Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT05357482 · ClinicalTrials.gov registry record · Phase 1
Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia
A Phase 1 study of Sickle Cell Anemia and Beta-Thalassemia, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- Active
- Registry status
- Phase 1
- Development phase
- 40
- Enrollment target
- 1
- Study location
NCT05357482 is a Phase 1 study of Sickle Cell Anemia and Beta-Thalassemia that is active but no longer recruiting, run by National Heart, Lung, and Blood Institute (NHLBI). The registered enrollment target is 40 participants, below the 99-participant average among 22 other Sickle Cell Anemia trials with a reported enrollment target (60% lower). The trial reports 1 study location across 1 state.
The verdict
NCT05357482, a Phase 1 study of Sickle Cell Anemia and Beta-Thalassemia, is active but no longer recruiting, sponsored by National Heart, Lung, and Blood Institute (NHLBI).
- ACTIVE NOT RECRUITING
- Registry status
- Phase 1
- Development phase
- 40 participants
- Enrollment target
- 1
- Study location
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 (briquilimab, 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.
Conditions Studied
Interventions
- DRUG Sirolimus
- DRUG Hydroxyurea
- RADIATION TBI
- BIOLOGICAL briquilimab
- DRUG Filgrastim (G-CSF)
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 40 participants |
| Start Date | 2022-05-12 |
| Est. Completion | 2027-01-31 |
| Phase | Phase 1 |
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT05357482
The ClinicalTrials.gov registry entry for NCT05357482 describes a study currently listed as active not recruiting, categorized as Phase 1. The registered enrollment target is 40 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 99-participant average among 22 other Sickle Cell Anemia trials with a reported enrollment target (60% lower). The listed sponsor is National Heart, Lung, and Blood Institute (NHLBI), which has 501 total studies on file at ClinicalTrials.gov.
The record links to 2 conditions, with Sickle Cell Anemia appearing as the primary indexed condition, and to 5 interventions - of which Sirolimus is the first listed.
NCT05357482 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.
Frequently Asked Questions
What is clinical trial NCT05357482 about?
NCT05357482 is a clinical study titled "Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia". 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 ant...
What is the current status of trial NCT05357482?
This trial is currently active not recruiting. It is a Phase 1 study. The enrollment target is 40 participants. The study started on 2022-05-12. Estimated completion is 2027-01-31.
What conditions does trial NCT05357482 study?
This clinical trial studies the following conditions: Sickle Cell Anemia, Beta-Thalassemia.
What interventions are being tested in trial NCT05357482?
The interventions under investigation include: Sirolimus (DRUG), Hydroxyurea (DRUG), TBI (RADIATION), briquilimab (BIOLOGICAL), Filgrastim (G-CSF) (DRUG).
Who is sponsoring clinical trial NCT05357482?
This trial is sponsored by National Heart, Lung, and Blood Institute (NHLBI), which has 501 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT05357482 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Sickle Cell Anemia
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and Other Anemias
RECRUITING · Phase 1
-
Haploidentical PBMC Transplant for Severe Congenital Anemias
ACTIVE NOT RECRUITING · Phase 1
-
Pharmacokinetics (PK) of Liquid Hydroxyurea in Pediatric Patients With Sickle Cell Anemia
COMPLETED · Phase 1
-
Nitric Oxide and Transfusion Therapy for Sickle Cell Patients With Pulmonary Hypertension
COMPLETED · Phase 1
-
Hematopoietic Stem Cell Transplant for Sickle Cell Disease
COMPLETED · Phase 1
-
Dose Escalation Study of PF-07209326 in Healthy Participants and Participants With Sickle Cell Disease
COMPLETED · Phase 1
Explore more on PlainTrial
Read our methodology - how this data is sourced, computed, and verified.
Related
Every figure on PlainTrial is rendered directly from the ClinicalTrials.gov registry, no number is typed in by an editor. This page mirrors this trial's own ClinicalTrials.gov registry record, live from the dataset. See our editorial standards & corrections policy, the methodology behind these numbers, or report a data error.