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Gene Therapy
Gene Therapy (ARU-1801) for Sickle Cell Anemia
Phase 1 & 2
Waitlist Available
Research Sponsored by Aruvant Sciences GmbH
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Has severe sickle cell disease, defined as one or more of the following: Minimum of two episodes of clinically diagnosed acute chest syndrome (ACS) requiring hospital admission, or one life threatening episode of ACS requiring intensive care unit (ICU) admission for exchange transfusion and/or intubation, or frequent ACS episodes which necessitate treatment with chronic transfusion therapy
Frequent painful vaso-occlusive episodes (VOEs) which significantly interfere with normal life activities, defined as a history of 2 or more severe acute sickle pain events per year requiring additional treatment at a medical facility outside of home pain management over the preceding 2-year period prior to study enrollment, or that necessitate chronic transfusion therapy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up baseline to year 15
Awards & highlights
Study Summary
This trial is testing a new treatment for sickle cell disease that involves collecting stem cells and transfering a corrective gene. The goal is to see if this is a safe and feasible treatment with the potential to improve quality of life for people with sickle cell disease.
Who is the study for?
This trial is for individuals with severe sickle cell disease, characterized by frequent pain episodes or acute chest syndrome. Participants must have tried and failed hydroxyurea therapy or chosen not to use it despite its benefits. They should be in stable health otherwise but cannot join if pregnant, breastfeeding, unwilling to use contraception for a year post-treatment, have active cancer (except certain skin cancers), HIV/hepatitis B/C, recent other study drugs intake, severe lung function issues, uncontrolled infections, high stroke risk or liver damage due to iron overload.Check my eligibility
What is being tested?
The trial tests ARU-1801's safety and effectiveness in gene transfer aimed at correcting the genetic defect of sickle cell anemia. It involves collecting stem cells from participants and modifying them with gamma-globin gene transfer before returning them back into the patient's body.See study design
What are the potential side effects?
Potential side effects are not explicitly listed but may include reactions related to stem cell collection and infusion processes such as discomfort at collection site, infection risk increase due to immune system impact from gene modification procedures; specific risks will depend on individual responses.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have severe sickle cell disease with frequent or severe episodes needing hospital care.
Select...
I have had 2 or more severe pain episodes from sickle cell each year that needed treatment beyond home care.
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I have been diagnosed with sickle cell disease.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ baseline to year 15
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~baseline to year 15
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Bone marrow aspirates with ≥1% gene-marked cells
Incidence of Adverse Events (AEs)
Incidence of Grade 3 allergic reaction
+11 moreSecondary outcome measures
Change in QoL (Quality of Life)
Change in proportion of antisickling/sickling hemoglobin
Frequency of opioid use pre-transplant versus post-transplant
+7 moreTrial Design
1Treatment groups
Experimental Treatment
Group I: ARU-1801Experimental Treatment1 Intervention
Autologous CD34+ hematopoietic stem cells transduced ex-vivo with gamma-globin lentiviral vector. Administered via IV infusion.
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Who is running the clinical trial?
Aruvant Sciences GmbHLead Sponsor
Children's Hospital Medical Center, CincinnatiLead Sponsor
815 Previous Clinical Trials
6,531,589 Total Patients Enrolled
12 Trials studying Sickle Cell Anemia
1,703 Patients Enrolled for Sickle Cell Anemia
Stella M. Davies, MB BS, PhD, MRCPStudy ChairChildren's Hospital Medical Center, Cincinnati
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Eligibility Criteria:
This trial includes the following eligibility criteria:- I am not pregnant or breastfeeding.I have liver damage shown by a biopsy or signs of liver fibrosis due to iron overload.I am a woman who can become pregnant and agree to use birth control for 1 year after the study drug and not to breastfeed during that time.I have or had hepatitis B, C, or HIV.I haven't had serious infections in the last month.I have had 2 or more severe pain episodes from sickle cell each year that needed treatment beyond home care.I have a history of stroke or am at high risk for a first stroke.I have severe sickle cell disease with frequent or severe episodes needing hospital care.I am on regular blood transfusions for severe symptoms affecting my daily life.I stopped taking hydroxyurea for my condition 2 months ago or never started it.I have been diagnosed with sickle cell disease.I have alpha thalassemia sickle cell disease.My organs are working well according to my last check-up.I have no active cancer except for certain skin cancers.
Research Study Groups:
This trial has the following groups:- Group 1: ARU-1801
Awards:
This trial has 1 awards, including:- No Placebo-Only Group - All patients enrolled in this study will receive some form of active treatment.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
What criteria must an individual meet in order to participate in this experiment?
"In order to be considered for inclusion in this medical trial, participants must possess the characteristics of anemia and sickle cell disease. Additionally, they should range between 18-45 years old. Approximately 7 individuals will be accepted into the study."
Answered by AI
Are septuagenarians eligible for enrolment in this trial?
"This study's minimal age requirement for participation is 18 years old and the maximal allowable age is 45."
Answered by AI
Is this trial still enrolling participants?
"According to clinicaltrials.gov, this medical trial is not actively enrolling patients at the moment. Initially published on July 1st 2014 and last updated November 12th 2022, 197 alternative trials are currently searching for participants."
Answered by AI
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