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Trial record 28 of 47 for:    "Diamond-Blackfan anemia"

Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells

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ClinicalTrials.gov Identifier: NCT02512679
Recruitment Status : Terminated (Optimal dose obtained for engraftment and minimizing toxicity)
First Posted : July 31, 2015
Results First Posted : June 17, 2016
Last Update Posted : February 27, 2017
Sponsor:
Collaborator:
Lucile Packard Children's Hospital
Information provided by (Responsible Party):
Neena Kapoor, M.D., Children's Hospital Los Angeles

Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Stem Cell Transplantation
Bone Marrow Transplantation
Peripheral Blood Stem Cell Transplantation
Allogeneic Transplantation
Genetic Diseases
Thalassemia
Pediatrics
Diamond-Blackfan Anemia
Combined Immune Deficiency
Wiskott-Aldrich Syndrome
Chronic Granulomatous Disease
X-linked Lymphoproliferative Disease
Metabolic Diseases
Interventions: Drug: Cyclophosphamide Dose Level 1
Drug: Cyclophosphamide Dose Level 2
Drug: Cyclophosphamide Dose Level 3
Drug: Cyclophosphamide Dose Level 4

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Patients for bone marrow transplantation who met the eligibility criteria of diagnosis and clinical status and had histocompatible sibling donor. These patients were recruited at Children's Hospital Los Angeles and Lucille Packard Children's Hospital at Stanford University. Patients were recruited between 2006 and 2013.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
This is a sequential dose de-escalation of Cyclophosphamide with first at 105 mg/kg total dose compared to standard 200mg/kg total dose (Arm 1). Prior to enrollment patients had to meet all eligibility criteria for allogeneic transplantation. Study closed after 1st level because all patients engrafted and none had toxicity related to transplant.

Reporting Groups
  Description
Cyclophosphamide Dose Level 1

Cyclophosphamide given by Intravenous (IV) at a total dose of 105 mg/kg, to be divided into three doses of one 35 mg/kg dose per day, for 3 days on the first level.

Drug to be given in combination of Busulfan, Campath and Fludarabine

Cyclophosphamide Dose Level 1: given by IV at a total dose of 105 mg/kg, to be divided into three doses of one 35 mg/kg dose per day, for 3 days on the first level. After ten patients the de-escalation will begin if the stopping rule is not met.

Cyclophosphamide Dose Level 2

De-escalation of Cyclophosphamide given by Intravenous (IV) at a total dose of 70 mg/kg, to be divided into two doses of one 35 mg/kg dose per day, for 2 days

Drug to be given in combination of Busulfan, Campath and Fludarabine

Cyclophosphamide Dose Level 1: given by IV at a total dose of 75 mg/kg, to be divided into two doses of one 35 mg/kg dose per day, for 2 days. After ten patients the de-escalation will begin if the stopping rule is not met.


Participant Flow:   Overall Study
    Cyclophosphamide Dose Level 1   Cyclophosphamide Dose Level 2
STARTED   20   0 
COMPLETED   20   0 
NOT COMPLETED   0   0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Cyclophosphamide Dose Level 1

Cyclophosphamide given by Intravenous (IV) at a total dose of 105 mg/kg, to be divided into three doses of one 35 mg/kg dose per day, for 3 days on the first level.

Drug to be given in combination of Busulfan, Campath and Fludarabine

Cyclophosphamide Dose Level 1: given by IV at a total dose of 105 mg/kg, to be divided into three doses of one 35 mg/kg dose per day, for 3 days on the first level. After ten patients the de-escalation will begin if the stopping rule is not met.


Baseline Measures
   Cyclophosphamide Dose Level 1 
Overall Participants Analyzed 
[Units: Participants]
 20 
Age 
[Units: Participants]
Count of Participants
 
<=18 years      19  95.0% 
Between 18 and 65 years      1   5.0% 
>=65 years      0   0.0% 
Age [1] 
[Units: Years]
Median (Full Range)
 2.3 
 (.4 to 26) 
[1] Deviation from study protocol - enrollment of 26-year-old subject with pediatric disease. Subject's data included in outcome measurements/analysis.
Gender 
[Units: Participants]
Count of Participants
 
Female      13  65.0% 
Male      7  35.0% 
Region of Enrollment [1] 
[Units: Participants]
 
United States   20 
[1] Enrollment of participants was conducted in California.


  Outcome Measures

1.  Primary:   Number of Participants With Neutrophil Engraftment (=/>500 Cells/uL) and Platelet Engraftment (>20K Cell/uL) at 30 Days   [ Time Frame: 30 days ]

2.  Primary:   Number of Participants With Disease Recurrence at 1 Year Post-transplant   [ Time Frame: 1 year ]

3.  Primary:   Number of Participants Who Developed Severe Mucositis, Veno-occlusive Disease (VOD), Toxicity of the Kidney, Liver, or Gastrointestinal (GI) Tract up to 1 Year Post-transplant   [ Time Frame: 1 year ]

4.  Secondary:   Number of Participants Who Developed Graft-Versus-Host-Disease (GVHD) as Determined by the Glucksberg Scale   [ Time Frame: 1 yr ]

5.  Secondary:   Number of Participants Who Were Disease Progression-Free and Death-Free at 1 Year Post-transplant   [ Time Frame: 1 yr ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
De-escalation was not ensued. All enrolled subjects engrafted without toxicity for dose level one.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Neena Kapoor, M.D.
Organization: Children's Hospital Los Angeles
phone: 343-361-2434
e-mail: nkapoor@chla.usc.edu


Publications of Results:

Responsible Party: Neena Kapoor, M.D., Children's Hospital Los Angeles
ClinicalTrials.gov Identifier: NCT02512679     History of Changes
Other Study ID Numbers: CCI-06-00177
First Submitted: May 21, 2015
First Posted: July 31, 2015
Results First Submitted: January 29, 2016
Results First Posted: June 17, 2016
Last Update Posted: February 27, 2017