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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: ClinicalTrials.gov
Last refreshed on: 12 December 2020
Main ID:  NCT02833805
Date of registration: 12/07/2016
Prospective Registration: Yes
Primary sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Public title: NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia
Scientific title: A Phase II Trial of Non-Myeloablative (NMA) Conditioning and Transplantation of Partially HLA-Mismatched/Haploidentical Related or Matched Unrelated Donor (MUD) Bone Marrow for Newly Diagnosed Patients With Severe Aplastic Anemia
Date of first enrolment: September 2016
Target sample size: 21
Recruitment status: Active, not recruiting
URL:  https://clinicaltrials.gov/show/NCT02833805
Study type:  Interventional
Study design:  Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).  
Phase:  Phase 2
Countries of recruitment
United States
Contacts
Name:     Amy E DeZern, MD
Address: 
Telephone:
Email:
Affiliation:  Johns Hopkins University
Key inclusion & exclusion criteria

Inclusion Criteria:

- Confirmed diagnosis of inherited or acquired severe aplastic anemia (SAA)

- One of the following available donors:

1. HLA-haploidentical relative

2. If recipient is >= 40 years old, may use HLA-matched related donor

3. For recipients with inherited bone marrow failure syndromes (IBMFS) with clear
evidence of same disorder in potential related donors, may use 10/10 matched
unrelated donor

- Recipient and/or legal guardian must sign protocol informed consent

- Donor must be willing to donate bone marrow

- Left ventricular ejection fraction (LVEF) >= 40%. For recipients < 13 years old,
shortening fraction >= 26% may be used instead.

- Bilirubin < 3 x upper limit of normal (ULN) for age, unless patient has Gilbert's
disease

- aspartate aminotransferase (AST) and alanine aminotransferase (ALT) < 5 x ULN for age

- For patients >= 13 years old: estimated creatinine clearance > 50 mL/min using
Cockcroft-Gault formula and actual body weight

- For patients >= 1 but < 13 years old: glomerular filtration rate (GFR) estimated by
updated Schwartz formula >= 90 mL/min/1.73 m^2. If estimated GFR is < 90 mL/min/1.73
m^2, 24-hour measured creatinine clearance must be > 50 mL/min/1.73 m^2.

- For patients >= 8 years old, diffusing capacity of the lung for carbon monoxide (DLCO)
(corrected for hemoglobin) > 40%; forced expiratory volume at one second (FEV1) > 50%;
forced vital capacity (FVC) > 50%

- For patients < 8 years old or unable to undergo pulmonary function testing: no
evidence of dyspnea at rest; no need for supplemental oxygen; oxygen saturation > 92%
on room air

- Karnofsky/Lansky status (depending on age) >= 70%

- Females and males of childbearing potential must agree to practice 2 effective methods
of contraception at the same time. If unwilling, they must agree to complete
abstinence.

Exclusion Criteria:

- Previous administration of immunosuppressive therapy for SAA.

- Fanconi anemia. At minimum, this diagnosis must be excluded by diepoxybutane (DEB) or
equivalent testing on peripheral blood or marrow in patients < 30 years old.

- Clonal cytogenetic abnormalities consistent with pre-myelodysplastic syndrome
(pre-MDS) or MDS on bone marrow examination

- Presence of anti-donor antibodies

- Prior allogeneic stem cell transplant

- Prior solid organ transplant

- Uncontrolled bacterial, viral, or fungal infection

- HIV seropositivity

- Active hepatitis B or C infection determined by serology and/or nucleic acid testing
(NAT)

- Pregnancy or active breastfeeding

- Prior malignancies except: resected basal carcinoma or treated cervical carcinoma in
situ; cancer treated with curative intent > 5 years previously. Other prior cancers
will not be allowed unless approved by the PI.



Age minimum: N/A
Age maximum: N/A
Gender: All
Health Condition(s) or Problem(s) studied
Aplastic Anemia
Bone Marrow Failure
Immunosuppression
Severe Aplastic Anemia
Intervention(s)
Drug: Cyclophosphamide
Drug: Fludarabine
Drug: Mycophenolate mofetil
Drug: Tacrolimus
Drug: Thymoglobulin
Radiation: Total body irradiation
Primary Outcome(s)
Overall survival and engraftment at one year [Time Frame: 1 year]
Secondary Outcome(s)
Estimate full donor chimerism [Time Frame: Day 60]
Estimate GVHD-free relapse-free survival (GRFS) [Time Frame: 1 year]
Estimate transplant-related mortality [Time Frame: 1 year]
Incidence of chronic GVHD, one year [Time Frame: 1 year]
Incidence of grades II-IV acute GVHD, Day 100 [Time Frame: Day 100]
Incidence of grades III-IV acute GVHD, Day 100 [Time Frame: 1 year]
Incidence of primary graft failure [Time Frame: 1 year]
Incidence of secondary graft failure [Time Frame: 1 year]
Overall survival at one year [Time Frame: 1 year]
Probability of neutrophil recovery [Time Frame: 1 year]
Probability of platelet recovery [Time Frame: 1 year]
Secondary ID(s)
IRB00107139
J1688
Source(s) of Monetary Support
Please refer to primary and secondary sponsors
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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