Prader-Willi SyndromePhase 3bestPWSNCT02179151
Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Beloranib) in Obese Subjects With Prader-Willi Syndrome
A Phase 3 study testing ZGN-440 for Injectable Suspension in people with Prader-Willi Syndrome and Obesity, run by Zafgen, Inc. at 15 sites in United States.
Trial summary
The purpose of this study is to evaluate efficacy and safety of ZGN-440 (beloranib) in obese adolescent and adult subjects with Prader-Willi Syndrome. Read more about this study
- Who is it for?
- Ages 12 to 65 years
- Conditions the study is for
- Prader-Willi Syndrome, Obesity
- What is being tested?
- ZGN-440 for Injectable Suspension
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 108 participants
- Where
- 15 sitesUnited States
- Study dates
- Sep 2014to Oct 2016
- Phase
- Phase 3
- 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 12 to 65 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Confirmed genetic diagnosis of Prader-Willi Syndrome
- Age 12-65
- Obesity
- Age 12-17: BMI ≥ 95th percentile for age and gender
- Age 18-65: BMI ≥27 to ≤60 kg/m2
Who cannot join
- Subjects living in a group home ≥ 50% of the time
- Recent use (within 3 months) of weight loss agents including herbal medication
- Poorly controlled severe psychiatric disorders
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
United States15 sites
Children's Hospital of Colorado
Aurora, Colorado, 80045
National Institute of Child Health
Bethesda, Maryland, 20892
Children's Hospital Boston
Boston, Massachusetts, 02115
University Hospitals Case Medical Center
Cleveland, Ohio, 44106
University of Florida
Gainesville, Florida, 32610
Texas Children's Hospital
Houston, Texas, 77030
Kansas University Medical Center
Kansas City, Kansas, 66160
Winthrop University
Mineola, New York, 11501
Vanderbilt University
Nashville, Tennessee, 37235
University of California, Davis
Sacramento, California, 95817
Children's Hospital and Clinics of Minnesota
Saint Paul, Minnesota, 55102
University of Utah
Salt Lake City, Utah, 84112
UCSD: Rady Children's Hospital
San Diego, California, 92123
Seattle Children's Research Institute
Seattle, Washington, 98105
Saint Louis University
St Louis, Missouri, 63104
About this study
Official title: Randomized, Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Subcutaneous Beloranib in Suspension) in Obese Subjects With Prader-Willi Syndrome to Evaluate Total Body Weight, Food-related Behavior, and Safety Over 6 Months
The purpose of this study is to evaluate efficacy and safety of ZGN-440 (beloranib) in obese adolescent and adult subjects with Prader-Willi Syndrome.
Read the detailed description
Phase 3, Randomized, Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Subcutaneous Beloranib in Suspension) in Obese Subjects with Prader-Willi Syndrome to Evaluate Total Body Weight, Food-related Behavior, and Safety Over 6 Months
- Conditions
- Prader-Willi Syndrome, Obesity
- Being tested
- ZGN-440 for Injectable Suspension, ZGN-440 Placebo for Injectable Suspension
- Keywords
- Prader-Willi Syndrome, Obesity, Hyperphagia
- Medical subject headings
- Prader-Willi Syndrome, Obesity, Hyperphagia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ZGN-440 for Injectable SuspensionDrug
Also called ZGN-440, Beloranib
Subjects will receive ZGN-440 twice weekly subcutaneous injections for up to 28 weeks.
ZGN-440 Placebo for Injectable SuspensionDrug
Also called Placebo
Subjects will receive placebo twice weekly subcutaneous injections for up to 28 weeks.
| Study group | Role | What they receive |
|---|---|---|
| Placebo | Placebo comparator | Intervention: ZGN-440 Placebo for Injectable Suspension Receives: Drug: ZGN-440 Placebo for Injectable Suspension |
| ZGN-440 Injectable Suspension (1.8 mg) | Experimental | Intervention: ZGN-440 for Injectable Suspension Receives: Drug: ZGN-440 for Injectable Suspension |
| ZGN-440 Injectable Suspension (2.4 mg) | Experimental | Intervention: ZGN-440 for Injectable Suspension Receives: Drug: ZGN-440 for Injectable Suspension |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in total body weight
Measured: Baseline to Week 29
Change in hyperphagia-related behavior as measured by total score of a Hyperphagia Questionnaire
Measured: Baseline to Week 29
Secondary outcomes (5)
Change in LDL cholesterol
Measured: Baseline to Week 29
Change in HDL cholesterol
Measured: Baseline to Week 29
Change in total body mass as measured by DXA
Measured: Baseline to Week 29
Change in total body fat mass as measured by DXA
Measured: Baseline to Week 29
Change in triglyceride
Measured: Baseline to Week 29
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jul 2014 (estimated)
- Study startSep 2014
- Primary completionDec 2015
- Study completionOct 2016
Why it stopped: FDA Clinical Hold
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 Prader-Willi SyndromeStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 108 participants (actual)
Sponsor and investigators
- Lead sponsor
- Zafgen, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Dennis Kim, MD, Study director, Zafgen, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02179151
- Sponsor's protocol ID
- ZAF-311
- Acronym
- bestPWS
- Status last verified
- Jan 2017
- First submitted
- 25 Jun 2014
- Last updated
- 26 Jan 2017
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
McCandless SE, Yanovski JA, Miller J, Fu C, Bird LM, Salehi P, Chan CL, Stafford D, Abuzzahab MJ, Viskochil D, Barlow SE, Angulo M, Myers SE, Whitman BY, Styne D, Roof E, Dykens EM, Scheimann AO, Malloy J, Zhuang D, Taylor K, Hughes TE, Kim DD, Butler MG. Effects of MetAP2 inhibition on hyperphagia and body weight in Prader-Willi syndrome: A randomized, double-blind, placebo-controlled trial. Diabetes Obes Metab. 2017 Dec;19(12):1751-1761. doi: 10.1111/dom.13021. Epub 2017 Jul 13.
PubMed 28556449
Study at a glance
From the registry record, last updated by the sponsor on 26 Jan 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Randomized, Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Subcutaneous Beloranib in Suspension) in Obese Subjects With Prader-Willi Syndrome to Evaluate Total Body Weight, Food-related Behavior, and Safety Over 6 Months
- ClinicalTrials.gov ID
- NCT02179151
- Acronym / sponsor ID
- bestPWS / ZAF-311
- Conditions
- Prader-Willi Syndrome, Obesity
- Keywords
- Prader-Willi Syndrome, Obesity, Hyperphagia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: FDA Clinical Hold
- Enrolment
- 108 participants (actual)
- Lead sponsor
- Zafgen, Inc.Industry (pharma, biotech, devices)
- Study start
- Sep 2014
- Primary completion
- Dec 2015 (actual)
- Study completion
- Oct 2016 (actual)
- First posted
- 1 Jul 2014 (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 study is to evaluate efficacy and safety of ZGN-440 (beloranib) in obese adolescent and adult subjects with Prader-Willi Syndrome.
Detailed description
Phase 3, Randomized, Double-Blind, Placebo Controlled, Phase 3 Trial of ZGN-440 (Subcutaneous Beloranib in Suspension) in Obese Subjects with Prader-Willi Syndrome to Evaluate Total Body Weight, Food-related Behavior, and Safety Over 6 Months
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo | Placebo comparator | Drug: ZGN-440 Placebo for Injectable SuspensionIntervention: ZGN-440 Placebo for Injectable Suspension |
| ZGN-440 Injectable Suspension (1.8 mg) | Experimental | Drug: ZGN-440 for Injectable SuspensionIntervention: ZGN-440 for Injectable Suspension |
| ZGN-440 Injectable Suspension (2.4 mg) | Experimental | Drug: ZGN-440 for Injectable SuspensionIntervention: ZGN-440 for Injectable Suspension |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (5 listed)
- Confirmed genetic diagnosis of Prader-Willi Syndrome
- Age 12-65
- Obesity
- Age 12-17: BMI ≥ 95th percentile for age and gender
- Age 18-65: BMI ≥27 to ≤60 kg/m2
Key exclusion criteria (3 listed)
- Subjects living in a group home ≥ 50% of the time
- Recent use (within 3 months) of weight loss agents including herbal medication
- Poorly controlled severe psychiatric disorders
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 |
|---|---|---|
| ZGN-440 for Injectable SuspensionAlso: ZGN-440, BeloranibSubjects will receive ZGN-440 twice weekly subcutaneous injections for up to 28 weeks. | Drug | ZGN-440 Injectable Suspension (1.8 mg), ZGN-440 Injectable Suspension (2.4 mg) |
| ZGN-440 Placebo for Injectable SuspensionAlso: PlaceboSubjects will receive placebo twice weekly subcutaneous injections for up to 28 weeks. | Drug | Placebo |
Primary outcome
Change in total body weight
Time frame: Baseline to Week 29
Change in hyperphagia-related behavior as measured by total score of a Hyperphagia Questionnaire
Time frame: Baseline to Week 29
5 secondary outcomes
Change in LDL cholesterol
Time frame: Baseline to Week 29
Change in HDL cholesterol
Time frame: Baseline to Week 29
Change in total body mass as measured by DXA
Time frame: Baseline to Week 29
Change in total body fat mass as measured by DXA
Time frame: Baseline to Week 29
Change in triglyceride
Time frame: Baseline to Week 29
Results and safety
The study was stopped early (reason given: FDA Clinical Hold). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Jan 2017.
- Derived from this study: PubMed 28556449McCandless SE, Yanovski JA, Miller J, Fu C, Bird LM, Salehi P, Chan CL, Stafford D, Abuzzahab MJ, Viskochil D, Barlow SE, Angulo M, Myers SE, Whitman BY, Styne D, Roof E, Dykens EM, Scheimann AO, Malloy J, Zhuang D, Taylor K, Hughes TE, Kim DD, Butler MG. Effects of MetAP2 inhibition on hyperphagia and body weight in Prader-Willi syndrome: A randomized, double-blind, placebo-controlled trial. Diabetes Obes Metab. 2017 Dec;19(12):1751-1761. doi: 10.1111/dom.13021. Epub 2017 Jul 13.
- Individual participant dataSharing planned: No
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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.
Dennis Kim, MD study director
Zafgen, Inc.
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing ZGN-440 for Injectable Suspension in people with Prader-Willi Syndrome and Obesity, run by Zafgen, Inc. at 15 sites 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 3 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: stopped early, as of the sponsor's last update on 26 Jan 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites 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. Some participants in this study may receive a placebo. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 was stopped early (reason given: FDA Clinical Hold). 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?
Change in total body weight (time frame: Baseline to Week 29). The study lists 2 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 NCT02179151, 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 26 Jan 2017.