Neutropenia, Severe ChronicPhase 1/2NCT02533362
ANF-Rho in the Treatment of Chronic Neutropenia
A Phase 1/2 study testing ANF-Rho in people with Neutropenia, Severe Chronic, run by Prolong Pharmaceuticals.
Trial summary
A 6 month safety, tolerability, efficacy and pharmacokinetic study with ANF-Rho to treat patients with chronic neutropenia. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Neutropenia, Severe Chronic
- What is being tested?
- ANF-Rho
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- Oct 2015to Oct 2016
- 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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients 1 years of age or older
- Patients with established chronic neutropenia defined as Median Absolute Neutrophil Count (ANC) < 0.5 x 109/L (both with and without demonstrated genetic lesion) having an indication for treatment, including:
- Independent of hematological parameters, all patients with: Shwachman-Diamond syndrome (SDS), Barth's syndrome or other inherited diseases associated with neutropenia (exclude Glycogen Storage Disease 1b)
- Patients on granulocyte-colony stimulating factor (GCSF) \& PEG-GCSF treatment are still eligible to participate after a washout period of 7 days, after the stopping of the drug
- Signed and dated informed written consent/assent by the patient/parent
- Women of childbearing potential with a negative serum pregnancy test and using a reliable method of contraception during the study period. Male study participants also agreeing to use contraception for the study period.
Who cannot join
- Evidence of chromosomal abnormalities, myelodysplasia, hematologic malignancy, aplastic anemia, systemic lupus erythematosus, or rheumatoid arthritis (Felty's syndrome) or if the neutropenia was drug-induced
- Progressive malignant disease or malignancy history
- Presence of macrophage activation syndrome before the diagnosis of neutropenia
- Clinical Significant Abnormal Renal, Cardiac, Hepatic or Blood Coagulation disease.
- Chronic infection such as hepatitis B virus (HBV), hepatitis C Virus (HCV) or Human immunodeficiency virus( HIV) or history of tuberculosis
- Association with anemia, thrombocytopenia (low blood platelets) before the diagnosis of neutropenia.
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- Drug abuse, substance abuse, or alcohol abuse
- Use of any other investigational drug at the time of enrollment, or within 5 half-lives prior to enrollment, whichever is longer
- Patients unwilling and/or who are not capable of ensuring compliance with the provisions of the study protocol
- Women who are pregnant or breastfeeding
- Women of childbearing potential who do not use an approved method of birth control. Periodic abstinence (e.g., calendar, ovulation, symptothermal, post ovulation methods) is not acceptable
- Patients with known DNA loss-of-function mutations in GCSFR and RUNX1 genes
About this study
Official title: A Phase 2, Open-Label, Single Center, Efficacy, Safety, Tolerability, and Pharmacokinetic Study of ANF-Rho™ in Patients With Chronic Neutropenia
A 6 month safety, tolerability, efficacy and pharmacokinetic study with ANF-Rho to treat patients with chronic neutropenia.
Read the detailed description
Chronic neutropenia patients who are eligible for participation will be screened and enrolled to received open-label ANF-Rho for a period of 6 months. Study participation will be separated into 2 periods, Induction (8 weeks) and Maintenance (16 weeks). Blood samples to measure neutrophil levels and biochemistry labs will be drawn throughout the study to monitor efficacy response and patient safety. Pharmacokinetic samples will be collected during both periods and questionnaires will be completed by the patient and investigator for bone pain, quality of life (QOL) and injection site reaction.
- Conditions
- Neutropenia, Severe Chronic
- Being tested
- ANF-Rho
- Medical subject headings
- Neutropenia, severe chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ANF-RhoDrug
Also called pegfilgrastim ANF
Pegylated version of recombinant human granulocyte-colony stimulating factor (G-CSF)
| Study group | Role | What they receive |
|---|---|---|
| ANF-Rho | Experimental | pegfilgrastim Anti-Neutropenic Factor (ANF) Receives: Drug: ANF-Rho |
What is being measured
The primary outcome is the main question the study is designed to answer.
Neutrophil response rate to ANF Rho following a 6 month treatment period (induction, stabilization and maintenance periods) in patients with Chronic Neutropenia.
Median Absolute Neutrophil Count (ANC) of ≥1.0 x 109/L over the 6 months total treatment period
Measured: 6 months
Secondary outcomes (8)
Serum concentration of ANF-Rho over time
Pharmacokinetic parameter measuring ANF-Rho levels over time
Measured: 6 months
The peak plasma concentration of ANF-Rho after administration (Cmax) over time
Pharmacokinetic Parameter measuring ANF-Rho levels over time
Measured: 6 months
Total ANF-Rho exposure (AUC)
Pharmacokinetic parameter measuring ANF-Rho levels from time 0 through last dose.
Measured: 6 months
Time to peak plasma concentration of ANF-Rho (Tmax)
Pharmacokinetic parameter measuring the time to Cmax.
Measured: 6 months
Half-Life of ANF-Rho (T1/2)
Pharmacokinetic parameter to measure the time required for the concentration of the drug to reach half of its original value
Measured: 6 months
Rate of subject reported infection related morbidities (infections, hospitalisations and antibiotic use) in Chronic Neutropenia patients treated with ANF Rho
Measured: 6 months
Evaluate the effect of ANF Rho on the Quality of Life and Bone Pain in Chronic Neutropenia patients.
Change in Quality of life over 6 months measured by the Short Form 36, change in bone pain over 6 months measured by the Bone Pain Questionnaire, Wong and Baker Scale and FLACC Behavioral Pain Assessment Scale
Measured: 6 months
Evaluate safety and tolerability of subcutaneous injections of ANF Rho in chronic neutropenia patients.
Composite endpoint with multiple vital signs, bone scans, biopsies, splenic ultrasounds, electrocardiographic assessments, clinical signs, and bio-analytical measures (e.g., biochemistry), and reported adverse events following repeated dosing.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Aug 2015 (estimated)
- Study startOct 2015
- Primary completionOct 2016 (estimated)
- Study completionOct 2016 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neutropenia, Severe ChronicStudies 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
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Prolong Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hemant Misra, PhD, Study director, Prolong Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02533362
- Sponsor's protocol ID
- PGCN-002
- Status last verified
- Aug 2015
- First submitted
- 10 Aug 2015
- Last updated
- 6 Mar 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 6 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 2, Open-Label, Single Center, Efficacy, Safety, Tolerability, and Pharmacokinetic Study of ANF-Rho™ in Patients With Chronic Neutropenia
- ClinicalTrials.gov ID
- NCT02533362
- Acronym / sponsor ID
- PGCN-002
- Conditions
- Neutropenia, Severe Chronic
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Prolong PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Oct 2015
- Primary completion
- Oct 2016 (estimated)
- Study completion
- Oct 2016 (estimated)
- First posted
- 26 Aug 2015 (estimated)
- 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.
A 6 month safety, tolerability, efficacy and pharmacokinetic study with ANF-Rho to treat patients with chronic neutropenia.
Detailed description
Chronic neutropenia patients who are eligible for participation will be screened and enrolled to received open-label ANF-Rho for a period of 6 months. Study participation will be separated into 2 periods, Induction (8 weeks) and Maintenance (16 weeks). Blood samples to measure neutrophil levels and biochemistry labs will be drawn throughout the study to monitor efficacy response and patient safety. Pharmacokinetic samples will be collected during both periods and questionnaires will be completed by the patient and investigator for bone pain, quality of life (QOL) and injection site reaction.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ANF-Rho | Experimental | Drug: ANF-Rhopegfilgrastim Anti-Neutropenic Factor (ANF) |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (6 listed)
- Patients 1 years of age or older
- Patients with established chronic neutropenia defined as Median Absolute Neutrophil Count (ANC) < 0.5 x 109/L (both with and without demonstrated genetic lesion) having an indication for treatment, including:
- Independent of hematological parameters, all patients with: Shwachman-Diamond syndrome (SDS), Barth's syndrome or other inherited diseases associated with neutropenia (exclude Glycogen Storage Disease 1b)
- Patients on granulocyte-colony stimulating factor (GCSF) \& PEG-GCSF treatment are still eligible to participate after a washout period of 7 days, after the stopping of the drug
- Signed and dated informed written consent/assent by the patient/parent
Key exclusion criteria (12 listed)
- Evidence of chromosomal abnormalities, myelodysplasia, hematologic malignancy, aplastic anemia, systemic lupus erythematosus, or rheumatoid arthritis (Felty's syndrome) or if the neutropenia was drug-induced
- Progressive malignant disease or malignancy history
- Presence of macrophage activation syndrome before the diagnosis of neutropenia
- Clinical Significant Abnormal Renal, Cardiac, Hepatic or Blood Coagulation disease.
- Chronic infection such as hepatitis B virus (HBV), hepatitis C Virus (HCV) or Human immunodeficiency virus( HIV) or history of tuberculosis
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 |
|---|---|---|
| ANF-RhoAlso: pegfilgrastim ANFPegylated version of recombinant human granulocyte-colony stimulating factor (G-CSF) | Drug | ANF-Rho |
Primary outcome
Neutrophil response rate to ANF Rho following a 6 month treatment period (induction, stabilization and maintenance periods) in patients with Chronic Neutropenia.
Time frame: 6 months
Median Absolute Neutrophil Count (ANC) of ≥1.0 x 109/L over the 6 months total treatment period
8 secondary outcomes
Serum concentration of ANF-Rho over time
Time frame: 6 months
The peak plasma concentration of ANF-Rho after administration (Cmax) over time
Time frame: 6 months
Total ANF-Rho exposure (AUC)
Time frame: 6 months
Time to peak plasma concentration of ANF-Rho (Tmax)
Time frame: 6 months
Half-Life of ANF-Rho (T1/2)
Time frame: 6 months
Rate of subject reported infection related morbidities (infections, hospitalisations and antibiotic use) in Chronic Neutropenia patients treated with ANF Rho
Time frame: 6 months
Evaluate the effect of ANF Rho on the Quality of Life and Bone Pain in Chronic Neutropenia patients.
Time frame: 6 months
Evaluate safety and tolerability of subcutaneous injections of ANF Rho in chronic neutropenia patients.
Time frame: 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 2019.
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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.
Hemant Misra, PhD study director
Prolong Pharmaceuticals
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ANF-Rho in people with Neutropenia, Severe Chronic, run by Prolong Pharmaceuticals.
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 6 inclusion and 12 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 2019. 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?
Neutrophil response rate to ANF Rho following a 6 month treatment period (induction, stabilization and maintenance periods) in patients with Chronic Neutropenia. (time frame: 6 months). 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 2019.