Trial Outcomes & Findings for Stem Cell Transplant Therapy With Campath-1H for Treating Advanced Mycosis Fungoides and Sezary Syndrome (NCT NCT00047060)

Last Updated: 2023-11-14

Results Overview

Proportion of subjects achieving a complete response. Complete response (CR) is defined as: disappearance of all signs and symptoms of cutaneous T-cell lymphomas (CTCL) for a period of at least one month.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

5 participants

Primary outcome timeframe

36 months

Results posted on

2023-11-14

Participant Flow

Participant milestones

Participant milestones
Measure	Stem Cell Transplant Therapy With Campath-1H Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Overall Study STARTED	5
Overall Study COMPLETED	3
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Stem Cell Transplant Therapy With Campath-1H for Treating Advanced Mycosis Fungoides and Sezary Syndrome

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 Participants Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Age, Categorical <=18 years	0 Participants n=99 Participants
Age, Categorical Between 18 and 65 years	5 Participants n=99 Participants
Age, Categorical >=65 years	0 Participants n=99 Participants
Sex: Female, Male Female	2 Participants n=99 Participants
Sex: Female, Male Male	3 Participants n=99 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=99 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	5 Participants n=99 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=99 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=99 Participants
Race (NIH/OMB) Asian	0 Participants n=99 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=99 Participants
Race (NIH/OMB) Black or African American	3 Participants n=99 Participants
Race (NIH/OMB) White	1 Participants n=99 Participants
Race (NIH/OMB) More than one race	0 Participants n=99 Participants
Race (NIH/OMB) Unknown or Not Reported	1 Participants n=99 Participants

PRIMARY outcome

Timeframe: 36 months

Population: Received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV 3x a week for 2 weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially w/target CSA levels in the therapeutic range (200 -400 ng/ml).

Outcome measures

Outcome measures
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 Participants Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Efficacy of Nonmyeloablative Preparative Regimen	4 Participants

SECONDARY outcome

Timeframe: up to 100 days

Number of participants who experienced acute GVHD grades II-IV Acute-GVHD was graded and staged prospectively using criteria from the 1994 Consensus Conference on Acute- GVHD Grading. Grades are defined as: Grade II: Skin = rash on 25-50 percent body surface area; Liver = Total Bilirubin 3.1-6.0 mg/dL; Lower GI = Diarrhea 1001-1500 mL/day. Grade III: Skin = Rash on \>50% of body surface; Liver = Total Bilirubin 6.1 - 15.0 mg/dL; Lower GI = Diarrhea \> 1500 mL/day. Grade IV: Skin = Generalized erythroderma plus bullous formation; Liver = Total Bilirubin \>15 mg/dL; Lower GI = Severe abdominal pain with or without ileus. Grade II GVHD as moderate, grade III as severe, and grade IV life-threatening.

Outcome measures

Outcome measures
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 Participants Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Number of Participants Who Experienced Acute GVHD Grades II-IV Grade II	0 Participants
Number of Participants Who Experienced Acute GVHD Grades II-IV Grade III-IV	1 Participants

SECONDARY outcome

Timeframe: Day 100 up to 3 years

Population: The analyses included only those participants that engrafted and survived over 100 days

Number of participant who experienced chronic graft versus host disease (GVHD) following stem cell transplant The diagnosis of clinical features of chronic-GVHD was determined prospectively and classified retrospectively into limited or extensive based on the Revised Seattle Classification. Chronic GvHD severity categorized as "limited" is defined as: localized skin lesions with or without limited hepatic involvement and "extensive" is defined as: generalized skin involvement, major hepatic complications, or involvement of any other organ.

Outcome measures

Outcome measures
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 Participants Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Number of Participant Who Experienced Chronic Graft Versus Host Disease Limited	3 Participants
Number of Participant Who Experienced Chronic Graft Versus Host Disease Extensive	0 Participants

SECONDARY outcome

Timeframe: up to 100 days

Number of participants that experienced graft failure. Graft failure is defined as: the failure to achieve sustained engraftment following stem cell transplantation.

Outcome measures

Outcome measures
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 Participants Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Number of Participants That Experienced Graft Failure	0 Participants

SECONDARY outcome

Timeframe: Up to 3 years

Overall response following stem cell transplant. Complete response (CR) is defined as: disappearance of all signs and symptoms of cutaneous T-cell lymphomas (CTCL) for a period of at least one month. Partial response (PR) - a 50% or greater decrease in the sum of the products of the longest perpendicular diameters of all measured lesions (a greater than 50% reduction in area of disease involvement in the case of cutaneous disease) lasting for a period of at least one month. No new metastatic lesions may appear. Stable disease is defined as: tumor measurements not meeting the criteria of CR, PR, or PD. Progressive disease (PD) - increase of 25% or greater in the sum of the products of the longest perpendicular diameters of all measured lesions (a greater than 25% increase in area of disease involvement in the case of cutaneous disease) compared to the smallest previous measurements, or the development of any new metastatic or cutaneous disease.

Outcome measures

Outcome measures
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 Participants Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Overall Response Complete Response (CR)	4 Participants
Overall Response Stable Disease	1 Participants
Overall Response Partial response (PR)	0 Participants
Overall Response Progressive disease (PD)	0 Participants

SECONDARY outcome

Timeframe: day 100

Number of Participants who experienced transplant related mortality by day 100

Outcome measures

Outcome measures
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 Participants Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Number of Participants Who Experienced Transplant Related Mortality	0 Participants

SECONDARY outcome

Timeframe: up to 5 years

Number of participants overall survival following stem cell transplant. Overall survival is defined as number participants alive following stem cell transplant

Outcome measures

Outcome measures
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 Participants Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Number of Participants Overall Survival	3 Participants

SECONDARY outcome

Timeframe: up to 100 days

Number of participants that remained Disease-free survival following stem cell transplant. Disease-free survival is defined as survival free of disease relapse or disease progression following stem cell transplant.

Outcome measures

Outcome measures
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 Participants Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Number of Participants That Remained Disease-free	4 Participants

SECONDARY outcome

Timeframe: up to 100 days

Number of participants that experienced engraftment following stem cell transplant. Engraftment is defined as neutrophil count is greater than 0.5 x10\^9.

Outcome measures

Outcome measures
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 Participants Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Number of Participants That Experienced Engraftment	5 Participants

SECONDARY outcome

Timeframe: up to 100 days

Number of participants that experienced platelet recovery up to day 100 following stem cell transplant. Platelet recovery is defined as platelet count is greater than 50 x 10\^9/l without platelet transfusion.

Outcome measures

Outcome measures
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 Participants Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Number of Participants That Experienced Platelet Recovery	5 Participants

SECONDARY outcome

Timeframe: up to 100 days

Number of participants that experienced red blood cell recovery following stem cell transplant. Red blood cell recovery is defined as achieving transfusion independence.

Outcome measures

Outcome measures
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 Participants Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Number of Participants That Experienced Red Blood Cell Recovery	5 Participants

SECONDARY outcome

Timeframe: Up to 22 months

Median time in months to achieve full myeloid and full donor T-cell Chimerism. Myeloid (CD34+) and T-cell (CD3+) chimerisms were determined by PCR analysis of short tandem repeats (STR). Full donor chimerism is defined as \>95% donor- derived cells in the peripheral blood in a specific lineage.

Outcome measures

Outcome measures
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 Participants Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg IV three times a week for two weeks followed by fludarabine 25mg/m\^2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x10\^6 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200-400 ng/ml)
Median Time in Months to Achieve Full Myeloid and Full Donor T-cell Chimerism Myeloid Chimerism	9 months Interval 3.0 to 18.0
Median Time in Months to Achieve Full Myeloid and Full Donor T-cell Chimerism Donor T-cell Chimerism	7 months Interval 4.0 to 22.0

Adverse Events

Stem Cell Transplant Therapy With Campath-1H

Serious events: 5 serious events

Other events: 0 other events

Deaths: 2 deaths

Serious adverse events

Serious adverse events
Measure	Stem Cell Transplant Therapy With Campath-1H n=5 participants at risk Subjects received a nonmyeloablative preparative regimen of alemtuzumab 30mg iv three times a week for two weeks followed by fludarabine 25mg/m2/day for five days followed by a PBPC graft targeted to deliver ≥ 5x106 CD34+ cells/kg. Cyclosporine A (CSA) for GVHD prophylaxis will be used initially with target CSA levels in the therapeutic range (200 -400 ng/ml).
Blood and lymphatic system disorders Febrile Neutropenia	20.0% 1/5 • Up to 13 years
Eye disorders Eyelid disorder	20.0% 1/5 • Up to 13 years
Gastrointestinal disorders Abdominal pain	20.0% 1/5 • Up to 13 years
Gastrointestinal disorders Vomiting	20.0% 1/5 • Up to 13 years
General disorders Disease Progression	40.0% 2/5 • Up to 13 years
General disorders Fatigue	20.0% 1/5 • Up to 13 years
General disorders Pain	40.0% 2/5 • Up to 13 years
Immune system disorders Acute graft versus host disease	20.0% 1/5 • Up to 13 years
Immune system disorders chronic graft versus host disease	20.0% 1/5 • Up to 13 years
Infections and infestations Candida infection	20.0% 1/5 • Up to 13 years
Infections and infestations Cytomegalovirus syndrome	60.0% 3/5 • Up to 13 years
Infections and infestations Herpes Zoster	20.0% 1/5 • Up to 13 years
Infections and infestations influenza	20.0% 1/5 • Up to 13 years
Infections and infestations Pneumonia	20.0% 1/5 • Up to 13 years
Infections and infestations Pneumonia bacterial	20.0% 1/5 • Up to 13 years
Infections and infestations Sepsis	60.0% 3/5 • Up to 13 years
Infections and infestations Skin infection	20.0% 1/5 • Up to 13 years
Infections and infestations Urinary tract infection	20.0% 1/5 • Up to 13 years
Injury, poisoning and procedural complications Fall	20.0% 1/5 • Up to 13 years
Metabolism and nutrition disorders Dehydration	40.0% 2/5 • Up to 13 years
Metabolism and nutrition disorders Hyponatremia	20.0% 1/5 • Up to 13 years
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Haematological malignancy	20.0% 1/5 • Up to 13 years
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Squamous cell carcinoma	40.0% 2/5 • Up to 13 years
Nervous system disorders Headache	20.0% 1/5 • Up to 13 years
Nervous system disorders Loss of consciousness	20.0% 1/5 • Up to 13 years
Psychiatric disorders Depression	20.0% 1/5 • Up to 13 years
Respiratory, thoracic and mediastinal disorders Pneumothorax	20.0% 1/5 • Up to 13 years
Vascular disorders Thromboembolic event	20.0% 1/5 • Up to 13 years

Other adverse events

Adverse event data not reported

Additional Information

Aue, Georg

National Heart Lung and Blood Institute

Phone: +1 301 451 7141

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place