Is NCT04232085 recruiting in Baltimore, MD?

Yes, NCT04232085 is currently recruiting participants at its Baltimore, MD location. This primary immune deficiency disorder clinical trial is actively enrolling qualified participants.

Is participation in this Baltimore clinical trial free?

Yes, all study-related care is provided at no cost to qualified participants. Many studies also offer compensation for time and travel expenses.

Primary Immune Deficiency Disorder Clinical Trial in Baltimore, MD

Q: How do I join the primary immune deficiency disorder clinical trial in Baltimore?

To join this clinical trial in Baltimore, complete the eligibility form on this page. A study coordinator will contact you to discuss next steps and determine if you qualify.

About This Primary Immune Deficiency Disorder Study in Baltimore

Phase II prospective trial to assess the rates of donor engraftment using reduced intensity conditioning (RIC) hematopoietic stem cell transplant (HSCT) and post-transplant cyclophosphamide (PTCy) for patients with primary immune deficiencies (PID), immune dysregulatory syndromes (IDS), inherited bone marrow failure syndromes (IBMFS), short telomere syndromes, Fanconi anemia, and non-Fanconi DNA double-strand break (DNA-dsb) repair disorder.

Sponsor: Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins

Who Can Participate

Inclusion Criteria

Cohort A: Primary Immune Deficiencies with indication for HCT:

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

DOCK8 deficiency Immune Dysregulatory Syndromes:

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)

Increased DNA breakage after exposure of patient cells to DNA cross-linking agents such as diepoxybutane or mitomycin C and germline mutation(s) in an identified Fanconi pathway gene. Available donor as follows:

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. A minimum match of 5/10 is therefore required, and will be considered sufficient evidence that the donor and recipient share one HLA haplotype.

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 \<13 years, shortening fraction (SF) \> 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.

Exclusion Criteria

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. The investigators recognize that patients with CAEBV may have ongoing EBV viremia at the time of initiating pre-transplant therapy, but other patients should have no uncontrolled bacterial, viral, or fungal infections.

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 the graft. Cord blood is not permitted.

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. This will be decided on a case-by-case basis by the PI and one of the immunogenetics directors.

In inherited disorders, family members must be tested for carrier and disease status of the underlying disorders. In the event that family members are unaffected carriers, eligibility as donors will be decided upon by the PI on a case-by-case basis

In the event that two or more eligible donors are identified, the donor will be selected per institutional standards. Suggested criteria include the following:

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.

Red blood cell compatibility, in order of preference:

RBC cross match compatible Minor ABO incompatibility, Major ABO incompatibility

If the patient is male, male donors are preferred.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Baltimore?

Yes, this clinical trial (NCT04232085) has an active research site in Baltimore, MD that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Primary Immune Deficiency Disorder Treatment Options in Baltimore, MD

If you're searching for primary immune deficiency disorder treatment options in Baltimore, MD, this clinical trial (NCT04232085) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Baltimore research site is actively enrolling participants for this phase 2 clinical trial. You'll receive care from experienced primary immune deficiency disorder specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all primary immune deficiency disorder clinical trials near you to find additional studies recruiting in your area.

Primary Immune Deficiency Disorder Clinical Trial in Baltimore, MD

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