Clinical trial for Sickle Cell Disease
Official title Study of HLA-Haploidentical Stem Cell Transplantation to Treat Clinically Aggressive Sickle Cell Disease
ClinicalTrials.gov ID: NCT03121001
What this study is testing
What is ATG?
ATG is an investigational medicine being studied as part of a treatment plan for sickle cell disease. is designed to suppress the immune system to help the body accept new stem cells.
Also referred to as Thymoglobulin.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study aims to see if a specific transplant method helps new healthy blood cells grow successfully in patients with sickle cell disease.
- Who it's for
- The study is looking for people who have sickle cell disease that is causing serious health problems.
- What's involved
- Participants will receive low-dose radiation and certain medicines to prepare their body, followed by a bone marrow transplant from a family member who is not a perfect genetic match.
- Time commitment
- The study focuses on checking if the new cells are working 60 days after the transplant, which involves regular medical visits and tests.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 16 to 60
You may be able to join if
- Patients with sickle cell disease are eligible if they have any of the following complications: 1.1 Stroke or central nervous system event lasting...
- Age 16-60 years
- Karnofsky performance status of 60 or higher (Appendix A)
- Adequate cardiac function, defined as left ventricular ejection fraction ≥ 40%
- Adequate pulmonary function, defined as diffusion lung capacity of carbon monoxide ≥ 50% predicted (after adjustment for hemoglobin concentration)
See the full eligibility criteria
- Patients with sickle cell disease are eligible if they have any of the following complications: 1.1 Stroke or central nervous system event lasting longer than 24 hours 1.2 Frequent vaso-occlusive pain episodes, defined...
- Age 16-60 years
- Karnofsky performance status of 60 or higher (Appendix A)
- Adequate cardiac function, defined as left ventricular ejection fraction ≥ 40%
- Adequate pulmonary function, defined as diffusion lung capacity of carbon monoxide ≥ 50% predicted (after adjustment for hemoglobin concentration)
- Estimated GFR ≥ 50mL/min/1.73m2 as calculated by the modified MDRD equation
- ALT ≤ 3x upper limit of normal
- HIV-negative
- Patient is not pregnant
- Patient is able and willing to sign informed consent
- Patient does not have a fully HLA-matched sibling donor
- Patient has an HLA-haploidentical relative Donor Eligibility Relatives (parents, offspring, siblings, aunts/uncles, cousins) will be tested by molecular typing of HLA class I (A, B, and C) and class II (DRB1) at low...
The study team makes the final eligibility decision.
Where it is enrolling
- Chicago, Illinois, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 16 years to 60 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Chicago, Illinois, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.