Chronic Inflammatory Demyelinating PolyradiculoneuropathyPhase 2/3POINTNCT01225276
Safety and Efficacy Study of Three Different Dosages of NewGam in Patients With CIDP
A Phase 2/3 study testing NewGam 10% in people with Chronic Inflammatory Demyelinating Polyradiculoneuropathy, run by Octapharma.
Trial summary
NewGam (current working title for a new IGIV formulation) is a newly developed human normal immunoglobulin solution ready for intravenous administration (IGIV). This study will evaluate the safety and efficacy of three different dosages of NewGam 10% in patients with Chronic Inflammatory Demyelinating… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Chronic Inflammatory Demyelinating Polyradiculoneuropathy
- What is being tested?
- NewGam 10%
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 2 participants
- Where
- No sites listed yet
- Study dates
- Oct 2011to Oct 2012
- Phase
- Phase 2/3
- 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
- Patients diagnosed as having CIDP based on fulfilment of clinical criteria of the INCAT Group and the definite electrophysiological criteria for CIDP ; patients with MADSAM or pure motor CIDP will be included provided they fulfil these criteria
- Worsening of disability and objective increase in weakness or sensory deficit during the 6 months prior to screening
- >=18 years of age
Who cannot join
- Unifocal forms of CIDP
- Pure sensory CIDP
- MMN with conduction block
- Treatment of CIDP with immunoglobulins (intravenous or subcutaneous) at any time prior to study entry
- Steroids of any type equivalent to prednisolone or prednisone > 10 mg/day or equivalent plasma exchange (PE) during the last 3 months prior to baseline visit
- Treatment with cyclosporin, methotrexate, mitoxantrone, mycophenolate mofetil, interferon or other immunosuppressive or immunomodulatory drugs during the three months prior to baseline visit
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- Clinical evidence of peripheral neuropathy from another
- Known diabetes mellitus
- Other serious medical condition complicating assessment or treatment
- Thromboembolic events: patients with a history of deep vein thrombosis (DVT) within the last year prior to baseline visit or pulmonary embolism ever
- Known IgA deficiency with antibodies to IgA
- History of hypersensitivity, anaphylaxis or severe systemic response to immunoglobulin, blood or plasma derived products, or any component of NewGam
- Known blood hyperviscosity
About this study
Official title: Double-blind, Placebo-controlled, Randomised, Multicentre, Adaptive, Two-stage Phase 2/3 Study Evaluating Safety and Efficacy of Three Dosages of NewGam in CIDP Patients
NewGam (current working title for a new IGIV formulation) is a newly developed human normal immunoglobulin solution ready for intravenous administration (IGIV). This study will evaluate the safety and efficacy of three different dosages of NewGam 10% in patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy.
Read the detailed description
This is a Phase 2/3 study that will take place in 2 stages. The primary objective of Stage 1 (Phase 2 dose-finding part)is to determine and select one dosage from three NewGam maintenance dosage arms in comparison with a placebo arm, based on the percentage of responders (response defined as a decrease, meaning improvement, in the adjusted INCAT disability score by at least 1 point). The selected NewGam dosage and placebo will be employed and compared in Stage 2.
The primary objective of Stage 2 (Phase 3 confirmatory part) is to demonstrate superiority of the maintenance dosage regimen selected at study Stage 1 over placebo in patients with CIDP as assessed by the percentage of responders.
The secondary objective is to evaluate the safety (measured by number of adverse events)and efficacy of NewGam administration in patients with CIDP compared to baseline.
- Being tested
- NewGam 10%, Placebo
- Keywords
- CIDP, IVIG
- Medical subject headings
- Polyradiculoneuropathy, Chronic Inflammatory Demyelinating
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NewGam 10%Drug
Loading dose at baseline (Week 0) will be 2.0 g/kg NewGam . The maintenance doses to be infused 7 times will be 2.0 g/kg every 21 (+/-4) days.
PlaceboDrug
Also called 0.9% saline solution
Loading dose at baseline (Week 0) in Placebo arm will be corresponding volume of an authorised 0.9% saline solution . The maintenance doses of the 0.9% saline solution to be infused 7 times will be given every 21 (+/-4) days.
NewGam 10%Drug
Loading dose at baseline (Week 0) will be 2.0 g/kg NewGam in all three NewGam treatment arms. The maintenance doses to be infused 7 times will be 1.0 g/kg every 21 (+/-4) days.
NewGam 10%Drug
Loading dose at baseline (Week 0) will be 2.0 g/kg NewGam in all three NewGam treatment arms. The maintenance doses to be infused 7 times will be 0.4 g/kg every 21 (+/-4) days.
| Study group | Role | What they receive |
|---|---|---|
| Dosage Arm 1 | Experimental | NewGam 10% 0.4 g/kg Receives: Drug: NewGam 10% |
| Dosage Arm 2 | Experimental | NewGam 10% 1.0 g/kg Receives: Drug: NewGam 10% |
| Dosage Arm 3 | Experimental | NewGam 10% 2.0 g/kg Receives: Drug: NewGam 10% |
| Dosage Arm 4 | Placebo comparator | Placebo 0.9% Saline Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adjusted INCAT disability score
Measured: Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)
Secondary outcomes (4)
Vital Signs
Measured: During each infusion - Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)
Grip Strength
Measured: Visit 9 & 13
Nerve Conduction Studies
Measured: Visti 9 & 13
Motor Impairment Assessment utlizing the Expanded MRC Sum Score
Expanded 'Medical Research Council sum score' will be measured as improvement in MRC units .
Measured: Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Oct 2010 (estimated)
- Study startOct 2011
- Primary completionOct 2012
- Study completionOct 2012
Why it stopped: Study Terminated.priority changes in product development.
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 Chronic Inflammatory Demyelinating PolyradiculoneuropathyStudies of the same condition, open and under way.
- More trials run by OctapharmaThe 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Wolfgang Frenzel, MD, Study director, Octapharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01225276
- Sponsor's protocol ID
- NGAM-03
- Acronym
- POINT
- Status last verified
- Feb 2017
- First submitted
- 6 Oct 2010
- Last updated
- 23 Feb 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No: no (IPD) data exist for this study
Publications
Bus SR, de Haan RJ, Vermeulen M, van Schaik IN, Eftimov F. Intravenous immunoglobulin for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev. 2024 Feb 14;2(2):CD001797. doi: 10.1002/14651858.CD001797.pub4.
PubMed 38353301
Study at a glance
From the registry record, last updated by the sponsor on 23 Feb 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Double-blind, Placebo-controlled, Randomised, Multicentre, Adaptive, Two-stage Phase 2/3 Study Evaluating Safety and Efficacy of Three Dosages of NewGam in CIDP Patients
- ClinicalTrials.gov ID
- NCT01225276
- Acronym / sponsor ID
- POINT / NGAM-03
- Keywords
- CIDP, IVIG
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study Terminated.priority changes in product development.
- Enrolment
- 2 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- Oct 2011
- Primary completion
- Oct 2012 (actual)
- Study completion
- Oct 2012 (actual)
- First posted
- 20 Oct 2010 (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.
NewGam (current working title for a new IGIV formulation) is a newly developed human normal immunoglobulin solution ready for intravenous administration (IGIV). This study will evaluate the safety and efficacy of three different dosages of NewGam 10% in patients with Chronic Inflammatory Demyelinating Polyradiculoneuropathy.
Detailed description
This is a Phase 2/3 study that will take place in 2 stages. The primary objective of Stage 1 (Phase 2 dose-finding part)is to determine and select one dosage from three NewGam maintenance dosage arms in comparison with a placebo arm, based on the percentage of responders (response defined as a decrease, meaning improvement, in the adjusted INCAT disability score by at least 1 point). The selected NewGam dosage and placebo will be employed and compared in Stage 2.
The primary objective of Stage 2 (Phase 3 confirmatory part) is to demonstrate superiority of the maintenance dosage regimen selected at study Stage 1 over placebo in patients with CIDP as assessed by the percentage of responders.
The secondary objective is to evaluate the safety (measured by number of adverse events)and efficacy of NewGam administration in patients with CIDP compared to baseline.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Dosage Arm 1 | Experimental | Drug: NewGam 10%NewGam 10% 0.4 g/kg |
| Dosage Arm 2 | Experimental | Drug: NewGam 10%NewGam 10% 1.0 g/kg |
| Dosage Arm 3 | Experimental | Drug: NewGam 10%NewGam 10% 2.0 g/kg |
| Dosage Arm 4 | Placebo comparator | Drug: PlaceboPlacebo 0.9% Saline |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Patients diagnosed as having CIDP based on fulfilment of clinical criteria of the INCAT Group and the definite electrophysiological criteria for CIDP ; patients with MADSAM or pure motor CIDP will be included provided they fulfil these criteria
- Worsening of disability and objective increase in weakness or sensory deficit during the 6 months prior to screening
- >=18 years of age
Key exclusion criteria (13 listed)
- Unifocal forms of CIDP
- Pure sensory CIDP
- MMN with conduction block
- Treatment of CIDP with immunoglobulins (intravenous or subcutaneous) at any time prior to study entry
- Steroids of any type equivalent to prednisolone or prednisone > 10 mg/day or equivalent plasma exchange (PE) during the last 3 months prior to baseline visit
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 |
|---|---|---|
| NewGam 10%Loading dose at baseline (Week 0) will be 2.0 g/kg NewGam . The maintenance doses to be infused 7 times will be 2.0 g/kg every 21 (+/-4) days. | Drug | Dosage Arm 1 |
| PlaceboAlso: 0.9% saline solutionLoading dose at baseline (Week 0) in Placebo arm will be corresponding volume of an authorised 0.9% saline solution . The maintenance doses of the 0.9% saline solution to be infused 7 times will be given every 21 (+/-4) days. | Drug | Dosage Arm 4 |
| NewGam 10%Loading dose at baseline (Week 0) will be 2.0 g/kg NewGam in all three NewGam treatment arms. The maintenance doses to be infused 7 times will be 1.0 g/kg every 21 (+/-4) days. | Drug | Dosage Arm 2 |
| NewGam 10%Loading dose at baseline (Week 0) will be 2.0 g/kg NewGam in all three NewGam treatment arms. The maintenance doses to be infused 7 times will be 0.4 g/kg every 21 (+/-4) days. | Drug | Dosage Arm 3 |
Primary outcome
Adjusted INCAT disability score
Time frame: Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)
4 secondary outcomes
Vital Signs
Time frame: During each infusion - Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)
Grip Strength
Time frame: Visit 9 & 13
Nerve Conduction Studies
Time frame: Visti 9 & 13
Motor Impairment Assessment utlizing the Expanded MRC Sum Score
Time frame: Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)
Results and safety
The study was stopped early (reason given: Study Terminated.priority changes in product development.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Feb 2017.
- Derived from this study: PubMed 38353301Bus SR, de Haan RJ, Vermeulen M, van Schaik IN, Eftimov F. Intravenous immunoglobulin for chronic inflammatory demyelinating polyradiculoneuropathy. Cochrane Database Syst Rev. 2024 Feb 14;2(2):CD001797. doi: 10.1002/14651858.CD001797.pub4.
- Individual participant dataSharing planned: No. no (IPD) data exist for this study
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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.
Wolfgang Frenzel, MD study director
Octapharma
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing NewGam 10% in people with Chronic Inflammatory Demyelinating Polyradiculoneuropathy, run by Octapharma.
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 3 inclusion and 13 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: stopped early, as of the sponsor's last update on 23 Feb 2017. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 stopped early (reason given: Study Terminated.priority changes in product development.). Results are not available in the source record.
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?
Adjusted INCAT disability score (time frame: Every 3 weeks for 48 weeks (stage 1) or 36 weeks (stage 2)). 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 NCT01225276, 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 23 Feb 2017.