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Gene Therapy

nula-cel Drug Product for Sickle Cell Disease (RESTORE Trial)

Phase 1 & 2

Recruiting

Research Sponsored by Kamau Therapeutics

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up over time, from 6 months to study completion, up to 24 months post-infusion

Awards & highlights

RESTORE Trial Summary

This trial is testing a new drug for safety and effectiveness in people with severe sickle cell disease.

Who is the study for?

This trial is for people aged 12-40 with severe Sickle Cell Disease who've had multiple acute chest syndrome episodes or recurrent severe pain crises, despite treatment. They must be generally well-functioning (good performance status). Those with prior gene therapy, a perfect sibling donor match, active infections, pregnancy/breastfeeding, or certain genetic risks are excluded.Check my eligibility

What is being tested?

The study tests nula-cel Drug Product in about 15 participants to see if it's safe and effective for treating severe Sickle Cell Disease. It's an early-phase trial where all enrolled patients receive the experimental treatment and are monitored for results.See study design

What are the potential side effects?

Since this is a first-in-human study of nula-cel, specific side effects aren't listed but may include typical stem cell transplant-related risks like infection risk increase due to immune system effects and potential infusion reactions.

RESTORE Trial Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have had 4 or more severe pain crises in the last 2 years despite treatment.

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I have had 2 or more acute chest syndrome episodes in the last 2 years.

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I can carry out most of my everyday activities without help.

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I am between 12 and 40 years old.

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I have severe sickle cell disease with frequent pain or lung problems.

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I am between 12 and 40 years old.

RESTORE Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ over time, from 6 months to 18 months post-infusion

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~over time, from 6 months to 18 months post-infusion

This trial's timeline: 3 weeks for screening, Varies for treatment, and over time, from 6 months to 18 months post-infusion for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Frequency and severity of AEs/SAEs

Incidence rate of treatment-related mortality

Overall survival

+1 more

Secondary outcome measures

Change in annualized packed red blood cell (pRBC) transfusion requirements (volume and frequency) for SCD indications

Evaluation of HbS as a percentage of total Hgb

Evaluation of adult Hgb as a percentage of total Hgb

+8 more

RESTORE Trial Design

1Treatment groups

Experimental Treatment

Group I: nula-cel Drug ProductExperimental Treatment1 Intervention

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

0 / 6Eligibility criteria met

Find a Location

Who is running the clinical trial?

Kamau TherapeuticsLead Sponsor

Graphite Bio, Inc.Lead Sponsor

Mathew Porteus, MD, PhDStudy DirectorKamau Therapeutics

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What is the cap for enrollment in this trial?

"This medical trial is searching for 15 participants that meet the listed eligibility criteria. Volunteers can locate a clinical site in Birmingham, Alabama at University of Alabama or Washington University in Saint Louis, Missouri."

Answered by AI

Is this research endeavor seeking out participants aged fifty and above?

"This experiment is open to individuals between the age of 12 and 40."

Answered by AI

Is this research initiative actively welcoming participants?

"The information hosted on clinicaltrials.gov affirms that this medical trial, which initially went live on November 15th 2021, is actively recruiting patients. The record was recently edited as of October 5th 2022."

Answered by AI

What criteria must be met to become eligible for this research study?

"This research is seeking 15 participants aged 12 to 40 who suffer from anemia and sickle cell. To be involved, applicants must display a minimum of 4 episodes of VOC in the 2 years preceding their application as well as at least two ACS episodes in that same period including one within the previous year. Additionally, they must have achieved a Lansky/Karnofsky Performance Status score above 80%."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2021. "Gene Correction in Autologous CD34+ Hematopoietic Stem Cells (HbS to HbA) to Treat Severe Sickle Cell Disease". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04819841.

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