Neurofibromatosis Type 1Early Phase 1NCT00657202
Ranibizumab for Neurofibromas Associated With Neurofibromatosis 1
A Early Phase 1 study testing Ranibizumab in people with Neurofibromatosis Type 1 and Cutaneous Neurofibromas, run by Massachusetts General Hospital at 1 site in United States.
Trial summary
The purpose of this research study is to determine if ranibizumab can prevent the growth of neurofibromas. We will also be collecting extra blood and serum samples to help us learn more about NF1. Ranibizumab is a drug that affects the development of blood vessels that feed tumors. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Neurofibromatosis Type 1, Cutaneous Neurofibromas
- What is being tested?
- Ranibizumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 11 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2008to Dec 2013
- Phase
- Early 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients must have diagnosis of NF1 based on NIH criteria with two or more of the following characteristics: a) Six or more cafe-au-lait macules b) Skin fold freckling in the axilla or groin c) Optic pathway glioma d) Two or more Lisch nodules of the iris e) Distinctive bony lesions such as dysplasia of the sphenoid wing or of a long bone such as the tibia f) Two or more neurofibromas of any type or 1 or more plexiform neurofibroma g) First degree relative with NF1
- At least four cutaneous neurofibromas on skin exam with the following qualities: a) the lesion must be discrete by clinical exam and must be at least 5mm away from another skin tumor b) the lesion must be amenable to measurement with calipers with minimum dimension of 5mm and maximum dimension of 20mm c)the lesions cannot be located on the face, scalp, or groin and must be located in an area that can be photographed d) histologic confirmation of tumor type is not required in the setting of compatible clinical setting
- Must be willing to have treated CNF surgically removed during the study
- 18 years of age or older
- Normal organ and marrow function as described in the protocol
Who cannot join
- Pregnant or breast-feeding women
- Chemotherapy or radiotherapy within 6 weeks prior to entering the study
- Receiving any other investigational agent
- History of allergic reactions attributed to compounds of similar chemical or biologic composition as ranibizumab
- Hypertension that cannot be controlled by medications
- Known coagulopathy that increases risk of bleeding or a history of clinically significant hemorrhages in the past
Show all 9Show fewer
- NCI CTCAE grade 3 hemorrhage within 4 weeks of starting the study treatment
- Pre-existing thyroid abnormality with thyroid function that cannot be maintained in the normal range with medication
- Uncontrolled intercurrent illness
Trial locations and contacts
1 site in 1 country.
United States1 site
Massachusetts General Hospital
Boston, Massachusetts, 02114
About this study
Official title: Pilot Study of Ranibizumab (Lucentis) for Neurofibromas Associated With Neurofibromatosis 1
The purpose of this research study is to determine if ranibizumab can prevent the growth of neurofibromas. We will also be collecting extra blood and serum samples to help us learn more about NF1. Ranibizumab is a drug that affects the development of blood vessels that feed tumors. It targets a substance in the body called VEGF (Vascular Endothelial Growth Factor). VEGF helps tumors to grow and survive by supporting the growth of blood vessels that bring nutrients to the tumor. VEGF is made by cancerous tumors and also by non-cancerous tumors such as neurofibromas.
Read the detailed description
- Participants will receive one injection of ranibizumab into 3 tumors on their skin (one dose per tumor) on day 1 of treatment. One other tumor will be injected with normal saline solution. The saline solution-called a control-is necessary to determine whether injections (without medicine) can cause a tumor to shrink. Tumors will be measured and photographed prior to treatment. Ranibizumab tumors will be removed on days 8, 15, and 29; the saline treated tumor will be removed on day 29.
- Participants will come into the clinic once a week for a total of 4 weeks and then again on Days 35. 57 and 85 for post-treatment visits. Some of the following tests and procedures will be performed: physical examination (including photographs of tumors), review of current medications, vital signs, routine blood tests, serum chemistry blood tests, interstitial fluid pressure measurements and tumor samples.
- Conditions
- Neurofibromatosis Type 1, Cutaneous Neurofibromas
- Being tested
- Ranibizumab
- Keywords
- NF1, neurofibroma, ranibizumab, Lucentis
- Medical subject headings
- Neurofibromatosis 1, Neurofibroma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RanibizumabDrug
Also called Lucentis
Given as an injection into three cutaneous neurofibromas on day 1 of the study treatment.
| Study group | Role | What they receive |
|---|---|---|
| Ranibizumab | Experimental | Not described Receives: Drug: Ranibizumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
Determine the effect of inhibiting VEGF signaling on tumor volume and tumor interstitial fluid pressure by local injection of ranibizumab into cutaneous neurofibromas.
Measured: 2 years
Secondary outcomes (1)
To identify angiogenic molecules upregulated in neurofibromas treated with ranibizumab.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Apr 2008 (estimated)
- Study startMar 2008
- Primary completionOct 2009
- Study completionDec 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
- More trials run by Massachusetts General HospitalThe 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
- Early Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Massachusetts General Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Scott Randall Plotkin, MD, PhD, Massachusetts General Hospital
- Investigators
- Scott Plotkin, MD, PhD, Principal investigator, Massachusetts General Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00657202
- Sponsor's protocol ID
- 07-332
- Status last verified
- Jul 2016
- First submitted
- 27 Mar 2008
- Last updated
- 7 Jul 2016
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 7 Jul 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Pilot Study of Ranibizumab (Lucentis) for Neurofibromas Associated With Neurofibromatosis 1
- ClinicalTrials.gov ID
- NCT00657202
- Acronym / sponsor ID
- 07-332
- Conditions
- Neurofibromatosis Type 1, Cutaneous Neurofibromas
- Keywords
- NF1, neurofibroma, ranibizumab, Lucentis
- Phase
- Early Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Massachusetts General HospitalUniversity, hospital or other
- Study start
- Mar 2008
- Primary completion
- Oct 2009 (actual)
- Study completion
- Dec 2013 (actual)
- First posted
- 14 Apr 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.
The purpose of this research study is to determine if ranibizumab can prevent the growth of neurofibromas. We will also be collecting extra blood and serum samples to help us learn more about NF1. Ranibizumab is a drug that affects the development of blood vessels that feed tumors. It targets a substance in the body called VEGF (Vascular Endothelial Growth Factor). VEGF helps tumors to grow and survive by supporting the growth of blood vessels that bring nutrients to the tumor. VEGF is made by cancerous tumors and also by non-cancerous tumors such as neurofibromas.
Detailed description
- Participants will receive one injection of ranibizumab into 3 tumors on their skin (one dose per tumor) on day 1 of treatment. One other tumor will be injected with normal saline solution. The saline solution-called a control-is necessary to determine whether injections (without medicine) can cause a tumor to shrink. Tumors will be measured and photographed prior to treatment. Ranibizumab tumors will be removed on days 8, 15, and 29; the saline treated tumor will be removed on day 29.
- Participants will come into the clinic once a week for a total of 4 weeks and then again on Days 35. 57 and 85 for post-treatment visits. Some of the following tests and procedures will be performed: physical examination (including photographs of tumors), review of current medications, vital signs, routine blood tests, serum chemistry blood tests, interstitial fluid pressure measurements and tumor samples.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ranibizumab | Experimental | Drug: Ranibizumab |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Patients must have diagnosis of NF1 based on NIH criteria with two or more of the following characteristics: a) Six or more cafe-au-lait macules b) Skin fold freckling in the axilla or groin c) Optic pathway glioma d) Two or more Lisch nodules of the iris e) Distinctive bony lesions such as dysplasia of the sphenoid wing or of a long bone such as the tibia f) Two or more neurofibromas of any type or 1 or more plexiform neurofibroma g) First degree relative with NF1
- At least four cutaneous neurofibromas on skin exam with the following qualities: a) the lesion must be discrete by clinical exam and must be at least 5mm away from another skin tumor b) the lesion must be amenable to measurement with calipers with minimum dimension of 5mm and maximum dimension of 20mm c)the lesions cannot be located on the face, scalp, or groin and must be located in an area that can be photographed d) histologic confirmation of tumor type is not required in the setting of compatible clinical setting
- Must be willing to have treated CNF surgically removed during the study
- 18 years of age or older
- Normal organ and marrow function as described in the protocol
Key exclusion criteria (9 listed)
- Pregnant or breast-feeding women
- Chemotherapy or radiotherapy within 6 weeks prior to entering the study
- Receiving any other investigational agent
- History of allergic reactions attributed to compounds of similar chemical or biologic composition as ranibizumab
- Hypertension that cannot be controlled by medications
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 |
|---|---|---|
| RanibizumabAlso: LucentisGiven as an injection into three cutaneous neurofibromas on day 1 of the study treatment. | Drug | Ranibizumab |
Primary outcome
Determine the effect of inhibiting VEGF signaling on tumor volume and tumor interstitial fluid pressure by local injection of ranibizumab into cutaneous neurofibromas.
Time frame: 2 years
1 secondary outcome
To identify angiogenic molecules upregulated in neurofibromas treated with ranibizumab.
Time frame: 2 years
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 7 Jul 2016.
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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.
Scott Plotkin, MD, PhD principal investigator
Massachusetts General Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Early Phase 1 study testing Ranibizumab in people with Neurofibromatosis Type 1 and Cutaneous Neurofibromas, run by Massachusetts General Hospital at 1 site in United States.
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 5 inclusion and 9 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 7 Jul 2016. 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 United States. 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?
Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.
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?
Determine the effect of inhibiting VEGF signaling on tumor volume and tumor interstitial fluid pressure by local injection of ranibizumab into cutaneous neurofibromas. (time frame: 2 years). 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 7 Jul 2016.