Chronic Granulomatous DiseaseEarly Phase 1NCT05600907
Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomatous Disease (CGD)
A Early Phase 1 study testing JSP191 in people with Chronic Granulomatous Disease and CGD, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
Trial summary
Background: Chronic granulomatous disease (CGD) is a rare immune disorder that can cause serious infections throughout the body. The only cure for CGD is a stem cell transplant. Transplants from a sibling are best, but many people must get transplants from unrelated donors. Read more about this study
- Who is it for?
- Ages 4 to 65 years
- Conditions the study is for
- Chronic Granulomatous Disease, CGD
- What is being tested?
- JSP191
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 7 participants
- Where
- 1 siteUnited States
- Study dates
- 2 Jan 2023to 30 Nov 2036
- Phase
- Early Phase 1
- Are there results?
- Not yet: the study is under way
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 4 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Has confirmed CGD.
- Has sufficient complications from underlying disease to warrant undergoing transplantation (either a history of or ongoing inflammation/CGD-related autoimmunity OR a CGD-related infection while on prophylaxis) OR has a Quartile 1 or 2 residual oxidase production level.
- Aged 4 years to 65 years.
- Has an unrelated matched donor available (but no matched related donor available).
- Must be able to stay within 1 hours travel of the NIH for the first 3 months after transplantation and have a family member or other designated companion to stay with during the post-transplant period.
- Must provide a durable power of attorney for health care decisions to an appropriate adult relative or guardian in accordance with form NIH 200 NIH Durable Power of Attorney for Health Care Decision Making.
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- If of childbearing potential, must agree to consistently use contraception from 1 month prior to baseline, throughout study participation, and for 1 year after receiving transplanted cells. Acceptable forms of contraception are:
- Contraceptive pills or patch, Norplant, Depo-Provera, or other FDA-approved contraceptive method.
- Male partner has previously undergone a vasectomy.
- If able to impregnate a partner, must agree to consistently use contraception from the time of enrollment through 3 months post-transplant. Acceptable forms of contraception are:
- Male condom with spermicide.
Who cannot join
- Eastern Cooperative Oncology Group (ECOG) or equivalent performance status >= 3 (see Supportive Care guidelines, available at https://training.seer.cancer.gov/followup/procedures/dataset/ecog.html).
- Left ventricular ejection fraction < 40%.
- Transaminases > 5x upper limit of normal based on the individual s clinical situation and at the discretion of the investigator.
- CRP > 100 mg/dL within 6 weeks of the transplant.
- Psychiatric disorder or mental deficiency severe enough as to make compliance with the HSCT unlikely, and/or to make regulatorily and legally effective informed consent impossible.
- Major anticipated illness or organ failure incompatible with survival from allogeneic HSCT.
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- Pregnant or breastfeeding.
- HIV positive.
- Uncontrolled seizure disorder.
- Any condition or circumstance that the PI feels would create difficulty in maintaining compliance with the requirements of this protocol.
- Individuals who are not willing to submit their information as part of the alemtuzumab (Campath) Distribution Program application or participants whom the Distribution Program committee has determined are not qualified to receive alemtuzumab.
- NOTE: Alemtuzumab (IV formulation) is no longer distributed commercially. In order to receive product, the physician must contact the program for the patient. If the patient is not willing to consent to submit their information (demographics, contact information, and rationale for use) to the program such that we can obtain the drug, then we cannot proceed with conditioning; therefore, the individual will not be eligible for this protocol.
Trial locations and contacts
1 site in 1 country.
United States1 site
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892
About this study
Official title: Pilot Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomatous Disease (CGD)
Background:
Chronic granulomatous disease (CGD) is a rare immune disorder that can cause serious infections throughout the body. The only cure for CGD is a stem cell transplant. Transplants from a sibling are best, but many people must get transplants from unrelated donors. However, these transplants can cause serious complications in people with CGD.
Read more
Objective:
To see if a study drug (JSP191) can help improve the success rates of stem cell transplants for people with CGD from an unrelated donor.
Eligibility:
People aged 4 to 65 years with CGD who require a transplant.
Design:
Participants will be screened. Part of the screening will help to identify the best match to a transplant donor. Participants will have a physical exam, including dental and eye exams. They will have blood and urine tests. They will have tests of their breathing and heart function. A bone marrow sample will be taken. They will have their stem cells collected.
Participants will have a catheter inserted into a vein in their chest. It will remain in place for the entire period of transplant and recovery.
Participants will be in the hospital 40 to 50 days for the transplant. This will include a conditioning phase, to prepare their body for the procedure, as well as the transplant and recovery phases. As part of the conditioning phase, participants will receive JSP191 through a vein for 1 hour.
After discharge, participants will have follow-up visits 2 times a week for 100 days. Additional follow-up visits will continue for 5 years....
Read the detailed description
Study Description:
This is a pilot study for individuals with chronic granulomatous disease (CGD) using an unmanipulated matched unrelated donor (MUD) graft. The conditioning regimen will use alemtuzumab (Campath-1H, Genzyme Corporation) and total body irradiation (TBI) combined with the investigational new drug JSP191.
Participants will receive a high-dose donor graft infusion followed by post-transplant cyclophosphamide (Cytoxan, Baxter International, Inc). This study will determine whether replacing busulfan with the new investigational drug, JSP191, within the alemtuzumab/TBI-based conditioning regimen yields engraftment in patients with CGD.
Objectives:
Primary Objective:
- To measure the engraftment rates using JSP191 in conjunction with a high cell-dose graft.
Secondary Objectives:
- To assess overall survival.
- To evaluate the safety and efficacy of JSP191 in a TBI- and alemtuzumab-based regimen for transplantation of unmanipulated unrelated donor grafts in patients with CGD.
- Assessment of graft versus host disease (GvHD).
- Evaluate the long-term engraftment of those receiving JSP191.
- To assess the level and kinetics of immune reconstitution.
- To evaluate the pharmacokinetic (PK) properties of JSP191 in patients with CGD.
Exploratory Objectives:
- To assess the impact of inflammatory cytokines on engraftment, inflammatory reactions, and graft rejection.
- To further elucidate the factors involved in the development of GvHD and graft rejection/failure.
Endpoints:
Primary Endpoint:
- Reduced incidence of graft failure or rejection (as defined by >= 20% engraftment by oxidase-positive neutrophils in >= 85% of participants by Day 100, 1 year, and 2 years after transplant). Myeloid chimerism and/or oxidase positivity of >= 20% will be considered engrafted.
Secondary Endpoints:
- Overall survival.
- Safety will be evaluated by recording of adverse events (AEs) related to JSP191 rather than to GvHD or engraftment. An increase in incidence of AEs or development of AEs not typically seen in patients receiving a busulfan-based regimen will be attributed to the use of JSP191.
- Assessment of rates of GvHD.
- Rates of grade 3 and 4 acute GvHD (aGvHD) of < 20% (see Appendix B on grading).
- Development and incidence of aGvHD and chronic GvHD (cGvHD) with a comparison to historical controls and incidence on parallel protocols using busulfan.
- Engraftment at >2 years and up to 5 years post-transplant.
- Reconstitution kinetics using absolute neutrophil and absolute lymphocyte recovery kinetics will be compared as well as impact on immunoglobulin (Ig) levels.
- PK will be measured and compared to historical data supplied by Jasper Therapeutics, Inc. and from other patient populations on other studies at the NIH.
Exploratory Endpoints:
- Evaluation of inflammatory markers as risk factors for engraftment syndrome and/or graft failure.
- Obtain samples for cytokine profiles at various timepoints post-transplant to assess the impact on inflammation profiles by JSP191 in the setting of the conditioning regimen.
- Conditions
- Chronic Granulomatous Disease, CGD
- Being tested
- JSP191
- Keywords
- Transplant, Hsct, Monoclonal Antibody
- Medical subject headings
- Granulomatous Disease, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
JSP191Biological
A monoclonal antibody that targets human CD117
| Study group | Role | What they receive |
|---|---|---|
| Single arm study | Experimental | Eligible CGD patients will receive one time infusion of study product along with Alemtuzumab and TBI as part of HSCT conditioning regimen Receives: Biological: JSP191 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Early and long term engraftment
To determine the engraftment rates, both early and long term, when using JSP191 in conjunction with TBI, Alemtuzumab and post transplant cyclophosphamide and sirolimus for unrelated donor transplantation of patients with Chronic Granulomatous Disease.
Measured: Day 100 and 2 years post transplant
Secondary outcomes (3)
Incidence of viral reactivation
To compare the incidence of peri and post transplant complications including viral reactivation with this new regimen compared to historical data.
Measured: Through study completion about 104 weeks
Incidence of graft-vs-host disease (GvHD)
To determine and compare the rates and severity of GvHD.
Measured: Through study completion about 104 weeks
Safety
To determine and assess the toxicity of JSP191 in combination with Campath, TBI and post transplant cyclophosphamide.
Measured: Through study completion about 104 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Nov 2022
- Study start2 Jan 2023
- Primary completion30 Nov 2036 (estimated)
- Study completion30 Nov 2036 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Chronic Granulomatous DiseaseStudies of the same condition, open and under way.
- More trials run by National Institute of Allergy and Infectious Diseases (NIAID)The 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 7 participants (actual)
Sponsor and investigators
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Elizabeth M Kang, M.D., Principal investigator, National Institute of Allergy and Infectious Diseases (NIAID)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05600907
- Sponsor's protocol ID
- 10000609
- Other IDs
- 000609-I
- Status last verified
- 19 Aug 2026
- First submitted
- 26 Oct 2022
- Last updated
- 27 Aug 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No: Presently there is no plan to share individual data on this study.
Study at a glance
From the registry record, last updated by the sponsor on 27 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study to Assess the Use of JSP191 in Matched Unrelated Donor Transplantation for Chronic Granulomatous Disease (CGD)
- ClinicalTrials.gov ID
- NCT05600907
- Acronym / sponsor ID
- 10000609
- Conditions
- Chronic Granulomatous Disease, CGD
- Keywords
- Transplant, Hsct, Monoclonal Antibody
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 7 participants (actual)
- Lead sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)US National Institutes of Health
- Study start
- 2 Jan 2023 (actual)
- Primary completion
- 30 Nov 2036 (estimated)
- Study completion
- 30 Nov 2036 (estimated)
- First posted
- 1 Nov 2022 (actual)
- 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.
Background:
Chronic granulomatous disease (CGD) is a rare immune disorder that can cause serious infections throughout the body. The only cure for CGD is a stem cell transplant. Transplants from a sibling are best, but many people must get transplants from unrelated donors. However, these transplants can cause serious complications in people with CGD.
Objective:
To see if a study drug (JSP191) can help improve the success rates of stem cell transplants for people with CGD from an unrelated donor.
Eligibility:
People aged 4 to 65 years with CGD who require a transplant.
Design:
Participants will be screened. Part of the screening will help to identify the best match to a transplant donor. Participants will have a physical exam, including dental and eye exams. They will have blood and urine tests. They will have tests of their breathing and heart function. A bone marrow sample will be taken. They will have their stem cells collected.
Participants will have a catheter inserted into a vein in their chest. It will remain in place for the entire period of transplant and recovery.
Participants will be in the hospital 40 to 50 days for the transplant. This will include a conditioning phase, to prepare their body for the procedure, as well as the transplant and recovery phases. As part of the conditioning phase, participants will receive JSP191 through a vein for 1 hour.
After discharge, participants will have follow-up visits 2 times a week for 100 days. Additional follow-up visits will continue for 5 years....
Detailed description
Study Description:
This is a pilot study for individuals with chronic granulomatous disease (CGD) using an unmanipulated matched unrelated donor (MUD) graft. The conditioning regimen will use alemtuzumab (Campath-1H, Genzyme Corporation) and total body irradiation (TBI) combined with the investigational new drug JSP191.
Participants will receive a high-dose donor graft infusion followed by post-transplant cyclophosphamide (Cytoxan, Baxter International, Inc). This study will determine whether replacing busulfan with the new investigational drug, JSP191, within the alemtuzumab/TBI-based conditioning regimen yields engraftment in patients with CGD.
Objectives:
Primary Objective:
- To measure the engraftment rates using JSP191 in conjunction with a high cell-dose graft.
Secondary Objectives:
- To assess overall survival.
- To evaluate the safety and efficacy of JSP191 in a TBI- and alemtuzumab-based regimen for transplantation of unmanipulated unrelated donor grafts in patients with CGD.
- Assessment of graft versus host disease (GvHD).
- Evaluate the long-term engraftment of those receiving JSP191.
- To assess the level and kinetics of immune reconstitution.
- To evaluate the pharmacokinetic (PK) properties of JSP191 in patients with CGD.
Exploratory Objectives:
- To assess the impact of inflammatory cytokines on engraftment, inflammatory reactions, and graft rejection.
- To further elucidate the factors involved in the development of GvHD and graft rejection/failure.
Endpoints:
Primary Endpoint:
- Reduced incidence of graft failure or rejection (as defined by >= 20% engraftment by oxidase-positive neutrophils in >= 85% of participants by Day 100, 1 year, and 2 years after transplant). Myeloid chimerism and/or oxidase positivity of >= 20% will be considered engrafted.
Secondary Endpoints:
- Overall survival.
- Safety will be evaluated by recording of adverse events (AEs) related to JSP191 rather than to GvHD or engraftment. An increase in incidence of AEs or development of AEs not typically seen in patients receiving a busulfan-based regimen will be attributed to the use of JSP191.
- Assessment of rates of GvHD.
- Rates of grade 3 and 4 acute GvHD (aGvHD) of < 20% (see Appendix B on grading).
- Development and incidence of aGvHD and chronic GvHD (cGvHD) with a comparison to historical controls and incidence on parallel protocols using busulfan.
- Engraftment at >2 years and up to 5 years post-transplant.
- Reconstitution kinetics using absolute neutrophil and absolute lymphocyte recovery kinetics will be compared as well as impact on immunoglobulin (Ig) levels.
- PK will be measured and compared to historical data supplied by Jasper Therapeutics, Inc. and from other patient populations on other studies at the NIH.
Exploratory Endpoints:
- Evaluation of inflammatory markers as risk factors for engraftment syndrome and/or graft failure.
- Obtain samples for cytokine profiles at various timepoints post-transplant to assess the impact on inflammation profiles by JSP191 in the setting of the conditioning regimen.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single arm study | Experimental | Biological: JSP191Eligible CGD patients will receive one time infusion of study product along with Alemtuzumab and TBI as part of HSCT conditioning regimen |
Who may qualify
- Ages
- 4 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- Has confirmed CGD.
- Has sufficient complications from underlying disease to warrant undergoing transplantation (either a history of or ongoing inflammation/CGD-related autoimmunity OR a CGD-related infection while on prophylaxis) OR has a Quartile 1 or 2 residual oxidase production level.
- Aged 4 years to 65 years.
- Has an unrelated matched donor available (but no matched related donor available).
- Must be able to stay within 1 hours travel of the NIH for the first 3 months after transplantation and have a family member or other designated companion to stay with during the post-transplant period.
Key exclusion criteria (12 listed)
- Eastern Cooperative Oncology Group (ECOG) or equivalent performance status >= 3 (see Supportive Care guidelines, available at https://training.seer.cancer.gov/followup/procedures/dataset/ecog.html).
- Left ventricular ejection fraction < 40%.
- Transaminases > 5x upper limit of normal based on the individual s clinical situation and at the discretion of the investigator.
- CRP > 100 mg/dL within 6 weeks of the transplant.
- Psychiatric disorder or mental deficiency severe enough as to make compliance with the HSCT unlikely, and/or to make regulatorily and legally effective informed consent impossible.
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 |
|---|---|---|
| JSP191A monoclonal antibody that targets human CD117 | Biological | Single arm study |
Primary outcome
Early and long term engraftment
Time frame: Day 100 and 2 years post transplant
To determine the engraftment rates, both early and long term, when using JSP191 in conjunction with TBI, Alemtuzumab and post transplant cyclophosphamide and sirolimus for unrelated donor transplantation of patients with Chronic Granulomatous Disease.
3 secondary outcomes
Incidence of viral reactivation
Time frame: Through study completion about 104 weeks
Incidence of graft-vs-host disease (GvHD)
Time frame: Through study completion about 104 weeks
Safety
Time frame: Through study completion about 104 Weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Aug 2026.
- NIH Clinical Center Detailed Web PageLink provided by the sponsor.
- Individual participant dataSharing planned: No. Presently there is no plan to share individual data on this study.
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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.
Elizabeth M Kang, M.D. principal investigator
National Institute of Allergy and Infectious Diseases (NIAID)
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing JSP191 in people with Chronic Granulomatous Disease and CGD, run by National Institute of Allergy and Infectious Diseases (NIAID) at 1 site in United States.
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 11 inclusion and 12 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: under way, not looking for participants, as of the sponsor's last update on 27 Aug 2026. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
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?
Early and long term engraftment (time frame: Day 100 and 2 years post transplant). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 27 Aug 2026.