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RecruitingNCT04339777Updated Nov 10, 2026

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity

A Phase 2 interventional study of Busulfan test dose and Fludarabine in Lymphoproliferative Disorders, Autoimmune Lymphoproliferative and Immune System Diseases, sponsored by National Cancer Institute (NCI). Recruiting at 1 site in United States. Open to participants aged 4 Years to 69 Years. Per ClinicalTrials.gov, last updated 2026-11-10.

Sponsored by National Cancer Institute (NCI) · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Started Sep 2020; still recruiting 6 years 1 month later.
Updated Nov 10, 2026Eligibility revisedPrimary completion moved+1 moreGo to Updates ↓
Phase
Phase 2
Study type
Interventional
Enrollment
66
Allocation
Non-randomized
Ages
4 Years to 69 Years
Sex
All
01

Study summary

Background:

During a transplant, blood "stem cells" from one person are given to someone else. The cells grow into the different cells that make up the immune system. This can cure people with certain immunodeficiencies. But transplant has many risks and complications.

Objective:

To see if stem cell transplant can be successfully performed in people with primary immunodeficiency disease and cure them.

Eligibility:

People ages 4-69 for whom a primary immunodeficiency (PID) or Primary Immune Regulatory Disorder (PIRD), has caused significant health problems and either standard management has not worked or there are no standard management options, along with their donors

Design:

Donors will be screened under protocol 01-C-0129. They will donate blood or bone marrow.

Participants will be screened with:

Medical history

Physical exam

Blood, urine, and heart tests

CT or PET scans

Before transplant, participants will have dental and eye exams. They will have a bone marrow biopsy. For this, a needle will be inserted through the skin into the pelvis to remove marrow.

Participants will be hospitalized before their transplant. They will have a central catheter put into a vein in their chest or neck. They will get medications through the catheter to prevent complications. Participants will get stem cells through the catheter. They will stay in the hospital for at least 4 weeks. They will give blood, urine, bone marrow, and stool samples. They may need blood transfusions. They may need more scans. They will take more medications.

Participants will have visits on days 30, 60, 100, 180, and 360, and 24 months after the transplant. Then they will have visits once a year for about 5 years

Read the detailed description

Background:

  • With the availability of whole exome sequencing (WES) and whole genome sequencing (WGS) for patients with suspected inborn errors of immunity (IEI), the number of recognized IEI has increased in recent years to over 400 distinct immune defects.
  • Allogeneic hematopoietic stem cell transplantation (HSCT) represents a potentially curative therapy for many hematologic diseases.
  • Hematopoietic stem cell transplant is now an accepted standard or an appropriate experimental approach for treatment of an increasing number of IEI
  • We propose to evaluate the efficacy and safety of allogeneic hematopoietic stem cell transplantation using selected conditioning regimens and selected donor sources in reconstituting normal hematopoiesis and immune function and reversing the disease phenotype in patients with IEI.

Objectives:

-To determine whether allogeneic HSCT in patients with IEI results in sustained donor engraftment defined as neutrophil recovery with ANC >= 500/mm\^3 for 3 consecutive days associated with > 50% donor T-cell and myeloid cell donor chimerism by day 100 for diseases characterized by loss of function, and >75% donor T-cell and myeloid cell chimerism for diseases characterized by gain-of-function mutations.

Eligibility:

  • Participants ages 4-69 years old with a known IEI, or with clinical evidence of an IEI with a history of recurrent infections requiring prolonged courses of therapy, or evidence of immune dysregulation manifested by autoimmune/autoinflammatory disease, atopy, hemophagocytic lymphohistiocytosis, hypogammaglobulinemia, or impaired response to vaccination. A virally-driven malignancy alone will also constitute a basis for inclusion.
  • Have an 8/8, 7/8, or 6/8 HLA-matched related or unrelated donor (HLA -A, -B, -C, DRB1, by high resolution typing) or a haploidentical related donor; unrelated donors are identified through the National Marrow Donor Program.

Design:

For Recipients with Fully Matched Donors

  • Patients with IEI receiving a high intensity transplant conditioning regimen will receive a regimen consisting of fludarabine 40 mg/m\^2 IV once daily for 4 days on days -6, -5, -4, and -3, busulfan IV once daily for 4 days on days -6, -5, -4, -3 (busulfan dose will be based on pharmacokinetic levels from the test dose or real time pharmacokinetics (PKs) and will be targeted to a daily AUC of 3200-4400 micro Mol*min/L (total busulfan exposure of 52-72 mg*h/L) (3.2 mg/kg IV per day will be the default dose), and HSCT on day 0.
  • Patients with IEI receiving an intermediate intensity transplant conditioning regimen will receive a regimen consisting of fludarabine 40 mg/m2 IV once daily for 4 days on days -6, -5, -4, and -3, busulfan IV once daily for 3 days on days -6, -5, -and -4 (busulfan dose will be based on pharmacokinetic levels from the test dose or real time PKs and will be targeted to a daily AUC of 3200-4400 micro*Mol min/L (total busulfan exposure of 39-54 mg*h/L) (3.2 mg/kg IV per day will be the default dose), and HSCT on day 0.
  • Patients with IEI receiving a low intensity transplant conditioning regimen will receive a regimen consisting of fludarabine 40 mg/m\^2 IV once daily for 4 days on days -6, -5, -4, and -3, busulfan IV once daily for 2 days on days -6, and -5 (busulfan dose will be based on pharmacokinetic levels from the test dose or real time PKs and will be targeted to a daily AUC of 3200-4400 micro Mol*min/L (total busulfan exposure of 26-36 mg*h/L) (3.2 mg/kg IV per day will be the default dose), and HSCT on day 0.

In all cohorts, alemtuzumab will be given per PI discretion to patients with clinical evidence of immune dysregulation, at the dose of 10 mg/m\^2 subcutaneously divided over 3 days, on days -14, -13 and -12.

For Recipients with 7/8 or 6/8 Matched Related or Unrelated Donors and Haploidentical Related Donors

  • Patients with IEI receiving a high intensity transplant conditioning regimen will receive a regimen consisting of fludarabine 40 mg/m\^2 IV once daily for 4 days on days -6, -5, -4, and -3, busulfan IV once daily for 4 days on days -6, -5, -4, and -3 (busulfan dose will be based on pharmacokinetic levels from the test dose or real time PKs and will be targeted to a daily AUC of 3200-4400 micro Mol*min/L (52-72 mg*h/L) (3.2 mg/kg IV per day will be the default dose), 200 cGy TBI on day -1, and HSCT on day 0.
  • Patients with IEI receiving an intermediate intensity transplant conditioning regimen will receive a regimen consisting of fludarabine 40 mg/m2 IV once daily for 4 days on days - 6, -5, -4, -and 3, busulfan IV once daily for 3 days on days -6, -5, and -4 (busulfan dose will be based on pharmacokinetic levels from the test dose or real time PKs and will be targeted to a daily AUC of 3200-4400 micro Mol*min/L (39-54 mg*h/L) (3.2 mg/kg IV per day will be the default dose), 200 cGy TBI on day -1, and HSCT on day 0.
  • Patients with IEI receiving a low intensity transplant conditioning regimen will receive a regimen consisting of fludarabine 40 mg/m2 IV once daily for 4 days on days -6, -5, -4, and -3, busulfan IV once daily for 2 days on day -6 and -5 (busulfan dose will be based on pharmacokinetic levels from the test dose or real time PKs and will be targeted to a daily AUC of 3200-4400 micro Mol*min/L (26-36 mg*h/L) (3.2 mg/kg IV per day will be the default dose), 200 cGy TBI on day -1, and HSCT on day 0.

In all cohorts, alemtuzumab will be given per PI discretion to patients with clinical evidence of immune dysregulation, at the dose of 10 mg/m2 subcutaneously divided over 3 days, on days -14, -13 and -12.

For Post-Transplant GVHD Prophylaxis

-Post-transplant GVHD prophylaxis in all groups will consist of cyclophosphamide 50 mg/kg IV once daily for 2 days on days +3 and +4, along with mycophenolate mofetil from day +5 to approximately day +35 and tacrolimus from day +5 to approximately day +180.

If there is no evidence of GVHD, tacrolimus will be stopped or tapered at approximately day +180.

02

Conditions studied

  • Lymphoproliferative Disorders
  • Autoimmune Lymphoproliferative
  • Immune System Diseases
  • Common Variable Immunodeficiency
  • Primary T-cell Immunodeficiency Disorders

Keywords

  • Autoimmunity
  • Haploidentical
  • Opportunistic Infection
  • Immune Dysregulation
  • Congenital
03

In context

Lymphoproliferative Disorders

216 studies on the registry are indexed under Lymphoproliferative Disorders; 49 are open to participants now.

This study's planned enrollment of 66 is above the median of 41 across 173 interventional studies indexed under Lymphoproliferative Disorders.

Browse Lymphoproliferative Disorders studies →

Lead sponsor

National Cancer Institute (NCI) is the lead sponsor of 3,556 studies on the registry; 336 are open to participants now.

Of its 411 completed or terminated interventional studies of FDA-regulated products, 373 (91%) have results posted.

Browse National Cancer Institute (NCI) studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

The criteria were revised on the registry on Nov 10, 2026. See what changed

Ages eligible
4 Years to 69 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Age >= 4 years and \<=69 yo with Weight >=12 kilograms
  • Mutation in a known monogenic (IEI) gene performed by a CLIA certified laboratory, who have failed standard medical management, or when no standard medical management is available.

OR

Patients without a known IEI mutation may be eligible if they have a clinical history that is characteristic of an individual with an immune defect including a history of infections requiring prolonged courses of therapy or evidence of immune dysregulation manifested by /autoinflammatory disease, atopy, hemophagocytic lymphohistiocytosis, hypogammaglobulinemia, or impaired response to vaccination. A virally-driven alone will also constitute basis for inclusion.

  • Availability of an 8/8, 7/8, or 6/8 HLA-matched related or unrelated donor (if the mismatch is at DQ this will be considered an 8/8 matched donor), or a haploidentical related donor. or Lansky performance status of >= 40%

  • Adequate end-organ function, as measured by:

    -- > 40%, preferably by 2-D echocardiogram (ECHO) obtained within 60 days prior to enrollment.

  • Creatinine: Adult patients: \<= 2.0 mg/dl and >= 30 ml/min; Pediatric patients (\<18 years old): creatinine \< 1.5 mg/dL and a creatinine clearance, using the Schwartz Formula > 30 mL/min/1.73m\^2.

    • Serum conjugated \< 2.5 mg/dl; serum and AST \<= 5 times upper

limit of normal.

--Pulmonary function tests: > 30% and DLCO >30%. Participants who are unable to have DLCO assessed due to age or inability to complete PFT, are still eligible if no evidence of dyspnea at rest and no need for supplemental oxygen at the discretion of the PI.

  • Ability of subject, parent/guardian, or legally authorized representative (LAR) to understand and the willingness to sign a written document. For subjects \<18 years old, their legal guardian must give informed consent. Pediatric patients will provide assent.
  • As therapeutic agents used in this trial may be harmful to a fetus, women of and men must agree to use adequate (hormonal or barrier method of birth control; abstinence) prior to study entry and for at least one year post-allo HCT. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in the study, she should inform her treating physician immediately.
  • Willingness to remain in the NIH hospital or, if discharged, stay close to the NIH, for a minimum of 100 days after transplant or longer, if there are complications. If outpatient in the first 100 days after transplant, patient must commit to having an adult caregiver with them at all times.

Exclusion criteria

EXCLUSION CRITERIA:

  • Patients who are receiving any other agents (with the exception of virus-specific therapy e.g. cytotoxic T-cells for the treatment of viral infection/reactivation prior to allo HCT).
  • Patients with known should be excluded from this clinical trial because of their poor prognosis and because they often develop progressive neurologic dysfunction that would confound the evaluation of neurologic and other adverse events.
  • -positive patients are ineligible because these patients are at increased risk of lethal infections when treated with marrow-suppressive therapy. Appropriate studies will be undertaken in patients receiving combination antiretroviral therapy when indicated.
  • History of allergic reactions attributed to compounds of similar chemical or biologic composition to agents (, cyclophosphamide, busulfan, tacrolimus, sirolimus, MMF, G-CSF, alemtuzumab) used in the study
  • Active psychiatric disorder which is deemed by the PI to have significant risk of compromising compliance with the transplant protocol or which does not allow for appropriate
  • Pregnant women are excluded from this study because the study agents have the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with the study agents, breastfeeding should be discontinued if the mother is treated with the study agents.
  • Uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia.
  • A psychiatric illness/social situations or any other circumstance that, in the opinion of the investigator, may compromise the safety or compliance of the participant with study requirements or would preclude the participant from successful study completion.
Terms in these criteria, in plain language(15)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Autoimmune disease

A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.

Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

Karnofsky performance status (KPS)

A 0–100 scale of how well someone can carry out everyday activities. 100 is no complaints and no signs of illness; 70 is able to look after yourself but not to work or keep up normal activity; 50 is needing considerable help and frequent medical care.

Studies often ask for 60 or 70 and above. Your doctor gives the score.

LVEF (left ventricular ejection fraction)

The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.

Creatinine clearance (CrCl)

How quickly the kidneys clear creatinine, a waste product, from the blood. A study may estimate it from a blood test with age, weight and sex (often by the Cockcroft–Gault formula), or measure it using blood and urine collected over a set time. Given in mL/min; higher is better.

Bilirubin

A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.

Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.

ALT and AST (liver enzymes)

Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.

Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.

FEV1

From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

Brain metastases

Cancer deposits in the brain from a cancer that started elsewhere in the body (CNS metastases include the spinal cord).

Studies differ: some leave out any brain metastases, some accept ones treated and stable for some weeks. Check what this study allows.

HIV

The human immunodeficiency virus, which weakens the immune system.

Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
66 participants (estimated)

Study arms

  • Active comparator
    Arm A

    Low Intensity, Intermediate Intensity and High Intensity Conditioning with or without alemtuzumab

    Drug: Busulfan test dose · Drug: Fludarabine · Drug: Busulfan · Drug: Alemtuzumab · Procedure: Allogeneic HSCT · Drug: Tacrolimus (Tacro) · Drug: Mycophenolate mofetil (MMF) · Drug: Cyclophosphamide (Cytoxan)

  • Active comparator
    Arm B

    Intermediate Intensity Conditioning with or without Alemtuzumab

    Drug: Busulfan test dose · Drug: Fludarabine · Drug: Busulfan · Drug: Alemtuzumab · Radiation: Total body Irradiation · Procedure: Allogeneic HSCT · Drug: Tacrolimus (Tacro) · Drug: Mycophenolate mofetil (MMF) · Drug: Cyclophosphamide (Cytoxan)

Interventions

  • DrugBusulfan test dose

    0.8 mg/kg IV infusion over 2 hours

  • DrugFludarabine

    40 mg/m2 IV infusion over 30 min once daily for 4 days

  • DrugBusulfan

    AUC Targeted Dose based on busulfan test dose PKs, IV infusion over 3 hours once daily (3.2 mg/kg IV per day will be the default dose) per the below time frame: For 10/10 Matched Related and Unrelated Donor Recipients For the High Intensity Arm, the busulfan dose will be given for 4 days (-6, -5, -4, and -3). For Intermediate Intensity Arm, the busulfan dose will be given for 3 days (-6,-5, and -4). For Low Intensity Arm, the busulfan dose will be given for 2 days on days (-6 and -5). 9/10 HLA Matched Related or Unrelated Donor Recipients For the High Intensity Arm, the busulfan dose will be given for 3 days (-6, -5, and -4). For the the Intermediate Intensity Arm, the busulfan dose will be given for 2 days (-6 and -5). For the Low Intensity Arm, the busulfan dose will be given for 1 day on day (-6).

  • DrugAlemtuzumab

    Alemtuzumab will be given if there is evidence of immune dysregulation 10 mg/m2 SC divided over three days (-14, -13, and -12)

  • RadiationTotal body Irradiation

    200 cGy Transplant Day -1 (Only for 9/10 HLA Matched Related or Unrelated Donor Recipients )

  • ProcedureAllogeneic HSCT

    Stem cell transplant

  • DrugTacrolimus (Tacro)

    Tacrolimus 0.02 mg/kg IV continuous infusion over 24 hours starting on day +5

  • DrugMycophenolate mofetil (MMF)

    Mycophenolate mofetil 15 mg/kg IV over 2 hours three times a day starting on day +5 will continue until Approximately+35 (+/- two days)

  • DrugCyclophosphamide (Cytoxan)

    Cyclophosphamide: 50 mg/kg IV once daily over 2 hours on days +3 and +4, dosed according to ideal body weight

06

What researchers measure

Primary outcomes

  1. Sustained donor engraftment

    neutrophil recovery with ANC \>/= 500/mm\^3 for 3 consecutive days with \>50% or \>75% T-cell and myeloid donor chimerism

    Time frame: baseline to day +100

Secondary outcomes

  1. Reversal of the immunological abnormalities

    Cumulative correction of disease-specific immunological abnormalities by 1-5 years post transplant

    Time frame: 1 through 5 years post transplant

  2. Reversal of the clinical phenotype

    Cumulative improvement of clinical phenotype consistent with the replacement of hematopoietic cells at 1 -5 years post transplant

    Time frame: 1 and 2 years post transplant

  3. regimen-related mortality

    Cumulative incidence of transplant-related mortality at 180 days and 1 year post transplant

    Time frame: +180 and 1 year post transplant

  4. Overall survival

    Time from transplant to death of any cause

    Time frame: 1 through 5 years post transplant

  5. infection and viral reactivation

    Cumulative incidence of transplant-related mortality at 180 days and 1 year post transplant

    Time frame: +180 and 1 year post transplant

  6. Incidence of Chronic Graft-versus-host disease

    Cumulative incidence of chronic graft versus host disease at 1 and 2 years post transplant

    Time frame: 1 and 2 years post transplant

  7. Incidence of Acute Graft-versus-host disease

    Cumulative incidence of acute graft versus host disease at day 100 post transplant

    Time frame: 100 days post transplant

  8. Event free survival

    Time from transplant to death of any cause, primary or secondary graft failure, or second transplant

    Time frame: 1 through 5 years post transplant

07

Study locations

1 of 1 sites recruiting
  • National Institutes of Health Clinical Center
    Bethesda, Maryland 20892, United States
    Recruiting
    • For more information at the NIH Clinical Center contact National Cancer Institute Referral Office · Contact888-624-1937
08

References and documents

Publications

  • Arnold DE, Kaviany S, Aluri J, Calvo KR, Mehta SS, Tobin JM, Schuettpelz LG, Saucier N, Schmitz EG, Murguia-Favela L, Wright NAM, De Ravin SS, Rao VK, Holland SM, Bleesing JJ, Loughran TP Jr, Feith DJ, Powell J, Ratan A, Warren JT, Pai SY, Bednarski JJ, Connelly JA, Cooper MA. Clinical characteristics, management, and hematopoietic cell transplantation of patients with TLR8 gain-of-function. Blood Adv. 2026 Mar 24;10(6):1967-1976. doi: 10.1182/bloodadvances.2025016338. PubMed 41370196 ↗

Individual participant data

Plan to share: Yes — All IPD recorded in the medical record will be shared with intramural investigators upon request.

Supporting information: Study protocol, Statistical analysis plan, Informed consent form

Time frame: Clinical data available during the study and indefinitely.

Access criteria: Clinical data will be made available via subscription to BTRIS and with the permission of the study PI.

09

Timeline

3 registry updates since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedDate before it movedWhere the date was before it movedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Started Sep 2020 (actual). Primary completion is estimated for Oct 2028, 96 months after the start.

Of 30 phase 2 interventional studies under Lymphoproliferative Disorders, half reached primary completion within 144 months of starting (the median), counting those still running; a quarter within 57 and three quarters within 356. This study's planned 96 months is below that.

Since we started tracking on Sep 25, 2026, the dates moved once, on Oct 19, 2026: primary completion Oct 2026 → Oct 2028 and completion Nov 2027 → Nov 2032.

Earlier changes are on the record history on ClinicalTrials.gov ↗

How these figures are worked out, and which studies are left out of them.

Registered
Apr 8, 2020
First posted
Apr 9, 2020
StartActual
Sep 22, 2020
Primary completionEst.
Oct 1, 2028was Oct 2026
CompletionEst.
Nov 30, 2032was Nov 2027
Last update
Nov 10, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
Primary completion
Oct 1, 2026→Oct 1, 2028
Oct 19, 2026
Study completion
Nov 30, 2027→Nov 30, 2032
Oct 19, 2026
Eligibility
2 inclusion criteria edited; 1 exclusion criterion edited, 1 added
What changed in the criteria
Inclusion criteria
  • Edited--Pulmonary function tests: FEV1 > 30% and DLCO >30%. ChildrenParticipants who are unable to have DLCO assessed due to age or inability to complete PFT, are still eligible if no evidence of dyspnea at rest and no need for supplemental oxygen at the discretion of the PI.
  • EditedAbility of subject or, parent/guardian, or legally authorized representative (LAR) to understand and the willingness to sign a written informed consent document. For subjects <18 years old, their legal guardian must give informed consent. Pediatric patients will provide assent.
Exclusion criteria
  • EditedUncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
  • AddedA psychiatric illness/social situations or any other circumstance that, in the opinion of the investigator, may compromise the safety or compliance of the participant with study requirements or would preclude the participant from successful study completion.
Nov 10, 2026
Show all 3 updates
  1. Nov 10, 2026
    Eligibility 2 inclusion criteria edited; 1 exclusion criterion edited, 1 added
    What changed in the criteria
    Inclusion criteria
    • Edited--Pulmonary function tests: FEV1 > 30% and DLCO >30%. ChildrenParticipants who are unable to have DLCO assessed due to age or inability to complete PFT, are still eligible if no evidence of dyspnea at rest and no need for supplemental oxygen at the discretion of the PI.
    • EditedAbility of subject or, parent/guardian, or legally authorized representative (LAR) to understand and the willingness to sign a written informed consent document. For subjects <18 years old, their legal guardian must give informed consent. Pediatric patients will provide assent.
    Exclusion criteria
    • EditedUncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, or cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
    • AddedA psychiatric illness/social situations or any other circumstance that, in the opinion of the investigator, may compromise the safety or compliance of the participant with study requirements or would preclude the participant from successful study completion.
    + 1 other change: description
  2. Oct 19, 2026
    Primary completion Oct 1, 2026→Oct 1, 2028
    Study completion Nov 30, 2027→Nov 30, 2032
    + 1 other change: verification date
  3. Sep 28, 2026
    Minor edits only
    + 1 other change: verification date

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT04339777
Lead sponsor
National Cancer Institute (NCI)
Responsible party
Sponsor
First posted
Apr 9, 2020
Start date
Sep 22, 2020
Primary completion
Oct 1, 2028 (estimated)
Completion
Nov 30, 2032 (estimated)
Last update
Nov 10, 2026

Study contacts

Shannon L Knight, R.N.
Contact
shannon.knight@nih.gov
(240) 921-5872
Sung-Yun Pai, M.D.
Contact
sung-yun.pai@nih.gov
(240) 858-7284
Sung-Yun Pai, M.D.
principal investigator · National Cancer Institute (NCI)

Oversight

FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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