Aplastic AnemiaMini-alloNCT01272817
Nonmyeloablative Allogeneic Transplant
A study testing Nonmyeloablative Allogeneic Transplant in people with Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, run by Scripps Health at 1 site in United States.
Trial summary
Allogeneic transplant from a matched sibling for the treatment of a variety of illnesses including bone marrow failure states, leukemias, myelodysplastic or myeloproliferative syndromes, lymphoma, or myeloma using a nonmyeloablative preparative regimen. Read more about this study
- Who is it for?
- Ages 18 to 72 years
- Conditions the study is for
- Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Acute Myelogenous Leukemia
- What is being tested?
- Nonmyeloablative Allogeneic Transplant
- Type of treatment
- 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
- Oct 2001to Oct 2015
- Phase
- Interventional
- Are there results?
- None posted in the registry
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 18 to 72 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age > 55 years or
- Age < 55 and LVEF < 45% or creatinine clearance < 60 ml/min
- Ability to cover the cost of the transplant, necessary medications, and transportation/housing.
- Caregiver must be available while outpatient
- Guidelines for Cladribine-Melphalan-based conditioning:
- Bone Marrow Failure States Severe Aplastic Anemia (relapsed following immunosuppressive therapy) Paroxysmal Nocturnal Hemoglobinuria (poor prognostic features or hemosiderosis)
Show all 26Show fewer
- AML (first CR except for t(15;17), inv16, t(8,21); second CR; relapse failing second induction attempt)
- ALL (first CR with at least one poor prognostic feature; second or greater CR; relapse failing reinduction attempt)
- MDS (RAEB, RAEBiT, CMMoL)
- CML (chronic phase; accelerated phase; blast phase following reinduction attempt; 2nd chronic or accelerated phase following gleevec therapy
- Hodgkin's lymphoma (first or greater relapse)
- Non-Hodgkin's Lymphoma
- Aggressive Histology (includes T Cell NHL) Incomplete response to induction Second CR Sensitive or refractory relapse
- Indolent Histology Second or greater relapse
- Mantle Cell Lymphoma (any Stage - must have received induction chemotherapy)
- Multiple Myeloma (10% residual plasmacytosis following anthracycline-based chemotherapy or residual disease following autologous transplant)
- Waldenstrom Macroglobulinemia (must have failed either purine analogue-based chemotherapy (Fludara or 2CdA) or standard CVP therapy; hyperviscosity or cytopenias)
- Guidelines for total lymphoid irradiation-based conditioning
- MDS (RA, RARS)
- CLL (Rai stage III or IV - must have received at least two different treatment regimens in the past)
- Breast Cancer (symptomatic metastatic disease, who have failed standard chemotherapy)
- Renal Cell Cancer (metastatic disease at multiple sites)
- Malignant Melanoma (metastatic disease at multiple sites)
- Sarcoma (all subtypes presently, unresectable metastatic disease)
- Ovarian Cancer (stage III or IV, platinum insensitive disease, i.e. progression within 6 months of initial platinum chemotherapy)
- Thymoma (unresectable disease)
Who cannot join
- Prior allogeneic stem cell or bone marrow transplant
- Current or past history of invasive mycotic infection
- Breast Feeding
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation Using Antithymocyte Globulin With Either Melphalan and Cladribine or Total Lymphoid Irradiation
Allogeneic transplant from a matched sibling for the treatment of a variety of illnesses including bone marrow failure states, leukemias, myelodysplastic or myeloproliferative syndromes, lymphoma, or myeloma using a nonmyeloablative preparative regimen.
Read the detailed description
same
- Conditions
- Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome, Chronic Myelogenous Leukemia
- Being tested
- Nonmyeloablative Allogeneic Transplant
- Keywords
- Nonmyeloablative transplant, Allogeneic transplant, Transplant
- Medical subject headings
- Anemia, Aplastic, Hemoglobinuria, Paroxysmal, Leukemia, Myeloid, Acute, Precursor Cell Lymphoblastic Leukemia-Lymphoma, Myelodysplastic Syndromes, Leukemia, Myelogenous, Chronic, BCR-ABL Positive, Leukemia, Lymphocytic, Chronic, B-Cell, Hodgkin Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nonmyeloablative Allogeneic TransplantProcedure or surgery
Cladribine 0.14 mg/kg/day for five days, melphalan 100 mg/m2 on one day
Nonmyeloablative Allogeneic TransplantProcedure or surgery
Total lymphoid irradiation 100cGy/day times 10 days (Monday through Friday)
| Study group | Role | What they receive |
|---|---|---|
| Cladribine + melphalan | Other | Cladribine + melphalan conditioning Receives: Procedure: Nonmyeloablative Allogeneic Transplant |
| TLI | Other | Total lymphoid irradiation conditioning Receives: Procedure: Nonmyeloablative Allogeneic Transplant |
What is being measured
The primary outcome is the main question the study is designed to answer.
Engraftment
Evaluation of engraftment of donor stem cells by bone marrow examinations at days 30, 100, and 360 after transplant.
Measured: One year
Secondary outcomes (1)
Graft-versus-host disease
Assess the incidence and severity of acute and/or chronic GVHD for patients transplanted on this protocol.
Measured: One year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jan 2011 (estimated)
- Study startOct 2001
- Primary completionOct 2015
- Study completionOct 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Aplastic AnemiaStudies of the same condition, open and under way.
- 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 N/A
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Scripps Health
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Jeffrey W. Andrey, M.D., Principal investigator, Scripps Health
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01272817
- Sponsor's protocol ID
- IRB#13-6255
- Acronym
- Mini-allo
- Status last verified
- Nov 2022
- First submitted
- 6 Jan 2011
- Last updated
- 23 Nov 2022
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation Using Antithymocyte Globulin With Either Melphalan and Cladribine or Total Lymphoid Irradiation
- ClinicalTrials.gov ID
- NCT01272817
- Acronym / sponsor ID
- Mini-allo / IRB#13-6255
- Conditions
- Aplastic Anemia, Paroxysmal Nocturnal Hemoglobinuria, Acute Myelogenous Leukemia, Acute Lymphocytic Leukemia, Myelodysplastic Syndrome, Chronic Myelogenous Leukemia
- Keywords
- Nonmyeloablative transplant, Allogeneic transplant, Transplant
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Scripps HealthUniversity, hospital or other
- Study start
- Oct 2001
- Primary completion
- Oct 2015 (actual)
- Study completion
- Oct 2015 (actual)
- First posted
- 10 Jan 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Allogeneic transplant from a matched sibling for the treatment of a variety of illnesses including bone marrow failure states, leukemias, myelodysplastic or myeloproliferative syndromes, lymphoma, or myeloma using a nonmyeloablative preparative regimen.
Detailed description
same
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Cladribine + melphalan | Other | Procedure: Nonmyeloablative Allogeneic TransplantCladribine + melphalan conditioning |
| TLI | Other | Procedure: Nonmyeloablative Allogeneic TransplantTotal lymphoid irradiation conditioning |
Who may qualify
- Ages
- 18 to 72 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (26 listed)
- Age > 55 years or
- Age < 55 and LVEF < 45% or creatinine clearance < 60 ml/min
- Ability to cover the cost of the transplant, necessary medications, and transportation/housing.
- Caregiver must be available while outpatient
- Bone Marrow Failure States Severe Aplastic Anemia (relapsed following immunosuppressive therapy) Paroxysmal Nocturnal Hemoglobinuria (poor prognostic features or hemosiderosis)
Key exclusion criteria (3 listed)
- Prior allogeneic stem cell or bone marrow transplant
- Current or past history of invasive mycotic infection
- Breast Feeding
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 |
|---|---|---|
| Nonmyeloablative Allogeneic TransplantCladribine 0.14 mg/kg/day for five days, melphalan 100 mg/m2 on one day | Procedure or surgery | Cladribine + melphalan |
| Nonmyeloablative Allogeneic TransplantTotal lymphoid irradiation 100cGy/day times 10 days (Monday through Friday) | Procedure or surgery | TLI |
Primary outcome
Engraftment
Time frame: One year
Evaluation of engraftment of donor stem cells by bone marrow examinations at days 30, 100, and 360 after transplant.
1 secondary outcome
Graft-versus-host disease
Time frame: One year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Nov 2022.
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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.
Jeffrey W. Andrey, M.D. principal investigator
Scripps Health
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Nonmyeloablative Allogeneic Transplant in people with Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, run by Scripps Health 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 26 inclusion and 3 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 23 Nov 2022. 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.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Engraftment (time frame: One year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 23 Nov 2022.