Phase 3 Study of Intranasal Carbetocin (LV-101) in Patients With Prader-Willi Syndrome (CARE-PWS)
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| ClinicalTrials.gov Identifier: NCT03649477 |
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Recruitment Status :
Completed
First Posted : August 28, 2018
Results First Posted : November 17, 2021
Last Update Posted : July 26, 2022
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Prader-Willi Syndrome | Drug: 3.2 mg intranasal carbetocin Drug: 9.6 mg intranasal carbetocin Drug: placebo | Phase 3 |
This is a Phase 3 randomized, double-blind study with an 8-week, placebo-controlled period designed to test the effectiveness, safety, and tolerability of LV-101 in participants with PWS.
Effectiveness will be measured using both caregiver-reported and clinician-reported measures of hyperphagia (extreme hunger), obsessive and compulsive behaviors, and anxiety. Safety and tolerability will be measured by adverse events, laboratory tests, and physical exams.
After the 8-week placebo-controlled period, there will be a long-term follow-up period of 56 weeks and an optional extension period after study week 64 during which all participants will receive active treatment with LV-101. At Week 8, participants who were randomized to placebo in the placebo-controlled period will be randomized to one of the two LV-101 doses, administered three times per day before meals.
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 130 participants |
| Allocation: | Randomized |
| Intervention Model: | Parallel Assignment |
| Intervention Model Description: | Three parallel groups (two different doses of carbetocin and placebo) for the first 8 weeks; two parallel groups (two different doses of carbetocin) during 56 weeks of follow-up and the optional extension period |
| Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | Phase 3, Randomized, Double-Blind, Placebo-Controlled, 8-week Clinical Study to Assess the Efficacy, Safety, and Tolerability, of Intranasal Carbetocin (LV-101) in Prader-Willi Syndrome (PWS) With Long Term Follow-Up (CARE-PWS) |
| Actual Study Start Date : | November 20, 2018 |
| Actual Primary Completion Date : | May 13, 2020 |
| Actual Study Completion Date : | July 9, 2022 |
| Arm | Intervention/treatment |
|---|---|
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Placebo Comparator: Placebo
matched placebo during first 8-weeks; prospectively randomized 1:1 to either one of the two doses of carbetocin during 56-week follow-up and optional extension periods
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Drug: placebo
three times per day with meals |
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Experimental: 3.2 mg of LV-101
3.2 mg of LV-101 during first 8-weeks; remain on same dose during 56-week follow-up and optional extension periods
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Drug: 3.2 mg intranasal carbetocin
three times per day with meals
Other Name: LV-101 |
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Experimental: 9.6 mg of LV-101
9.6 mg of LV-101 during first 8-weeks; remain on same dose during 56-week follow-up and optional extension periods
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Drug: 9.6 mg intranasal carbetocin
three times per day with meals
Other Name: LV-101 |
- Hyperphagia Behavior [ Time Frame: Baseline to Week 8 ]
Change in hyperphagia (extreme hunger) as measured by the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) Total Score versus placebo.
Score range: 0-36; higher scores mean a worse outcome. Reduction in score indicates improvement.
- Obsessive and Compulsive Behaviors [ Time Frame: baseline to Week 8 ]
Change in obsessive and compulsive behaviors as measured by the Children's Yale-Brown Obsessive-Compulsive Scale (CY-BOCS) Total Score versus placebo.
Score range: 0-40; higher scores mean a worse outcome. Reduction in score indicates improvement.
- Anxiety [ Time Frame: Baseline to Week 8 ]
Change in participant anxiety as measured by the PWS Anxiety and Distress Questionnaire (PADQ) Total Score versus placebo.
Score range: 0-56; higher scores mean a worse outcome. Reduction in score indicates improvement.
- Global Impression [ Time Frame: Week 8 ]Clinical Global Impression of Change (CGI-C) score versus placebo. Score range: 1-7; higher scores mean a worse outcome. Reduction in score indicates improvement.
- Hyperphagia Behavior (Subset) [ Time Frame: Baseline to Week 8 ]
Change in hyperphagia as measured by the change in specified subsets of HQ-CT questions versus placebo.
Score range: 0-24; higher scores mean a worse outcome. Reduction in score indicates improvement.
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| Ages Eligible for Study: | 7 Years to 18 Years (Child, Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Genetically-confirmed Prader-Willi syndrome
- Provide voluntary, written informed consent (parent(s) / legal guardian(s) of participant); provide voluntary, written assent (participants, as appropriate)
- PWS Nutritional Phase 3 (hyperphagic, rarely feels full)
Exclusion Criteria:
- Living in a group home
- Genetically diagnosed Schaaf-Yang syndrome or other genetic, hormonal, or chromosomal cognitive impairment
- New food-related interventions, including environment or dietary restrictions, within 1 month of screening
- Dose of any allowed chronic concomitant medications or supplements that have not been stable for ≥3 months prior to the study or is not expected to remain stable while participating in the study; adjustments in growth hormone dose ≤10% are not exclusionary
- Presence of cardiovascular disorders, epilepsy, frequent migraines, or severe asthma
- More than 3 episodes of sinusitis in the 12 months prior to Screening Visit or presence of nasal diseases that may affect deposition of intranasal medication
- Unwilling to abstain from nasal saline, other nasal irrigation, or other intranasal medications for 2 weeks prior to the Baseline visit and during the 8-week, placebo-controlled period of the study
- Use of weight loss medication, oxytocin, carbetocin, or vasopressin in the 6 months prior to screening
- Participation in an interventional research study involving another investigational medication or device in the 6 months prior to screening or during the study
- Based on the judgment of the Investigator, is unsuitable for the study for any reason, including but not limited to unstable medical condition, inability to comply with the protocol, or other risk to subject or to the integrity of the study
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03649477
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Documents provided by Levo Therapeutics, Inc.:
| Responsible Party: | Levo Therapeutics, Inc. |
| ClinicalTrials.gov Identifier: | NCT03649477 |
| Other Study ID Numbers: |
LV-101-3-01 |
| First Posted: | August 28, 2018 Key Record Dates |
| Results First Posted: | November 17, 2021 |
| Last Update Posted: | July 26, 2022 |
| Last Verified: | August 2021 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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PWS Prader-Willi syndrome carbetocin |
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Prader-Willi Syndrome Syndrome Disease Pathologic Processes Intellectual Disability Neurobehavioral Manifestations Neurologic Manifestations Nervous System Diseases Abnormalities, Multiple Congenital Abnormalities Chromosome Disorders |
Genetic Diseases, Inborn Obesity Overweight Overnutrition Nutrition Disorders Carbetocin Oxytocin Oxytocics Reproductive Control Agents Physiological Effects of Drugs |