Only 4 spots left

~4 spots leftby Jul 2026

Gene Therapy for Sickle Cell Disease
(Restore Trial)

Recruiting in Palo Alto (17 mi)

+3 other locations

Age: < 65

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 1 & 2

Recruiting

Sponsor: Kamau Therapeutics

Disqualifiers: Malignancy, Immunodeficiency, Infection, Pregnancy, others

No Placebo Group

Trial Summary

What is the purpose of this trial?

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.

Do I have to stop taking my current medications for the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

What data supports the effectiveness of the treatment Nula-cel for sickle cell disease?

Research shows that gene therapy strategies, like those used in Nula-cel, can correct the genetic mutation causing sickle cell disease. Studies have demonstrated that modifying the genes in stem cells can lead to the production of healthy hemoglobin, reducing the symptoms of the disease.¹ ² ³ ⁴ ⁵

What makes the GPH101 drug unique for treating sickle cell disease?

The GPH101 drug, also known as Nula-cel, is a gene therapy specifically designed to treat sickle cell disease by addressing the genetic cause of the condition, which is different from traditional treatments that mainly focus on managing symptoms.⁶ ⁷ ⁸ ⁹ ¹⁰

Research Team

Mathew Porteus, MD, PhD

Principal Investigator

Kamau Therapeutics

Eligibility Criteria

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.

Inclusion Criteria

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

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

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

See 3 more

Exclusion Criteria

I have had a stem cell transplant or gene therapy.

I have or had another cancer, blood disorder, clotting issue, or immune system disorder.

I have a genetic change that could lead to blood cancer, as my doctor has determined.

See 3 more

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Conditioning

Participants undergo myeloablative conditioning prior to receiving nula-cel infusion

1-2 weeks

Treatment

Participants receive nula-cel via IV infusion

1 day

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Treatment Details

Interventions

GPH101 Drug Product (Gene Therapy)

Trial OverviewThe 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.

Participant Groups

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.

Find a Clinic Near You

Research Locations NearbySelect from list below to view details:

Nationwide Children's HospitalColumbus, OH

Washington UniversitySt. Louis, MO

Lucile Packard Children's HospitalPalo Alto, CA

Washington UniversitySaint Louis, MO

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Who Is Running the Clinical Trial?

Kamau Therapeutics

Lead Sponsor

Trials

Patients Recruited

20+

Graphite Bio, Inc.

Lead Sponsor

Trials

Patients Recruited

20+

Findings from Research

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Dose-escalation study of ICA-17043 in patients with sickle cell disease.Ataga, KI., Orringer, EP., Styles, L., et al.[2022]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Development of β-globin gene correction in human hematopoietic stem cells as a potential durable treatment for sickle cell disease.Lattanzi, A., Camarena, J., Lahiri, P., et al.[2022]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Preclinical Evaluation of a Novel Lentiviral Vector Driving Lineage-Specific BCL11A Knockdown for Sickle Cell Gene Therapy.Brendel, C., Negre, O., Rothe, M., et al.[2020]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease.Ramadier, S., Chalumeau, A., Felix, T., et al.[2023]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

An α-chain modification rivals the effect of fetal hemoglobin in retarding the rate of sickle cell fiber formation.Worth, EH., Fugate, MK., Grasty, KC., et al.[2023]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

A pharmacokinetic model of filgrastim and pegfilgrastim application in normal mice and those with cyclophosphamide-induced granulocytopaenia.Scholz, M., Ackermann, M., Engel, C., et al.[2021]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

[Haptenic agranulocytosis: treatment with hemopoietic growth factors].Pivnik, AV., Tonkoglaz, VN., Boĭko, DV.[2011]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Identification of Low-Level Product-Related Variants in Filgrastim Products Presently Available in Highly Regulated Markets.Hausberger, A., Lamanna, WC., Hartinger, M., et al.[2017]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Enteral bioavailability of human granulocyte colony stimulating factor conjugated with poly(ethylene glycol).Jensen-Pippo, KE., Whitcomb, KL., DePrince, RB., et al.[2019]

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Characterisation of the site-specific monoPEGylated rhG-CSF analogue pegteograstim.Hong, J., Lee, B., Kang, K., et al.[2022]