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Monoclonal Antibodies

JSP191 + Stem Cell Transplant for Sickle Cell Anemia

Phase 1 & 2
Recruiting
Led By John F Tisdale, M.D.
Research Sponsored by National Heart, Lung, and Blood Institute (NHLBI)
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients with beta-thalassemia who have grade 2 or 3 iron overload
Ages >=4 years (>=18 years for phase 1 portion of the study)
Must not have
ECOG performance status of 3 or more, or Lanksy performance status of <40
Major anticipated illness or organ failure incompatible with survival from PBSC transplant
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 1 and 2 years post transplant
Awards & highlights
No Placebo-Only Group

Summary

This trial is testing if a new antibody drug (JSP191) improves the success of a blood stem cell transplant for people with sickle cell disease.

Who is the study for?
This trial is for people aged 13 or older with sickle cell disease (SCD) at high risk due to organ damage or complications, and those with beta-thalassemia who have significant iron overload. Participants must be eligible for a blood stem cell transplant and have a matched family donor. They should understand the study procedures, agree to use birth control during the study, and not be pregnant or breastfeeding.
What is being tested?
The trial tests whether adding briquilimab (JSP191), an antibody drug, improves outcomes of nonmyeloablative hematopoietic cell transplantation in treating SCD and beta-thalassemia. It involves hospitalization for about 30 days for the transplant procedure followed by close monitoring up to three months post-transplant.
What are the potential side effects?
Potential side effects may include reactions related to immune suppression such as increased infection risk, possible infusion-related reactions from the antibody drug, and typical risks associated with bone marrow transplants like graft-versus-host disease.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
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I have beta-thalassemia with moderate to severe iron overload.
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I am at least 4 years old (or 18 for the first part of the study).
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My donor is a complete HLA match.
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I have sickle cell disease with complications not improved by current treatments.
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I have a family donor who is a complete match for me.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
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I am mostly bedridden due to my health condition.
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I do not have any major illnesses that would make a stem cell transplant impossible.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~1 and 2 years post transplant
This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 and 2 years post transplant for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
percent myeloid (CD14/15) chimerism
Secondary study objectives
Alemtuzumab levels
Days to neutrophil engraftment
JSP antibody PK levels
+8 more

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

2Treatment groups
Experimental Treatment
Active Control
Group I: briquilimab in stem cell transplant recipients for SCDExperimental Treatment7 Interventions
Affected SCD and beta-thal subjects will receive briquilimab
Group II: Stem cell Donors of Recipients undergoing stem cell transplantActive Control1 Intervention
Participants donate stem cells for recipient to undergo stem cell transplant
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
TBI
2014
Completed Phase 2
~1040
Hydroxyurea
2006
Completed Phase 4
~3490
Filgrastim (G-CSF)
1995
Completed Phase 3
~460
Sirolimus
2013
Completed Phase 4
~2750
Alemtuzumab
2004
Completed Phase 4
~1880
Plerixafor
2011
Completed Phase 3
~710

Find a Location

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,935 Previous Clinical Trials
47,792,241 Total Patients Enrolled
John F Tisdale, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
14 Previous Clinical Trials
1,237 Total Patients Enrolled

Media Library

JSP191 (Monoclonal Antibodies) Clinical Trial Eligibility Overview. Trial Name: NCT05357482 — Phase 1 & 2
Beta Thalassemia Research Study Groups: briquilimab in stem cell transplant recipients for SCD, Stem cell Donors of Recipients undergoing stem cell transplant
Beta Thalassemia Clinical Trial 2023: JSP191 Highlights & Side Effects. Trial Name: NCT05357482 — Phase 1 & 2
JSP191 (Monoclonal Antibodies) 2023 Treatment Timeline for Medical Study. Trial Name: NCT05357482 — Phase 1 & 2
Beta Thalassemia Patient Testimony for trial: Trial Name: NCT05357482 — Phase 1 & 2
~60 spots leftby Nov 2032
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