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New treatment option for HbS Disease
Official title A Trial to Assess Haploidentical T-depleted Stem Cell Transplantation in Patients With SCD
ClinicalTrials.gov ID: NCT04201210
What this study is testing
- What it's testing
- HSCT is currently the only curative option for SCD but less than 20% of SCD patients have a MD donor available. So far, all curative approaches beyond a MSD HSCT at young age are non-satisfactory.
- 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 2 to 35
You may be able to join if
- Age 2yrs to 35yrs
- Homozygous hemoglobin S disease or heterozygous hemoglobin SC or S 0/+
- Study specific consent given
- Preexisting severe or moderate SCD related complications:
- Clinically significant neurological event (stroke) or deficit
You likely can't join if
- Karnofsky or Lansky Performance Score \< 70%
- Patients with donor-specific antibodies (DSA) against the potential stem cell donor by either
- Cell-based crossmatched assays (Complement-dependent cytotoxicity; CDC) or
- Flow cytometry crossmatch test or
- Solid-phase immunoassays (SPI) or
- Modified SPI such as C4d and C1q assays Whichever method the participating center is experienced in.
See the full eligibility criteria
- Age 2yrs to 35yrs
- Homozygous hemoglobin S disease or heterozygous hemoglobin SC or S 0/+
- Study specific consent given
- Preexisting severe or moderate SCD related complications:
- Clinically significant neurological event (stroke) or deficit
- Silent crisis, neurocognitive deficit
- Pathological angio-MRI with TOF Sequence
- TCD velocity \>200 cm/s at 2 occasions \>1 month apart
- More than 5 vaso-occlusive crises (VOC) in the past 1 year or more than 20 VOC in a lifetime
- Two or more episodes of acute chest syndrome (ACS) in a lifetime or one episode of ACS in the past 24 months
- Chronic transfusion requirement or more than 8 transfusions or one exchange transfusion in a lifetime
- Transfusion-refractory allo-immunization
- More than five SCD-related hospitalizations in a lifetime
- Beginning pulmonary hypertension
- Osteonecrosis at more than 2 sites
- Beginning SCD Nephropathy
- Recurrent priapism (\>2)
- Karnofsky or Lansky Performance Score \< 70%
- Patients with donor-specific antibodies (DSA) against the potential stem cell donor by either
- Cell-based crossmatched assays (Complement-dependent cytotoxicity; CDC) or
- Flow cytometry crossmatch test or
- Solid-phase immunoassays (SPI) or
- Modified SPI such as C4d and C1q assays Whichever method the participating center is experienced in.
- Patients with major AB0 incompatibility defined according to EBMT Handbook, Edition 2019 Tab 23.1.: ABO incompatibility Recipient Donor Major O A O B O AB A AB B AB
- Cardiac function:
- Ejection fraction at rest \<45.0% on echocardiography or
- Shortening fraction of \<27.0% by echocardiogram or radionuclide scan (MUGA)
- Patients with \> grade II hypertension by Common Toxicity Criteria (CTC)
- Renal function:
- Estimated creatinine clearance (for patients \> 12 years) greater than 50.0 mL/minute
- for pediatric patients (\> 1 year to 12 years), GFR estimated by the updated Schwartz formula ≥ 90.0 mL/min/1.73 m2. If \ 70.0 mL/min/1.73 m2 or
- Creatinine clearance below threshold defined for stem cell transplantation according to local clinical standard
- Pulmonary function:
- DLCO \>50% (adjusted for hemoglobin), and FVC and FEV1≥50%; children unable to perform for PFTs, O2 saturation \<92% on room air.
- Liver function:
- Total bilirubin \> 2x the upper limit of normal (unless elevated bilirubin is attributed to Gilbert's Syndrome) and ALT/AST \> 2.5x the upper limit of normal.
- Chronic active viral hepatitis
- Women who are pregnant (positive serum or urine βHCG) or breastfeeding. Note: Women of childbearing potential must have a negative serum pregnancy test at study entry.
- Adults of reproductive potential not willing to use an effective method of birth control during study treatment and for at least 12 months thereafter,
- History of uncontrolled autoimmune disease or on active treatment
- Patient unable to comply with the treatment protocol
- Prior autologous or allogeneic hematopoietic stem cell transplant
- Vaccination with a live virus vaccine during the trial
- HIV infection
- Patients with a history of psychiatric illness or a condition which could interfere with their ability to understand the requirements of the study (this includes alcoholism/drug addiction)
- Patients unwilling or unable to comply with the protocol or unable to give informed consent.
- Concurrent severe or uncontrolled medical disease (e.g. uncontrolled diabetes, congestive heart failure, myocardial infarction within 6 months prior to the study, unstable and uncontrolled hypertension, chronic renal...
The study team makes the final eligibility decision.
Where it's taking place
- Vienna, Austria
- Aachen, Germany
- Berlin, Germany
- Düsseldorf, Germany
- Frankfurt, Germany
- Heidelberg, Germany
- Regensburg, Germany
- Tübingen, Germany
- Würzburg, Germany
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 - 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, 2 years to 35 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Vienna, Austria; Aachen, Germany; Berlin, Germany; Düsseldorf, Germany; Frankfurt, Germany; Heidelberg, Germany and 3 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.