JAK1 GOFPhase 1/2SAFE-JAKiNCT07670156
Upadacitinib in Treatment of JAK/STAT Pathway Disorders With Activating Mutations
A Phase 1/2 study testing Upadacitinib in people with JAK1 GOF and STAT1 GOF, run by Lisa Satter at 3 sites in United States.
Trial summary
This study focuses on a genetic condition that affects the Janus Kinase-Signal Transducer and Activator of Transcription (JAK-STAT) immune signaling pathway. A specific change in the DNA leads to overactivation of this pathway, which can result in immune dysregulation and related clinical symptoms. Read more about this study
- Who is it for?
- Ages 12 to 65 years
- What is being tested?
- Upadacitinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 30 participants
- Where
- 3 sitesUnited States
- Study dates
- Oct 2026to Aug 2028
- Phase
- Phase 1/2
- 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 12 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- At least 30kg
- Patients with a confirmed JAK/STATGOF mutation with evidence of immune dysregulation
- Current disease status meeting criteria as defined in the disease scoring module
- Expected survival of more than 12 months
- Willingness to allow storage of biological samples for future research
- Agreement to use highly effective contraception (for female participants)
Who cannot join
- Hypersensitivity to the study drug or any medication in the same class
- Active infections
- Central nervous system (CNS) manifestations
- Pregnancy
- Medical conditions or use of concomitant medications that may interfere with the effect or evaluation of the study drug
- Laboratory abnormalities as specified in the protocol
- Receipt of a live vaccine within 30 days prior to study treatment
- High risk or history of osteoporosis, thrombosis, gastrointestinal (GI) perforation, or malignancy
Trial locations and contacts
3 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States3 sites
Baylor College of Medicine
Houston, Texas, 77030
Daisy Vita, 8328241319, dxtran1@texaschildrens.org
Supriya Parikh, 8328242589, Supriya.Parikh@bcm.edu
Columbia University
New York, New York, 10032
Joseph Oved, MD, 332-292-7978, jho2001@cumc.columbia.edu
Joseph Oved, MD
Washington University in St.Louis
St Louis, Missouri, 63110
Megan Cooper, MD, PhD, 314-286-0262, cooper_m@wustl.edu
Megan Cooper, MD, PhD
About this study
Official title: Safety and Efficacy of Upadacitinib in Treatment of JAK/STAT Pathway Disorders With Activating Mutations
This study focuses on a genetic condition that affects the Janus Kinase-Signal Transducer and Activator of Transcription (JAK-STAT) immune signaling pathway. A specific change in the DNA leads to overactivation of this pathway, which can result in immune dysregulation and related clinical symptoms.
Currently, five genetic mutations are known to cause these JAK-STAT pathway driven immune disorders: STAT1, STAT3, STAT5B, STAT6, and JAK1 (collectively referred to as JAK-STAT disorders). This study is a basket clinical trial, meaning patients with these different but related genetic conditions are enrolled in the same study and treated with the same investigational therapy.
Read more
The purpose of this study is to evaluate the safety and tolerability (ability to tolerate) of a drug called Upadacitinib in patients with JAK-STAT disorders with activating mutations. This drug belongs to a class of drug called Janus kinase (JAK) inhibitors, also known as JAKinibs. It is a type of immune system modulating medication that regulates (fixes) the JAK- STAT signaling pathway. Upadacitinib has been approved by the FDA for multiple immunological diseases and disorders. Presently, there is no FDA approved treatment for this group of JAK-STAT disorders. The study will also investigate immune factors in the blood to develop diagnosis methods that can be used in the future for better medical management of these disorders.
The study consists of four phases: screening phase, open label phase, randomized withdrawal phase and maintenance phase. The study will last approximately 12 months. While in the study, participants will receive a once daily dose of Upadacitinib that best helps control their disease. During the study participants will be asked to answer questions about their health and medical history. They will also complete physical exams, blood tests, and other questionnaires.
Read the detailed description
This is a phase 1/2, multi-center trial with an open label dose-escalation phase (OL) followed by a double-blind placebo-controlled randomized withdrawal phase (RW) to study the safety, tolerability, and preliminary efficacy of Upadacitinib in patients with confirmed and symptomatic gain of function mutations in the JAK-STAT pathway. Upon completion of the randomized withdrawal phase, each patient will enter the maintenance phase (MP) where all patients will receive Upadacitinib.
OL Phase (16 weeks): The subject will be escalated two times if tolerated and clinically indicated based upon disease response. The starting dose will be 15 mg per day for patients greater than or equal to 12 years of age (and at least 30 kg in weight). The subject will remain at this dose level for 28 days. The subject will visit the clinical site on day 1 of each dose escalation visit and then will be remotely monitored (phone call and laboratory monitoring to check complete blood counts) on Day 3 and Day 10.
Patients must have a disease status score of 2 or higher utilizing the Primary Immune Regulatory Deficiency (PIRD) Disease Modules prior to initiation of dose-escalation of the drug. The PIRD score is a disease scoring method that capture the clinical manifestations of all PIRDs including JAK/STAT GOF disorders. The PIRD scores for clinical improvement across organ systems for each patient will standardize the ability to quantify improvement across divergent disease phenotypes and assess outcomes based on clinical improvement within each disorder. Patients who demonstrate a response to study treatment, as determined by improvement in PIRD score, may proceed to the RW Phase. Patients who do not meet eligibility criteria for the RW Phase will enter MP.
During the OL Phase, the optimal treatment dose (OTD) for each patient will be determined. OTD will be defined as the dose that achieved complete response or partial response based on the PIRD scores.
RW Phase (8 weeks): For the double-blind randomized withdrawal phase, patients will be randomized to receive either the Optimum tolerated dose (OTD) dose from the OL phase or Placebo using a 1:1 randomization ratio. Subject will come to the study site every 4 weeks. The subject can discontinue the RW phase if he/she experiences a flare (worsening of disease symptoms) and will enter the Maintenance Phase outlined below. For measuring disease flare Primary Immune Regulatory Disorders (PIRD) score will be used. It will be deemed a flare if the score increases by ≥1 in disease manifestation. During the RW-phase, the reference for a disease flare (for the primary endpoint assessment) will be the end of the OL phase PIRD score.
MP Phase (28 weeks): Subject will be monitored closely for any drug related toxicities (blood counts, lipid and metabolic panels) and continue to take the highest tolerated dose level attained during the escalation phase. The subject will return to the clinical site on day 168, day 217, day 266, day 315 and day 364. Upon conclusion of the maintenance phase at day 364 the study team will safely transition the subject to clinically viable treatment alternatives that are commercially available (after a 3 days washout period for Upadacitinib).
- Conditions
- JAK1 GOF, STAT1 GOF, STAT3 GOF, STAT5B GOF, STAT6 GOF
- Being tested
- Upadacitinib, Placebo
- Keywords
- Upadacitinib, JAK-STAT Pathway Disorders, Immunodeficiency, Primary Immune Regulatory Disorder (PIRD), Inborn Error of Immunity (IEI), early onset autoimmunity, autoimmune cytopenia, immune dysregulation, STAT1, STAT3, lymphoproliferation, STAT5b, STAT6, JAK1
- Medical subject headings
- Immunologic Deficiency Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
UpadacitinibDrug
Also called Rinvoq
Participants will receive the dose that worked best for them (optimal treatment dose, OTD) during the earlier open-label phase of the study, which may be 15 mg, 30 mg, or 45 mg taken once daily. The study drug will be taken by mouth as tablets.
PlaceboDrug
Participants assigned to the placebo group will receive placebo tablets taken once daily. The placebo will be matched to the participant's optimal treatment dose (OTD) determined during the earlier open-label phase of the study.
| Study group | Role | What they receive |
|---|---|---|
| Upadacitinib (Drug) | Active comparator | During the 8-week randomized withdrawal (RW) phase, participants in this arm will receive upadacitinib at the dose that was determined to work best for them (optimal treatment dose, OTD) during the earlier open-label phase of the study (15 mg, 30 mg, or 45 mg OTD). This study is not designed to compare the different dose levels. Therefore, participants receiving any of the three dose levels will be considered as one arm because each participant is receiving their own OTD. Receives: Drug: Upadacitinib |
| Matching Placebo | Placebo comparator | During the 8-week RW phase, participants assigned to receive placebo will receive placebo tablets that match their OTD selected during the earlier phase of the study. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment Efficacy - TIme to first occurrence of disease reactivation
All patients that complete the open label dose-escalation phase and are randomized will be included in the efficacy analysis population.
Efficacy of the Optimal Tolerated Dose (OTD) will be assessed by comparing the time to first occurrence of disease reactivation using Primary Immune Regulatory Deficiency (PIRD) score during the 8-week Randomized Withdrawal (RW) phase between the Upadacitinib and placebo groups.
The PIRD score is a disease scoring method that capture the clinical manifestations of all PIRDs including JAK/STAT GOF disorders. The presence of a disease state and its severity is graded on a scale of 1-5. 1 indicates the absence of the disease. Disease flare is defined as an increase in PIRD score by ≥1 in disease manifestations.
Measured: During the 8 weeks of the randomized withdrawal phase
Safety and Tolerability - Percentage of Patients with Adverse Events of Special Interest
All patients that receive at least one dose of study drug will be included in the analyses to evaluate safety and tolerability of the study drug.
Safety and tolerability will be summarized for each phase of the study and will be assessed primarily based on adverse events of special interest. Assessment of safety and tolerability will primarily be done by calculating the percentage of patients with adverse events of special interest.
Measured: Throughout the whole treatment period (1 year)
Safety and Tolerability - Percentage of Patients with Organ Toxicity
All patients that receive at least one dose of study drug will be included in the analyses to evaluate safety and tolerability of the study drug.
Safety and tolerability will be summarized for each phase of the study and will be assessed based on organ toxicity. Organ toxicity will be assessed by clinical labs. Assessment of safety and tolerability will primarily be done by calculating the percentage of patients with organ specific toxicities.
Measured: Throughout the whole treatment period (1 year)
Secondary outcomes (2)
Change in Quality of life during treatment- adults
Quality of life (QOL) will be assessed using validated age-adjusted tools (SF-36 for adults) at screening, beginning of the Randomized Withdrawal (RW) phase, beginning of the Maintenance Phase (MP), end of the MP.
The SF-36 (36-Item Short Form Health Survey) is an open-source patient-reported questionnaire to measure quality of life (QoL) across multiple health domains. It contains 36 items grouped into eight scales, each assessing a different aspect of health.
To assess the change in quality of life during the Open-Label (OL) phase, change from screening values will be calculated using assessments at the beginning of the RW phase. To assess the change in quality of life during the MP, the change in the QOL scores from beginning of the MP to the end of the MP will be calculated.
Measured: During the 16 weeks of OL Phase and 28 weeks of MP
Change in Quality of life during treatment- children
Quality of life (QOL) in children will be assessed using validated age-adjusted tools (Peds-QL for children) at screening, beginning of the Randomized Withdrawal (RW) phase, beginning of the Maintenance Phase (MP), end of the MP.
The PedsQL Measurement Model is a modular approach to measuring health-related quality of life (HRQOL) in healthy children and adolescents. It uses age-appropriate questionnaires to assess physical, emotional, social, and school functioning from the child's and/or parent's perspective. Its modular design allows it to be adapted for various conditions, making it widely used in clinical research and healthcare settings.
To assess the change in quality of life during the Open-Label (OL) phase, change from screening values will be calculated using assessments at the beginning of the RW phase. To assess the change in quality of life during the MP, the change in the QOL scores from beginning of the MP to the end of the MP will be calculated.
Measured: During the 16 weeks of OL Phase and 28 weeks of MP
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Jun 2026
- Study startOct 2026 (estimated)
- Primary completionAug 2028 (estimated)
- Study completionAug 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for JAK1 GOFStudies of the same condition, open and under way.
- 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
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- The study will follow a basket clinical trial design for JAK-STAT pathway disorders. The study consists of: (1) Open Label (OL) Dose Escalation Phase (3 dose escalations or continuation; each lasting 28 days); (2) Double Blind Placebo Controlled Randomized Withdrawal (RW) Phase; (3) Maintenance Phase (MP). For the double-blind randomized withdrawal phase, patients will be randomized to receive either the optimal treatment dose from the OL phase or Placebo using a 1:1 randomization ratio using variable block sizes.
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Lisa Satter
- Sponsor type
- University, hospital or other
- Collaborators
- National Center for Advancing Translational Sciences (NCATS), AbbVie
- Responsible party
- Sponsor-investigator, Lisa Satter, Baylor College of Medicine
- Investigators
- Lisa F Satter, MD, Study chair, Baylor College of Medicine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07670156
- Sponsor's protocol ID
- H-52366
- Other IDs
- 4UH3TR003908
- Acronym
- SAFE-JAKi
- Status last verified
- Sep 2026
- First submitted
- 19 Jun 2026
- Last updated
- 8 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy of Upadacitinib in Treatment of JAK/STAT Pathway Disorders With Activating Mutations
- ClinicalTrials.gov ID
- NCT07670156
- Acronym / sponsor ID
- SAFE-JAKi / H-52366
- Conditions
- JAK1 GOF, STAT1 GOF, STAT3 GOF, STAT5B GOF, STAT6 GOF
- Keywords
- Upadacitinib, JAK-STAT Pathway Disorders, Immunodeficiency, Primary Immune Regulatory Disorder (PIRD), Inborn Error of Immunity (IEI), early onset autoimmunity, autoimmune cytopenia, immune dysregulation
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- Lisa SatterUniversity, hospital or other
- Collaborators
- National Center for Advancing Translational Sciences (NCATS), AbbVie
- Study start
- Oct 2026 (estimated)
- Primary completion
- Aug 2028 (estimated)
- Study completion
- Aug 2028 (estimated)
- First posted
- 26 Jun 2026 (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.
This study focuses on a genetic condition that affects the Janus Kinase-Signal Transducer and Activator of Transcription (JAK-STAT) immune signaling pathway. A specific change in the DNA leads to overactivation of this pathway, which can result in immune dysregulation and related clinical symptoms.
Currently, five genetic mutations are known to cause these JAK-STAT pathway driven immune disorders: STAT1, STAT3, STAT5B, STAT6, and JAK1 (collectively referred to as JAK-STAT disorders). This study is a basket clinical trial, meaning patients with these different but related genetic conditions are enrolled in the same study and treated with the same investigational therapy.
The purpose of this study is to evaluate the safety and tolerability (ability to tolerate) of a drug called Upadacitinib in patients with JAK-STAT disorders with activating mutations. This drug belongs to a class of drug called Janus kinase (JAK) inhibitors, also known as JAKinibs. It is a type of immune system modulating medication that regulates (fixes) the JAK- STAT signaling pathway. Upadacitinib has been approved by the FDA for multiple immunological diseases and disorders. Presently, there is no FDA approved treatment for this group of JAK-STAT disorders. The study will also investigate immune factors in the blood to develop diagnosis methods that can be used in the future for better medical management of these disorders.
The study consists of four phases: screening phase, open label phase, randomized withdrawal phase and maintenance phase. The study will last approximately 12 months. While in the study, participants will receive a once daily dose of Upadacitinib that best helps control their disease. During the study participants will be asked to answer questions about their health and medical history. They will also complete physical exams, blood tests, and other questionnaires.
Detailed description
This is a phase 1/2, multi-center trial with an open label dose-escalation phase (OL) followed by a double-blind placebo-controlled randomized withdrawal phase (RW) to study the safety, tolerability, and preliminary efficacy of Upadacitinib in patients with confirmed and symptomatic gain of function mutations in the JAK-STAT pathway. Upon completion of the randomized withdrawal phase, each patient will enter the maintenance phase (MP) where all patients will receive Upadacitinib.
OL Phase (16 weeks): The subject will be escalated two times if tolerated and clinically indicated based upon disease response. The starting dose will be 15 mg per day for patients greater than or equal to 12 years of age (and at least 30 kg in weight). The subject will remain at this dose level for 28 days. The subject will visit the clinical site on day 1 of each dose escalation visit and then will be remotely monitored (phone call and laboratory monitoring to check complete blood counts) on Day 3 and Day 10.
Patients must have a disease status score of 2 or higher utilizing the Primary Immune Regulatory Deficiency (PIRD) Disease Modules prior to initiation of dose-escalation of the drug. The PIRD score is a disease scoring method that capture the clinical manifestations of all PIRDs including JAK/STAT GOF disorders. The PIRD scores for clinical improvement across organ systems for each patient will standardize the ability to quantify improvement across divergent disease phenotypes and assess outcomes based on clinical improvement within each disorder. Patients who demonstrate a response to study treatment, as determined by improvement in PIRD score, may proceed to the RW Phase. Patients who do not meet eligibility criteria for the RW Phase will enter MP.
During the OL Phase, the optimal treatment dose (OTD) for each patient will be determined. OTD will be defined as the dose that achieved complete response or partial response based on the PIRD scores.
RW Phase (8 weeks): For the double-blind randomized withdrawal phase, patients will be randomized to receive either the Optimum tolerated dose (OTD) dose from the OL phase or Placebo using a 1:1 randomization ratio. Subject will come to the study site every 4 weeks. The subject can discontinue the RW phase if he/she experiences a flare (worsening of disease symptoms) and will enter the Maintenance Phase outlined below. For measuring disease flare Primary Immune Regulatory Disorders (PIRD) score will be used. It will be deemed a flare if the score increases by ≥1 in disease manifestation. During the RW-phase, the reference for a disease flare (for the primary endpoint assessment) will be the end of the OL phase PIRD score.
MP Phase (28 weeks): Subject will be monitored closely for any drug related toxicities (blood counts, lipid and metabolic panels) and continue to take the highest tolerated dose level attained during the escalation phase. The subject will return to the clinical site on day 168, day 217, day 266, day 315 and day 364. Upon conclusion of the maintenance phase at day 364 the study team will safely transition the subject to clinically viable treatment alternatives that are commercially available (after a 3 days washout period for Upadacitinib).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- The study will follow a basket clinical trial design for JAK-STAT pathway disorders. The study consists of: (1) Open Label (OL) Dose Escalation Phase (3 dose escalations or continuation; each lasting 28 days); (2) Double Blind Placebo Controlled Randomized Withdrawal (RW) Phase; (3) Maintenance Phase (MP). For the double-blind randomized withdrawal phase, patients will be randomized to receive either the optimal treatment dose from the OL phase or Placebo using a 1:1 randomization ratio using variable block sizes.
| Arm | Type | Receives |
|---|---|---|
| Upadacitinib (Drug) | Active comparator | Drug: UpadacitinibDuring the 8-week randomized withdrawal (RW) phase, participants in this arm will receive upadacitinib at the dose that was determined to work best for them (optimal treatment dose, OTD) during the earlier open-label phase of the study (15 mg, 30 mg, or 45 mg OTD). This study is not designed to compare the different dose levels. Therefore, participants receiving any of the three dose levels will be considered as one arm because each participant is receiving their own OTD. |
| Matching Placebo | Placebo comparator | Drug: PlaceboDuring the 8-week RW phase, participants assigned to receive placebo will receive placebo tablets that match their OTD selected during the earlier phase of the study. |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- At least 30kg
- Patients with a confirmed JAK/STATGOF mutation with evidence of immune dysregulation
- Current disease status meeting criteria as defined in the disease scoring module
- Expected survival of more than 12 months
- Willingness to allow storage of biological samples for future research
Key exclusion criteria (8 listed)
- Hypersensitivity to the study drug or any medication in the same class
- Active infections
- Central nervous system (CNS) manifestations
- Pregnancy
- Medical conditions or use of concomitant medications that may interfere with the effect or evaluation of the study drug
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 |
|---|---|---|
| UpadacitinibAlso: RinvoqParticipants will receive the dose that worked best for them (optimal treatment dose, OTD) during the earlier open-label phase of the study, which may be 15 mg, 30 mg, or 45 mg taken once daily. The study drug will be taken by mouth as tablets. | Drug | Upadacitinib (Drug) |
| PlaceboParticipants assigned to the placebo group will receive placebo tablets taken once daily. The placebo will be matched to the participant's optimal treatment dose (OTD) determined during the earlier open-label phase of the study. | Drug | Matching Placebo |
Primary outcome
Treatment Efficacy - TIme to first occurrence of disease reactivation
Time frame: During the 8 weeks of the randomized withdrawal phase
All patients that complete the open label dose-escalation phase and are randomized will be included in the efficacy analysis population. Efficacy of the Optimal Tolerated Dose (OTD) will be assessed by comparing the time to first occurrence of disease reactivation using Primary Immune Regulatory Deficiency (PIRD) score during the 8-week Randomized Withdrawal (RW) phase between the Upadacitinib and placebo groups. The PIRD score is a disease scoring method that capture the clinical manifestations of all PIRDs including JAK/STAT GOF disorders. The presence of a disease state and its severity is graded on a scale of 1-5. 1 indicates the absence of the disease. Disease flare is defined as an increase in PIRD score by ≥1 in disease manifestations.
Safety and Tolerability - Percentage of Patients with Adverse Events of Special Interest
Time frame: Throughout the whole treatment period (1 year)
All patients that receive at least one dose of study drug will be included in the analyses to evaluate safety and tolerability of the study drug. Safety and tolerability will be summarized for each phase of the study and will be assessed primarily based on adverse events of special interest. Assessment of safety and tolerability will primarily be done by calculating the percentage of patients with adverse events of special interest.
Safety and Tolerability - Percentage of Patients with Organ Toxicity
Time frame: Throughout the whole treatment period (1 year)
All patients that receive at least one dose of study drug will be included in the analyses to evaluate safety and tolerability of the study drug. Safety and tolerability will be summarized for each phase of the study and will be assessed based on organ toxicity. Organ toxicity will be assessed by clinical labs. Assessment of safety and tolerability will primarily be done by calculating the percentage of patients with organ specific toxicities.
2 secondary outcomes
Change in Quality of life during treatment- adults
Time frame: During the 16 weeks of OL Phase and 28 weeks of MP
Change in Quality of life during treatment- children
Time frame: During the 16 weeks of OL Phase and 28 weeks of MP
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 8 Sep 2026.
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.
Lisa F Satter, MD study chair
Baylor College of Medicine
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Upadacitinib in people with JAK1 GOF and STAT1 GOF, run by Lisa Satter at 3 sites 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 6 inclusion and 8 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: opening soon, as of the sponsor's last update on 8 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Contact Supriya Parikh (8328242589, supriya.parikh@bcm.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Treatment Efficacy - TIme to first occurrence of disease reactivation (time frame: During the 8 weeks of the randomized withdrawal phase). The study lists 3 primary outcomes in all. 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 8 Sep 2026.