NCT04819841

Brief 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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_1

Timeline
32mo left

Started Nov 2021

Longer than P75 for phase_1

Geographic Reach
1 country

4 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress63%
Nov 2021Dec 2028

First Submitted

Initial submission to the registry

March 24, 2021

Completed
5 days until next milestone

First Posted

Study publicly available on registry

March 29, 2021

Completed
8 months until next milestone

Study Start

First participant enrolled

November 15, 2021

Completed
5.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2026

Expected
2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2028

Last Updated

March 11, 2026

Status Verified

March 1, 2026

Enrollment Period

5.1 years

First QC Date

March 24, 2021

Last Update Submit

March 9, 2026

Conditions

Sickle Cell Disease

Keywords

sickle cell diseasesickle cell anemiagene correctiongene therapyCRISPR

Outcome Measures

Primary Outcomes (5)

  • 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

Secondary Outcomes (11)

  • Time to neutrophil engraftment

    through study completion, up to 24 months post-infusion

  • Time to platelet engraftment

    through study completion, up to 24 months post-infusion

  • Evaluation of gene correction levels in peripheral myeloid cells

    through study completion, up to 24 months post-infusion

  • Evaluation of adult Hgb as a percentage of total Hgb

    through study completion, up to 24 months post-infusion

  • Evaluation of HbS as a percentage of total Hgb

    through study completion, up to 24 months post-infusion

  • +6 more secondary outcomes

Study Arms (1)

nula-cel Drug Product

EXPERIMENTAL

nula-cel Drug Product is a human autologous CRISPR-Cas9 edited and sickle mutation-corrected HSPC product.

Genetic: nula-cel Drug Product

Interventions

nula-cel Drug ProductGENETIC

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

nula-cel Drug Product

Eligibility Criteria

Age12 Years - 40 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • ≥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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (4)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

Lucile Packard Children's Hospital

Palo Alto, California, 94304, United States

RECRUITING

Washington University

St Louis, Missouri, 63110, United States

RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

RECRUITING

MeSH Terms

Conditions

Anemia, Sickle Cell

Condition Hierarchy (Ancestors)

Anemia, Hemolytic, CongenitalAnemia, HemolyticAnemiaHematologic DiseasesHemic and Lymphatic DiseasesHemoglobinopathiesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Mathew Porteus, MD, PhD

    Kamau Therapeutics

    STUDY DIRECTOR

Central Study Contacts

Restore Clinical Study Support

CONTACT

650-442-2283RestoreStudySupport@kamautx.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 24, 2021

First Posted

March 29, 2021

Study Start

November 15, 2021

Primary Completion (Estimated)

December 31, 2026

Study Completion (Estimated)

December 31, 2028

Last Updated

March 11, 2026

Record last verified: 2026-03

Locations

US(4)