Trial Outcomes & Findings for Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders (NCT NCT01626092)
NCT ID: NCT01626092
Last Updated: 2017-12-05
Results Overview
Number of patients who achieve hematopoietic engraftment - assessment of nucleated peripheral blood cells for donor (allogeneic) chimerism following this reduced-intensity HCT.
Recruitment status
COMPLETED
Study phase
NA
Target enrollment
3 participants
Primary outcome timeframe
Day 100 Following Hematopoietic Cell Transplant (HCT)
Results posted on
2017-12-05
Participant Flow
Participant milestones
| Measure |
Intent-To-Treat Patients
Patients with high-risk lysosomal and peroxisomal disorders treated with preparative regimen (Campath-1H 0.3 mg/kg intravenous (IV) on days -12 through -8, clofarabine 40 mg/m\^2 IV on days -9 through -5, melphalan 140 mg/m\^2 IV on day -4 and Total Body Irradiation with Marrow Boosting \[ first dose of 200 cGy single dose; 5 doses of 160cGy for marrow boosting - 1000cGy cumulative exposure\] by Volumetric-Modulated Arc Therapy \[VMAT\] on days -3 through -1). Hematopoietic stem cell transplantation will be infused on Day 0. Post-transplant immunosuppression to follow: Mycophenolate mofetil (MMF) begin day -3 and continue to day +30 or 7 days after engraftment, whichever is later; Cyclosporine A (CsA) begin day -3 and then taper at day +100 if related donor, day +180 for unrelated donor.
Campath-1H: A daily dose of 0.3 mg/kg IV over 2 hours will be administered on days - 12, -11, -10, -9, and -8.
Clofarabine: A daily dose of 40 mg/m2 will be administered IV over 2 hours on days -9,
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|---|---|
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Overall Study
STARTED
|
3
|
|
Overall Study
COMPLETED
|
3
|
|
Overall Study
NOT COMPLETED
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Reduced-Intensity Hematopoietic Stem Cell Transplant for High Risk Lysosomal and Peroxisomal Disorders
Baseline characteristics by cohort
| Measure |
Intent-To-Treat Patients
n=3 Participants
Patients with high-risk lysosomal and peroxisomal disorders treated with preparative regimen (Campath-1H 0.3 mg/kg intravenous (IV) on days -12 through -8, clofarabine 40 mg/m\^2 IV on days -9 through -5, melphalan 140 mg/m\^2 IV on day -4 and Total Body Irradiation with Marrow Boosting \[ first dose of 200 cGy single dose; 5 doses of 160cGy for marrow boosting - 1000cGy cumulative exposure\] by Volumetric-Modulated Arc Therapy \[VMAT\] on days -3 through -1). Hematopoietic stem cell transplantation will be infused on Day 0. Post-transplant immunosuppression to follow: Mycophenolate mofetil (MMF) begin day -3 and continue to day +30 or 7 days after engraftment, whichever is later; Cyclosporine A (CsA) begin day -3 and then taper at day +100 if related donor, day +180 for unrelated donor.
Campath-1H: A daily dose of 0.3 mg/kg IV over 2 hours will be administered on days - 12, -11, -10, -9, and -8
Clofarabine: A daily dose of 40 mg/m2 will be administered IV over 2 hours on days -9
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|---|---|
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Age, Categorical
<=18 years
|
3 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
0 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
3 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Day 100 Following Hematopoietic Cell Transplant (HCT)Number of patients who achieve hematopoietic engraftment - assessment of nucleated peripheral blood cells for donor (allogeneic) chimerism following this reduced-intensity HCT.
Outcome measures
| Measure |
Intent-To-Treat Patients
n=3 Participants
Patients with high-risk lysosomal and peroxisomal disorders treated with preparative regimen (Campath-1H 0.3 mg/kg intravenous (IV) on days -12 through -8, clofarabine 40 mg/m\^2 IV on days -9 through -5, melphalan 140 mg/m\^2 IV on day -4 and Total Body Irradiation with Marrow Boosting \[ first dose of 200 cGy single dose; 5 doses of 160cGy for marrow boosting - 1000cGy cumulative exposure\] by Volumetric-Modulated Arc Therapy \[VMAT\] on days -3 through -1). Hematopoietic stem cell transplantation will be infused on Day 0. Post-transplant immunosuppression to follow: Mycophenolate mofetil (MMF) begin day -3 and continue to day +30 or 7 days after engraftment, whichever is later; Cyclosporine A (CsA) begin day -3 and then taper at day +100 if related donor, day +180 for unrelated donor.
Campath-1H: A daily dose of 0.3 mg/kg IV over 2 hours will be administered on days - 12, -11, -10, -9, and -8.
Clofarabine: A daily dose of 40 mg/m2 will be administered IV over 2 hours on days -9,
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|---|---|
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Donor (Allogeneic) Hematopoietic Engraftment
|
1 participants
|
SECONDARY outcome
Timeframe: Day 100 following HCTIncidence of death due to complications of HCT following this reduced-intensity conditioning regimen.
Outcome measures
| Measure |
Intent-To-Treat Patients
n=3 Participants
Patients with high-risk lysosomal and peroxisomal disorders treated with preparative regimen (Campath-1H 0.3 mg/kg intravenous (IV) on days -12 through -8, clofarabine 40 mg/m\^2 IV on days -9 through -5, melphalan 140 mg/m\^2 IV on day -4 and Total Body Irradiation with Marrow Boosting \[ first dose of 200 cGy single dose; 5 doses of 160cGy for marrow boosting - 1000cGy cumulative exposure\] by Volumetric-Modulated Arc Therapy \[VMAT\] on days -3 through -1). Hematopoietic stem cell transplantation will be infused on Day 0. Post-transplant immunosuppression to follow: Mycophenolate mofetil (MMF) begin day -3 and continue to day +30 or 7 days after engraftment, whichever is later; Cyclosporine A (CsA) begin day -3 and then taper at day +100 if related donor, day +180 for unrelated donor.
Campath-1H: A daily dose of 0.3 mg/kg IV over 2 hours will be administered on days - 12, -11, -10, -9, and -8.
Clofarabine: A daily dose of 40 mg/m2 will be administered IV over 2 hours on days -9,
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|---|---|
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Transplant-Related Mortality
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0 participants
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SECONDARY outcome
Timeframe: Changes from Baseline, Days 30, 60, 100, Year 1, Year 2, Year 3 Following HCTPopulation: None of the 3 patients enrolled in the study were evaluable for this outcome. Two had a repeat transplant and one was lost to follow-up.
Depending upon underlying primary disease, a combination of evaluative tools (e.g. brain magnetic resonance imaging (MRI), clinical neurologic exam, neuropsychologic testing, electromyography) will be applied for assessment of neurologic function and how it may be affected by this reduced-intensity HCT regimen.
Outcome measures
Outcome data not reported
Adverse Events
Intent-To-Treat Patients
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Intent-To-Treat Patients
n=3 participants at risk
Patients with high-risk lysosomal and peroxisomal disorders treated with preparative regimen (Campath-1H 0.3 mg/kg intravenous (IV) on days -12 through -8, clofarabine 40 mg/m\^2 IV on days -9 through -5, melphalan 140 mg/m\^2 IV on day -4 and Total Body Irradiation with Marrow Boosting \[ first dose of 200 cGy single dose; 5 doses of 160cGy for marrow boosting - 1000cGy cumulative exposure\] by Volumetric-Modulated Arc Therapy \[VMAT\] on days -3 through -1). Hematopoietic stem cell transplantation will be infused on Day 0. Post-transplant immunosuppression to follow: Mycophenolate mofetil (MMF) begin day -3 and continue to day +30 or 7 days after engraftment, whichever is later; Cyclosporine A (CsA) begin day -3 and then taper at day +100 if related donor, day +180 for unrelated donor.
Campath-1H: A daily dose of 0.3 mg/kg IV over 2 hours will be administered on days - 12, -11, -10, -9, and -8
Clofarabine: A daily dose of 40 mg/m2 will be administered IV over 2 hours on days -9
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|---|---|
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Ear and labyrinth disorders
Hearing loss
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33.3%
1/3
|
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Eye disorders
Decreased vision
|
66.7%
2/3
|
|
Gastrointestinal disorders
Abdominal pain
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33.3%
1/3
|
|
Gastrointestinal disorders
Nausea
|
100.0%
3/3
|
|
Gastrointestinal disorders
Vomiting
|
66.7%
2/3
|
|
Gastrointestinal disorders
Mucositis
|
100.0%
3/3
|
|
Gastrointestinal disorders
Acid reflux
|
33.3%
1/3
|
|
Gastrointestinal disorders
Loose stools
|
33.3%
1/3
|
|
General disorders
Fever
|
66.7%
2/3
|
|
Infections and infestations
Positive blood culture - Staphylococcus aureus
|
33.3%
1/3
|
|
Infections and infestations
Positive blood culture - Escherichia coli
|
33.3%
1/3
|
|
Infections and infestations
Fever
|
33.3%
1/3
|
|
Infections and infestations
BK viruria
|
33.3%
1/3
|
|
Infections and infestations
Adenoviremia
|
33.3%
1/3
|
|
Injury, poisoning and procedural complications
Acute radiation syndrome
|
33.3%
1/3
|
|
Injury, poisoning and procedural complications
Graft failure
|
100.0%
3/3
|
|
Investigations
Elevated lipase
|
33.3%
1/3
|
|
Investigations
Serum amylase increased
|
66.7%
2/3
|
|
Investigations
Hypogammaglobulinemia
|
33.3%
1/3
|
|
Investigations
Elevated ALT
|
33.3%
1/3
|
|
Investigations
Elevated AST
|
33.3%
1/3
|
|
Metabolism and nutrition disorders
Anorexia
|
66.7%
2/3
|
|
Nervous system disorders
Dizziness
|
33.3%
1/3
|
|
Nervous system disorders
Muscle spasms
|
33.3%
1/3
|
|
Nervous system disorders
Headache
|
33.3%
1/3
|
|
Psychiatric disorders
Anxiety
|
66.7%
2/3
|
|
Psychiatric disorders
Agitation
|
33.3%
1/3
|
|
Renal and urinary disorders
Dysuria
|
33.3%
1/3
|
|
Respiratory, thoracic and mediastinal disorders
Decreased oxygen saturation
|
33.3%
1/3
|
|
Skin and subcutaneous tissue disorders
Rash
|
33.3%
1/3
|
|
Vascular disorders
Hypertension
|
100.0%
3/3
|
|
Vascular disorders
Hypotension
|
33.3%
1/3
|
Additional Information
Weston P Miller, MD
Masonic Cancer Center, University of Minnesota
Phone: 612-626-2778
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place