JSP191 for Anemia, Sickle Cell

Phase-Based Progress Estimates
1
Effectiveness
1
Safety
National Institutes of Health Clinical Center, Bethesda, MD
Anemia, Sickle Cell+4 More
JSP191 - Biological
Eligibility
Any Age
All Sexes
What conditions do you have?
Select

Study Summary

Background: Sickle cell disease (SCD) is an inherited disorder of the blood. It can damage a person s organs and cause serious illness and death. A blood stem cell transplant is the only potential cure for SCD. Treatments that improve survival rates are needed. Objective: To find out if a new antibody drug (JSP191) improves the success of a blood stem cell transplant Eligibility: People aged 13 or older who are eligible for a blood stem cell transplant to treat SCD. Healthy family members over age 13 who are matched to transplant recipients are also needed to donate blood. Design: Participants receiving transplants will undergo screening. They will have blood drawn. They will have tests of their breathing and heart function. They may have chest x-rays. A sample of marrow will be collected from a pelvic bone. Participants will remain in the hospital about 30 days for the transplant and recovery. They will have a large intravenous line inserted into the upper arm or chest. The line will remain in place for the entire transplant and recovery period. The line will be used to draw blood as needed. It will also be used to administer the transplant stem cells as well as various drugs and blood transfusions. Participants will also receive some drugs by mouth. Participants must remain within 1 hour of the NIH for 3 months after transplant. During that time, they will visit the clinic up to 2 times a week. Follow-up visits will include tests to evaluate participants mental functions. They will have MRI scans of their brain and heart.

Eligible Conditions

  • Anemia, Sickle Cell
  • Thalassemia
  • Sickle Cell Anemia

Treatment Effectiveness

Effectiveness Progress

1 of 3

Study Objectives

1 Primary · 11 Secondary · Reporting Duration: 1 and 2 years post transplant

Year 2
Non-transplant related mortality
Proportion of patients with donor myeloid chimerism at or above 75%
Rate of bacterial infection
Rate of chronic GVHD
Rates of acute GVHD
Rates of graft failure
Transplant related mortality
1 year post transplant
percent myeloid (CD14/15) chimerism
day 100
Alemtuzumab levels
Days to neutrophil engraftment
JSP antibody PK levels
Rate of viral infection and/or reactivation

Trial Safety

Safety Progress

1 of 3

Trial Design

3 Treatment Groups

Stem cell Donors of Recipients undergoing stem cell transplant
1 of 3
JSP191 in pediatrics stem cell transplant recipients for SCD
1 of 3
JSP191 in adult stem cell transplant recipients for SCD
1 of 3
Active Control
Experimental Treatment

90 Total Participants · 3 Treatment Groups

Primary Treatment: JSP191 · No Placebo Group · Phase 1 & 2

JSP191 in pediatrics stem cell transplant recipients for SCDExperimental Group · 7 Interventions: Alemtuzumab, JSP191, Hydroxyurea, Sirolimus, Plerixafor, TBI, Filgrastim (G-CSF) · Intervention Types: Biological, Biological, Drug, Drug, Drug, Radiation, Drug
JSP191 in adult stem cell transplant recipients for SCDExperimental Group · 7 Interventions: Alemtuzumab, JSP191, Hydroxyurea, Sirolimus, Plerixafor, TBI, Filgrastim (G-CSF) · Intervention Types: Biological, Biological, Drug, Drug, Drug, Radiation, Drug
Stem cell Donors of Recipients undergoing stem cell transplantNoIntervention Group · 1 Intervention: Stem cell Donors of Recipients undergoing stem cell transplant · Intervention Types:
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Alemtuzumab
2004
Completed Phase 4
~1980
Hydroxyurea
2006
Completed Phase 4
~3620
Sirolimus
2013
Completed Phase 4
~2750
Plerixafor
2011
Completed Phase 3
~770
TBI
2014
Completed Phase 2
~890
Filgrastim (G-CSF)
1995
Completed Phase 3
~460

Trial Logistics

Logistics

Participation is compensated

You will be compensated for participating in this trial.

Trial Timeline

Approximate Timeline
Screening: ~3 weeks
Treatment: Varies
Reporting: 1 and 2 years post transplant
Closest Location: National Institutes of Health Clinical Center · Bethesda, MD
Photo of maryland 1Photo of maryland 2Photo of maryland 3
2016First Recorded Clinical Trial
29 TrialsResearching Anemia, Sickle Cell
271 CompletedClinical Trials

Who is running the clinical trial?

National Heart, Lung, and Blood Institute (NHLBI)Lead Sponsor
3,555 Previous Clinical Trials
46,915,099 Total Patients Enrolled
148 Trials studying Anemia, Sickle Cell
46,433 Patients Enrolled for Anemia, Sickle Cell
John F Tisdale, M.D.Principal InvestigatorNational Heart, Lung, and Blood Institute (NHLBI)
13 Previous Clinical Trials
1,008 Total Patients Enrolled
11 Trials studying Anemia, Sickle Cell
841 Patients Enrolled for Anemia, Sickle Cell

Eligibility Criteria

Age Any Age · All Participants · 10 Total Inclusion Criteria

Mark “yes” if the following statements are true for you:
You must meet all of the inclusion criteria for this condition.
You have had more than 1 hospital admission per year while on a therapeutic dose of sickle cell treatment /medication.
ACS (Any ACS) while on sickle cell treatment /medication.
The patient must be on sickle cell treatment or medication where total hemoglobin increase less than 1 g/dL or fetal hemoglobin increases <2.5 times the baseline level.

About The Reviewer

Michael Gill preview

Michael Gill - B. Sc.

First Published: October 9th, 2021

Last Reviewed: August 12th, 2022

Michael Gill holds a Bachelors of Science in Integrated Science and Mathematics from McMaster University. During his degree he devoted considerable time modeling the pharmacodynamics of promising drug candidates. Since then, he has leveraged this knowledge of the investigational new drug ecosystem to help his father navigate clinical trials for multiple myeloma, an experience which prompted him to co-found Power Life Sciences: a company that helps patients access randomized controlled trials.