Fanconi AnemiaPhase 1/2AFANNCT06648096
Afatinib in Patients With Fanconi Anemia (FA) and Advanced Head and Neck Squamous Cell Carcinoma (HNSCC)
A Phase 1/2 study testing Afatinib in people with Fanconi Anemia and Head and Neck Squamous Cell Carcinoma, run by Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau at 2 sites in 2 countries.
Trial summary
This research study is a phase Ib/II, single-arm, non-randomized, non-blind, multicenter study designed to determine whether Afatinib is effective and safe in patients with locoregionally unresectable and / or metastatic HNSCC with Fanconi Anemia. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fanconi Anemia, Head and Neck Squamous Cell Carcinoma
- What is being tested?
- Afatinib
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 2 sitesin 2 countries
- Study dates
- 8 Nov 2024to Dec 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Written informed consent according to local guidelines, must be signed and dated by the participant and investigator prior to performing any protocol procedure.
- Patient is ≥ 18 years of age.
- Confirmed diagnosis of Fanconi anemia.
- Histologically or cytologically confirmed unresectable or locoregionally advanced squamous cell carcinoma of the oral cavity, oropharynx, hypopharynx, larynx, nasopharynx, paranasal sinuses or salivary glands. Patients with distal metastasis (M1, American Joint Cancer Committee (AJCC) 8th ed.) are also eligible.
- Tumor not a candidate for resection prior to Afatinib due to technical inability to resect (tumor fixation / invasion in the skull base, cervical vertebrae, nasopharynx or fixed lymph nodes) and / or low surgical cure [T3-T4, N2-N3; , AJCC 8th ed.]).
- Patients must have at least 1 measurable lesion by computed tomography (CT) scan or magnetic resonance imaging (MRI) as defined by RECIST v1.1.
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- Previous anticancer treatment is allowed if it ends 6 weeks or 5 half-lives, whichever is shorter, before the expected date of start of the study treatment.
- Previous locoregional treatments such as radiotherapy are allowed.
- Eastern Cooperative Oncology Group (ECOG) performance status < 2 at inclusion.
- Adequate organ and bone marrow functions, as defined below:
- Neutrophils > 1000 cells / microliter.
- Platelets > 50,000 cells / microliter.
- Hemoglobin > 8 g / dL
- Creatinine < 1.5 x upper limit normal (ULN) with clearance > 50 mL / min.
- Total bilirubin < 1.5 x ULN. Note: patients with Gilbert's may be included with bilirubin <2 x ULN.
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) < 2.5 x ULN or < 5 ULN if liver metastases are present.
- International normalized ratio (INR) and prothrombin time (PT) <1.5 x ULN.
- Female patients must either:
- Be of non-childbearing potential:
- Postmenopausal (defined as at least 1 year without any menses) prior to screening , or Documented surgically sterile (e.g.hysterectomy, bilateral salpingectomy, bilateral oophorectomy, or bilateral tubal occlusion).
- Those who are amenorrheic due to an alternative medical cause are not considered postmenopausal and must follow the criteria for childbearing potential subjects.
- OR
- If of childbearing potential:
- Agree not to try to become pregnant during the study and for at least 1 months after the final study drug administration, And have a negative urine or serum pregnancy test within 7 days prior to Day 1 (females with false positive results and documented verification of negative pregnancy status are eligible for participation), And if heterosexually active, agree to abstinence (if in line with the usual preferred lifestyle of the patient) or consistently use a condom plus 1 form of highly effective birth control per locally accepted standards starting at screening and throughout the study period and for at least 1 month after the final study drug administration.
- Female patients must agree not to breastfeed or donate ovules starting at screening and throughout the study period, and for at least 1 month after the final study drug administration.
- Male patients must not donate sperm starting at screening and throughout the study period, and for at least 1 month after the final study drug administration.
- Male patients with a partner with childbearing potential, or who is pregnant or breastfeeding must agree to abstinence or use a condom plus 1 form of highly effective birth control throughout the study period and for at least 1 month after the final study drug administration.
- Patient agrees not to participate in another interventional study while on treatment in the present study.
Who cannot join
- Patients who are candidates for surgery with curative intent are not eligible.
- Less than two weeks from surgical resection or other major surgical procedure at start of treatment. Planned surgery for other diseases.
- Previous treatment with EGFR small molecule inhibitors, EGFR inhibitory antibodies and / or any investigational agents for the treatment of HNSCC within 4 weeks prior to the selection was not allowed.
- Note: Previous treatment with chemotherapy and/or radiotherapy is allowed.
- Patient must have recovered from any previous treatment toxicity to Grade ≤ 2.
- Existence of any other intercurrent malignant disease is not allowed within the previous 2 years to inclusion.
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- Note: Patients with non melanoma skin cancer, curatively treated localized prostate cancer, or carcinoma in situ of any type (if complete resection was performed) are allowed.
- Active severe Severe infectious disease in the 4 weeks prior to the initiation of study treatment, including . Known human immunodeficiency virus (HIV) infection or chronic Hepatitis B or C.
- Patient has documented history of a cerebral vascular event (stroke or transient ischemic attack), or the following criteria for cardiac disease:
- Myocardial infarction or unstable angina pectoris within 6 months of enrollment.
- History of serious ventricular arrhythmia (ie, ventricular tachycardia or ventricular fibrillation), high-grade atrioventricular block, or other cardiac arrhythmias requiring antiarrhythmic medications (except for atrial fibrillation that is well controlled with antiarrhythmic medication); history of QT interval prolongation.
- New York Heart Association (NYHA) class III or greater congestive heart failure or left ventricular ejection fraction of < 40%.
- Participants with QTc interval (corrected) > 470 msec at screening.
- History of interstitial lung disease requiring corticosteroids or pneumonitis.
- Gastrointestinal disorders that may interfere with the absorption of the study drug or chronic diarrhea.
- Patient has known hypersensitivity to afatinib or to any excipient contained in the drug formulation.
- Female patients who are or intend to be pregnant or breastfeeding during their participation in the study or 1 month after the final study drug administration.
- Patients unable to comply with the protocol as determined by the investigator.
- The patient is currently participating in another clinical trial that would interfere with the radiological imaging schedule or any other determinations required in this protocol.
- Patient has other underlying medical conditions that, in the opinion of the investigator, would impair the ability of the patient to receive or tolerate the planned treatment and follow-up.
- Patients with psychiatric disorders that may interfere with monitoring.
Trial locations and contacts
2 sites in 2 countries. Contact the study team or a site near you to ask about taking part.
2 sites in 2 countries
Germany1 site, 1 recruiting
- Recruiting
Medizinische Hochschule Hannover
Hanover
A Resposible Person Designated by the Sponsor, +34 93 434 44 12, investigacion@mfar.net
Principal Investigator Designated by the Sponsor, M.D.; Ph.D.
Spain1 site, 1 recruiting
- Recruiting
Hospital de la Santa Creu i Sant Pau
Barcelona, Catalonia
A Responsible Person Designated by the Sponsor, +34 93 434 44 12, investigacion@mfar.net
Principal Investigator Designated by the Sponsor, M.D.;Ph.D.
About this study
Official title: Phase Ib/II Study to Investigate the Safety and Efficacy of Afatinib When Administered as Therapy in Fanconi Anemia Patients With Unresectable and / or Metastatic Locoregionally Advanced Squamous Cell Carcinoma of the Oral Cavity, Oropharynx or Hypopharynx or Larynx.
This research study is a phase Ib/II, single-arm, non-randomized, non-blind, multicenter study designed to determine whether Afatinib is effective and safe in patients with locoregionally unresectable and / or metastatic HNSCC with Fanconi Anemia.
The main hypothesis, based on preclinical evidence, is that treatment with afatinib, an epithelial growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI), could be an effective treatment option to control cancer for patients with FA - HNSCC.
- Being tested
- Afatinib
- Medical subject headings
- Fanconi Anemia, Squamous Cell Carcinoma of Head and Neck
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AfatinibDrug
Afatinib starting at 20 mg (weeks 1-2), escalating to 30 mg after two weeks (weeks 3-4) and escalating to 40 mg after one month (week 5 - thereafter) if no hematologic or other relevant toxicities are observed (CTCAE V5.0 < grade 2)
| Study group | Role | What they receive |
|---|---|---|
| Afatinib | Experimental | Afatinib starting at 20 mg (weeks 1-2), escalating to 30 mg after two weeks (weeks 3-4) and escalating to 40 mg after one month (week 5 - thereafter) if no hematologic or other relevant toxicities are observed (CTCAE V5.0 < grade 2) Receives: Drug: Afatinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective Response Rate (ORR)
Assessed by the investigator through imaging follow-up (CT scan/MRI) using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. This will be considered as the percentage/proportion of patients with confirmed complete response (CR) or partial response (PR) as their overall best response throughout the first 9 months after the first dose of afatinib. Objective responses will be assessed locally by the investigator according to RECIST, V1.1, and indicating the change in size of tumors as compared with baseline, at the first dose of study treatment.
Measured: At 9-months after the first dose of study treatment
Secondary outcomes (8)
Disease control rate (DCR)
Percentage/proportion of patients with complete response (CR) or partial response (PR), according to ORR definition for the trial, or maintained stable disease (SD) as their overall best response, assessed by imaging follow-up (CT scan/MRI) and RECIST V1.1 criteria. Stable disease should be maintained for at least 4 months to be considered as a DCR event.
Measured: Throughout the study period, at least 9 months
Duration of response (DoR)
Time from first confirmed response (CR or PR), according to ORR definition for the trial, to the date of the documented progression of the disease (PD) as determined using RECIST V1.1 criteria or death due to any cause, whichever occurs first. Those patients with response and without PD or death event will be censored on the date of their last tumor assessment.
Measured: Throughout the study period, at least 9 months
Disease-free survival (DFS)
Time from first dosing date to the date of PD /secondary primary tumor (SPT) according to RECIST V1.1. Patients alive and free of events at the date of the analysis will be censored at their last known tumor assessment. Patients who start a new treatment line without progression /SPT will be censored on the date of first dose of the subsequent anticancer treatment.
Measured: Throughout the study period, at least 9 months
Overall survival (OS)
defined as the time elapsed from the first dose of study treatment until death from any cause. We will assess the median OS, estimated by Kaplan-Meier. Patients alive and free of events at the date of the analysis will be censored at their last known contact.
Measured: Throughout the study period, at least 9 months
Patient reported health-related quality of life (HRQoL) QLQ-C30
assessed through the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire C30 (EORTC QLQ-C30), version 3. The endpoint will report a numerical value that ranges from 0 to 100. Higher values indicate better performances status for all items but for symptoms, in which higher indicates greater or stronger symptoms.
Measured: At baseline and every 8 weeks until tumor recurrence, approximately 9 months
Patient reported health-related quality of life (HRQoL) QLQ-HN43
assessed through the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire EORTC head and neck cancer specific complementary module QLQ-HN43. The endpoint will report a numerical value that ranges from 0 to 100. Higher values indicate better performances status for all items but for symptoms, in which higher indicates greater or stronger symptoms.
Measured: At baseline and every 8 weeks until tumor recurrence, approximately 9 months
Patient reported health-related quality of life (HRQoL) EQ-5D
assessed through the EQ-5D Health Status Questionnaire. The endpoint will report a numerical value that ranges from 0 to 100. Higher values indicate better performances status for all items but for symptoms, in which higher indicates greater or stronger symptoms.
Measured: At baseline and every 8 weeks until tumor recurrence, approximately 9 months
Rate of severe toxicities
Percentage of patients experiencing Grade 3-5 treatment-related Adverse events (AEs) ) assessed by national cancer institute (NCI) Common Terminology Criteria for Adverse Events CTCAE v5.0
Measured: Throughout the study period, at least 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Oct 2024
- Study start8 Nov 2024
- Primary completionDec 2028 (estimated)
- Study completionDec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Fanconi AnemiaStudies of the same condition, open and under way.
- More trials run by Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant PauThe 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
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Collaborators
- MFAR Clinical Research S.L.
- Responsible party
- Sponsor
- Investigators
- Jordi Surrallés, M.D.; Ph.D., Study chair, Sant Pau's Hospital Research Institute
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06648096
- Sponsor's protocol ID
- IRSSP-001
- Other IDs
- 2024-511477-29-00
- Acronym
- AFAN
- Status last verified
- May 2026
- First submitted
- 15 Oct 2024
- Last updated
- 27 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No: No individual patient data will be shared
Publications
Anguera G, Gallego O, Llobet M, Berga N, Moreno-Martinez ME, Leon X, Kratz C, Garcia-Escudero R, Minguillon J, Surralles J. Opening of a phase Ib/II study to investigate the safety and efficacy of Afatinib in patients with Fanconi anemia and unresectable locally advanced or metastatic head and neck squamous cell carcinoma. BMC Cancer. 2025 Aug 26;25(1):1374. doi: 10.1186/s12885-025-14619-6.
PubMed 40859225
Study at a glance
From the registry record, last updated by the sponsor on 27 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase Ib/II Study to Investigate the Safety and Efficacy of Afatinib When Administered as Therapy in Fanconi Anemia Patients With Unresectable and / or Metastatic Locoregionally Advanced Squamous Cell Carcinoma of the Oral Cavity, Oropharynx or Hypopharynx or Larynx.
- ClinicalTrials.gov ID
- NCT06648096
- Acronym / sponsor ID
- AFAN / IRSSP-001
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant PauUniversity, hospital or other
- Collaborators
- MFAR Clinical Research S.L.
- Study start
- 8 Nov 2024 (actual)
- Primary completion
- Dec 2028 (estimated)
- Study completion
- Dec 2028 (estimated)
- First posted
- 18 Oct 2024 (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 research study is a phase Ib/II, single-arm, non-randomized, non-blind, multicenter study designed to determine whether Afatinib is effective and safe in patients with locoregionally unresectable and / or metastatic HNSCC with Fanconi Anemia.
The main hypothesis, based on preclinical evidence, is that treatment with afatinib, an epithelial growth factor receptor (EGFR) tyrosine kinase inhibitor (TKI), could be an effective treatment option to control cancer for patients with FA - HNSCC.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Afatinib | Experimental | Drug: AfatinibAfatinib starting at 20 mg (weeks 1-2), escalating to 30 mg after two weeks (weeks 3-4) and escalating to 40 mg after one month (week 5 - thereafter) if no hematologic or other relevant toxicities are observed (CTCAE V5.0 < grade 2) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (28 listed)
- Written informed consent according to local guidelines, must be signed and dated by the participant and investigator prior to performing any protocol procedure.
- Patient is ≥ 18 years of age.
- Confirmed diagnosis of Fanconi anemia.
- Histologically or cytologically confirmed unresectable or locoregionally advanced squamous cell carcinoma of the oral cavity, oropharynx, hypopharynx, larynx, nasopharynx, paranasal sinuses or salivary glands. Patients with distal metastasis (M1, American Joint Cancer Committee (AJCC) 8th ed.) are also eligible.
- Tumor not a candidate for resection prior to Afatinib due to technical inability to resect (tumor fixation / invasion in the skull base, cervical vertebrae, nasopharynx or fixed lymph nodes) and / or low surgical cure [T3-T4, N2-N3; , AJCC 8th ed.]).
Key exclusion criteria (21 listed)
- Patients who are candidates for surgery with curative intent are not eligible.
- Less than two weeks from surgical resection or other major surgical procedure at start of treatment. Planned surgery for other diseases.
- Previous treatment with EGFR small molecule inhibitors, EGFR inhibitory antibodies and / or any investigational agents for the treatment of HNSCC within 4 weeks prior to the selection was not allowed.
- Note: Previous treatment with chemotherapy and/or radiotherapy is allowed.
- Patient must have recovered from any previous treatment toxicity to Grade ≤ 2.
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 |
|---|---|---|
| AfatinibAfatinib starting at 20 mg (weeks 1-2), escalating to 30 mg after two weeks (weeks 3-4) and escalating to 40 mg after one month (week 5 - thereafter) if no hematologic or other relevant toxicities are observed (CTCAE V5.0 < grade 2) | Drug | Afatinib |
Primary outcome
Objective Response Rate (ORR)
Time frame: At 9-months after the first dose of study treatment
Assessed by the investigator through imaging follow-up (CT scan/MRI) using Response Evaluation Criteria in Solid Tumors (RECIST) v1.1. This will be considered as the percentage/proportion of patients with confirmed complete response (CR) or partial response (PR) as their overall best response throughout the first 9 months after the first dose of afatinib. Objective responses will be assessed locally by the investigator according to RECIST, V1.1, and indicating the change in size of tumors as compared with baseline, at the first dose of study treatment.
8 secondary outcomes
Disease control rate (DCR)
Time frame: Throughout the study period, at least 9 months
Duration of response (DoR)
Time frame: Throughout the study period, at least 9 months
Disease-free survival (DFS)
Time frame: Throughout the study period, at least 9 months
Overall survival (OS)
Time frame: Throughout the study period, at least 9 months
Patient reported health-related quality of life (HRQoL) QLQ-C30
Time frame: At baseline and every 8 weeks until tumor recurrence, approximately 9 months
Patient reported health-related quality of life (HRQoL) QLQ-HN43
Time frame: At baseline and every 8 weeks until tumor recurrence, approximately 9 months
Patient reported health-related quality of life (HRQoL) EQ-5D
Time frame: At baseline and every 8 weeks until tumor recurrence, approximately 9 months
Rate of severe toxicities
Time frame: Throughout the study period, at least 9 months
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 May 2026.
- Derived from this study: PubMed 40859225Anguera G, Gallego O, Llobet M, Berga N, Moreno-Martinez ME, Leon X, Kratz C, Garcia-Escudero R, Minguillon J, Surralles J. Opening of a phase Ib/II study to investigate the safety and efficacy of Afatinib in patients with Fanconi anemia and unresectable locally advanced or metastatic head and neck squamous cell carcinoma. BMC Cancer. 2025 Aug 26;25(1):1374. doi: 10.1186/s12885-025-14619-6.
- Individual participant dataSharing planned: No. No individual patient data will be shared
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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.
Jordi Surrallés, M.D.; Ph.D. study chair
Sant Pau's Hospital Research Institute
2 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Afatinib in people with Fanconi Anemia and Head and Neck Squamous Cell Carcinoma, run by Fundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau at 2 sites in 2 countries.
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 28 inclusion and 21 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: looking for participants now, as of the sponsor's last update on 27 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Germany, Spain. 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 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 A Responsible Person Designated by the Sponsor (+34 93 434 44 12, investigacion@mfar.net), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) (time frame: At 9-months after the first dose of study treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06648096, which also lists 1 linked publication.
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 May 2026.