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New treatment option for Aplastic Anemia
Official title Haploidentical HCT for Severe Aplastic Anemia
ClinicalTrials.gov ID: NCT04558736
What this study is testing
What is Anti-Thymocyte Globulin (Rabbit)?
Anti-Thymocyte Globulin (Rabbit) is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for aplastic anemia.
Also referred to as thymoglobulin, rabbit ATG.
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 is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation. The purpose of this study is to learn more about newer methods of transplanting blood forming cells donated by a family member that is not fully matched to the patient.
- 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 up to 21
You may be able to join if
- for Transplant Recipient
- Age less than or equal to 21 years at time of enrollment.
- Confirmed diagnosis of SAA or a single lineage cytopenia (a) SAA or single lineage cytopenia will be defined as follows:
- i. Bone marrow cellularity \< 25% or hypocellular marrow for age, AND
- ii. One or more of the following (in peripheral blood): (i) Neutrophils \< 0.5 x10\^9/L (ii) Platelets \< 20 x10\^9/L, or platelet transfusion...
You likely can't join if
- for Transplant Recipient:
- Diagnosis of Fanconi anemia. Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing.
- Known clinical or genetic diagnosis of dyskeratosis congenita
- Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre- MDS) or MDS on marrow examination (e.g. Monosomy 7).
- Diagnosis of myelodysplastic syndrome (MDS).
- Presence of anti-donor HLA antibodies (positive anti-donor HLA antibody is defined as a positive cross-match test of any titer by complement-...
See the full eligibility criteria
- for Transplant Recipient
- Age less than or equal to 21 years at time of enrollment.
- Confirmed diagnosis of SAA or a single lineage cytopenia (a) SAA or single lineage cytopenia will be defined as follows:
- i. Bone marrow cellularity \< 25% or hypocellular marrow for age, AND
- ii. One or more of the following (in peripheral blood): (i) Neutrophils \< 0.5 x10\^9/L (ii) Platelets \< 20 x10\^9/L, or platelet transfusion dependence (iii) Hemoglobin \<8g/dL, or red blood cell transfusion dependence
- Does not have a suitable HLA-matched sibling donor (MSD) or volunteer 10/10 HLA-matched unrelated donor (MUD) available in the necessary time for progenitor cell donation.
- Failed at least one trial of immunosuppressive therapy (IST) by being refractory (persistence of severe cytopenias and fulfillment of SAA disease criteria at least 3 months after initial IST) or having relapsed (initial...
- Has a suitable single haplotype matched (≥ 3 of 6) family member donor.
- Patient and/or legal guardian must sign informed consent for HCT.
- Adequate organ function defined as:
- Left ventricular ejection fraction \> 40% or shortening fraction ≥ 25%.
- Creatinine clearance (CrCl) or glomerular filtration rate (GFR) ≥ 50 ml/ min/1.73m2.
- Forced vital capacity (FVC) ≥ 50% of predicted value; or pulse oximetry
- ≥ 92% on room air if patient is unable to perform pulmonary function testing.
- Karnofsky or Lansky (age-dependent) performance score ≥ 50.
- Bilirubin ≤ 3 times the upper limit of normal for age.
- Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) ≤ 5 times the upper limit of normal for age.
- Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time or agree to abstinence until after the last dose of chemotherapy has been administered
- for Transplant Recipient:
- Diagnosis of Fanconi anemia. Fanconi anemia must be excluded by diepoxybutane (DEB) or equivalent testing.
- Known clinical or genetic diagnosis of dyskeratosis congenita
- Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome (pre- MDS) or MDS on marrow examination (e.g. Monosomy 7).
- Diagnosis of myelodysplastic syndrome (MDS).
- Presence of anti-donor HLA antibodies (positive anti-donor HLA antibody is defined as a positive cross-match test of any titer by complement- dependent cytotoxicity or flow cytometric testing or the presence of anti-...
- Prior allogeneic hematopoietic cell transplant.
- Prior solid organ transplant.
- Known life-threatening reaction (i.e., anaphylaxis) to ATG that would prohibit use for the patient.
- Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as progression or no clinical improvement on appropriate medical treatment.
- Female patients who are pregnant (per institutional practice) or breast- feeding.
- Prior malignancies except resected basal cell carcinoma or treated cervical carcinoma in situ. Cancer treated with curative intent \> 5 years previously will be allowed. Cancer treated with curative intent ≤ 5 years...
- Alemtuzumab or ATG within 2 weeks of enrollment. Inclusion Criteria for Haploidentical Donor
- At least single haplotype matched (≥ 3 of 6) family member.
- At least 18 years of age.
- HIV negative.
- Not pregnant as confirmed by negative serum or urine pregnancy test within 14 days prior to enrollment (if female).
- Not breast feeding.
- Related donors must be ruled out for telomere disease by appropriate clinical and diagnostic measures (for example, clinical evaluation, telomere length testing, genetic testing, and/or bone marrow examination).
- The HAPLO donor and/or legal guardian must be able to sign informed consent documents.
- The potential HAPLO donor must be willing and able to donate PBSCs.
The study team makes the final eligibility decision.
Where it's taking place
- Memphis, Tennessee, 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 - 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, up to 21 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Memphis, Tennessee, United States. 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.