A Phase 1/2 Study of Inhaled KB707 in Patients

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Krystal Biotech Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Neoplasms [C04]

Decision date (initial)

2026-07-02

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

No

Funding sources

Krystal Biotech, Inc.

External identifiers

EU CT number

2025-522723-98-00

ClinicalTrials.gov

NCT06228326

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Efficacy, Safety

To evaluate the safety and tolerability of inhaled KB707 monotherapy and in combination regimens (e.g., KB707 plus immune checkpoint inhibitors [with or without chemotherapy]; or docetaxel)

Secondary objectives 2

1. To evaluate whether the proposed dose ranges include the maximum tolerated dose of KB707
2. To evaluate the preliminary efficacy of inhaled KB707 monotherapy and in combination regimens by RECIST v1.1

Conditions and MedDRA coding

solid tumor

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. 1. The subject or legally authorized representative must have read, understood, and signed an Institutional Review Board (IRB) approved Informed Consent Form and must be willing and able to comply with study procedures and instructions.
2. 2. Age 18 years or older at the time of informed consent.
3. 3. Life expectancy >12 weeks.
4. 4. Have an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1 at Screening
5. 5. Have at least one measurable lung lesion per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST v1.1) at Screening.
6. 6. Demonstrated adequate organ function at Screening, as defined below: a. WBC count ≥2000/μL (after at least 7 days without growth factor support) b. Absolute neutrophil count ≥1500/μL (after at least 7 days without growth factor support) c. Platelet count ≥100×103 μL d. Hemoglobin ≥9.0 g/dL e. Serum creatinine ≤2 mg/dL (or glomerular filtration rate ≥40 mL/min) f. AST and ALT ≤3x upper limit of normal (ULN)g. Total bilirubin within normal limits unless associated with hepatobiliary metastases or Gilbert’s syndrome, in that case total bilirubin ≤2x ULN
7. 7. Histologically confirmed diagnosis of advanced solid tumor malignancy affecting the lungs and the individual has progressed on standard of care therapy, cannot tolerate standard of care therapy, refused standard of care therapy, or has no standard of care therapy.
8. 8. Histologically or cytologically confirmed diagnosis of stage 3 or 4 NSCLC, as per American Joint Committee on Cancer (AJCC) staging system (8th edition)
9. 9. Subject must meet the following criteria of prior lines of therapy: a. Subject has previously received no more than one line of prior immune checkpoint inhibitor (ICI) with or without platinum-based chemotherapy, or no more than two prior lines of therapy when given the ICI and platinum-based chemotherapy sequentially as two separate lines. b. Subjects with an actionable mutation (e.g., EGFR, KRAS, ALK, or ROS1 genomic alteration), are permitted to have received one additional line of approved targeted therapy.

Exclusion criteria 12

1. 1. Not fully recovered from prior surgery or radiotherapy, including all radiation-related toxicities.
2. 2. Have known medical history of positive test for, or diagnosis of, human immunodeficiency virus (HIV-1/2).
3. 3. The subject is pregnant, nursing, or plans to become pregnant during study treatment and through three months after the last dose of KB707.
4. 4. Subject who is unwilling to comply with contraception requirements per-protocol.
5. 5. Any clinical condition or clinically significant abnormality that, in the opinion of the Investigator, would impact a subject’s ability to complete all study-related procedures and/or poses an additional risk to the assessment of safety of KB707.
6. 6. Subject is known to be noncompliant or is unlikely to comply with the requirements of the study protocol in the opinion of the Investigator.
7. 7. Subject has a known additional malignancy that is progressing or requires active treatment.
8. 8. Subject has active brain metastases or leptomeningeal metastases.
9. 9. Prior anti-PD-1/PD-L1 therapy was intolerable and required discontinuation of treatment.
10. 10. Subject has active, known, or suspected autoimmune disease requiring systemic treatment
11. 11. Subject has known acute or chronic hepatitis.
12. 12. Subject has active pneumonitis or history of ICI-induced pneumonitis that required steroids.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Frequency, severity, and relatedness of Adverse Events (AEs) and Serious Adverse Events (SAEs)

Secondary endpoints 2

1. Incidence of Dose-Limiting Toxicity (DLT) by dose cohorts
2. Assessment of the objective response rate (ORR), complete response (CR), clinical benefit rate (CBR), duration of response (DOR), time to response (TTR), progression free survival (PFS), and overall survival (OS)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Krystal Biotech Inc.

Sponsor organisation

Krystal Biotech Inc.

Address

2100 Wharton Street Suite 701

City

Pittsburgh

Postcode

15203-1973

Country

United States

Scientific contact point

Organisation

Krystal Biotech Inc.

Contact name

Suma Krishnan

Public contact point

Organisation

Krystal Biotech Inc.

Contact name

Patient Advocacy

Third parties 3

Locations

2 EU/EEA countries · 15 investigational sites

By country

Investigational sites

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 12 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

1 submissions — initial application plus substantial / non-substantial modifications