Niemann-Pick Disease, Type C1Phase 1NCT03893071
Open-Label Study of Long-Term Safety and Efficacy of Intravenous Trappsol Cyclo (HPβCD) in Niemann-Pick Disease Type C
A Phase 1 study testing Hydroxypropyl-β-cyclodextrin in people with Niemann-Pick Disease, Type C1, run by Cyclo Therapeutics, Inc. at 1 site in United States.
Trial summary
The purpose of this study is to provide continued access to treatment for NPC-1 after participation and completion of the Phase I trial CTD-TCNPC-101, when administered at doses of 1500 mg/kg and 2500 mg/kg by slow IV infusion over a period of 8 to 9 hours every two weeks. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Niemann-Pick Disease, Type C1
- What is being tested?
- Hydroxypropyl-β-cyclodextrin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteUnited States
- Study dates
- 23 May 2019to Mar 2022
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Completion of study CTD-TCNPC-101 with no safety concerns at the final visit
- Negative urine pregnancy test for females of child bearing potential
- Patients must be legally resident in the USA with access to healthcare
- Written, informed consent
Who cannot join
- Inability to comply with the proposed protocol assessments or any uncertainty about their ability to give meaningful, informed consent (legal guardian may give consent with subject assent)
- Concurrent medical conditions representing a contraindication to any of the study medications
- Grade 3 renal impairment or worse as indicated by estimated Glomerular filtration rate (eGFR) < 60mL/min/1.73m2
- Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or International Normalised Ratio (INR) >1.8
- Male patients and female patients of childbearing potential who are not willing to use appropriate birth control (i.e. double barrier birth control) from enrollment until the follow-up visit
Trial locations and contacts
1 site in 1 country.
United States1 site
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
About this study
Official title: An Open-Label Extension Study of the Long-Term Safety and Efficacy of Intravenous Trappsol® Cyclo (HP-β-CD) in Patients With Niemann-Pick Disease Type C (NPC-1)
The purpose of this study is to provide continued access to treatment for NPC-1 after participation and completion of the Phase I trial CTD-TCNPC-101, when administered at doses of 1500 mg/kg and 2500 mg/kg by slow IV infusion over a period of 8 to 9 hours every two weeks.
Read the detailed description
This is an open-label extension study of intravenous HP-β-CD (administered as Trappsol® Cyclo(TM)) in patients with NPC-1 to evaluate long-term safety and efficacy by providing continued access to treatment following completion of study CTD-TCNPC-101. Patients will receive medication at their home or local site under the supervision of a home nurse professional or at the parent site under the supervision of the site principal investigator. Patients will visit the parent site for safety and efficacy evaluations after receiving treatment for 3 months in the extension protocol.
Thereafter, visits to the parent site will be every 6 months until cessation. The study will continue until Trappsol® Cyclo (TM) becomes commercially available or study/site/patient discontinuation. All patients who complete study CTD-TCNCP-101 and pass the screening criteria will be eligible for this study. The study will be directed from parent site in the US. Vital signs, AEs Adverse Events (AEs) and concomitant meds will be recorded by the home nurse professional or deputy at the local site and reported immediately to the parent site for entry into the Electronic Data Capture (EDC) and reporting to the sponsor.
For logistical reasons home infusions will be permitted as long as AEs, infusion details and concomitants medications are reported directly to the parent site by an authorised deputy according to local guidelines. A Safety Review Committee (SRC) consisting of an independent advisor, the medical monitor, and the principal investigator will be established to review AEs and laboratory data throughout the study.
- Conditions
- Niemann-Pick Disease, Type C1
- Being tested
- Hydroxypropyl-β-cyclodextrin
- Medical subject headings
- Niemann-Pick Disease, Type C
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Hydroxypropyl-β-cyclodextrinDrug
Also called Hydroxypropyl-β-cyclodextrin (HP-β-CD), Trappsol® Cyclo
HP-β-CD will be administered as Trappsol® Cyclo 25% (250mg/mL) by slow intravenous infusion over a period of 8 up to 9 hours.
| Study group | Role | What they receive |
|---|---|---|
| Hydroxypropyl-β-cyclodextrin IV | Experimental | Hydroxypropyl-β-cyclodextrin will be administered as Trappsol® Cyclo 25% (250mg/mL) by slow intravenous infusion over a period of 8 up to 9 hours. Receives: Drug: Hydroxypropyl-β-cyclodextrin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product
Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product
Measured: 1-104 weeks
Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product
Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product
Measured: 1-104 weeks
Number of discontinuations due to AEs: Patient Discontinuation, Study Treatment Discontinuation, Study and Site Discontinuation
Discontinuation, Study and Site Discontinuation
Measured: 1-104 weeks
Auditory capacity will be measured by behavioral auditory assessment
Auditory capacity will be measured by behavioral auditory assessment
Measured: 1-104 weeks
Secondary outcomes (1)
Change from baseline in total score as well as individual National Institute of Health (NIH) Niemann-Pick Disease Type C (NPC) Severity Scale (NCSS)
Change from baseline in total score as well as individual National Institute of Health (NIH) Niemann-Pick Disease Type C (NPC) Severity Scale (NCSS)
Measured: 1-104 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Mar 2019
- Study start23 May 2019
- Primary completionMar 2022 (estimated)
- Study completionMar 2022 (estimated)
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 Niemann-Pick Disease, Type C1Studies 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
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Cyclo Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Caroline Hastings, MD, Principal investigator, UCSF Benioff Children's Hospital Oakland
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03893071
- Sponsor's protocol ID
- CTD-TCNPC-102
- Status last verified
- Feb 2021
- First submitted
- 20 Feb 2019
- Last updated
- 18 Jun 2021
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Hastings C, Liu B, Hurst B, Cox GF, Hrynkow S. Intravenous 2-hydroxypropyl-beta-cyclodextrin (Trappsol(R) Cyclo) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial. Mol Genet Metab. 2022 Dec;137(4):309-319. doi: 10.1016/j.ymgme.2022.10.004. Epub 2022 Oct 17.
PubMed 36279795
Study at a glance
From the registry record, last updated by the sponsor on 18 Jun 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension Study of the Long-Term Safety and Efficacy of Intravenous Trappsol® Cyclo (HP-β-CD) in Patients With Niemann-Pick Disease Type C (NPC-1)
- ClinicalTrials.gov ID
- NCT03893071
- Acronym / sponsor ID
- CTD-TCNPC-102
- Conditions
- Niemann-Pick Disease, Type C1
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- Cyclo Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 23 May 2019 (actual)
- Primary completion
- Mar 2022 (estimated)
- Study completion
- Mar 2022 (estimated)
- First posted
- 28 Mar 2019 (actual)
- 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 study is to provide continued access to treatment for NPC-1 after participation and completion of the Phase I trial CTD-TCNPC-101, when administered at doses of 1500 mg/kg and 2500 mg/kg by slow IV infusion over a period of 8 to 9 hours every two weeks.
Detailed description
This is an open-label extension study of intravenous HP-β-CD (administered as Trappsol® Cyclo(TM)) in patients with NPC-1 to evaluate long-term safety and efficacy by providing continued access to treatment following completion of study CTD-TCNPC-101. Patients will receive medication at their home or local site under the supervision of a home nurse professional or at the parent site under the supervision of the site principal investigator. Patients will visit the parent site for safety and efficacy evaluations after receiving treatment for 3 months in the extension protocol.
Thereafter, visits to the parent site will be every 6 months until cessation. The study will continue until Trappsol® Cyclo (TM) becomes commercially available or study/site/patient discontinuation. All patients who complete study CTD-TCNCP-101 and pass the screening criteria will be eligible for this study. The study will be directed from parent site in the US. Vital signs, AEs Adverse Events (AEs) and concomitant meds will be recorded by the home nurse professional or deputy at the local site and reported immediately to the parent site for entry into the Electronic Data Capture (EDC) and reporting to the sponsor.
For logistical reasons home infusions will be permitted as long as AEs, infusion details and concomitants medications are reported directly to the parent site by an authorised deputy according to local guidelines. A Safety Review Committee (SRC) consisting of an independent advisor, the medical monitor, and the principal investigator will be established to review AEs and laboratory data throughout the study.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hydroxypropyl-β-cyclodextrin IV | Experimental | Drug: Hydroxypropyl-β-cyclodextrinHydroxypropyl-β-cyclodextrin will be administered as Trappsol® Cyclo 25% (250mg/mL) by slow intravenous infusion over a period of 8 up to 9 hours. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Completion of study CTD-TCNPC-101 with no safety concerns at the final visit
- Negative urine pregnancy test for females of child bearing potential
- Patients must be legally resident in the USA with access to healthcare
- Written, informed consent
Key exclusion criteria (5 listed)
- Inability to comply with the proposed protocol assessments or any uncertainty about their ability to give meaningful, informed consent (legal guardian may give consent with subject assent)
- Concurrent medical conditions representing a contraindication to any of the study medications
- Grade 3 renal impairment or worse as indicated by estimated Glomerular filtration rate (eGFR) < 60mL/min/1.73m2
- Clinical evidence of acute liver disease including symptoms of jaundice or right upper quadrant pain or International Normalised Ratio (INR) >1.8
- Male patients and female patients of childbearing potential who are not willing to use appropriate birth control (i.e. double barrier birth control) from enrollment until the follow-up 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 |
|---|---|---|
| Hydroxypropyl-β-cyclodextrinAlso: Hydroxypropyl-β-cyclodextrin (HP-β-CD), Trappsol® CycloHP-β-CD will be administered as Trappsol® Cyclo 25% (250mg/mL) by slow intravenous infusion over a period of 8 up to 9 hours. | Drug | Hydroxypropyl-β-cyclodextrin IV |
Primary outcome
Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product
Time frame: 1-104 weeks
Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product
Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product
Time frame: 1-104 weeks
Serious adverse events (SAEs): Frequency, severity, time to onset, duration and relatedness to study product
Number of discontinuations due to AEs: Patient Discontinuation, Study Treatment Discontinuation, Study and Site Discontinuation
Time frame: 1-104 weeks
Discontinuation, Study and Site Discontinuation
Auditory capacity will be measured by behavioral auditory assessment
Time frame: 1-104 weeks
Auditory capacity will be measured by behavioral auditory assessment
1 secondary outcome
Change from baseline in total score as well as individual National Institute of Health (NIH) Niemann-Pick Disease Type C (NPC) Severity Scale (NCSS)
Time frame: 1-104 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Jun 2021.
- Derived from this study: PubMed 36279795Hastings C, Liu B, Hurst B, Cox GF, Hrynkow S. Intravenous 2-hydroxypropyl-beta-cyclodextrin (Trappsol(R) Cyclo) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial. Mol Genet Metab. 2022 Dec;137(4):309-319. doi: 10.1016/j.ymgme.2022.10.004. Epub 2022 Oct 17.
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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.
Caroline Hastings, MD principal investigator
UCSF Benioff Children's Hospital Oakland
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Hydroxypropyl-β-cyclodextrin in people with Niemann-Pick Disease, Type C1, run by Cyclo Therapeutics, Inc. 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 4 inclusion and 5 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: status not confirmed, as of the sponsor's last update on 18 Jun 2021. 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?
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?
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?
Adverse events (AEs): Frequency, severity, time to onset, duration, and relatedness to study product (time frame: 1-104 weeks). The study lists 4 primary outcomes in all. 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 NCT03893071, 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 18 Jun 2021.