Low GelsolinPhase 1/2NCT00671307
Ascending Single Dose Study of Rhu-pGelsolin in Patients With Decreased Gelsolin Levels
A Phase 1/2 study testing rhu-pGelsolin in people with Low Gelsolin and Trauma, run by Critical Biologics Corporation at 1 site in Hong Kong.
Trial summary
This study is designed to assess the pharmacokinetics and safety of an infusion of a single dose of recombinant plasma gelsolin (rhu-pGelsolin) when given to patients admitted to the Intensive Care Unit with documented low levels of natural gelsolin. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Low Gelsolin, Trauma, Infection
- What is being tested?
- rhu-pGelsolin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 40 participants
- Where
- 1 siteHong Kong
- Study dates
- Mar 2008to May 2010
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- >18 years of age
- Documented gelsolin level <100 mg/mL
- Admission to ICU
- Women of child-bearing age have a negative pregnancy test
- Multiple Organ Failure score < 4
- Catheter present through which blood samples can be taken
- Written Informed Consent obtained
Who cannot join
- Participation in other investigational treatment protocols
- Patients <18 years of age
- Patients who have a modified Multiple Organ Failure score of >=4
- Patient, who in the opinion of the Principal Investigator, are unlikely to be in the ICU for >48 hours
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Phase 1b Randomized, Double-blind, Placebo-controlled, Ascending Single Dose Infusion Trial of the Pharmacokinetics of Rhu-pGelsolin in Patients With Decreased Natural Gelsolin Levels
This study is designed to assess the pharmacokinetics and safety of an infusion of a single dose of recombinant plasma gelsolin (rhu-pGelsolin) when given to patients admitted to the Intensive Care Unit with documented low levels of natural gelsolin. It is believed that this drug will raise the gelsolin levels in these patients and decrease the probability that they will develop complications from their underlying disease such as organ system failure or death.
Read the detailed description
This is a randomized, double-blind, placebo-controlled, ascending single dose of rhu-pGelsolin infusion study. Thirteen subjects (10 active and 3 placebo) will be dosed at 3 mg/kg and 5 subjects (3 active and 2 placebo) will be dosed at 6 mg/kg. In addition, twenty-two subjects (18 active and 4 placebo) will be dosed at 6 mg/kg per day for 3 days. The Data Safety Monitoring Committee (DSMC) and Steering Committee will review the preceding dose safety data, and agree the tolerability prior to the enrolment for next higher dose. Blood samples will be collected during 1 hour infusion through 72 hours post dose for PK assessment after each dose.
- Conditions
- Low Gelsolin, Trauma, Infection, Burns
- Being tested
- rhu-pGelsolin, Placebo
- Keywords
- Gelsolin, Trauma, Pneumonia, Peritonitis, Burn, Infection, Sepsis, Systemic Inflammatory Response Syndrome
- Medical subject headings
- Wounds and Injuries, Infections, Burns, Meretoja syndrome, Pneumonia, Peritonitis, Sepsis, Systemic Inflammatory Response Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rhu-pGelsolinDrug
Also called Recombinant plasma gelsolin, CBC-100, Solinex
IV formulation of recombinant plasma gelsolin formulated as 10 mg/ml given as a single IV infusion over 1 hour
PlaceboDrug
Also called Vehicle control
Vehicle control given as IV infusion over 1 hour
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Placebo Receives: Drug: Placebo |
| Low dose | Experimental | 3 mg/kg of rhu-pGelsolin given as an IV infusion over 1 hour Receives: Drug: rhu-pGelsolin |
| Mid-dose | Experimental | 6 mg/kg of rhu-pGelsolin given as an IV infusion over 1 hour Receives: Drug: rhu-pGelsolin |
| High dose | Experimental | 6 mg/kg of rhu-pGelsolin given a an IV infusion over 1 hour once a day for 3 days Receives: Drug: rhu-pGelsolin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetics of plasma gelsolin
Measured: 72 hours
Secondary outcomes (3)
Adverse events and the development of anti-rhu-pGelsolin antibodies
Measured: 3 months
Pharmacodynamics of sepsis biomarkers
Measured: 72 hours
Clinical outcomes (mortality, duration of ICU stay, duration of ventilator support, duration of hospital stay)
Measured: 28 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2008 (estimated)
- Study startMar 2008
- Primary completionMar 2010
- Study completionMay 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (7)Papers the sponsor has linked to this study.
- Other trials for Low GelsolinStudies 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 40 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Critical Biologics Corporation
- Sponsor type
- Industry (pharma, biotech, devices)
- Investigators
- Wai M Chan, MBBS, Principal investigator, Queen Mary Hospital, Hong KongSelene Tam, PhD, Study director, University of Hong Kong; Clinical Trials CentreRichard C Straube, MD, Study chair, Critical Biologics Corporation
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00671307
- Sponsor's protocol ID
- CBC101
- Status last verified
- Apr 2010
- First submitted
- 30 Apr 2008
- Last updated
- 29 Sep 2010
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Lee PS, Waxman AB, Cotich KL, Chung SW, Perrella MA, Stossel TP. Plasma gelsolin is a marker and therapeutic agent in animal sepsis. Crit Care Med. 2007 Mar;35(3):849-55. doi: 10.1097/01.CCM.0000253815.26311.24.
PubMed 17205019Lee PS, Drager LR, Stossel TP, Moore FD, Rogers SO. Relationship of plasma gelsolin levels to outcomes in critically ill surgical patients. Ann Surg. 2006 Mar;243(3):399-403. doi: 10.1097/01.sla.0000201798.77133.55.
PubMed 16495706Christofidou-Solomidou M, Scherpereel A, Solomides CC, Christie JD, Stossel TP, Goelz S, DiNubile MJ. Recombinant plasma gelsolin diminishes the acute inflammatory response to hyperoxia in mice. J Investig Med. 2002 Jan;50(1):54-60. doi: 10.2310/6650.2002.33518.
PubMed 11813829Rothenbach PA, Dahl B, Schwartz JJ, O'Keefe GE, Yamamoto M, Lee WM, Horton JW, Yin HL, Turnage RH. Recombinant plasma gelsolin infusion attenuates burn-induced pulmonary microvascular dysfunction. J Appl Physiol (1985). 2004 Jan;96(1):25-31. doi: 10.1152/japplphysiol.01074.2002. Epub 2003 May 2.
PubMed 12730154Mounzer KC, Moncure M, Smith YR, Dinubile MJ. Relationship of admission plasma gelsolin levels to clinical outcomes in patients after major trauma. Am J Respir Crit Care Med. 1999 Nov;160(5 Pt 1):1673-81. doi: 10.1164/ajrccm.160.5.9807137.
PubMed 10556139DiNubile MJ, Stossel TP, Ljunghusen OC, Ferrara JL, Antin JH. Prognostic implications of declining plasma gelsolin levels after allogeneic stem cell transplantation. Blood. 2002 Dec 15;100(13):4367-71. doi: 10.1182/blood-2002-06-1672. Epub 2002 Aug 1.
PubMed 12393536Suhler E, Lin W, Yin HL, Lee WM. Decreased plasma gelsolin concentrations in acute liver failure, myocardial infarction, septic shock, and myonecrosis. Crit Care Med. 1997 Apr;25(4):594-8. doi: 10.1097/00003246-199704000-00007.
PubMed 9142022
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 1b Randomized, Double-blind, Placebo-controlled, Ascending Single Dose Infusion Trial of the Pharmacokinetics of Rhu-pGelsolin in Patients With Decreased Natural Gelsolin Levels
- ClinicalTrials.gov ID
- NCT00671307
- Acronym / sponsor ID
- CBC101
- Conditions
- Low Gelsolin, Trauma, Infection, Burns
- Keywords
- Gelsolin, Trauma, Pneumonia, Peritonitis, Burn, Infection, Sepsis, Systemic Inflammatory Response Syndrome
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 40 participants (estimated)
- Lead sponsor
- Critical Biologics CorporationIndustry (pharma, biotech, devices)
- Study start
- Mar 2008
- Primary completion
- Mar 2010 (actual)
- Study completion
- May 2010 (actual)
- First posted
- 5 May 2008 (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.
This study is designed to assess the pharmacokinetics and safety of an infusion of a single dose of recombinant plasma gelsolin (rhu-pGelsolin) when given to patients admitted to the Intensive Care Unit with documented low levels of natural gelsolin. It is believed that this drug will raise the gelsolin levels in these patients and decrease the probability that they will develop complications from their underlying disease such as organ system failure or death.
Detailed description
This is a randomized, double-blind, placebo-controlled, ascending single dose of rhu-pGelsolin infusion study. Thirteen subjects (10 active and 3 placebo) will be dosed at 3 mg/kg and 5 subjects (3 active and 2 placebo) will be dosed at 6 mg/kg. In addition, twenty-two subjects (18 active and 4 placebo) will be dosed at 6 mg/kg per day for 3 days. The Data Safety Monitoring Committee (DSMC) and Steering Committee will review the preceding dose safety data, and agree the tolerability prior to the enrolment for next higher dose. Blood samples will be collected during 1 hour infusion through 72 hours post dose for PK assessment after each dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: PlaceboPlacebo |
| Low dose | Experimental | Drug: rhu-pGelsolin3 mg/kg of rhu-pGelsolin given as an IV infusion over 1 hour |
| Mid-dose | Experimental | Drug: rhu-pGelsolin6 mg/kg of rhu-pGelsolin given as an IV infusion over 1 hour |
| High dose | Experimental | Drug: rhu-pGelsolin6 mg/kg of rhu-pGelsolin given a an IV infusion over 1 hour once a day for 3 days |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- >18 years of age
- Documented gelsolin level <100 mg/mL
- Admission to ICU
- Women of child-bearing age have a negative pregnancy test
- Multiple Organ Failure score < 4
Key exclusion criteria (4 listed)
- Participation in other investigational treatment protocols
- Patients <18 years of age
- Patients who have a modified Multiple Organ Failure score of >=4
- Patient, who in the opinion of the Principal Investigator, are unlikely to be in the ICU for >48 hours
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 |
|---|---|---|
| rhu-pGelsolinAlso: Recombinant plasma gelsolin, CBC-100, SolinexIV formulation of recombinant plasma gelsolin formulated as 10 mg/ml given as a single IV infusion over 1 hour | Drug | High dose, Low dose, Mid-dose |
| PlaceboAlso: Vehicle controlVehicle control given as IV infusion over 1 hour | Drug | Placebo |
Primary outcome
Pharmacokinetics of plasma gelsolin
Time frame: 72 hours
3 secondary outcomes
Adverse events and the development of anti-rhu-pGelsolin antibodies
Time frame: 3 months
Pharmacodynamics of sepsis biomarkers
Time frame: 72 hours
Clinical outcomes (mortality, duration of ICU stay, duration of ventilator support, duration of hospital stay)
Time frame: 28 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2010.
- Background: PubMed 17205019Lee PS, Waxman AB, Cotich KL, Chung SW, Perrella MA, Stossel TP. Plasma gelsolin is a marker and therapeutic agent in animal sepsis. Crit Care Med. 2007 Mar;35(3):849-55. doi: 10.1097/01.CCM.0000253815.26311.24.
- Background: PubMed 16495706Lee PS, Drager LR, Stossel TP, Moore FD, Rogers SO. Relationship of plasma gelsolin levels to outcomes in critically ill surgical patients. Ann Surg. 2006 Mar;243(3):399-403. doi: 10.1097/01.sla.0000201798.77133.55.
- Background: PubMed 11813829Christofidou-Solomidou M, Scherpereel A, Solomides CC, Christie JD, Stossel TP, Goelz S, DiNubile MJ. Recombinant plasma gelsolin diminishes the acute inflammatory response to hyperoxia in mice. J Investig Med. 2002 Jan;50(1):54-60. doi: 10.2310/6650.2002.33518.
- Background: PubMed 12730154Rothenbach PA, Dahl B, Schwartz JJ, O'Keefe GE, Yamamoto M, Lee WM, Horton JW, Yin HL, Turnage RH. Recombinant plasma gelsolin infusion attenuates burn-induced pulmonary microvascular dysfunction. J Appl Physiol (1985). 2004 Jan;96(1):25-31. doi: 10.1152/japplphysiol.01074.2002. Epub 2003 May 2.
- Background: PubMed 10556139Mounzer KC, Moncure M, Smith YR, Dinubile MJ. Relationship of admission plasma gelsolin levels to clinical outcomes in patients after major trauma. Am J Respir Crit Care Med. 1999 Nov;160(5 Pt 1):1673-81. doi: 10.1164/ajrccm.160.5.9807137.
- Background: PubMed 12393536DiNubile MJ, Stossel TP, Ljunghusen OC, Ferrara JL, Antin JH. Prognostic implications of declining plasma gelsolin levels after allogeneic stem cell transplantation. Blood. 2002 Dec 15;100(13):4367-71. doi: 10.1182/blood-2002-06-1672. Epub 2002 Aug 1.
- Background: PubMed 9142022Suhler E, Lin W, Yin HL, Lee WM. Decreased plasma gelsolin concentrations in acute liver failure, myocardial infarction, septic shock, and myonecrosis. Crit Care Med. 1997 Apr;25(4):594-8. doi: 10.1097/00003246-199704000-00007.
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.
Wai M Chan, MBBS principal investigator
Queen Mary Hospital, Hong Kong
Selene Tam, PhD study director
University of Hong Kong; Clinical Trials Centre
Richard C Straube, MD study chair
Critical Biologics Corporation
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing rhu-pGelsolin in people with Low Gelsolin and Trauma, run by Critical Biologics Corporation at 1 site in Hong Kong.
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 7 inclusion and 4 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: finished, as of the sponsor's last update on 29 Sep 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Hong Kong. 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. Some participants in this study may receive a placebo. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Pharmacokinetics of plasma gelsolin (time frame: 72 hours). 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 NCT00671307, which also lists 7 linked publications.
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 29 Sep 2010.