Fanconi AnemiaPhase 1/2NCT04522375
A Dose Escalation Study of FP-045 in Patients With Fanconi Anemia
A Phase 1/2 study testing FP-045 in people with Fanconi Anemia, run by Foresee Pharmaceuticals Co., Ltd..
Trial summary
This is a multi-center, Phase 1/2 study to determine the Optimal Biologic Dose (OBD) and to evaluate the safety, tolerability, PK, and preliminary activity of FP 045 when administered orally in young adult/adolescent and pediatric patients with Fanconi anemia. Read more about this study
- Who is it for?
- Ages 3 to 35 years
- Conditions the study is for
- Fanconi Anemia
- What is being tested?
- FP-045
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 30 Jun 2023to Dec 2025
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
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 3 to 35 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- male or female aged 3-35
- documented Fanconi anemia by chromosome breakage analysis
- females of child-bearing potential and males required to use highly effective birth control
- mild to moderate bone marrow failure with at least one cytopenia of > grade 1 severity
Who cannot join
- history of any malignancy except focal squamous cell or basal cell carcinoma of the skin or carcinoma in situ of cervix
- has myelodysplastic syndrome or acute leukemia per world health organization (WHO) criteria
- has history of any significant medical conditions
- has aspartate aminotransferase (AST)/alanine aminotransferase (ALT) > 5x upper limit of normal (ULN) or calculated creatinine clearance (Clcr) of < 50 mL/min
- has active Hepatitis B or C
- has an ongoing systemic infection
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- requires a strong CYP3A4 inhibitor
- has had major surgery within 30 days
- Active graft versus host disease requiring systemic treatment
- Has a history of bone marrow or stem cell transplant
About this study
Official title: A Multinational, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Activity of FP 045 in Patients With Fanconi Anemia (FusuciA Study)
This is a multi-center, Phase 1/2 study to determine the Optimal Biologic Dose (OBD) and to evaluate the safety, tolerability, PK, and preliminary activity of FP 045 when administered orally in young adult/adolescent and pediatric patients with Fanconi anemia. The study will enroll a total of 4 young adult/adolescent patients and a minimum of 8 and up to 12 pediatric patients with mild-moderate bone marrow failure who have not undergone hematopoietic cell transplant. This makes the total patient number between 12-16 total. Dose escalation will occur individually for each patient, within each age group. Each patient will receive each of 3 dose levels of FP 045 (intra-patient dose escalation), beginning with Dose Level 1, followed by Dose Levels 2 and 3. Each dose level will be administered for 28 days prior to escalation to the next higher dose level for that patient.
Read the detailed description
Dose escalation will begin with young adult/adolescent patients. The initial two patients enrolled in the study will be > 15 years of age. These patients must complete the entire 28-day period of treatment at Dose Level 1 prior to additional young adult/adolescent patients being enrolled. All 4 young adult/adolescent patients must complete 28 days of treatment at Dose Level 1, and cumulative safety must be reviewed by the Safety Review Committee (SRC), prior to the enrollment of pediatric patients. The initial two pediatric patients enrolled will be > 6 years of age. These patients must complete the entire 28-day period of treatment at Dose Level 1 prior to additional pediatric patients being enrolled. A minimum of 8 and maximum of 12 pediatric patients will be enrolled to allow for at least 4 patients between the ages of 3-6.
Study assessments will be conducted at each visit. Patients will be observed closely for Dose Limiting Toxicity (DLT) during each dosing period. Any patient experiencing a DLT will have study drug interrupted and will not be allowed to escalate to the next higher dose level. The patient may resume treatment at one dose level lower once the DLT has resolved to baseline or to ≤ Grade 1 in severity. The MTD will be defined as the dose level immediately below the dose level at which DLT occurred. Patients requiring an interruption in treatment of > 3 weeks following a DLT will be withdrawn from the study. The MTD will be assessed separately for each individual patient.
Following the completion of dose escalation, each patient will continue treatment at either the highest dose or their individual MTD, and then transition to the OBD for their age group (once defined), for a total of 3 months. Patients failing to receive 75% of planned doses for reasons other than adverse effects may be replaced.
- Conditions
- Fanconi Anemia
- Being tested
- FP-045
- Medical subject headings
- Fanconi Anemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FP-045Drug
activator of aldehyde dehydrogenase
| Study group | Role | What they receive |
|---|---|---|
| FP-045 | Experimental | The study will enroll a total of 6 young adult/adolescent patients progressing through three dose levels, followed by a minimum of 8 and up to 12 pediatric patients progressing through up to three dose levels. Receives: Drug: FP-045 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Optimal Biologic Dose (OBP) of FP-045
The OBP of FP-045 in adolescent and pediatric subjects
Measured: 28 days x up to 3 doses
stabilizing or improving cytopenia in FA
Change from baseline in hemoglobin
Measured: 3 months
Secondary outcomes (2)
Safety and tolerability
Frequency of adverse events and serious adverse events
Measured: 3-6 months
pharmacokinetic profile
Mean AUC of FP-045 by dose level
Measured: 3- 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Aug 2020
- Study start30 Jun 2023
- Primary completionJun 2025 (estimated)
- Study completionDec 2025 (estimated)
Why it stopped: Twenty months after investigative site initiations, and best efforts to identify study participants, no participants were identified.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Fanconi AnemiaStudies 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
- Not applicable
- Intervention model
- Single group
- Model description
- Phase of dose escalation to OBD followed by 3 months of treatment
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Foresee Pharmaceuticals Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Susan Whitaker, Study director, Foresee Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04522375
- Sponsor's protocol ID
- FP045C-19-001
- Status last verified
- Mar 2025
- First submitted
- 3 Aug 2020
- Last updated
- 6 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multinational, Multicenter, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Activity of FP 045 in Patients With Fanconi Anemia (FusuciA Study)
- ClinicalTrials.gov ID
- NCT04522375
- Acronym / sponsor ID
- FP045C-19-001
- Conditions
- Fanconi Anemia
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Twenty months after investigative site initiations, and best efforts to identify study participants, no participants were identified.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Foresee Pharmaceuticals Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 30 Jun 2023 (actual)
- Primary completion
- Jun 2025 (estimated)
- Study completion
- Dec 2025 (estimated)
- First posted
- 21 Aug 2020 (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 is a multi-center, Phase 1/2 study to determine the Optimal Biologic Dose (OBD) and to evaluate the safety, tolerability, PK, and preliminary activity of FP 045 when administered orally in young adult/adolescent and pediatric patients with Fanconi anemia. The study will enroll a total of 4 young adult/adolescent patients and a minimum of 8 and up to 12 pediatric patients with mild-moderate bone marrow failure who have not undergone hematopoietic cell transplant. This makes the total patient number between 12-16 total. Dose escalation will occur individually for each patient, within each age group. Each patient will receive each of 3 dose levels of FP 045 (intra-patient dose escalation), beginning with Dose Level 1, followed by Dose Levels 2 and 3. Each dose level will be administered for 28 days prior to escalation to the next higher dose level for that patient.
Detailed description
Dose escalation will begin with young adult/adolescent patients. The initial two patients enrolled in the study will be > 15 years of age. These patients must complete the entire 28-day period of treatment at Dose Level 1 prior to additional young adult/adolescent patients being enrolled. All 4 young adult/adolescent patients must complete 28 days of treatment at Dose Level 1, and cumulative safety must be reviewed by the Safety Review Committee (SRC), prior to the enrollment of pediatric patients. The initial two pediatric patients enrolled will be > 6 years of age. These patients must complete the entire 28-day period of treatment at Dose Level 1 prior to additional pediatric patients being enrolled. A minimum of 8 and maximum of 12 pediatric patients will be enrolled to allow for at least 4 patients between the ages of 3-6.
Study assessments will be conducted at each visit. Patients will be observed closely for Dose Limiting Toxicity (DLT) during each dosing period. Any patient experiencing a DLT will have study drug interrupted and will not be allowed to escalate to the next higher dose level. The patient may resume treatment at one dose level lower once the DLT has resolved to baseline or to ≤ Grade 1 in severity. The MTD will be defined as the dose level immediately below the dose level at which DLT occurred. Patients requiring an interruption in treatment of > 3 weeks following a DLT will be withdrawn from the study. The MTD will be assessed separately for each individual patient.
Following the completion of dose escalation, each patient will continue treatment at either the highest dose or their individual MTD, and then transition to the OBD for their age group (once defined), for a total of 3 months. Patients failing to receive 75% of planned doses for reasons other than adverse effects may be replaced.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Phase of dose escalation to OBD followed by 3 months of treatment
| Arm | Type | Receives |
|---|---|---|
| FP-045 | Experimental | Drug: FP-045The study will enroll a total of 6 young adult/adolescent patients progressing through three dose levels, followed by a minimum of 8 and up to 12 pediatric patients progressing through up to three dose levels. |
Who may qualify
- Ages
- 3 to 35 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- male or female aged 3-35
- documented Fanconi anemia by chromosome breakage analysis
- females of child-bearing potential and males required to use highly effective birth control
- mild to moderate bone marrow failure with at least one cytopenia of > grade 1 severity
Key exclusion criteria (10 listed)
- history of any malignancy except focal squamous cell or basal cell carcinoma of the skin or carcinoma in situ of cervix
- has myelodysplastic syndrome or acute leukemia per world health organization (WHO) criteria
- has history of any significant medical conditions
- has aspartate aminotransferase (AST)/alanine aminotransferase (ALT) > 5x upper limit of normal (ULN) or calculated creatinine clearance (Clcr) of < 50 mL/min
- has active Hepatitis B or C
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 |
|---|---|---|
| FP-045activator of aldehyde dehydrogenase | Drug | FP-045 |
Primary outcome
The Optimal Biologic Dose (OBP) of FP-045
Time frame: 28 days x up to 3 doses
The OBP of FP-045 in adolescent and pediatric subjects
stabilizing or improving cytopenia in FA
Time frame: 3 months
Change from baseline in hemoglobin
2 secondary outcomes
Safety and tolerability
Time frame: 3-6 months
pharmacokinetic profile
Time frame: 3- 6 months
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Mar 2025.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
Susan Whitaker study director
Foresee Pharmaceuticals
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing FP-045 in people with Fanconi Anemia, run by Foresee Pharmaceuticals Co., Ltd..
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 4 inclusion and 10 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: withdrawn, as of the sponsor's last update on 6 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.
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?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Optimal Biologic Dose (OBP) of FP-045 (time frame: 28 days x up to 3 doses). The study lists 2 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 6 Mar 2025.