Dyskeratosis CongenitaPhase 2/3NCT00455312
Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA
A Phase 2/3 study testing Campath 1H, Cyclophosphamide and Fludarabine in people with Dyskeratosis Congenita and Aplastic Anemia, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
Trial summary
Transplantation with stem cells is a standard therapy in many centers around the world. Previous experience with stem cell transplantation therapy for leukemias, lymphomas, other cancers, aplastic anemia and other non-malignant diseases, has led to prolonged disease-free survival or cure for some patients. Read more about this study
- Who is it for?
- Ages Up to 70 years
- Conditions the study is for
- Dyskeratosis Congenita, Aplastic Anemia
- What is being tested?
- Campath 1H, Cyclophosphamide and Fludarabine
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 36 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2007to Jun 2016
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 70 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients with dyskeratosis congenita (DC) or severe aplastic anemia (SAA) 0-70 years of age with an acceptable hematopoietic stem cell (HSC) donor
- HSC source
- Human leukocyte antigen (HLA) identical or 1 antigen mismatched sibling or other relative eligible to donate bone marrow (BM), umbilical cord blood (UCB) or mobilized peripheral blood (PB) at cell doses that meet current institutional standards.
- HLA identical or up to a 1 antigen mismatched unrelated donor.
- Two units of unrelated umbilical cord blood (UCB) that are (a) up to 2 HLA antigens mismatched to the patient (b) up to 2 HLA antigens mismatched to each other, (c) minimum cell dose of ≥ 3.5 x 10^7 nucleated cells/kg and optimal cell dose ≥ 5 x 10^7 nucleated cells/kg.
- If two units are not available: single unrelated UCB unit selected according to Minnesota Bone Marrow Transplant (BMT) program guidelines
Show all 33Show fewer
- Disease Characteristics for DC (both of the following):
- Evidence of BM failure:
- Requirement for red blood cell and/or platelet transfusions,
- Requirement for granulocyte colony-stimulating factor (G-CSF) or granulocyte-macrophage colony-stimulating factor (GM-CSF) or erythropoietin, or
- Refractory cytopenias defined as two out of three: platelets <40,000/microliter (uL) or transfusion dependent, Absolute neutrophil count <500/uL without hematopoietic growth factor support, Hemoglobin <9g/uL or transfusion dependent
- Diagnosis of DC:
- A triad of mucocutaneous features: oral leukoplakia, nail dystrophy, abnormal reticular skin hyperpigmentation.
- Or one of the following: Short telomeres (under a research study), Dyskerin mutation, Telomerase RNA (TERC) mutation
- Disease Characteristics for SAA (both of the following):
- Evidence of BM failure:
- Refractory cytopenia defined by bone marrow cellularity <25-50% (with < 30% residual hematopoietic cells)
- Diagnosis of SAA:
- Refractory cytopenias defined as two out of three: Platelets <20,000/uL or transfusion dependent, Absolute neutrophil count <500/uL without hematopoietic growth factor support, Absolute reticulocyte count <20,000/uL
- Patients with early myelodysplastic features.
- Patients with or without clonal cytogenetic abnormalities.
- Patient Exclusion Criteria:
- Patients with one or more of the following:
- Decompensated congestive heart failure; left ventricular ejection fraction <35%
- Acute hepatitis or evidence of moderate or severe portal fibrosis or cirrhosis on biopsy
- Carbon Monoxide Diffusing Capacity (DLCO) <30% predicted, and oxygen requirement
- Glomerular filtration rate (GFR) <30% predicted
- Pregnant or lactating female
- Active serious infection whereby patient has been on intravenous antibiotics for at least one week prior to study entry. Any patient with AIDS or HIV seropositivity. If recent mold infection e.g. Aspergillus - must have >30 days of appropriate treatment before HSC transplantation and infection must be controlled and cleared by the Infectious Disease consultant.
- Cannot receive total body irradiation (TBI) due to prior radiation therapy
- Diagnosis of Fanconi anemia based on diepoxybutane (DEB).
- DC patients with advanced myelodysplastic syndrome (MDS) or acute myeloid leukemia with >30 blasts.
- History of non hematopoietic malignancy within 2 years except resected basal cell carcinoma or treated carcinoma in situ.
Who cannot join
None listed.
Trial locations and contacts
1 site in 1 country.
United States1 site
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, 55455
About this study
Official title: Hematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic Anemia
Transplantation with stem cells is a standard therapy in many centers around the world. Previous experience with stem cell transplantation therapy for leukemias, lymphomas, other cancers, aplastic anemia and other non-malignant diseases, has led to prolonged disease-free survival or cure for some patients. However, the high doses of pre-transplant radiation and chemotherapy drugs used, and the type of drugs used, often cause many side effects that are intolerable for some patients. Slow recovery of blood counts is a frequent complication of high dose pre-transplant regimens, resulting in a longer period of risk for bleeding and infection plus a longer time in the hospital.
Recent studies have shown that using lower doses of radiation and chemotherapy (ones that do not completely kill all of the patient's bone marrow cells) before blood or bone marrow transplant, may be a better treatment for high risk patients, such as those with Dyskeratosis Congenita (DC) or Severe Aplastic Anemia(SAA). These low dose transplants may result in shorter periods of low blood counts, and blood counts that do not go as low as with traditional pre-transplant radiation and chemotherapy. Furthermore, in patients with Dyskeratosis Congenita or SAA, the stem cell transplant will replace the blood forming cells with healthy cells.
It has recently been shown that healthy marrow can take and grow after transplantation which uses doses of chemotherapy and radiation that are much lower than that given to patients with leukemia. While high doses of chemotherapy and radiation may be necessary to get rid of leukemia, this may not be important to patients with Dyskeratosis Congenita or SAA. The purpose of this research is to see if this lower dose chemotherapy and radiation regimen followed by transplant is a safe and effective treatment for patients with Dyskeratosis Congenita or SAA.
Read the detailed description
This is an open label, single arm, phase II clinical trial designed to evaluate the safety and efficacy of the treatment regimen. Efficacy will be measured by long-term engraftment of the transplanted cells.The primary endpoint of neutrophil engraftment is defined as an absolute neutrophil count (ANC) >5 x 108/L (first of three consecutive laboratory measurements on different days) with at least 10% donor cells by day 100. We will evaluate the proportion of success (P) and its 95% confidence interval (CI) for the entire group. The null hypothesis of 90% engraftment will be rejected if 4 or more patients fail to engraft out of 15 evaluable patients. The secondary endpoints of regimen related mortality, acute and chronic graft-versus-host disease (GVHD) and secondary malignancies will be estimated by cumulative incidence treating non-event deaths as a competing risk. Survival will be estimated by Kaplan-Meier methods. Immune reconstitution will be summarized with descriptive statistics.
SAA and DC arms will be analyzed separately.
- Conditions
- Dyskeratosis Congenita, Aplastic Anemia
- Being tested
- Campath 1H, Cyclophosphamide, Fludarabine, Total Body Irradiation, Stem Cell Transplantation
- Keywords
- Dyskeratosis Congenita, Hematopoietic Stem Cell Transplantation, Severe Aplastic Anemia
- Medical subject headings
- Dyskeratosis Congenita, Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Campath 1HDrug
Also called Alemtuzumab
10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).
CyclophosphamideDrug
Also called Cytoxan
7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).
FludarabineDrug
Also called Fludara
6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)
Total Body IrradiationProcedure or surgery
Also called Radiation therapy, therapeutic radiation
1 day before the transplant one dose (200 cGy) of total body irradiation is given
Stem Cell TransplantationProcedure or surgery
Also called Bone Marrow Transplant
Infusion of stem cells on Day 0.
antithymocyte globulinDrug
Also called Atgam, Thymoglobulin, ATG
ATG (rabbit) 3 mg/kg for 3 days.
2mg/kg IV is given before each dose of antithymocyte globulin (ATG).
| Study group | Role | What they receive |
|---|---|---|
| Patients with DC | Experimental | Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation. Receives: Drug: Campath 1H; Drug: Cyclophosphamide; Drug: Fludarabine; Procedure: Total Body Irradiation; Procedure: Stem Cell Transplantation |
| Patients with SAA | Experimental | Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation. Receives: Drug: Cyclophosphamide; Drug: Fludarabine; Procedure: Total Body Irradiation; Procedure: Stem Cell Transplantation; Drug: antithymocyte globulin; Drug: Methylprednisolone |
What is being measured
The primary outcome is the main question the study is designed to answer.
Neutrophil Engraftment
Defined as an absolute neutrophil count (ANC) >5 x 10^8/L (first of three consecutive laboratory measurements on different days) with at least 10% donor cells by day 100. Demonstrate sustained engraftment after a fludarabine based preparative regimen in patients with dyskeratosis congenita followed by hematopoietic cell transplantation.
Measured: Day 100
Secondary outcomes (9)
Incidence of Regimen Related Mortality at 100 Days
all deaths without previous relapse or progression
Measured: 100 days
Incidence of Chronic GVHD
Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host.
Measured: 6 months
Incidence of Chronic GVHD
Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host.
Measured: 1 year
Incidence of Late Secondary Malignancies
Defined as patients who have a secondary malignancy (cancer) occurring.
Measured: 1 Year
Incidence of Grade 2-4 Acute Graft Versus Host Disease (GVHD)
Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
Measured: Day 100
Incidence of Grade 3-4 Acute Graft Versus Host Disease (GVHD)
Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
Measured: Day 100
Overall Survival
Overall survival is defined as time from date of transplant to date of death or censored at the date of last documented contact for patients still alive.
Measured: Day 100
Overall Survival
Overall survival is defined as time from date of transplant to date of death or censored at the date of last documented contact for patients still alive.
Measured: 1 Year
Incidence of Pulmonary Complications
Defined as patients who exhibit a pulmonary (lung) adverse event.
Measured: 6 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2007 (estimated)
- Study startAug 2007
- Primary completionMar 2015
- Study completionJun 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Dyskeratosis CongenitaStudies of the same condition, open and under way.
- More trials run by Masonic Cancer Center, University of MinnesotaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Masonic Cancer Center, University of Minnesota
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jakub Tolar, M.D., Ph.D., Principal investigator, Masonic Cancer Center, University of Minnesota
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00455312
- Sponsor's protocol ID
- MT2006-06
- Other IDs
- 0612M98727
- Status last verified
- Dec 2017
- First submitted
- 30 Mar 2007
- Last updated
- 5 Dec 2017
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Dietz AC, Orchard PJ, Baker KS, Giller RH, Savage SA, Alter BP, Tolar J. Disease-specific hematopoietic cell transplantation: nonmyeloablative conditioning regimen for dyskeratosis congenita. Bone Marrow Transplant. 2011 Jan;46(1):98-104. doi: 10.1038/bmt.2010.65. Epub 2010 Apr 12.
PubMed 20383216
Study at a glance
From the registry record, last updated by the sponsor on 5 Dec 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hematopoietic Stem Cell Transplant For Patients With Dyskeratosis Congenita and Severe Aplastic Anemia
- ClinicalTrials.gov ID
- NCT00455312
- Acronym / sponsor ID
- MT2006-06
- Conditions
- Dyskeratosis Congenita, Aplastic Anemia
- Keywords
- Dyskeratosis Congenita, Hematopoietic Stem Cell Transplantation, Severe Aplastic Anemia
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Masonic Cancer Center, University of MinnesotaUniversity, hospital or other
- Study start
- Aug 2007
- Primary completion
- Mar 2015 (actual)
- Study completion
- Jun 2016 (actual)
- First posted
- 3 Apr 2007 (estimated)
- Results
- Posted 31 May 2017
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Transplantation with stem cells is a standard therapy in many centers around the world. Previous experience with stem cell transplantation therapy for leukemias, lymphomas, other cancers, aplastic anemia and other non-malignant diseases, has led to prolonged disease-free survival or cure for some patients. However, the high doses of pre-transplant radiation and chemotherapy drugs used, and the type of drugs used, often cause many side effects that are intolerable for some patients. Slow recovery of blood counts is a frequent complication of high dose pre-transplant regimens, resulting in a longer period of risk for bleeding and infection plus a longer time in the hospital.
Recent studies have shown that using lower doses of radiation and chemotherapy (ones that do not completely kill all of the patient's bone marrow cells) before blood or bone marrow transplant, may be a better treatment for high risk patients, such as those with Dyskeratosis Congenita (DC) or Severe Aplastic Anemia(SAA). These low dose transplants may result in shorter periods of low blood counts, and blood counts that do not go as low as with traditional pre-transplant radiation and chemotherapy. Furthermore, in patients with Dyskeratosis Congenita or SAA, the stem cell transplant will replace the blood forming cells with healthy cells.
It has recently been shown that healthy marrow can take and grow after transplantation which uses doses of chemotherapy and radiation that are much lower than that given to patients with leukemia. While high doses of chemotherapy and radiation may be necessary to get rid of leukemia, this may not be important to patients with Dyskeratosis Congenita or SAA. The purpose of this research is to see if this lower dose chemotherapy and radiation regimen followed by transplant is a safe and effective treatment for patients with Dyskeratosis Congenita or SAA.
Detailed description
This is an open label, single arm, phase II clinical trial designed to evaluate the safety and efficacy of the treatment regimen. Efficacy will be measured by long-term engraftment of the transplanted cells.The primary endpoint of neutrophil engraftment is defined as an absolute neutrophil count (ANC) >5 x 108/L (first of three consecutive laboratory measurements on different days) with at least 10% donor cells by day 100. We will evaluate the proportion of success (P) and its 95% confidence interval (CI) for the entire group. The null hypothesis of 90% engraftment will be rejected if 4 or more patients fail to engraft out of 15 evaluable patients. The secondary endpoints of regimen related mortality, acute and chronic graft-versus-host disease (GVHD) and secondary malignancies will be estimated by cumulative incidence treating non-event deaths as a competing risk. Survival will be estimated by Kaplan-Meier methods. Immune reconstitution will be summarized with descriptive statistics.
SAA and DC arms will be analyzed separately.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Patients with DC | Experimental | Drug: Campath 1H, Drug: Cyclophosphamide, Drug: Fludarabine, Procedure: Total Body Irradiation, Procedure: Stem Cell TransplantationPatients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation. |
| Patients with SAA | Experimental | Drug: Cyclophosphamide, Drug: Fludarabine, Procedure: Total Body Irradiation, Procedure: Stem Cell Transplantation, Drug: antithymocyte globulin, Drug: MethylprednisolonePatients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation. |
Who may qualify
- Ages
- Up to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (33 listed)
- Patients with dyskeratosis congenita (DC) or severe aplastic anemia (SAA) 0-70 years of age with an acceptable hematopoietic stem cell (HSC) donor
- Patients with one or more of the following:
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Campath 1HAlso: Alemtuzumab10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours). | Drug | Patients with DC |
| CyclophosphamideAlso: Cytoxan7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours). | Drug | Patients with DC, Patients with SAA |
| FludarabineAlso: Fludara6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour) | Drug | Patients with DC, Patients with SAA |
| Total Body IrradiationAlso: Radiation therapy, therapeutic radiation1 day before the transplant one dose (200 cGy) of total body irradiation is given | Procedure or surgery | Patients with DC, Patients with SAA |
| Stem Cell TransplantationAlso: Bone Marrow TransplantInfusion of stem cells on Day 0. | Procedure or surgery | Patients with DC, Patients with SAA |
| antithymocyte globulinAlso: Atgam, Thymoglobulin, ATGATG (rabbit) 3 mg/kg for 3 days. | Drug | Patients with SAA |
| Methylprednisolone2mg/kg IV is given before each dose of antithymocyte globulin (ATG). | Drug | Patients with SAA |
Primary outcome
Neutrophil Engraftment
Time frame: Day 100
Defined as an absolute neutrophil count (ANC) >5 x 10^8/L (first of three consecutive laboratory measurements on different days) with at least 10% donor cells by day 100. Demonstrate sustained engraftment after a fludarabine based preparative regimen in patients with dyskeratosis congenita followed by hematopoietic cell transplantation.
9 secondary outcomes
Incidence of Regimen Related Mortality at 100 Days
Time frame: 100 days
Incidence of Chronic GVHD
Time frame: 6 months
Incidence of Chronic GVHD
Time frame: 1 year
Incidence of Late Secondary Malignancies
Time frame: 1 Year
Incidence of Grade 2-4 Acute Graft Versus Host Disease (GVHD)
Time frame: Day 100
Incidence of Grade 3-4 Acute Graft Versus Host Disease (GVHD)
Time frame: Day 100
Overall Survival
Time frame: Day 100
Overall Survival
Time frame: 1 Year
Incidence of Pulmonary Complications
Time frame: 6 Months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Dec 2017.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 20383216Dietz AC, Orchard PJ, Baker KS, Giller RH, Savage SA, Alter BP, Tolar J. Disease-specific hematopoietic cell transplantation: nonmyeloablative conditioning regimen for dyskeratosis congenita. Bone Marrow Transplant. 2011 Jan;46(1):98-104. doi: 10.1038/bmt.2010.65. Epub 2010 Apr 12.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jakub Tolar, M.D., Ph.D. principal investigator
Masonic Cancer Center, University of Minnesota
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Campath 1H, Cyclophosphamide and Fludarabine in people with Dyskeratosis Congenita and Aplastic Anemia, run by Masonic Cancer Center, University of Minnesota at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 33 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Dec 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 31 May 2017). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Neutrophil Engraftment (time frame: Day 100). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00455312, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Dec 2017.