JSP191 Antibody Targeting Conditioning in SCID Patients
Stopped: Sponsor Decision - terminated due to changes in company priorities and not related to safety concerns.
United States23 participantsStarted 2017-03-20
Plain-language summary
A Phase 1/2 study to evaluate the safety, tolerability, and efficacy of an antibody conditioning regimen, known as JSP191, in patients with Severe Combined Immune Deficiency undergoing blood stem cell transplantation
Who can participate
Age range
3 Months – 12 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Typical SCID as defined by Primary Immune Deficiency Treatment Consortia including but not limited to the following subtypes:
. T-, B+, NK-: IL-2Rcγ deficient, JAK3-deficient (no longer enrolling)
. T-, B+, NK+: IL7Rα deficient, CD3 subunit deficient, CD45 deficient (no longer enrolling) OR 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.
. Patients with human leukocyte antigen (HLA) matched related or unrelated donors
. Adequate end organ function as defined in study protocol
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Phase 1: Safety and tolerability of JSP191 as conditioning therapy in SCID patients undergoing HCT: adverse events
Timeframe: Up to 5 years post Donor Cell Transplant (28 days dose limiting toxicity period)
2
Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients
Timeframe: Up to 24 weeks post Donor Cell Transplant
3
Phase 2: Efficacy of JSP191 as conditioning therapy in SCID patients
Timeframe: Weeks 36-104 post Donor Cell Transplant