Safety: Death
Primary
· Average of approximately 31 months for those who received an initial transplant
How many, if any, participants die during study participation.
| Group | Value | 95% CI |
|---|---|---|
| BOLT-BMT Protocol | 4 |
Last reviewed · How we verify
NCT03330795: BOLT-BMT
Bilateral Orthotopic Lung Transplant - Bone Marrow Transplant
Completed
Phase 1, PHASE2
Results posted
Last updated 22 July 2025
What this trial tests
Phase 1, PHASE2 trial testing CD3/CD19 neg allogeneic BMT in Primary Immunodeficiency in 5 participants. Completed in 1 March 2024.
Timeline
1 December 2017
Primary endpoint
1 March 2024
1 March 2024
1 March 2024
Quick facts
| Lead sponsor | National Institute of Allergy and Infectious Diseases (NIAID) |
|---|---|
| Phase | Phase 1, PHASE2 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | na |
| Design | single group |
| Masking | none |
| Primary purpose | treatment |
| Enrollment | 5 |
| Start date | 1 December 2017 |
| Primary completion | 1 March 2024 |
| Estimated completion | 1 March 2024 |
| Sites | 1 location across United States |
Drugs / interventions tested
- CD3/CD19 neg allogeneic BMT — full drug profile →
Conditions studied
- Primary Immunodeficiency — all drugs for Primary Immunodeficiency →
- PID — all drugs for PID →
Sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
Who can join
Adults 10 to 45, any sex, with Primary Immunodeficiency or PID. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Safety: Engraftment Syndrome
Primary
· Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
How many, if any, participants develop engraftment syndrome.
| Group | Value | 95% CI |
|---|---|---|
| CD3/CD19 Neg Allogeneic BMT | 0 |
Engraftment Failure
Primary
· Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
| Group | Value | 95% CI |
|---|---|---|
| CD3/CD19 Neg Allogeneic BMT | 1 |
Grade 4 or 5 Events Potentially Attributable to Rituximab
Primary
· Average of ~25 months.
| Group | Value | 95% CI |
|---|---|---|
| BOLT-BMT Protocol | 4 |
BOS at 1 Year Post BOLT
Primary
· throughout the first year post BOLT
Diagnosis of BOS (Bronchiolitis Obliterans Syndrome) at any time up to 1 year post BOLT. BOS is one of the undesirable complications of lung transplantation.
| Group | Value | 95% CI |
|---|---|---|
| CD3/CD19 Neg Allogeneic BMT | 0 |
Efficacy: Count of Participants With Requirement for Supplemental Oxygen and/or Ventilatory Support
Primary
· at 1 Year Post Lung Transplant (BOLT)
The number of participant(s) who need either supplemental oxygen and/or ventilator support (noninvasive/invasive) will be assessed using the Bronchiolitis Obliterans Syndrome (BOS) Classification Score for pulmonary function (e.g., the Forced Expiratory Volume in 1 Second (FEV1). FEV1 is air volume exhaled in 1 second during spirometry, a lung function test. (Continuing dependence on supplemental oxygen or ventilatory support is an undesirable outcome of lung transplantation).
| Group | Value | 95% CI |
|---|---|---|
| CD3/CD19 Neg Allogeneic BMT | 0 |
Efficacy: Count of Participants With T-cell Chimerism
Primary
· 1 Year Post Bone Marrow Transplant (BMT)
The number of participants who have ≥ 25% donor T-cell chimerism.
| Group | Value | 95% CI |
|---|---|---|
| CD3/CD19 Neg Allogeneic BMT | 1 |
Efficacy: Count of Participants With Myeloid Chimerism
Primary
· 1 Year Post Bone Marrow Transplant (BMT)
The number of participants with myeloid disorders (e.g. Chronic Granulomatous Disease \[CGD\]) who attain ≥ 10% myeloid chimerism.
| Group | Value | 95% CI |
|---|---|---|
| CD3/CD19 Neg Allogeneic BMT | 1 |
Efficacy: Count of Participants B-cell Chimerism
Primary
· 1 Year Post Bone Marrow Transplant (BMT)
The number of participants with B-cell disorders who attain ≥ 10% B-cell chimerism.
| Group | Value | 95% CI |
|---|---|---|
| CD3/CD19 Neg Allogeneic BMT | 0 |
Count of Participants Able to Proceed to BMT
Secondary
· 6 Months Post Lung Transplant (BOLT)
The number of participants for which it is feasible to proceed to BMT within 6 months following lung transplant.
| Group | Value | 95% CI |
|---|---|---|
| Lung Transplant | 1 |
Count of Participants Who Achieve Tolerance
Secondary
· Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
The number of participants who develop tolerance to both the host and pulmonary graft. Definition of tolerance: A participant's successful withdrawal from systemic immunosuppression for 6 weeks with no increase cGVHD score and stable or improving PFTs.
| Group | Value | 95% CI |
|---|---|---|
| CD3/CD19 Neg Allogeneic BMT | 0 |
Long Term Complications of Combined Solid Organ and Bone Marrow Transplant
Secondary
· Average of ~7.5 months of time post BMT for those receiving both lung and BMT transplants.
Summary of long-term complications of combined solid organ and bone marrow transplant (BMT).
| Group | Value | 95% CI |
|---|---|---|
| CD3/CD19 Neg Allogeneic BMT | 0 |
Adverse events — posted to ClinicalTrials.gov
Time frame: Average of approximately 25 months. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
BOLT-BMT Protocol
Serious: 5/5 (100%)
Deaths: 4/5
Serious adverse events (32 terms)
| Reaction | System | BOLT-BMT Protocol |
|---|---|---|
| Acute kidney injury | Renal and urinary disorders | — |
| Gastrointestinal haemorrhage | Gastrointestinal disorders | — |
| Pneumonia | Respiratory, thoracic and mediastinal disorders | — |
| Acute graft versus host disease in intestine | Immune system disorders | — |
| Bone marrow disorder | Blood and lymphatic system disorders | — |
| Bronchial haemorrhage | Respiratory, thoracic and mediastinal disorders | — |
| Bronchitis | Respiratory, thoracic and mediastinal disorders | — |
| Colitis | Gastrointestinal disorders | — |
| Dehydration | Metabolism and nutrition disorders | — |
| Diarrhoea | Gastrointestinal disorders | — |
| Feeling abnormal | General disorders | — |
| Fluid balance positive | Investigations | — |
| Fungal sepsis | Infections and infestations | — |
| Hyperkalaemia | Metabolism and nutrition disorders | — |
| Infection | Infections and infestations | — |
| Jugular vein thrombosis | Vascular disorders | — |
| Lower urinary tract symptoms | Renal and urinary disorders | — |
| Meningitis | Nervous system disorders | — |
| Myocardial infarction | Cardiac disorders | — |
| Neutrophil count decreased | Investigations | — |
| Otitis externa | Ear and labyrinth disorders | — |
| Platelet count decreased | Investigations | — |
| Pulmonary mass | Respiratory, thoracic and mediastinal disorders | — |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | — |
| Respiratory distress | Respiratory, thoracic and mediastinal disorders | — |
Other adverse events (279 terms — click to expand)
| Reaction | System | BOLT-BMT Protocol |
|---|---|---|
| Diarrhoea | Gastrointestinal disorders | — |
| Hyperglycaemia | Metabolism and nutrition disorders | — |
| Pneumothorax | Respiratory, thoracic and mediastinal disorders | — |
| Anaemia | Blood and lymphatic system disorders | — |
| Hyperkalaemia | Metabolism and nutrition disorders | — |
| Hypertension | Vascular disorders | — |
| Hypocalcaemia | Metabolism and nutrition disorders | — |
| Hypotension | Vascular disorders | — |
| Leukocytosis | Blood and lymphatic system disorders | — |
| Lymphocyte count decreased | Investigations | — |
| Nausea | Gastrointestinal disorders | — |
| Neutrophil count increased | Investigations | — |
| Non-cardiac chest pain | General disorders | — |
| Oedema peripheral | General disorders | — |
| Vomiting | Gastrointestinal disorders | — |
| White blood cell count decreased | Investigations | — |
| Abdominal pain | Gastrointestinal disorders | — |
| Anxiety | Psychiatric disorders | — |
| Atelectasis | Respiratory, thoracic and mediastinal disorders | — |
| Back pain | Musculoskeletal and connective tissue disorders | — |
| Blood potassium increased | Investigations | — |
| Confusional state | Psychiatric disorders | — |
| Constipation | Gastrointestinal disorders | — |
| Cough | Respiratory, thoracic and mediastinal disorders | — |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | — |
| Fungal infection | Infections and infestations | — |
| Haematuria | Renal and urinary disorders | — |
| Headache | Nervous system disorders | — |
| Hyperchloraemia | Metabolism and nutrition disorders | — |
| Hypoalbuminaemia | Metabolism and nutrition disorders | — |
| Hypomagnesaemia | Metabolism and nutrition disorders | — |
| Neutrophil count decreased | Investigations | — |
| Platelet count decreased | Investigations | — |
| Pulmonary oedema | Respiratory, thoracic and mediastinal disorders | — |
| Respiratory disorder | Respiratory, thoracic and mediastinal disorders | — |
| Tremor | Nervous system disorders | — |
| Abdominal distension | Gastrointestinal disorders | — |
| Acidosis | Metabolism and nutrition disorders | — |
| Acute kidney injury | Renal and urinary disorders | — |
| Alanine aminotransferase increased | Investigations | — |
Most-reported serious reactions: Acute kidney injury, Gastrointestinal haemorrhage, Pneumonia, Acute graft versus host disease in intestine, Bone marrow disorder, Bronchial haemorrhage, Bronchitis, Colitis.
Data from ClinicalTrials.gov NCT03330795 adverse events section.
Sponsor's own description
The purpose of this study is to determine whether bilateral orthotopic lung transplantation (BOLT) followed by cadaveric partially-matched CD3+/CD19+ depleted bone marrow transplant (BMT) is safe and effective for individuals aged 10 through 45 years with the diagnosis of primary immunodeficiency (PID) and end-stage lung disease. The enrollment goal: 8 participants who receive both BOLT and BMT.
Publications & conference data
No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.
Verify or expand the search:
- PubMed search for NCT03330795
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
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Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
Data sources for this page
- Trial protocol + status: ClinicalTrials.gov NCT03330795 (US National Library of Medicine, public domain)
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by National Institute of Allergy and Infectious Diseases (NIAID)
- Last refreshed: 22 July 2025
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03330795.
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