GlioblastomaPhase 1NCT02869243
A Dose Escalation Phase I Study Of Human- Recombinant Bone Morphogenetic Protein 4 Administrated Via CED In GBM Patients
A Phase 1 study testing hrBMP4 in people with Glioblastoma, run by Stemgen at 4 sites in 3 countries.
Trial summary
The purpose of the study is to evaluate the feasibility and safety of intra-tumor and interstitial therapy with hBMP4 in increasing doses in patients with progressive and/or multiple recurrent Glioblastoma multiforme (GBM). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Glioblastoma
- What is being tested?
- hrBMP4
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 4 sitesin 3 countries
- Study dates
- 18 Jul 2017to 30 Jun 2021
- Phase
- Phase 1
- 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 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- To be eligible for inclusion into this study, each patient must fulfil the following inclusion criteria ≤ 28 days prior to the anticipated surgery date:
- Malignant glioma (WHO grade III or IV) who have undergone conventional treatment, including surgery (gross total resection or unintentional partial resection with residual tumour) or biopsy (with residual tumour), and/or radiation therapy, and/or chemotherapy, and/or Temozolomide and have progressive and/or multiple recurrent GBM. Preoperative assessment by clinical presentation and CT/MRI appearance of the lesion will identify suitable candidates.
- Age 18-75 years.
- Karnofsky >70 (see APPENDIX C: EXAMPLE OF PERFORMANCE STATUS: KARNOFSKY SCALE).
- Stable dose of corticosteroids no longer than 4 weeks prior to enrolment.
- Females of childbearing potential must have a negative serum or urine pregnancy test.
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- Females who have undergone surgical sterilization or who have been postmenopausal for at least 2 years are not considered to be of childbearing potential.
- Females of childbearing potential and males who have not undergone surgical sterilization must agree to practice a form of effective contraception prior to entry into the study and for 1 month after the end of infusion.
- Effective contraception:
- If female, is non-lactating, has a negative urine pregnancy test result, and does not plan on becoming pregnant during the study, or not of childbearing potential (hysterectomy or tubal ligation at least 6 months prior to entry to the study or post-menopausal for 1 year); if of childbearing potential (including peri-menopausal women who have had a menstrual period within one year) must practice or be willing to continue to practice acceptable birth control from screening and until 1 month after the study medication has been discontinued.
- Acceptable birth control includes:
- Combined (oestrogen and progestogen containing) hormonal contraception;
- Associated with inhibition of ovulation; oral OR intravaginal OR transdermal;
- Progestogen-only hormonal contraception associated with inhibition of ovulation: oral OR injectable OR implantable;
- Progestogen-only oral hormonal contraception, where inhibition of ovulation is not the primary mode of action;
- Intrauterine device (IUD);
- Intrauterine hormone-releasing system (IUS);
- Bilateral tubal occlusion;
- Vasectomised partner;
- Sexual abstinence;
- Male or female condom with or without spermicide;
- Cap, diaphragm or sponge with spermicide. Complies with The Heads of Medicines Agencies (HMA) Recommendations Related to Contraception and Pregnancy Testing in Clinical Trials (Sept 2014).
- The definition of effective contraception will be based on the judgment of the investigator.
- Ability to understand and the willingness to sign a written informed consent document.
Who cannot join
- To be eligible for inclusion into this study, each patient must violate none of the following exclusion criteria ≤ 28 days prior to the anticipated surgery date:
- Patients who had chemotherapy, radiotherapy or other anti-neoplastic therapy (within 4 weeks or 5x half-life whichever is shorter) prior to study treatment or those who have not recovered to Grade ≤1 or returned to baseline from any acute treatment-related toxicities of the previous therapy except for alopecia and Grade 2 neuropathy.
- Patients who are receiving any other investigational agents.
- Life expectancy <3 months
- Haematological dysfunction defined as:
- White blood cell (WBC) count <3.0 x 109/L;
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- Absolute neutrophil count <1.5 x 109/L;
- Haemoglobin level <10.0 g/dL;
- Platelet count <100 x 109/L.
- Liver dysfunction defined as:
- Aspartate transaminase (AST) >2.5 x the upper limit of normal (ULN) for age and gender;
- Alanine transaminase (ALT) >2.5 x the ULN for age and gender;
- Bilirubin >1.5 x the ULN for age and gender.
- Renal dysfunction defined as:
- Creatinine clearance <60 mL/min/1.73 m2 for patients with creatinine levels above institutional normal for age and gender.
- Serology indicating active infection with Hepatitis B or C, or HIV.
- Significant co-morbidity, including coagulation disorders.
- Pregnancy or unwillingness to practice reliable birth control.
- Presence of another active malignancy less than 2 years previously (exception: non-melanoma skin cancer).
- Multifocal, bilateral
- Midline shift more than 5 mm
Trial locations and contacts
4 sites in 3 countries.
4 sites in 3 countries
Netherlands2 sites
VU University Medical Center
Amsterdam, 1081 HV
Erasmus University Medical Center, Department of Neurosurgery
Rotterdam, 3015 GD
About this study
Official title: A Dose Escalation Phase I Study Of Human- Recombinant Bone Morphogenetic Protein 4 Administrated Via Convection-Enhanced Delivery In Patients With Progressive And/Or Multiple Recurrent Glioblastoma Multiforme
The purpose of the study is to evaluate the feasibility and safety of intra-tumor and interstitial therapy with hBMP4 in increasing doses in patients with progressive and/or multiple recurrent Glioblastoma multiforme (GBM).
Read the detailed description
This multicentre, open-label, dose escalating, Phase I study will enrol approximately 18 patients with progressive and/or multiple recurrent GBM, who after failure of standard therapies will receive GMP Human- recombinant Bone Morphogenetic Protein 4 via intra-tumour and interstitial delivery by CED.
Patients will undergo a resection or biopsy of the tumour, confirmation of viable malignant glioma tumour cells during frozen section performed by the institutional pathologists, and intra-tumor and interstitial placement under neuronavigational guidance of 2 or 3 catheters. No catheters will be placed at time of resection. After resection, catheters will be placed during a separate procedure several days later based upon the patient's condition as determined by both a clinical examination and routine MRI scan.
Patients will receive intra-tumour and interstitial CED of increasing amounts of hrBMP4 solutions (at a starting dose of 0.5 mg) and 1:70 gadolinium-diethylenetriamine pentaacetic acid (Gd-DTPA) in a total of 44-66 ml over up to 4-6 days at the discretion of the investigator.
Gd-DTPA will be co-infused with hrBMP4 to determine the extent of intra-tumour and interstitial drug delivery. Patients will be hospitalized from the time of the original surgery until the end of infusion of hrBMP4.
Patients will be followed for the duration of the study. The primary endpoint of the study is monitoring feasibility and patient safety for the incidence of Dose-Limiting Toxicity (DLT) following intra-tumour and interstitial therapy with hrBMP4 and to determine whether there is a Maximum Tolerated Dose (MTD).
- Conditions
- Glioblastoma
- Being tested
- hrBMP4
- Keywords
- A malignant growing astrocytoma of central nervous system
- Medical subject headings
- Glioblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
hrBMP4Drug
Also called Recombinant Bone Morphogenic Protein-4, Device:Intracranial Catheter
Patients will undergo a resection or biopsy of the tumour, confirmation of viable malignant glioma tumour cells and intratumour/interstitial placement under neuronavigational guidance of 2 or 3 catheters.
Catheters will be placed during a second procedure a few days later based upon the patient's condition.
Patients will receive intra-tumour and interstitial CED of increasing amounts of hrBMP4 solutions (starting dose of 0.5 mg) and 1:70 gadolinium-diethylenetriamine pentaacetic acid (Gd-DTPA) in a total of 44-66ml over up to 4-6 days. Gd-DTPA will be co-infused with BMP4 to determine the extent of intra-tumour and interstitial drug delivery. HrBMP4 will be delivered as a continuous infusion via the intracranial catheters.
| Study group | Role | What they receive |
|---|---|---|
| hrBMP4 | Experimental | Intra-tumour and interstitial convection enhanced delivery (CED) as a continuous infusion via intracranial catheters of hrBMP4 solution and gadolinium Receives: Drug: hrBMP4 |
What is being measured
The primary outcome is the main question the study is designed to answer.
DLTs
Number of patients who experienced Dose-Limiting Toxicities. DLT is collected to determine Maximum-Tolerated Dose (MTD)
Measured: Up to 8 Weeks after each cohort of 3 patients
Secondary outcomes (8)
Tumor response
MRI scans with the following variables: volumetric T1, T2, volumetric Fluid Attenuated Inversion Recovery (FLAIR) and volumetric T1 with contrast. Preoperative assessment by clinical presentation and MRI appearance of the tumour burden will be taken and will be used as the reference point to determine the objective tumour response.
Objective tumour response: Duplicates of all scans are to be made at the time of each scan and to be retained for the option of a central reading at the end of the trial. Tumour responses must be confirmed by repeat evaluations which should be performed no less than 4 weeks after the criteria of response was first met.
Tumor response will be evaluated in accordance to Macdonald criteria
Measured: Within 28 days before resection, intraoperative, up to 24 hours post start infusion, after 4-6 days post start infusion (end of infusion) and 4, 12, 24, 36, 48 and 52 weeks after hospitalization
EORTC QLQ-C30 Summary Score
The EORTC quality of life questionnaire (QLQ-C30) will be used to assess quality of life.
The questionnaires will be available in local languages as per country requirements.
Measured: After 4-6 days post start infusion (end of infusion) and during 4,8,12 and 52 weeks after hospitalization
Maximum observed plasma concentration of BMP4 (Cmax)
Blood samples were obtained and plasma concentrations were determined using a validated high-pressure liquid chromatography method.
Measured: Prior to the infusion, after 4-6 days post start infusion (end of infusion) and 4 weeks after hospitalization
Lowest concentration of BMP4 in the blood (Ctrough)
Blood samples were obtained and plasma concentrations were determined using a validated high-pressure liquid chromatography method.
Measured: Prior to the infusion, after 4-6 days post start infusion (end of infusion) and 4 weeks after hospitalization
Area under the curve (AUC∞)
Blood samples were obtained and plasma concentrations were determined using a validated high-pressure liquid chromatography method.
Measured: Prior to the infusion, after 4-6 days post start infusion (end of infusion) and 4 weeks after hospitalization
Central and peripheral volumes of distribution (Vd)
Blood samples were obtained and plasma concentrations were determined using a validated high-pressure liquid chromatography method.
Measured: Prior to the infusion, after 4-6 days post start infusion (end of infusion) and 4 weeks after hospitalization
Clearance
Blood samples were obtained and plasma concentrations were determined using a validated high-pressure liquid chromatography method.
Measured: Prior to the infusion, after 4-6 days post start infusion (end of infusion) and 4 weeks after hospitalization
Half-life (t1/2)
Blood samples were obtained and plasma concentrations were determined using a validated high-pressure liquid chromatography method.
Measured: Prior to the infusion, after 4-6 days post start infusion (end of infusion) and 4 weeks after hospitalization
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Aug 2016 (estimated)
- Study start18 Jul 2017
- Primary completion16 Oct 2020
- Study completion30 Jun 2021
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 GlioblastomaStudies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Stemgen
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- ORION Clinical Services
- Responsible party
- Sponsor
- Investigators
- Francesco DiMeco, PI, Principal investigator, Istituto Neurologico C. Besta Milan Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02869243
- Sponsor's protocol ID
- hrBMP4-001
- Status last verified
- Feb 2024
- First submitted
- 2 Aug 2016
- Last updated
- 16 Feb 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Bos EM, Binda E, Verploegh ISC, Wembacher E, Hoefnagel D, Balvers RK, Korporaal AL, Conidi A, Warnert EAH, Trivieri N, Visioli A, Zaccarini P, Caiola L, van Wijck R, van der Spek P, Huylebroeck D, Leenstra S, Lamfers MLM, Ram Z, Westphal M, Noske D, Legnani F, DiMeco F, Vescovi AL, Dirven CMF. Local delivery of hrBMP4 as an anticancer therapy in patients with recurrent glioblastoma: a first-in-human phase 1 dose escalation trial. Mol Cancer. 2023 Aug 10;22(1):129. doi: 10.1186/s12943-023-01835-6.
PubMed 37563568
Study at a glance
From the registry record, last updated by the sponsor on 16 Feb 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Dose Escalation Phase I Study Of Human- Recombinant Bone Morphogenetic Protein 4 Administrated Via Convection-Enhanced Delivery In Patients With Progressive And/Or Multiple Recurrent Glioblastoma Multiforme
- ClinicalTrials.gov ID
- NCT02869243
- Acronym / sponsor ID
- hrBMP4-001
- Conditions
- Glioblastoma
- Keywords
- A malignant growing astrocytoma of central nervous system
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- StemgenIndustry (pharma, biotech, devices)
- Collaborators
- ORION Clinical Services
- Study start
- 18 Jul 2017 (actual)
- Primary completion
- 16 Oct 2020 (actual)
- Study completion
- 30 Jun 2021 (actual)
- First posted
- 16 Aug 2016 (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.
The purpose of the study is to evaluate the feasibility and safety of intra-tumor and interstitial therapy with hBMP4 in increasing doses in patients with progressive and/or multiple recurrent Glioblastoma multiforme (GBM).
Detailed description
This multicentre, open-label, dose escalating, Phase I study will enrol approximately 18 patients with progressive and/or multiple recurrent GBM, who after failure of standard therapies will receive GMP Human- recombinant Bone Morphogenetic Protein 4 via intra-tumour and interstitial delivery by CED.
Patients will undergo a resection or biopsy of the tumour, confirmation of viable malignant glioma tumour cells during frozen section performed by the institutional pathologists, and intra-tumor and interstitial placement under neuronavigational guidance of 2 or 3 catheters. No catheters will be placed at time of resection. After resection, catheters will be placed during a separate procedure several days later based upon the patient's condition as determined by both a clinical examination and routine MRI scan.
Patients will receive intra-tumour and interstitial CED of increasing amounts of hrBMP4 solutions (at a starting dose of 0.5 mg) and 1:70 gadolinium-diethylenetriamine pentaacetic acid (Gd-DTPA) in a total of 44-66 ml over up to 4-6 days at the discretion of the investigator.
Gd-DTPA will be co-infused with hrBMP4 to determine the extent of intra-tumour and interstitial drug delivery. Patients will be hospitalized from the time of the original surgery until the end of infusion of hrBMP4.
Patients will be followed for the duration of the study. The primary endpoint of the study is monitoring feasibility and patient safety for the incidence of Dose-Limiting Toxicity (DLT) following intra-tumour and interstitial therapy with hrBMP4 and to determine whether there is a Maximum Tolerated Dose (MTD).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| hrBMP4 | Experimental | Drug: hrBMP4Intra-tumour and interstitial convection enhanced delivery (CED) as a continuous infusion via intracranial catheters of hrBMP4 solution and gadolinium |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (24 listed)
- To be eligible for inclusion into this study, each patient must fulfil the following inclusion criteria ≤ 28 days prior to the anticipated surgery date:
- Malignant glioma (WHO grade III or IV) who have undergone conventional treatment, including surgery (gross total resection or unintentional partial resection with residual tumour) or biopsy (with residual tumour), and/or radiation therapy, and/or chemotherapy, and/or Temozolomide and have progressive and/or multiple recurrent GBM. Preoperative assessment by clinical presentation and CT/MRI appearance of the lesion will identify suitable candidates.
- Age 18-75 years.
- Karnofsky >70 (see APPENDIX C: EXAMPLE OF PERFORMANCE STATUS: KARNOFSKY SCALE).
- Stable dose of corticosteroids no longer than 4 weeks prior to enrolment.
Key exclusion criteria (21 listed)
- To be eligible for inclusion into this study, each patient must violate none of the following exclusion criteria ≤ 28 days prior to the anticipated surgery date:
- Patients who had chemotherapy, radiotherapy or other anti-neoplastic therapy (within 4 weeks or 5x half-life whichever is shorter) prior to study treatment or those who have not recovered to Grade ≤1 or returned to baseline from any acute treatment-related toxicities of the previous therapy except for alopecia and Grade 2 neuropathy.
- Patients who are receiving any other investigational agents.
- Life expectancy <3 months
- Haematological dysfunction defined as:
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 |
|---|---|---|
| hrBMP4Also: Recombinant Bone Morphogenic Protein-4, Device:Intracranial CatheterPatients will undergo a resection or biopsy of the tumour, confirmation of viable malignant glioma tumour cells and intratumour/interstitial placement under neuronavigational guidance of 2 or 3 catheters. Catheters will be placed during a second procedure a few days later based upon the patient's condition. Patients will receive intra-tumour and interstitial CED of increasing amounts of hrBMP4 solutions (starting dose of 0.5 mg) and 1:70 gadolinium-diethylenetriamine pentaacetic acid (Gd-DTPA) in a total of 44-66ml over up to 4-6 days. Gd-DTPA will be co-infused with BMP4 to determine the extent of intra-tumour and interstitial drug delivery. HrBMP4 will be delivered as a continuous infusion via the intracranial catheters. | Drug | hrBMP4 |
Primary outcome
DLTs
Time frame: Up to 8 Weeks after each cohort of 3 patients
Number of patients who experienced Dose-Limiting Toxicities. DLT is collected to determine Maximum-Tolerated Dose (MTD)
8 secondary outcomes
Tumor response
Time frame: Within 28 days before resection, intraoperative, up to 24 hours post start infusion, after 4-6 days post start infusion (end of infusion) and 4, 12, 24, 36, 48 and 52 weeks after hospitalization
EORTC QLQ-C30 Summary Score
Time frame: After 4-6 days post start infusion (end of infusion) and during 4,8,12 and 52 weeks after hospitalization
Maximum observed plasma concentration of BMP4 (Cmax)
Time frame: Prior to the infusion, after 4-6 days post start infusion (end of infusion) and 4 weeks after hospitalization
Lowest concentration of BMP4 in the blood (Ctrough)
Time frame: Prior to the infusion, after 4-6 days post start infusion (end of infusion) and 4 weeks after hospitalization
Area under the curve (AUC∞)
Time frame: Prior to the infusion, after 4-6 days post start infusion (end of infusion) and 4 weeks after hospitalization
Central and peripheral volumes of distribution (Vd)
Time frame: Prior to the infusion, after 4-6 days post start infusion (end of infusion) and 4 weeks after hospitalization
Clearance
Time frame: Prior to the infusion, after 4-6 days post start infusion (end of infusion) and 4 weeks after hospitalization
Half-life (t1/2)
Time frame: Prior to the infusion, after 4-6 days post start infusion (end of infusion) and 4 weeks after hospitalization
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 16 Feb 2024.
- Derived from this study: PubMed 37563568Bos EM, Binda E, Verploegh ISC, Wembacher E, Hoefnagel D, Balvers RK, Korporaal AL, Conidi A, Warnert EAH, Trivieri N, Visioli A, Zaccarini P, Caiola L, van Wijck R, van der Spek P, Huylebroeck D, Leenstra S, Lamfers MLM, Ram Z, Westphal M, Noske D, Legnani F, DiMeco F, Vescovi AL, Dirven CMF. Local delivery of hrBMP4 as an anticancer therapy in patients with recurrent glioblastoma: a first-in-human phase 1 dose escalation trial. Mol Cancer. 2023 Aug 10;22(1):129. doi: 10.1186/s12943-023-01835-6.
- 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.
Francesco DiMeco, PI principal investigator
Istituto Neurologico C. Besta Milan Italy
4 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing hrBMP4 in people with Glioblastoma, run by Stemgen at 4 sites in 3 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 24 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: finished, as of the sponsor's last update on 16 Feb 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Netherlands, Israel, Italy. 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 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.
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?
DLTs (time frame: Up to 8 Weeks after each cohort of 3 patients). 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 NCT02869243, 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 16 Feb 2024.