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Protocol Details

A Phase 1/2 Study to Evaluate the Safety, Tolerability and Efficacy, of JSP191 for Hematopoietic Cell Transplantation Conditioning to Achieve Engraftment and Immune Reconstitution in Subjects with SCID

This study is currently recruiting participants.

Summary | Eligibility | Citations | Contacts




Sponsoring Institute

National Institute of Allergy and Infectious Diseases (NIAID)

Recruitment Detail

Type: Participants currently recruited/enrolled
Gender: Male & Female
Min Age: 3 Years
Max Age: N/A

Referral Letter Required


Population Exclusion(s)




Recruitment Keyword(s)



Severe Combined Immunodeficiency (SCID)

Investigational Drug(s)


Investigational Device(s)



Biological/Vaccine: JSP191

Supporting Site

National Institute of Allergy and Infectious Diseases


For people with severe combined immune deficiency (SCID), their immune system does not work properly. The only cure for most SCIDs is stem cell transplant. These transplants can have serious complications. Researchers want to see if the drug JSP191 can help.


To learn if the JSP191 monoclonal antibody is safe and effective for stem cell transplant conditioning for people with certain types of SCID.


People ages 3 and older who have SCID and have received stem cell transplant but need a retransplant. Their original donor will provide the new sample.


Participants will be screened with:

Physical exam

Eye exam

Blood and heart tests


Bone marrow sample


Participants will be admitted to the hospital. They will receive a central venous catheter if they do not have one. They will receive the JSP191 infusion through a vein. About 10-21 days later, they will have a bone marrow biopsy. Then they will have a stem cell transplant.

Participants will stay at the hospital for 30 days after transplant to recover. They must remain local to the hospital for 8 weeks after discharge for monitoring. They will have several blood tests.

After the 8 weeks, participants will have follow-up visits 1-2 times a year for 5 years. These will include repeats of the screening tests.

Donors will have visits 6 days in a row. They will be injected with a drug to help them make more stem cells. They will donate the cells via apheresis. For this, blood will be drawn and circulated through a machine over 2-6 hours.

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1. Typical SCID as defined by Primary Immune Deficiency Treatment Consortia (1A) (Shearer, Dunn et al. 2014) (PIDTC) including but not limited to the following subtypes:

a. T-, B+, NK-: IL-2Rcy deficient, JAK3-deficient

b. T-, B-, NK+: RAG1/2 deficient, Artemis-deficient

c. T-, B+, NK+: IL7Ralpha deficient, CD3 subunit deficient, CD45 deficient


Variant SCID with absent or low T cell function, Omenn syndrome, Leaky SCID, Reticular dysgenesis, Adenosine deaminase deficiency, and Purine nucleoside phosphorylase deficiency may be included after consultation with the medical monitor.

2. Organ function defined as:

a. Serum Creatinine < 1.5 x upper limit of normal (ULN) or 24-hour creatinine clearance >= 50 mL/min

b. Forced expiratory volume in 1 second (FEV1), forced vital capacity (FVC), and diffusing capacity of the lung for carbon monoxide (DLCO) corrected for hemoglobin and volume, > 40% predicted by pulmonary function tests (PFTs)

c. For subjects unable to cooperate for PFTs, criteria are no evidence of dyspnea at rest, no exercise intolerance, and no requirement for supplemental oxygen

d. Shortening fraction of >= 27% or ejection fraction of (Bullet) 50% by echocardiogram

e. Serum total bilirubin of < 2 x ULN, unless attributable to Gilbert's syndrome

f. Alanine aminotransferase (ALT) or aspartate aminotransferase (AST) <= 5 x ULN

g. Absolute neutrophil count > 500 per microliter of blood

h. Platelet count > 100,000 per microliter of blood

i. Hemoglobin > 10 grams/deciliter

3. Life expectancy of at least 8 weeks

4. Female subjects of childbearing potential and sexually mature male subjects willing to use an effective contraceptive method for the duration of study participation

5. Subjects and/or parents or legal guardians willing to provide written informed consent

6. Authorize use and disclosure of personal health information in accordance with Health Insurance Portability and Accountability Act

Inclusion Criteria Specific to Group A:

1. Prior donor of appropriate age (>= 5 years old) available for re-collection of stem cells by apheresis

2. Previous allogeneic HCT (>= 6 months post initial transplant) with poor graft function defined as one of the following:

a. Inadequate B cell engraftment, defined by any of the following parameters:

i. Absence (< 5%) of donor B cells

ii. IgA and IgM < normal values for age

iii. Isohemagglutinin titer < 1:8

iv. Anti-tetanus antibody titers post-vaccination < 3 x pre-vaccination titer or below protective level off Ig replacement for 3 months OR persistent severe hypogammaglobulinemia requiring routine immunoglobulin replacement

b. Incomplete T cell reconstitution, defined as any one of the following criteria:

i. CD3+ cells < 600/uL

ii. CD4+ cells < 200/uL

iii. Phytohemagglutinin response < 30% lower limit of normal

c. Severe clinical symptoms explained by poor immunity defined as one of the following criteria:

i. Autoimmunity unrelated to chronic GVHD

ii. Hospitalizations more than 1 per year for infection or courses of antimicrobial treatment >= 3 per year over the preceding 2 years

iii. Chronic viral infection.

3. Prothrombin time international normalized ratio (PT INR) and partial thromboplastin time (PTT) <= 1.5 x ULN

4. No evidence of stringent donor myeloid chimerism in peripheral blood by STR analysis. These stringent donor myeloid chimerism analyses will be performed, at a central lab, on flow-cytometric sorted CD15+ myeloid cells, followed by STR PCR-based assay. Due to a potential for stringent myeloid chimerism assay (flow-cytometric sorting and PCR) variability (+/- 1%), subjects with stringent donor myeloid chimerism of 1% may be enrolled in the trial with medical monitor approval.

Inclusion criteria specific to Group B:

1. Age >= 3 months SCID, without history of allogeneic HCT

2. Haploidentical donor of appropriate age (>= 5 years old) or HLA-matched non-sibling donor (8/8 at allelic level at HLA-A, -B, -C, and -DR), or HLA-matched sibling donor for subjects >= 18 months of age available for apheresis

a. For NK+ SCID subjects who have transplacental maternal engraftment (TME), the mother is the preferred donor. Alternative donors may be considered after consultation with the medical monitor.

b. For RAG 1/2-deficient and Artemis-deficient subjects who have TME, the mother is the preferred donor. Alternative donors may be considered after consultation with the medical monitor.

c. TME is defined as evidence of maternal genotype in isolated CD3 cells analyzed by polymerase chain reaction (PCR), short-tandem-repeat (STR) analysis, or variable number tandem repeats (VNTR) based assays.


1. Subjects with any acute or uncontrolled infections.

Acute infection will refer to a bacterial, fungal or viral infection newly acquired within 1 month of enrollment that is uncontrolled as defined by signs and symptoms of the infection not improving or getting worse over that month. Uncontrolled infection, whether acute or chronic as an exclusion criterion will be defined as any infection of a severity or with acute progression in the month prior to enrollment that in the opinion of the PI poses an unacceptable risk to a subject undergoing conditioning and transplant. Acute infections that have resolved with or without specific treatment by the time of enrollment are not exclusions.

2. Subjects receiving any other investigational agents

3. Subjects with active malignancies

4. Pregnant women

5. Women who are nursing and do not wish to discontinue breast feeding

6. Lansky/Karnofsky performance score < 50%

7. Active GVHD within 6 months prior to enrollment or on immunosuppressive therapy for GVHD.

8. Any other medical condition that, in the opinion of the Principal Investigator, could pose a significant safety risk to the subject or jeopardize the integrity of the study.

9. Subjects, who in the opinion of the Principal Investigator, may not be able to comply with the safety monitoring requirements of the study.

Exclusion criteria specific to Group B:

1. Subjects <18 months old with an HLA-matched sibling eligible to donate hematopoietic cells

Donor Inclusion Criteria

1. For Group A, only the original donor will be acceptable.

2. For Group B, donors must be HLA-haploidentical relative or an HLA-matched non-sibling donor or an HLA-matched sibling donor for subjects >= 18 months of age.

3. HLA-matched and HLA-haploidentical relative donors must be willing to undergo granulocyte colony stimulating factor (G-CSF) and Plerixafor administration and multiple apheresis procedures.

Donor Exclusion Criteria

1. Evidence of medical conditions (including a severe existing autoimmune disorder) that preclude them from donation.

2. Any contraindication to collection by apheresis of 20 x 108 mobilized mononuclear cells.

3. Live/attenuated vaccination within 2 months prior to mobilization of peripheral blood stem cells.

4. For Group B only, HLA-matched sibling donors for subjects less than 18 months of age are excluded.

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Not Provided

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Principal Investigator

Referral Contact

For more information:

Harry L. Malech, M.D.
National Institute of Allergy and Infectious Diseases (NIAID)
NIHBC 10 - CRC BG RM 5-3750
(301) 480-6916

Sandra Anaya-O'Brien, R.N.
National Institute of Allergy and Infectious Diseases (NIAID)
National Institutes of Health
Building 10
Room 5-3732
10 Center Drive
Bethesda, Maryland 20892
(301) 346-9781

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: TTY Users Dial 7-1-1

Clinical Trials Number:


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