLA-matched sibling donor * Prior HSCT or gene therapy * Prior or current malignancy or myeloproliferative or a significant coagulation or immunodeficiency disorder * Clinically significant and active bacterial, viral, fungal or parasitic infection * Pregnancy or breastfeeding in a postpartum female * Presence of a chromosomal abnormality/mutation that may put the participant at an increased risk for MDS or AML per investigator's judgment

Primary outcome measure(s)

  • Proportion of patients who reach neutrophil engraftment — 42 days post-infusion
  • Incidence rate of treatment-related mortality — 100 days post-infusion
  • Incidence rate of treatment-related mortality — 12 months post-infusion
  • Overall survival — 24 months post-infusion
  • Frequency and severity of AEs/SAEs — 24 months post-infusion

Trial sites (6)

FacilityCityRegionStatus
Children's Hospital Los Angeles Los Angeles California Recruiting
Lucile Packard Children's Hospital Palo Alto California Recruiting
Washington University St Louis Missouri Recruiting
Columbia University Irving Medical Center New York New York Recruiting
Memorial Sloan Kettering New York New York Recruiting
Nationwide Children's Hospital Columbus Ohio Recruiting

Other trials for the same condition

A Phase I/II Study of ITU512 in Healthy Participants and Patients With Sickle Cell Disease Recruiting
A Study to Investigate the Effect of Mitapivat on Transfusion Burden in Subjects With Sickle Cell Disease (SCD) Recruiting
Long-term Follow-up (LTFU) of Patients Treated With Genome-edited Autologous Hematopoietic Stem and Progenitor Cells (HSPC) Active, not recruiting
A Phase 1b, Open-Label Study of DISC-3405 in Participants With Sickle Cell Disease (SCD) Recruiting
A Study to Evaluate How Well Etavopivat Works in People With Sickle Cell Disease Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT04819841 on ClinicalTrials.gov ↗ ← All trials in the USA