Research Trials

5-Amino-1-methylquinolinium (5-amino-1MQ, 5A1MQ, 5-AMQ) is a small-molecule, membrane-permeable inhibitor of nicotinamide N-methyltransferase (NNMT) developed at the University of Texas Medical Branch (Watowich, Neelakantan and colleagues). All published efficacy and safety data come from cell culture and rodent models: diet-induced obese mice (Neelakantan et al., 2018; Sampson et al., 2021; Babula et al., 2024), aged mice with muscle injury or sarcopenia (Neelakantan et al., 2019; Dimet-Wiley et al., 2024), rat pharmacokinetics (Awosemo et al., 2021), and mouse tumour models. No human clinical trial of 5-amino-1MQ has been published or registered, and reviews of NNMT inhibition note that no clinical trials targeting NNMT have been reported. Adverse-effect information below is therefore limited to animal and in-vitro observations.

AOD9604 (Tyr-hGH177-191) is a synthetic 16-amino-acid fragment of the C-terminus of human growth hormone developed by Metabolic Pharmaceuticals (Australia) as an oral or intravenous anti-obesity candidate. Between 2001 and 2006 six randomised, double-blind, placebo-controlled trials (about 893 participants) were run; safety data were summarised in a sponsor-authored 2013 review (Stier et al.), but the efficacy results were never published in a peer-reviewed journal. The largest Phase 2b trial (502 randomised, 24 weeks) did not show a statistically significant difference in weight loss versus placebo and the sponsor terminated obesity development in February 2007; an FDA 2024 compounding advisory review concluded there is a lack of evidence of effectiveness. The published human data concern oral and intravenous routes only; no human data for subcutaneous administration were identified by FDA. A 'GRAS' designation cited by the sponsor was a self-affirmed expert-panel conclusion; no AOD9604 entry was located in the FDA GRAS Notice Inventory.

Cagrilintide is a long-acting amylin analogue developed by Novo Nordisk for once-weekly subcutaneous administration. As monotherapy it has been studied in a Phase 1b multiple-ascending-dose trial and a 26-week Phase 2 dose-finding RCT (Lau et al., Lancet 2021); most subsequent evidence concerns its fixed-dose co-administration with semaglutide 2.4 mg (CagriSema) in Phase 2 and Phase 3 trials (REDEFINE and REIMAGINE programmes), several of which included a cagrilintide-alone arm. Cagrilintide is investigational and not approved by any regulator.

MOTS-c is a 16-amino-acid mitochondrial-encoded peptide characterised in 2015. Interventional evidence comes from mouse studies (intraperitoneal dosing in diet-induced obesity and ageing models); human data are limited to observational measurements of endogenous circulating MOTS-c in obesity, coronary endothelial dysfunction, ageing and exercise. A synthetic analogue, CB4211 (CohBar), completed a phase 1a/1b safety study in healthy and NAFLD subjects, but no results have been posted or published in indexed journals. No completed human efficacy trial of MOTS-c itself exists.

Retatrutide (LY3437943) is a once-weekly single-peptide agonist of the GIP, GLP-1 and glucagon receptors developed by Eli Lilly. It has been studied in randomised, double-blind, placebo-controlled Phase 2 trials in adults with obesity (NEJM 2023) and type 2 diabetes (Lancet 2023), a Phase 2a MASLD substudy (Nature Medicine 2024), and a Phase 3 programme (TRIUMPH obesity trials; TRANSCEND type 2 diabetes trials), of which TRANSCEND-T2D-1 is peer-reviewed (Lancet 2026) and TRIUMPH-1 has so far been reported only in a sponsor press release. It is investigational and not approved by any regulator.

Tirzepatide is a once-weekly GIP and GLP-1 receptor agonist that is FDA-approved as Mounjaro (type 2 diabetes) and Zepbound (chronic weight management; obstructive sleep apnoea). It has been studied in large Phase 3 programmes: SURPASS in type 2 diabetes (including the head-to-head SURPASS-2 trial versus semaglutide 1 mg) and SURMOUNT in obesity/overweight (SURMOUNT-1 versus placebo; SURMOUNT-5 versus semaglutide 2.4 mg). Adverse-event, interaction and contraindication data below are taken from the trial abstracts and the current Zepbound prescribing information.

CJC-1295 (DAC:GRF) is a tetrasubstituted analogue of human GHRH(1-29) carrying a C-terminal maleimidopropionamide 'Drug Affinity Complex' that covalently binds serum albumin after injection, extending its half-life to roughly 6-8 days. It was developed by ConjuChem (Canada). Published human data consist of two small randomised placebo-controlled ascending-dose studies in healthy adults (Teichman et al., JCEM 2006) and a single-dose GH-pulsatility study in healthy young men (Ionescu & Frohman, JCEM 2006). A Phase 2 trial in HIV-associated visceral obesity (NCT00267527) was halted in July 2006 after the death of a participant in Argentina; the trial data were never published and development ceased. Note: products sold as 'CJC-1295 without DAC' or 'Mod GRF (1-29)' lack the albumin-binding group and are not the compound studied in these trials; their closest published analogue data are those for sermorelin (GHRH 1-29).

Ipamorelin is a synthetic pentapeptide growth hormone secretagogue (ghrelin-receptor agonist) originally developed by Novo Nordisk and characterised in rat, swine and in-vitro models (Raun et al., 1998). Human evidence is thin: a single-dose intravenous pharmacokinetic/pharmacodynamic study in 40 healthy male volunteers (Gobburu et al., 1999) and one Phase 2 proof-of-concept RCT of intravenous ipamorelin for postoperative ileus after bowel resection (n=117; Beck et al., 2014), which found no significant efficacy difference from placebo. Development was not pursued and ipamorelin is not approved anywhere; there are no published human data on subcutaneous, repeated or long-term use, nor on body composition or 'anti-ageing' end points.

Sermorelin (GHRH 1-29 NH2) is the shortest synthetic fragment of human growth hormone-releasing hormone retaining full biological activity. It was FDA-approved as Geref (EMD Serono): a 0.05 mg diagnostic ampoule (NDA 19-863, approved 1990) for testing pituitary GH secretion and 0.5/1.0 mg vials (NDA 20-443, approved 1997) for idiopathic growth hormone deficiency in children with growth failure. Both were discontinued in 2008 and the NDAs withdrawn in 2009; FDA determined in 2013 that withdrawal was not for safety or effectiveness reasons. The efficacy evidence is a 1-year open-label multicentre paediatric study (Thorner et al., 1996) plus smaller paediatric trials, one of which found sermorelin less effective than somatropin; adult data are limited to small studies in healthy elderly volunteers. No approved sermorelin product exists today and the original prescribing information is not available on FDA or DailyMed sites, so adverse-effect data below come from published trials and a peer-reviewed drug review.

Tesamorelin (TH9507) is a synthetic analogue of growth hormone-releasing hormone (GHRH 1-44) that is FDA-approved as Egrifta / Egrifta SV / Egrifta WR for reduction of excess abdominal fat in HIV-infected adults with lipodystrophy. Its evidence base consists of two 26-week Phase 3 placebo-controlled trials (806 randomised) with 26-week safety extensions, a 6-month NIH-funded RCT examining visceral and liver fat (Stanley et al., JAMA 2014), and the FDA prescribing information, which carries contraindications (hypothalamic-pituitary axis disruption, active malignancy, pregnancy, hypersensitivity) and warnings on IGF-1 elevation, fluid retention and glucose intolerance.

No controlled clinical study of CJC-1295 and ipamorelin administered together has been published; a search of the indexed literature for the two names returns only narrative reviews and anti-doping papers. Each component has separate human data: CJC-1295 (a GHRH(1-29) analogue with albumin binding) was studied in two randomized, placebo-controlled ascending-dose trials in healthy adults (Teichman 2006), and ipamorelin (a ghrelin-receptor agonist pentapeptide) in a healthy-volunteer PK/PD infusion study (Gobburu 1999) and a phase 2 postoperative-ileus trial (Beck 2014). The individual component entries should be consulted for those data; nothing here describes the combination.

BPC-157 (pentadecapeptide GEPPPGKPADDAGLV) has been studied almost entirely in rodent models, most often in rats given 10 µg/kg or 10 ng/kg intraperitoneally, intragastrically or in drinking water, across tendon, ligament, muscle, gastrointestinal and NSAID-toxicity models. No completed, published randomised controlled trial in humans exists; the human literature consists of a retrospective chart review of 17 patients given intra-articular injections (Lee & Padgett 2021) and a two-participant intravenous pilot (Lee & Burgess 2025), both in the same non-indexed journal. A Phase 1 oral study (NCT02637284) was registered in 2015 but no results have been posted. In September 2023 the U.S. FDA placed BPC-157 in Category 2 of its 503A bulk-substance interim policy, citing immunogenicity and impurity concerns and 'no, or only limited, safety-related information'; the nomination has since been withdrawn.

No controlled human study of the BPC-157 + TB-500 combination exists. The combination has been tested once in animals: a 2026 rat Achilles-tendon transection study (Biçer et al.) gave BPC-157 10 µg/kg/day, TB-500 60 µg/kg/day, or both intraperitoneally for four weeks and found the combination conferred no additional benefit over either agent alone. A 2021 retrospective chart review of 17 knee-pain patients described intra-articular BPC 157 'alone or combined with thymosin beta-4' (full-length, not the fragment) with only a telephone pain survey and no control group. Component evidence is summarised in the individual entries; FDA Category 2 statements cover both components.

KPV is the C-terminal tripeptide (Lys-Pro-Val) of α-melanocyte-stimulating hormone. It has been studied in mouse and rat colitis models (oral in drinking water, and rectal or oral nanoparticle/hydrogel formulations) and in human intestinal epithelial and T-cell lines, where nanomolar concentrations reduced NF-κB activation. No human clinical trial of KPV has been published or registered; FDA's Category 2 entry states it 'has not identified any human exposure data on drug products containing KPV administered via any route'.

TB-500 is a synthetic 7-amino-acid fragment (Ac-LKKTETQ, residues 17-23) of the 43-amino-acid protein thymosin beta-4 (Tβ4). The two are not interchangeable in the evidence base: every completed human trial has used full-length recombinant or synthetic Tβ4 (RegeneRx/ReGenTree's RGN-259 eye drops and RGN-137 dermal gel) applied topically, whereas the fragment has been studied only in animals (e.g., a 2026 rat Achilles-tendon study at 60 µg/kg/day intraperitoneally). FDA's Category 2 statement on the fragment says it 'has not identified any human exposure data' for it. One Phase 1/2 trial of the fragment (NCT07487363, cardiovascular biomarkers) began recruiting in 2026 and has no results. Adverse-event data below therefore come from full-length Tβ4 trials and are labelled as such.

GHK-Cu is the copper(II) complex of the endogenous tripeptide Gly-His-Lys, first isolated from human plasma. Human data are limited to topical use: a multicentre randomised, evaluator-blinded trial of a GHK-Cu gel (Iamin) on diabetic neuropathic ulcers (Mulder 1994) and a small randomised study of GHK-Cu skin-care products after CO2 laser resurfacing (Miller 2006, n=13), plus in-vitro human skin penetration work. The remaining literature is in-vitro (fibroblast and gene-expression studies) and animal wound models, summarised in Pickart & Margolina's 2018 review. No human trial of injectable GHK-Cu has been published; FDA's Category 2 entry for injectable GHK-Cu cites immunogenicity risk and 'limited data in humans'.

No controlled study, in humans or animals, has evaluated the three-way combination of BPC-157, the TB-500 fragment and GHK-Cu. The only combination data located is a 2026 rat Achilles-tendon study of BPC-157 plus TB-500 (without GHK-Cu), in which the pair did not outperform either peptide alone. Evidence for each component is summarised in its own entry; none of the components has a completed, published human randomised trial for the marketed form (GHK-Cu human data are topical only). FDA has listed all three components (BPC-157, TB-500 fragment, injectable GHK-Cu) under its Category 2 safety statements citing immunogenicity/impurity concerns and limited or absent human safety data.

No controlled study, in humans or animals, has evaluated the four-way combination of BPC-157, the TB-500 fragment, GHK-Cu and KPV. The only combination data located is a 2026 rat Achilles-tendon study of BPC-157 plus TB-500 alone, in which the pair did not outperform either peptide individually. KPV has no human exposure data at all (FDA statement), and the other components have no completed human randomised trials for the marketed forms. Evidence for each component is summarised in its own entry; FDA Category 2 safety statements exist for all four components.

Melanotan II is a cyclic alpha-MSH analogue studied at the University of Arizona in the 1990s in a pilot phase I tanning study (3 men) and in three small double-blind, placebo-controlled crossover studies of penile erection in men with erectile dysfunction (10-20 men each), all using subcutaneous doses of 0.01-0.03 mg/kg. Development was abandoned in favour of bremelanotide. It has no marketing approval anywhere; the US FDA has treated it as an unapproved new drug since 2007 and Australia's TGA warns against its use. Published case reports link unlicensed MT-II use to melanoma, though causality is not established.

Thymosin alpha-1 is a 28-amino-acid thymic peptide marketed as thymalfasin (Zadaxin, SciClone) and reported to be approved in more than 35 countries, chiefly for chronic hepatitis B and C and as an immune adjuvant; it has no FDA-approved labeling in the United States (no entry in DailyMed) although it carries US orphan-drug designations. Randomized trials, mostly from the 1990s-2000s and largely in Asia and Italy, used 1.6 mg subcutaneously twice weekly for 6-12 months in hepatitis B, in combination with interferon in hepatitis C, and as an adjunct in severe sepsis (ETASS, n=361). Adverse-event reporting across these trials is limited but consistently describes injection-site discomfort as the main event.

Selank is a synthetic tuftsin analogue developed in Russia and studied there as an intranasal anxiolytic. The indexed human evidence consists of Russian-language comparative clinical studies (2008-2015) in generalised anxiety disorder, neurasthenia and anxiety-phobic/somatoform disorders, benchmarked against benzodiazepines (medazepam, phenazepam) rather than placebo, plus one placebo-controlled fMRI study in healthy volunteers. Doses and routes are not reported in most English abstracts, and no Western replication has been published.

Semax is a synthetic ACTH(4-10) fragment analogue developed in Russia and used there as an intranasal preparation. The human evidence consists of Russian-language, PubMed-indexed clinical studies from the 1990s-2010s in acute ischaemic stroke, chronic cerebrovascular insufficiency, motor neuron disease and other neurological conditions, most of them open-label or with non-randomised control groups; mechanistic work is largely in rat ischaemia models. No Western, placebo-controlled replication of the stroke findings has been published in the indexed literature, and only English abstracts of most trials are available.

DSIP is a nonapeptide isolated from rabbit cerebral venous blood in 1977. Human interventional studies were conducted mainly between 1981 and 1992, almost all using slow intravenous infusion of synthetic DSIP at 25-30 nmol/kg, in small groups of healthy volunteers, chronic insomniacs, patients with chronic pain, and inpatients in alcohol or opiate withdrawal. Results were mixed: Schneider-Helmert's group reported sleep normalisation, whereas two later double-blind studies (Monti 1987; Bes 1992) found little or no clinically meaningful effect. A 2009 anaesthesia study is the only modern human trial. All evidence is old, small, and predates current trial-reporting standards.

Bremelanotide is a cyclic melanocortin-receptor agonist approved by the US FDA in 2019 (Vyleesi) for premenopausal women with acquired, generalized hypoactive sexual desire disorder. Approval rested on two identical 24-week, randomized, placebo-controlled phase 3 trials (RECONNECT; n=1,247 in the safety population) with 52-week open-label extensions. The FDA label defines the regimen, adverse-reaction rates, contraindications and drug interactions summarised here. Earlier phase 1/2 work in men used intranasal and subcutaneous formulations.

Reduced glutathione (γ-glutamyl-cysteinyl-glycine) has been studied in humans by oral, inhaled and intravenous routes in randomised controlled trials: oral 250-1,000 mg/day for 6 months in healthy adults (Richie 2015) and 500 mg twice daily for 4 weeks (Allen & Bradley 2011, no change in biomarkers); inhaled 646 mg twice daily for 6 months in cystic fibrosis (Griese 2013, n=153, primary endpoint not met); intravenous 1,400 mg three times weekly for 4 weeks in Parkinson's disease (Hauser 2009, n=21); and intravenous 1,500 mg/m2 before oxaliplatin in colorectal cancer (Cascinu 2002, n=52). Trials generally report tolerability comparable to placebo. Separate safety signals relate to compounded intravenous products: in 2019 FDA reported seven patients with nausea, vomiting and, in one case, breathing difficulty requiring hospitalisation after IV glutathione compounded from a powder suspected of high endotoxin levels.

Human data must be separated by molecule. Direct NAD+ administration has been studied only in small studies: a pharmacokinetic pilot of a 6-hour intravenous infusion at 3 µmol/min (Grant 2019) and a retrospective tolerability review of 500 mg IV NAD+ versus IV nicotinamide riboside on four consecutive days in a commercial clinic (Reyna 2026). The larger randomised, placebo-controlled trials involve oral NAD+ precursors rather than NAD+ itself: nicotinamide riboside (NR) 100-1,000 mg/day for 8 weeks (Conze 2019), 1,000 mg/day for 6 weeks in a crossover (Martens 2018) and 2,000 mg/day for 12 weeks in obese men (Dollerup 2018), and nicotinamide mononucleotide (NMN) 250 mg/day for 10 weeks in prediabetic postmenopausal women (Yoshino 2021). Oral precursor trials report tolerability similar to placebo; intravenous NAD+ is associated with infusion-rate-dependent symptoms (gastrointestinal upset, chest pressure, increased heart rate) that resolve when the infusion ends.