Is NCT05163288 recruiting in Rochester, MN?

Yes, NCT05163288 is currently recruiting participants at its Rochester, MN location. This niemann-pick disease, type c clinical trial is actively enrolling qualified participants.

How do I join the niemann-pick disease, type c clinical trial in Rochester?

To join this clinical trial in Rochester, complete the eligibility form on this page. A study coordinator will contact you to discuss next steps and determine if you qualify.

Is participation in this Rochester clinical trial free?

Yes, all study-related care is provided at no cost to qualified participants. Many studies also offer compensation for time and travel expenses.

Niemann-Pick Disease, Type C Clinical Trial in Rochester, MN

About This Niemann-Pick Disease, Type C Study in Rochester

A pivotal, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 4 and older with a confirmed diagnosis of Niemann Pick disease type C (NPC). The objective of this study is to evaluate the safety, tolerability and efficacy of N-acetyl-L-leucine (IB1001) compared to standard of care. Following this randomized, double-blind, placebo-controlled "Parent Study", an extension phase is conducted for (1) patients who completed the "Parent Study" and (2) patients who are enrolled directly into the Extension Phase. Currently, the Extension Phase provides patients with 3 years of open-label treatment.

Sponsor: IntraBio Inc

Who Can Participate

Inclusion Criteria

Written informed consent signed by the patient and/or their legal representative/ parent/ impartial witness

Male or female aged ≥4 years with a confirmed genetic diagnosis of NPC at the time of signing informed consent.

Females of childbearing potential, defined as a premenopausal female capable of becoming pregnant, will be included if they are either sexually inactive (sexually abstinent for 14 days prior to the first dose and confirm to continue through 28 days after the last dose) or using one of the following highly effective contraceptives (i.e. results in \<1% failure rate when used consistently and correctly) 14 days prior to the first dose continuing through 28 days after the last dose:

intrauterine device (IUD);

surgical sterilization of the partner (vasectomy for 6 months minimum);

combined (estrogen or progestogen containing) hormonal contraception associated with the inhibition of ovulation (either oral, intravaginal, or transdermal);

progestogen only hormonal contraception associated with the inhibition of ovulation (either oral, injectable, or implantable);

intrauterine hormone releasing system (IUS);

bilateral tubal occlusion.

Females of non-childbearing potential who have undergone one of the following sterilization procedures at least 6 months prior to the first dose:

hysteroscopic sterilization;

bilateral tubal ligation or bilateral salpingectomy;

hysterectomy;

bilateral oophorectomy; OR be postmenopausal with amenorrhea for at least 1 year prior to the first dose and follicle stimulating hormone (FSH) serum levels consistent with postmenopausal status. FSH analysis for postmenopausal women will be done at screening. FSH levels should be in the postmenopausal range as determined by the central laboratory.

Non-vasectomized male patient agrees to use a condom with spermicide or abstain from sexual intercourse during the study until 90 days beyond the last dose of study medication and the female partner agrees to comply with inclusion criteria 3 or 4. For a vasectomized male who has had his vasectomy 6 months or more prior to study start, it is required that they use a condom during sexual intercourse. A male who has been vasectomized less than 6 months prior to study start must follow the same restrictions as a non-vasectomized male.

If male, patient agrees not to donate sperm from the first dose until 90 days after their last dose.

Patients must fall within: a) A SARA score of 7 ≤ X ≤ 34 points (out of 40) AND b) Either: i. Within the 2-7 range (0-8 range) of the Gait subtest of the SARA scale OR ii. Be able to perform the 9-Hole Peg Test with Dominant Hand (9HPT-D) (SCAFI subtest) in 20 ≤ X ≤150 seconds.

Weight ≥15 kg at screening.

Patients are willing to disclose their existing medications/therapies for (the symptoms) of NPC including those on the prohibited medication list. Non-prohibited medications/therapies (authorized medicines for NPC \[e.g. miglustat\], speech therapy, and physiotherapy) are permitted provided:

The Investigator does not believe the medication/therapy will interfere with the study protocol/results

Patients have been on a stable dose/duration and type of therapy for at least 42 days before Visit 1 (Baseline 1)

Patients are willing to maintain a stable dose/do not change their therapy throughout the duration of the study.

An understanding of the implications of study participation, provided in the written patient information and informed consent by patients or their legal representative/parent, and demonstrates a willingness to comply with instructions and attend required study visits (for children this criterion will also be assessed in parents or appointed guardians).

Exclusion Criteria

Patients who are unable to consistently Patients who have any known hypersensitivity or history of hypersensitivity to:

Acetyl-Leucine (DL-, L-, D-) or derivatives.

Excipients the IB1001 sachet (namely isomalt, hypromellose, and strawberry flavour).

Excipients the placebo sachet (namely isomalt, hypromellose, strawberry flavour, citric acid, microcrystalline cellulose, lactose, denatonium benzoate).

Simultaneous participation in another clinical study or participation in any clinical study involving administration of an investigational medicinal product (IMP; 'study drug') for at least 42 days prior to Visit 1. At the discretion of the investigator, Medical Monitor, and Sponsor, the washout period for specific IMPs may be longer based on the pharmacological activity and pharmacokinetics of the drug.

Patients with a physical, cognitive, or psychiatric condition which, at the investigator's discretion and in consultation with the Medical Monitor and Sponsor (as applicable), may put the patient at risk, may confound the study results, or may interfere with the patient's participation in the clinical study, i.e. reliably perform study assessments.

Known or persistent use, misuse, or dependency of medication, drugs, or alcohol.

Current or planned pregnancy or women who are breastfeeding.

Patients with severe vision or hearing impairment (that is not corrected by glasses or hearing aids) that, at the investigator's discretion, interferes with their ability to perform study assessments.

Patients who have been diagnosed with arthritis or other musculoskeletal disorders affecting joints, muscles, ligaments, and/or nerves that by themselves affects patient's mobility and, at the investigator's discretion, interferes with their ability to perform study assessments.

Patients unwilling and/or not able to undergo a 42 day period from any of the following prohibited medication prior to Visit 1 (Baseline 1) and remain without prohibited medication through Visit 6.

N-Acetyl-DL-Leucine (e.g. Tanganil®);

N-Acetyl-L-Leucine (prohibited if not provided as IMP in the IB1001-301 trial);

Sulfasalazine;

Rosuvastatin. \-

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Rochester?

Yes, this clinical trial (NCT05163288) has an active research site in Rochester, MN that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Niemann-Pick Disease, Type C Treatment Options in Rochester, MN

If you're searching for niemann-pick disease, type c treatment options in Rochester, MN, this clinical trial (NCT05163288) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Rochester research site is actively enrolling participants for this clinical trial. You'll receive care from experienced niemann-pick disease, type c specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all niemann-pick disease, type c clinical trials near you to find additional studies recruiting in your area.

Niemann-Pick Disease, Type C Clinical Trial in Rochester, MN

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