Authorised Therapeutic confirmatory (Phase III) Niemann Pick Disease Type C1

Study to Evaluate the Safety, Tolerability, and Efficacy of 2000 mg/kg of Trappsol® Cyclo™ and Standard of Care Compared to Placebo and Standard of Care in Patients with Niemann Pick Disease Type C1

EU CTIS ID: 2024-518266-27-00

What this study is testing

This study has dual primary objectives, such that the primary objective is to evaluate the effectiveness of Trappsol Cyclo and standard of care (SOC) compared to placebo and SOC as measured by an improvement in the 4-domain (4D-NPC-SS) or 5-domain (5D-NPC-SS) Niemann-Pick disease Type C Severity Scale: The 4D-NPC-SS will be utilized as the primary objective for the US. The 5D-NPC-SS will be utilized as an exploratory objective for the US. The 5D-NPC-SS will be utilized as the primary objective for the EU and RoW. The 4D-NPC-SS will be utilized as an exploratory objective for the EU and RoW All randomized patients, regardless of country, will be assessed by both the 4D-NPC-SS and the 5D-NPC-SS assessment tools per the Schedule of Assessments (SoA).

  • Therapeutic confirmatory (Phase III)

A plain-language read of the study's public EU CTIS listing. The study team confirms the details.

Who can take part

You may be able to join if

  • Patients ≥3 years of age at Screening.
  • Contraception requirements: a. All sexually active WOCBP (post menarche) must use highly effective contraception during the study and until 3 months after the last dose of study treatment b. Highly effective birth control methods include combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal); progestogen only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable); intrauterine device; intrauterine hormone-releasing system; bilateral tubal occlusion; or vasectomized partner c. All sexually active male patients with WOCBP partners (post menarche) must use a condom with or without spermicide in addition to the birth control used by their partners during the study and until 3 months after the last dose of study treatment d. Sexual abstinence is considered a highly effective birth control method only if it is defined as refraining from heterosexual intercourse during the study and for 3 months after the last dose of study treatment for WOCBP and for male patients with WOCBP partners. The reliability of sexual abstinence needs to be evaluated by the Investigator in relation to the duration of the clinical study and the preferred and usual lifestyle of the patient
  • The patient or legally authorized representative has read and signed the informed consent (or assent, as applicable) form prior to any study-related procedures
  • The legally authorized representative (if applicable) agrees for the patient to participate in all aspects of the study
  • The patient’s caregiver (as applicable) agrees to participate in all of the protocol-specified assessment scales, questionnaires, and interviews for the duration of the trial
  • Diagnosis of NPC1 confirmed by: a. Genetically confirmed (deoxyribonucleic acid sequence analysis) by mutations in both alleles of NPC1 OR b. Mutation in only 1 allele of NPC1 and either positive filipin staining in skin or vertical supranuclear gaze palsy (VSGP).

You likely can't join if

  • Recipient of a liver transplant within <12 months or planned liver transplantation. Patients who have received a successful transplant over 12 months or longer ago can be screened.
  • Current participation in another study is not permitted unless it is a noninterventional study and the sole purpose of the trial is for long term follow up describing clinical features or survival data (registry)
  • Patients with uncontrolled, severe epileptic seizure periods (at least 3 consecutive severe epileptic seizures that required medication) within 2 months prior to completion of informed consent (or assent, as applicable). This includes patients with ongoing seizures that are not stable in frequency or type or duration over a 2-month period prior to enrollment, requiring change in dose of antiepileptic medication (other than adjustment for weight) over a 2-month period prior to enrollment, or requiring 3 or more antiepileptic medications to control seizures over a 2-month period prior to enrollment
  • Neurologically asymptomatic patients
  • Inability to participate in the primary study assessment (4D-NPC-SS and 5D-NPC-SS) as determined by the Investigator.
  • Patients with active liver disease from any cause other than NPC1 or prolonged icterus or malformation of organs other than NPC1
See the full eligibility criteria
Who can join
  • Patients ≥3 years of age at Screening.
  • Contraception requirements: a. All sexually active WOCBP (post menarche) must use highly effective contraception during the study and until 3 months after the last dose of study treatment b. Highly effective birth control methods include combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, or transdermal); progestogen only hormonal contraception associated with inhibition of ovulation (oral, injectable, or implantable); intrauterine device; intrauterine hormone-releasing system; bilateral tubal occlusion; or vasectomized partner c. All sexually active male patients with WOCBP partners (post menarche) must use a condom with or without spermicide in addition to the birth control used by their partners during the study and until 3 months after the last dose of study treatment d. Sexual abstinence is considered a highly effective birth control method only if it is defined as refraining from heterosexual intercourse during the study and for 3 months after the last dose of study treatment for WOCBP and for male patients with WOCBP partners. The reliability of sexual abstinence needs to be evaluated by the Investigator in relation to the duration of the clinical study and the preferred and usual lifestyle of the patient
  • The patient or legally authorized representative has read and signed the informed consent (or assent, as applicable) form prior to any study-related procedures
  • The legally authorized representative (if applicable) agrees for the patient to participate in all aspects of the study
  • The patient’s caregiver (as applicable) agrees to participate in all of the protocol-specified assessment scales, questionnaires, and interviews for the duration of the trial
  • Diagnosis of NPC1 confirmed by: a. Genetically confirmed (deoxyribonucleic acid sequence analysis) by mutations in both alleles of NPC1 OR b. Mutation in only 1 allele of NPC1 and either positive filipin staining in skin or vertical supranuclear gaze palsy (VSGP).
  • Patients with an ASIS between 0.5 to 2.0 (inclusive) at Screening using the 17 Domain Niemann-Pick Type C Severity Scale (17D-NPC-SS) composite score. For patients who remain incontinent due to inability to train to become continent, the relative contribution to the 17-D-NPC-CSS composite score can be adjusted per the Investigator's judgment as not applicable, following conferring with the Medical Monitor. A not applicable score will be scored as a "0" for this domain.
  • Treated or not treated with miglustat. a. If a patient is receiving treatment with miglustat, the dose must have been stable for at least 3 continuous months prior to the first Screening Visit. b. If a patient has been discontinued from prescribed treatment with miglustat, she/he must have been discontinued for at least 3 continuous months prior to the first Screening Visit.
  • Body weight >4.5 kg to ≤125 kg
  • Presenting at least 1 neurological symptom of the disease (including, but not limited to, hearing loss, VSGP, ataxia, dementia, dystonia, history of seizures, cataplexy, dysarthria, or dysphagia)
  • Willing and capable to participate in all aspects of study design, including blood sampling (efficacy, PK, blood biomarkers, and safety laboratory tests). Adequate compliance with the assessments to obtain complete data can become a discussion between the Investigator and the medical monitor prior to randomization at the Baseline Visit the Medical Monitor prior to randomization at the Baseline Visit.
  • Patients who have previously been treated with hydroxypropyl-β- cyclodextrin (HPβCD) are eligible for participation in the study if their last intrathecal administration was 3 months or longer ago or if their last IV administration was 6 months or longer ago. No more than approximately 10% of the total number of randomized patients can previously have been exposed to HPβCD.
  • Ability to travel to the corresponding clinical study site at the scheduled visit times for evaluation and follow-up.
What rules you out
  • Recipient of a liver transplant within <12 months or planned liver transplantation. Patients who have received a successful transplant over 12 months or longer ago can be screened.
  • Current participation in another study is not permitted unless it is a noninterventional study and the sole purpose of the trial is for long term follow up describing clinical features or survival data (registry)
  • Patients with uncontrolled, severe epileptic seizure periods (at least 3 consecutive severe epileptic seizures that required medication) within 2 months prior to completion of informed consent (or assent, as applicable). This includes patients with ongoing seizures that are not stable in frequency or type or duration over a 2-month period prior to enrollment, requiring change in dose of antiepileptic medication (other than adjustment for weight) over a 2-month period prior to enrollment, or requiring 3 or more antiepileptic medications to control seizures over a 2-month period prior to enrollment
  • Neurologically asymptomatic patients
  • Inability to participate in the primary study assessment (4D-NPC-SS and 5D-NPC-SS) as determined by the Investigator.
  • Patients with active liver disease from any cause other than NPC1 or prolonged icterus or malformation of organs other than NPC1
  • Clinical evidence of acute liver disease including associated symptoms of jaundice or right upper quadrant pain or international normalized ratio >1.8
  • Stage 3 chronic kidney disease or worse as indicated by an estimated glomerular filtration rate (eGFR) <60 mL/min/1.73 m2. In patients aged ≤18 years, eGFR is calculated according to the Schwartz equation (Schwartz and Work, 2009), and in patients aged >18 years eGFR is calculated using the Modification of Diet in Renal Disease equation
  • Use of curcumin or fish oil supplements within 12 weeks prior to enrollment
  • Known or suspected allergy or intolerance to the study treatment
  • Treatment with any investigational drug during the 3 months prior to entering the study. If the investigational drug has a short half-life (<8 hours) and would be expected to be cleared from the body within 1 month, then the wash-out period is 1 month. Treatment with any form of leucine, whether as an investigational drug or other formulation is not allowed. Please consult with the Medical Monitor on a case-by-case basis.
  • Treatment with any other investigational drug during the study
  • Pregnancy or breastfeeding

The study team makes the final eligibility decision.

Where it's taking place

  • Australia
  • Israel
  • Brazil
  • Turkey
  • Argentina
  • Taiwan
  • Saudi Arabia
  • United States
  • United Kingdom

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 65+ years, 18-64 years, 0-17 years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Australia; Israel; Brazil; Turkey; Argentina; Taiwan and 3 more location(s). Enter your location above to see the nearest site and check your eligibility.

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BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.