Sickle Cell DiseasePhase 1NCT02065596

Hematopoietic Stem Cell Transplant for Sickle Cell Disease

A Phase 1 study testing Fludarabine and Hematopoietic Stem Cell Transplant (HSCT) in people with Sickle Cell Disease and Sickle Cell Anemia, run by Case Comprehensive Cancer Center at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Ages 18 to 65 years
Conditions the study is for: Sickle Cell Disease, Sickle Cell Anemia, SCD
What is being tested?: Fludarabine and Hematopoietic Stem Cell Transplant (HSCT)
Type of treatment: Surgery or procedure
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 4 participants
Where: 1 siteUnited States
Study dates: 19 Oct 2015to 17 Feb 2023
Phase: Phase 1
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 65 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

Patients must have one of the following inherited hemoglobin gene disorders:
a. Hemoglobin SS
b. Hemoglobin SC
c. Hemoglobin S-Beta-zero-Thalassemia or
d. Hemoglobin S-Beta-plus Thalassemia with an episode of multi-organ failure within 5 years of eligibility
Patients must meet one of the following risk criteria:

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Low Risk (Red Light. Stop and consider therapy closely): Must have matched sibling donor grafts, failed conventional therapy as determined by the PI, and evidence for morbid disease (one of the following):
a. 2 or more painful episode/year (requiring Emergency Department or inpatient care) x 2 years or
b. 1 or more diagnoses of Acute Chest Syndrome within 5 years, or
c. 2-year mortality 5-10% or
d. Baseline LDH>600 IU or
e. History of sepsis, with or without a WBC>13.5, or
f. On chronic transfusions
Moderate Risk (Yellow Light. Reasonable to proceed, but with caution): May have alternate donor grafts (haploidentical or matched unrelated donor), if MSD is not available. Must have history of high-level vasculopathy, as defined by at least one of the criteria below:
a. Urine Albumin to Creatinine Ratio of >300mg/g or eGFR 50-90 ml/min x 2 evaluations within 3 months or
b. History of overt clinical stroke, or progressive cerebral vasculopathy radiographically or
c. 1 or more diagnoses of Acute Chest Syndrome, multi-organ failure, or sickle hepatopathy within 7 years, or
d. Excessively morbid disease manifest as VOCs at a rate of 2 or more per year x 2-years or uncontolled retinal disease attributed to SCD. These patients can be considered for moderate-risk alternate donor transplants. The palliative nature of the transplant will be explicit in the consent.
e. 2-year mortality >10-15%
i. Baseline WBC>13.5 and on chronic transfusions or baseline LDH>600 or age >35 years old,
ii. Baseline TRV ≥3 m/s,
iii. Chronic transfusion therapy and age >35 years old or male gender,
iv. Baseline LDH>600 and age >35 years old or history of sepsis
v. History of sepsis and age >35 years old or male gender.
f. History of multi-organ failure
High Risk (Green light, proceed if possible): All donor types are eligible. Must have high risk disease and a >15% risk of 2-year mortality as defined by at least one of the criteria below.
a. Baseline TRV ≥3 m/s and baseline WBC >13.5 or on chronic transfusions or history of sepsis or age >35 years old,
b. Baseline WBC>13.5 and chronic transfusions or baseline LDH>600 or age >35 years old
c. Age >35 years old and chronic transfusions
To determine eligibility as a bone marrow transplant patient:
Available suitable donor
a. 6/6 HLA-matched sibling donor (HLA A, B, and DRB1), bone marrow only
b. 8/8 HLA-matched unrelated donor (HLA A, B, C, DRB1), bone marrow only
c. 4/8, 5/8, 6/8, 7/8 Haploidentical donor, bone marrow only
Patients must have adequate hematologic, hepatic, and renal function as defined below:
Direct bilirubin within 3 X normal institutional limits
ALT (SGPT) < 3 X institutional upper limit of normal
Creatinine clearance >21 mL/min/1.73 m^2 for subjects with creatinine clearance values below 50 mL/min/1.73 m^2, the principal investigator may use discretion for appropriate fludarabine dose adjustment as noted.
Patients must have adequate pulmonary function as defined by Pulmonary function: DLCO r40% (adjusted for hemoglobin) and FEV1r50%.
Contraception/Child Bearing The effects of Fludarabine, cytoxan, ATG, tacrolimus/sirolimus and MTX are cumulatively known to be deleterious to the health of the developing human fetus. For this reason, and because of teratogenic potential, all women of child-bearing potential and men must agree to use adequate contraception (double barrier method of birth control or abstinence) for the duration of study participation and for 12 months after completing treatment.
Performance Status: Eastern Cooperative Oncology Group (ECOG) Performance Status ≤ 2
Subjects must have the ability to understand and the willingness to sign a written informed consent document.

Who cannot join

Red cell alloimmunization to a degree that precludes extended transfusion
Patients with uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
Subjects must not have evidence of impaired liver function due to iron overload, +/- hepatitis. Patients will be evaluated by liver consult if ferritin >1500, history of hepatitis,or ALTis ≥3 X Upper limit of normal (ULN). Recommended evaluations could include liver biopsy if there is evidence for significant hepatic iron deposition or fibrosis/cirrhosis on T2* MRI of the liver.
eGFR <21 ml/min
≥2.0 liter-per-minute pm home oxygen requirement
An estimated Left Ventricular Ejection Fraction ≤40% (echo or MUGA)

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Hepatic cirrhosis (Biopsy Proven)
HIV positive, ineligible because of the increased risk of lethal infections when treated with marrow suppressive therapy. Appropriate studies will be undertaken in patients receiving combination antiretroviral therapy when indicated
Pregnant or breastfeeding women are excluded from this study because the immunomodulatory treatment, preparative regimen, and anti-GVHD therapy contain agents with the potential for teratogenic or abortifacient effects.
Evidence of uncontrolled bacterial, viral, or fungal infections (currently taking medication and progression of clinical symptoms) within 1 month prior to starting the conditioning regimen. Patients with fever or suspected minor infection should await resolution of symptoms before starting the conditioning regimen.
Prior allogeneic marrow or stem cell transplantation.

Trial locations and contacts

1 site in 1 country.

United States1 site

University Hospitals Seidman Cancer Center, Case Comprehensive Cancer Center
Cleveland, Ohio, 44106

Other trials in United States

About this study

This is a study of patients with sickle cell disease. It aims to find out if people with sickle cell disease can be cured by changing their immune system before they have blood stem cell transplants. Doctors will give patients a new drug (fludarabine) to see if this drug changes patients immune system and reduces the patient's cells (host) from rejecting donor cells (graft) after the patient gets a Hematopoietic (blood) stem cell transplant.

Read the detailed description

Primary Objective

To evaluate the safety and feasibility of hematopoietic stem cell transplant (HSCT) after treatment with fludarabine in adult patients with Sickle Cell Disease (SCD).

Secondary Objective(s), in HSCT for SCD

To evaluate the rates of disease-free and overall survival in both MSD and alternate graft donor (MUD, haploidentical, or cord blood-derived) recipients
To evaluate fertility in matched sibling and alternate-donor graft recipients
To evaluate GVHD rates in MSD and Alternate Graft Donor recipients in SCD.
To evaluate cerebral, pulmonary, renal, and generalized vasculopathy before and after HSCT in SCD.
To evaluate hematopoiesis and erythropoiesis before and before HSCT in SCD.
Modulation of SCD Phenotype by Allogeneic Transplantation. Rigorous clinical follow-up will be performed, per routine care, to evaluate those consequences of SCD that will be modified by allogeneic transplantation in the short-term (4-12 weeks), in the medium-term (12-24 weeks) and in the long-term (>24 weeks). Short-term changes would include disappearance of stress hematopoiesis and erythropoiesis; medium- and long-term changes would include effects on pain, fertility (TSH, LH), cognition (routine cognitive assessments), and end-organ damage (including urine albumin-to-creatinine ratios and tricuspid regurgitant jet velocities, as indicated).

Procedures:

The study will start with at least 10 and up to 25 patients. They will be given the lowest starting dose of fludarabine. This is done to make sure it is safe. Researchers will watch the patients during what is called the dose-limiting toxicity (DLT) period. Their safety will be monitored by a Safety Monitoring Committee, which is made up of people who run research studies. The study will not take new patients until the DLT period is done.

If at least 3 of the 10 patients enrolled do not benefit, the maximum tolerated dose (MTD) will be considered exceeded. After the DLT period is complete, patients will receive a stem cell transplant from a genetically matched donor. Patients will be continued to be monitored for a year after the transplant.

To prepare for the transplant patients will have to undergo the following treatments:

an exchange transfusion
a stem cell graft infusion from either a:
perfectly matched sibling donor (called MSD),
perfectly matched but unrelated donor (called MUD),
a half-matched related donor (called Haploidentical), or
a cord blood donor
rabbit antithymocyte globulin (ATG)
cytoxan (a type of chemotherapy)
Fludarabine (you get this medicine a few weeks before transplant and again, as part of the routine chemotherapy treatment). This is the main drug being studies in this research
total body irradiation (also called TBI)
tacrolimus, mycophenolate (MMF) and/or methotrexate (MTX). These drugs will weaken your immune system. They are given to lower your chances of getting GVHD and rejecting the donor cells.

Patients will be in the study for approximately 14 months.

Conditions: Sickle Cell Disease, Sickle Cell Anemia, SCD
Being tested: Fludarabine, Hematopoietic Stem Cell Transplant (HSCT)
Keywords: Sickle Cell Disease, Sickle Cell Anemia, GvHD, fludarabine
Medical subject headings: Anemia, Sickle Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

FludarabineDrug
Also called Fludarabine monophosphate
the study will begin with enrollment of an initial safety cohort of at least 10 subjects at the lowest dose, after which enrollment will pause until the dose-limiting toxicity (DLT) period has been completed. If a patient experiences DLT, defined as failure to engraft. In which case, the patient may be advanced to two higher doses.
Hematopoietic Stem Cell Transplant (HSCT)Procedure or surgery
Three weeks after Immunomodulation patients will be infused with matched bone marrow from a sibling, unrelated donor, haploidentical donor, or cord blood. Patients will be followed for the following year.

Study groups in NCT02065596
Study group	Role	What they receive
Immunomodulation with Fludarabine prior to HSCT	Experimental	Fludarabine given beginning at 25mgm/m2 three times per day. Patients may be escalated up to 25mgm/m2 five times per day depending on dose-limiting toxicity Receives: Drug: Fludarabine; Procedure: Hematopoietic Stem Cell Transplant (HSCT)

What is being measured

The primary outcome is the main question the study is designed to answer.

Probability of Engraftment
The number of patients with engraftment (as defined as recovery of ANC to 500 cells per cubic mm) compared to the total number of patients treated as a function of the patient's age.
Measured: 42 Days after transplant

Secondary outcomes (12)

Mean time to engraftment
The average time to for engraftment (as defined as recovery of ANC to 500 cells per cubic mm) to occur estimated using a Kaplan-Meier curve as a function of the patient's age.
Measured: 42 Days after transplant
Disease Progression
The average time (in days) to disease progression estimated using a Kaplan-Meier curve
Measured: 1 year
Overall Survival
The average time (in days) patients are alive after treatment estimated using a Kaplan-Meier curve
Measured: 1 year
Follicular Stimulating Hormone Levels
Estimated difference in changes in Follicular Stimulating Hormone Levels after transplant, among patients with matched sibling donors compared to patients with alternate donors.
Measured: 1 year
Luteinizing Hormone Levels
Estimated difference in changes in Luteinizing hormone Levels after transplant, among patients with matched sibling donors compared to patients with alternate donors.
Measured: 1 year
Testosterone Levels
Estimated difference in changes in Testosterone Levels after transplant, among male patients with matched sibling donors compared to patients with alternate donors.
Measured: 1 year
Graft versus Host Disease
The number patients with Grade III-IVGraft versus Host Disease as defined by CTCAE v4.0 in matched sibling compared to alternate-donor graft recipients
Measured: 1 year
Cerebral Vasculopathy
Vasculopathy may clinically manifest as a history of stroke. The difference in number of strokes before and after transplantation will be evaluated with paired statistical tests (t-test or McNemar).
Measured: 1 year
Renal Vasculopathy
Vasculopathy may clinically manifest as macroalbuminuria (≥300mg/g urinary albumin-to-creatinine ratio) or as a depressed estimated glomerular flow rate (eGFR). Evidence of renal vasculopathy before and after transplantation will be evaluated with paired statistical tests (t-test or McNemar).
Measured: 1 year
Pulmonary Vasculopathy
Vasculopathy may clinically manifest as pulmonary arterial systolic pressure (PASP) by echo. Difference in PASP levels before and after transplantation will be evaluated with paired statistical tests (t-test or McNemar).
Measured: 1 year
Hematopoiesis
Levels of stress hematopoiesis will be assessed by measuring telomere lengths with PCR before and after transplantation. These levels will be evaluated with paired statistical tests (t-test or McNemar).
Measured: 1 year
Erythropoiesis
Levels of stress erythropoiesis will be assessed by measuring telomere lengths with PCR before and after transplantation. These levels will be evaluated with paired statistical tests (t-test or McNemar).
Measured: 1 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

19 Oct 201517 Feb 2023

First posted19 Feb 2014 (estimated)
Study start19 Oct 2015
Primary completion17 Feb 2023
Study completion17 Feb 2023

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
More trials run by Case Comprehensive Cancer CenterThe 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 1
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 4 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02065596
Sponsor's protocol ID: CASE12Z13
Status last verified: Apr 2024
First submitted: 14 Feb 2014
Last updated: 29 Apr 2024
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

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All Sickle Cell Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 29 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Hematopoietic Stem Cell Transplant for Sickle Cell Disease
ClinicalTrials.gov ID: NCT02065596
Acronym / sponsor ID: CASE12Z13
Conditions: Sickle Cell Disease, Sickle Cell Anemia, SCD
Keywords: Sickle Cell Disease, Sickle Cell Anemia, GvHD, fludarabine
Phase: Phase 1
Study type: Interventional
Recruitment status: Completed
Enrolment: 4 participants (actual)
Lead sponsor: Case Comprehensive Cancer CenterUniversity, hospital or other
Study start: 19 Oct 2015 (actual)
Primary completion: 17 Feb 2023 (actual)
Study completion: 17 Feb 2023 (actual)
First posted: 19 Feb 2014 (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.

Detailed description

Primary Objective

To evaluate the safety and feasibility of hematopoietic stem cell transplant (HSCT) after treatment with fludarabine in adult patients with Sickle Cell Disease (SCD).

Secondary Objective(s), in HSCT for SCD

To evaluate the rates of disease-free and overall survival in both MSD and alternate graft donor (MUD, haploidentical, or cord blood-derived) recipients
To evaluate fertility in matched sibling and alternate-donor graft recipients
To evaluate GVHD rates in MSD and Alternate Graft Donor recipients in SCD.
To evaluate cerebral, pulmonary, renal, and generalized vasculopathy before and after HSCT in SCD.
To evaluate hematopoiesis and erythropoiesis before and before HSCT in SCD.
Modulation of SCD Phenotype by Allogeneic Transplantation. Rigorous clinical follow-up will be performed, per routine care, to evaluate those consequences of SCD that will be modified by allogeneic transplantation in the short-term (4-12 weeks), in the medium-term (12-24 weeks) and in the long-term (>24 weeks). Short-term changes would include disappearance of stress hematopoiesis and erythropoiesis; medium- and long-term changes would include effects on pain, fertility (TSH, LH), cognition (routine cognitive assessments), and end-organ damage (including urine albumin-to-creatinine ratios and tricuspid regurgitant jet velocities, as indicated).

Procedures:

To prepare for the transplant patients will have to undergo the following treatments:

an exchange transfusion
a stem cell graft infusion from either a:
perfectly matched sibling donor (called MSD),
perfectly matched but unrelated donor (called MUD),
a half-matched related donor (called Haploidentical), or
a cord blood donor
rabbit antithymocyte globulin (ATG)
cytoxan (a type of chemotherapy)
Fludarabine (you get this medicine a few weeks before transplant and again, as part of the routine chemotherapy treatment). This is the main drug being studies in this research
total body irradiation (also called TBI)
tacrolimus, mycophenolate (MMF) and/or methotrexate (MTX). These drugs will weaken your immune system. They are given to lower your chances of getting GVHD and rejecting the donor cells.

Patients will be in the study for approximately 14 months.

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Immunomodulation with Fludarabine prior to HSCT	Experimental	Drug: Fludarabine, Procedure: Hematopoietic Stem Cell Transplant (HSCT)Fludarabine given beginning at 25mgm/m2 three times per day. Patients may be escalated up to 25mgm/m2 five times per day depending on dose-limiting toxicity

Who may qualify

Ages: 18 to 65 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (42 listed)

Patients must have one of the following inherited hemoglobin gene disorders:
Low Risk (Red Light. Stop and consider therapy closely): Must have matched sibling donor grafts, failed conventional therapy as determined by the PI, and evidence for morbid disease (one of the following):
Moderate Risk (Yellow Light. Reasonable to proceed, but with caution): May have alternate donor grafts (haploidentical or matched unrelated donor), if MSD is not available. Must have history of high-level vasculopathy, as defined by at least one of the criteria below:
High Risk (Green light, proceed if possible): All donor types are eligible. Must have high risk disease and a >15% risk of 2-year mortality as defined by at least one of the criteria below.
Available suitable donor

Key exclusion criteria (11 listed)

Red cell alloimmunization to a degree that precludes extended transfusion
Patients with uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements
Subjects must not have evidence of impaired liver function due to iron overload, +/- hepatitis. Patients will be evaluated by liver consult if ferritin >1500, history of hepatitis,or ALTis ≥3 X Upper limit of normal (ULN). Recommended evaluations could include liver biopsy if there is evidence for significant hepatic iron deposition or fibrosis/cirrhosis on T2* MRI of the liver.
eGFR <21 ml/min
≥2.0 liter-per-minute pm home oxygen requirement

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
FludarabineAlso: Fludarabine monophosphatethe study will begin with enrollment of an initial safety cohort of at least 10 subjects at the lowest dose, after which enrollment will pause until the dose-limiting toxicity (DLT) period has been completed. If a patient experiences DLT, defined as failure to engraft. In which case, the patient may be advanced to two higher doses.	Drug	Immunomodulation with Fludarabine prior to HSCT
Hematopoietic Stem Cell Transplant (HSCT)Three weeks after Immunomodulation patients will be infused with matched bone marrow from a sibling, unrelated donor, haploidentical donor, or cord blood. Patients will be followed for the following year.	Procedure or surgery	Immunomodulation with Fludarabine prior to HSCT

Primary outcome

Probability of Engraftment
Time frame: 42 Days after transplant
The number of patients with engraftment (as defined as recovery of ANC to 500 cells per cubic mm) compared to the total number of patients treated as a function of the patient's age.

12 secondary outcomes

Mean time to engraftment
Time frame: 42 Days after transplant
Disease Progression
Time frame: 1 year
Overall Survival
Time frame: 1 year
Follicular Stimulating Hormone Levels
Time frame: 1 year
Luteinizing Hormone Levels
Time frame: 1 year
Testosterone Levels
Time frame: 1 year
Graft versus Host Disease
Time frame: 1 year
Cerebral Vasculopathy
Time frame: 1 year
Renal Vasculopathy
Time frame: 1 year
Pulmonary Vasculopathy
Time frame: 1 year
Hematopoiesis
Time frame: 1 year
Erythropoiesis
Time frame: 1 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 29 Apr 2024.

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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.

Molly Gallogly, MD, PhD principal investigator

Case Comprehensive Cancer Center

1 site in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

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 42 inclusion and 11 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 29 Apr 2024. 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 phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

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?

Probability of Engraftment (time frame: 42 Days after transplant). 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 NCT02065596.

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 29 Apr 2024.