Clinical trial for Primary Immune Deficiency Disorder
Official title Regenerative Medicine to Restore Hematopoiesis and Immune Function in Immunodeficiencies and Inherited Bone Marrow Failures
ClinicalTrials.gov ID: NCT04232085
What this study is testing
What is Alemtuzumab?
An investigational drug being studied as part of a reduced‑intensity conditioning regimen for stem cell transplant in patients with primary immune deficiencies.
Also referred to as Campath.
Plain-language explanation of the investigational treatment. It is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- The study will see how well donor stem cells take hold after a gentler transplant and a special drug given after the transplant.
- Who it's for
- It looks for adults or children who have a primary immune deficiency, an inherited bone‑marrow failure, or a short‑telomere syndrome such as Fanconi anemia.
- What's involved
- Participants will receive a reduced‑intensity stem‑cell transplant from a donor and then take cyclophosphamide after the transplant. They will have regular blood tests and clinic visits to monitor the transplant’s success.
- Time commitment
- The trial lasts about 12 months, with visits before the transplant, during the first 100 days after, and at regular intervals up to a year.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 4 to 50
You may be able to join if
- Chronic granulomatous disease (CGD)
- Wiskott-Aldrich syndrome (WAS)
- Hyper-IgM syndrome
- Common variable immunodeficiency (CVID)
- Leukocyte adhesion deficiency-1 (LAD-1)
You likely can't join if
- Patients will not be excluded on the basis of sex, racial or ethnic background.
- Positive leukocytotoxic crossmatch.
- Prior allogeneic stem cell transplant.
- Uncontrolled bacterial, viral, or fungal infection at the time of enrollment.
- Diagnosis of idiopathic aplastic anemia
- Seropositivity for the human immunodeficiency virus (HIV)
See the full eligibility criteria
- Chronic granulomatous disease (CGD)
- Wiskott-Aldrich syndrome (WAS)
- Hyper-IgM syndrome
- Common variable immunodeficiency (CVID)
- Leukocyte adhesion deficiency-1 (LAD-1)
- Severe Combined Immunodeficiency (SCID)
- CTLA-4 deficiency
- CARD9 deficiency
- Immunodysregulation polyendocrinopathy enteropathy X-linked (IPEX) syndrome
- Hemophagocytic lymphohistiocytosis (HLH) or related disorder with indication for transplant
- CAEBV: Patients with chronic EBV infection (CAEBV) with indication for BMT: Inherited Bone marrow failure disorders
- Congenital amegakaryocytic thrombocytopenia (CAMT)
- Diamond Blackfan anemia (DBA)
- Shwachman Diamond Syndrome (SDS)
- Thrombocytopenia Absent Radii (TAR)
- Glanzmans thrombasthenia (GT)
- Kostmann syndrome
- Other indications and/or other PID, IDS, and IBMFS diagnoses as deemed appropriate by the PI. Cohort B: Short telomere syndrome Cohort C: Confirmed diagnosis of Fanconi anemia or non-Fanconi DNA-dsb repair disorders
- Fanconi anemia
- Non-Fanconi DNA-dsb repair disorders
- Cerunnos-XRCC4-like factor deficiency (XLF or NHEJ1)
- DNA ligase IV deficiency (LIG4)
- Nijmegen breakage syndrome (NBS)
- Fully HLA matched sibling or other first-degree family member.
- Fully HLA matched unrelated 10/10 donor using high-resolution DNA-based typing at the following genetic loci: HLA-A, -B, -C, DRB1, and DQB1.
- Mismatched unrelated donor at 8 or 9/10 alleles, using high-resolution typing as above.
- HLA-haploidentical family members of any degree who match at least one allele of each of the following genetic loci: HLA-A, -B, -C, DRB1, and DQB1.
- The patient and/or legal guardian must sign informed consent for BMT.
- Patients with adequate organ function as measured by
- Cardiac: Left ventricular ejection fraction (LVEF) at rest must be ≥ 35%. For patients aged \ 25% by echocardiogram or LVEF by MUGA may be used.
- Hepatic: Bilirubin ≤ 3.0 mg/dL; and ALT, AST, and Alkaline Phosphatase \< 5 x ULN.
- Renal: Serum creatinine within normal range for age, or if serum creatinine outside normal range for age, then renal function (creatinine clearance or GFR) \> 40 mL/min/1.73m2.
- Pulmonary: PFT with FEV1 and FVC \>/= 50% of normal and DLCO corrected for Hgb \>/= 40% of normal. Patients unable to undergo PFTs should have stable resp status with SaO2 \>90% on a max of 2L/min supplemental O2.
- Karnofsky or Lansky performance status ≥70%
- Females and males of childbearing potential must agree to practice 2 effective methods of contraception at the same time, or agree to abstinence.
- Patients will not be excluded on the basis of sex, racial or ethnic background.
- Positive leukocytotoxic crossmatch.
- Prior allogeneic stem cell transplant.
- Uncontrolled bacterial, viral, or fungal infection at the time of enrollment. Uncontrolled is defined as currently taking medication and with progression or no clinical improvement on adequate medical treatment.
- Diagnosis of idiopathic aplastic anemia
- Seropositivity for the human immunodeficiency virus (HIV)
- Active Hepatitis B or C determined by serology and/or NAT
- Female patients who are diagnosed as pregnant by beta bHCG testing (per institutional practice) or who are breast-feeding.
- Active malignancy or within the timeframe for significant concern for relapse of prior malignancy
- For Cohort B and C: liver biopsy (if performed, not required) with moderate-severe fibrosis/cirrhosis Donor Eligibility
- Donor must be medically, socially, and psychologically fit to donate
- Bone marrow is the preferred graft source, however, PBSCs may be requested. In particular, PBSCs may be preferred for patients with active viral reactivations and/or for patients who would benefit from a higher count in...
- First-degree relatives should be tested for degree of HLA match, CMV serology, ABO type, and complete blood count (CBC). An unrelated donor search should be initiated at the time the patient is referred for BMT.
- Age ≥5 years
- Donors must meet the selection criteria as defined by the Foundation for the Accreditation of Hematopoietic Cell Therapy (FACT).
- Lack of recipient anti-donor HLA antibody in recipient
- Note: In some instances, low level, non-cytotoxic HLA specific antibodies may be permissible if found to be at a level well below that detectable by flow cytometry.
- In inherited disorders, family members must be tested for carrier and disease status of the underlying disorders.
- Related is preferred over unrelated.
- The potential donor that is youngest in age is preferred.
- For CMV seronegative patients, a CMV seronegative donor is preferred. For CMV seropositive patients, a CMV seropositive donor is preferred.
- RBC cross match compatible Minor ABO incompatibility, Major ABO incompatibility
- If the patient is male, male donors are preferred.
The study team makes the final eligibility decision.
Where it is enrolling
- Baltimore, Maryland, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part, but confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 4 months to 50 years. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Baltimore, Maryland, United States. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.